[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"beta-thalassemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:beta-thalassemia":29},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,17,0,[8,47,61,92,120,142,170,200,225,248,274,307,328,350,386,408,429],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":30,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100053445","phase-1-partial-stem-cell-transplant-for-sickle-cell-disease-from-matched-donors-100053445",false,"NCT07599176","Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors","Matched Related Donor Non-myeloablative Hematopoietic Cell Transplantation With Alemtuzumab, 400 cGy TBI, and Abatacept for Sickle Cell Disease and Beta-Thalassemia","* INCLUSION CRITERIA\n\nRECIPIENT:\n\nParticipants must fulfill one disease category (1 or 2) and 3\n\n1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F):\n\n   A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb\n\n   B. Abnormal trans-cranial Doppler examination (\\>=200 cm\u002Fs); OR\n\n   C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR\n\n   D. Sickle cell related renal insufficiency defined by a creatinine level \\>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \\\u003C60mL\u002Fmin\u002F1.73m2 for patients \\\u003C16 years of age or \\\u003C50mL\u002Fmin for patients \\>16 years of age OR requiring peritoneal or hemodialysis; OR\n\n   Age (Years): \\\u003C= 5 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 0.8\n\n   Age (Years): 5 \\\u003C age \\\u003C= 10 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.0\n\n   Age (Years): 10 \\\u003C age \\\u003C= 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.2\n\n   Age (Years): \\> 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.3\n\n   E. Tricuspid regurgitant jet velocity (TRV) of \\>=2.5 m\u002Fs in patients at least 3 weeks after a vaso-occlusive crisis; OR\n\n   F. Recurrent severe priapism defined as at least two episodes of an erection lasting \\>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR\n\n   G. Sickle hepatopathy defined as EITHER ferritin \\>1000mcg\u002FL OR direct bilirubin \\>0.4 mg\u002FdL at baseline; OR\n\n   H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment \u002Fmedication; OR\n\n   I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment \u002Fmedication\n2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:\n\n   * Portal fibrosis by liver biopsy\n   * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)\n   * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans\n3. Non disease specific\n\n   * Ages \\>=4 years and less than 65 years old\n   * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10)\n   * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable\n   * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.\n\n     * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant\u002Finjection from start of screening until immunosuppression is stopped.\n     * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped.\n\nDONOR:\n\n* Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.\n* Donors age 4 or older and \\>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.\n* Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA\n\nRECIPIENT:\n\n* Karnofsky or Lanksy performance status of \\\u003C40\n* Diffusing capacity of carbon monoxide \\\u003C35% predicted: DLCO corrected for hemoglobin or KCO (corrected for lung volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.\n* Baseline oxygen saturation of \\\u003C85% or PaO2 \\\u003C70\n* Left ventricular ejection fraction: \\\u003C35% estimated by ECHO\n* Transaminases \\>5x upper limit of normal for age\n* Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen\n* Major anticipated illness or organ failure incompatible with survival from HCT\n* Pregnant or breastfeeding\n\nDONOR:\n\n* Pregnant or breastfeeding\n* Cognitively impaired subjects","ALL","4 Years","65 Years",{"count":20,"type":21},90,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cells to stably remain in the recipients long term.",[28,29],"Sickle Cell Disease","Beta-thalassemia",[28,31,32,33],"Beta-Thalassemia","Stem Cell Transplant","Chimerism","RECRUITING","2026-07-10",{"date":37,"type":38},"2026-07-13","ACTUAL",{"date":40,"type":38},"2026-07-08",{"date":42,"type":21},"2035-06-30",{"name":44,"class":45},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":48,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":50,"targetDuration":4,"studyType":22,"phases":51,"briefSummary":26,"conditions":52,"keywords":53,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":54,"lastUpdatePostDateStruct":55,"startDateStruct":57,"completionDateStruct":59,"leadSponsor":60,"locationsCount":46},"100641763","* INCLUSION CRITERIA\n\nRECIPIENT:\n\nParticipants must fulfill one disease category (1 or 2) and 3\n\n1. Patients with sickle cell disease at high risk for disease related morbidity or mortality, defined by having an end-organ damage (A, B, C, D, OR E) or complication(s) not ameliorated by sickle cell-specific therapies (F):\n\n   A. Stroke defined as a clinically significant neurologic event that is accompanied by an infarct on cerebral MRI ORb\n\n   B. Abnormal trans-cranial Doppler examination (\\>=200 cm\u002Fs); OR\n\n   C. Silent cerebral infarct defined as an infarct-like lesion based on an MRI signal abnormality at least 3 mm in one dimension and visible in two planes on FLAIR or T2- weighted images (or similar image with 3D imaging) and documented neurological examination performed by a neurologist demonstrating the participant has a normal neurologic examination, or an abnormality on examination that could not be explained by the location of the brain lesion(s); OR\n\n   D. Sickle cell related renal insufficiency defined by a creatinine level \\>=1.5 times the upper limit of normal and kidney biopsy consistent with sickle cell nephropathy OR nephrotic syndrome OR creatinine clearance \\\u003C60mL\u002Fmin\u002F1.73m2 for patients \\\u003C16 years of age or \\\u003C50mL\u002Fmin for patients \\>16 years of age OR requiring peritoneal or hemodialysis; OR\n\n   Age (Years): \\\u003C= 5 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 0.8\n\n   Age (Years): 5 \\\u003C age \\\u003C= 10 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.0\n\n   Age (Years): 10 \\\u003C age \\\u003C= 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.2\n\n   Age (Years): \\> 15 \u002F Upper limit of normal serum creatinine (mg\u002Fdl): 1.3\n\n   E. Tricuspid regurgitant jet velocity (TRV) of \\>=2.5 m\u002Fs in patients at least 3 weeks after a vaso-occlusive crisis; OR\n\n   F. Recurrent severe priapism defined as at least two episodes of an erection lasting \\>=4 hours requiring medical intervention (e.g. aspiration, injection of vasoconstrictor, prior penile surgery.); OR\n\n   G. Sickle hepatopathy defined as EITHER ferritin \\>1000mcg\u002FL OR direct bilirubin \\>0.4 mg\u002FdL at baseline; OR\n\n   H. Vaso-occlusive crises: more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment \u002Fmedication; OR\n\n   I. Acute chest syndrome (ACS): any ACS while on sickle cell treatment \u002Fmedication\n2. Patients with beta-thalassemia who have grade 2 or 3 iron overload, determined by the presence of 2 or more of the following:\n\n   * Portal fibrosis by liver biopsy\n   * Inadequate chelation history (defined as failure to maintain adequate compliance with chelation with deferoxamine initiated within 18 months of the first transfusion and administered at least 5 days each week)\n   * Hepatomegaly of greater than 2 cm below the costochondral margin or by other imaging scans\n3. Non disease specific\n\n   * Ages \\>=4 years and less than 65 years old\n   * Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10\n   * Ability to comprehend and willing to sign an informed consent, assent obtained from minors when applicable. Negative serum or urine beta-HCG, when applicable\n   * Agree to use birth control throughout the study and 3 months after abatacept or sirolimus administration.\n\n     * Female subjects must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or implant\u002Finjection from start of screening until immunosuppression is stopped.\n     * Male subjects must agree to use effective contraception (including condoms) from start of screening until immunosuppression is stopped.\n\nDONOR:\n\n* Fully matched human leukocyte antigen (HLA) donors at A, B, C, and DR loci (8 of 8 or 10 of 10) are intended for this study.\n* Donors age 4 or older and \\>=15 kg (or weight deemed acceptable by IR for line placement, DTM for apheresis, and pediatric consult service) eligible to donate hematopoietic stem cells, are eligible for this study.\n* Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Donors will sign on a separate protocol, 20-H-0099 NHLBI standard of care protocol for the mobilization and collection of HSCs. Note that participation in this study is offered to all eligible donors, but is not required for a donor to make a stem cell donation, so it is possible that not all donors will enroll onto this study.\n\nEXCLUSION CRITERIA\n\nRECIPIENT:\n\n* Karnofsky or Lanksy performance status of \\\u003C40\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C35% predicted (corrected for hemoglobin and alveolar volume). This criterion may be omitted in young children (e.g. near age 5) or other individuals who may have difficulty understanding or complying with instructions of testing.\n* Baseline oxygen saturation of \\\u003C85% or PaO2 \\\u003C70\n* Left ventricular ejection fraction: \\\u003C35% estimated by ECHO\n* Transaminases \\>5x upper limit of normal for age\n* Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within one month prior to starting the conditioning regimen\n* Major anticipated illness or organ failure incompatible with survival from HCT\n* Pregnant or breastfeeding\n\nDONOR:\n\n* Pregnant or breastfeeding\n* Cognitively impaired subjects",{"count":20,"type":21},[24,25],[28,29],[28,31,32,33],"2026-07-01",{"date":56,"type":38},"2026-07-02",{"date":58,"type":21},"2026-07-07",{"date":42,"type":21},{"name":44,"class":45},{"id":62,"slug":63,"hasResults":11,"nctId":64,"briefTitle":65,"officialTitle":66,"acronym":4,"eligibilityCriteria":67,"healthyVolunteers":11,"sex":16,"minAge":68,"maxAge":4,"enrollmentInfo":69,"targetDuration":4,"studyType":22,"phases":71,"briefSummary":73,"conditions":74,"keywords":77,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":82,"lastUpdatePostDateStruct":83,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":91},"100367421","phase-3-a-study-to-evaluate-long-term-safety-in-participants-who-have-participated-in-other-luspatercept-ace-536-clinical-trials-100367421","NCT04064060","A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials","A Phase 3b, Open-label, Single-arm, Rollover Study to Evaluate Long-term Safety in Subjects Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials","Inclusion Criteria:\n\nParticipants must meet all the following criteria to be enrolled in this study:\n\n1. Participant is ≥ 18 years at the time of signing the informed consent form (ICF).\n2. Participant is willing and able to adhere to the study visit schedule and other protocol requirements.\n3. Participant has been participating in a luspatercept trial and continues to fulfill all the requirements of the parent protocol and the participant has been either:\n\n   1. Assigned to luspatercept treatment, continues to receive clinical benefit in the opinion of the investigator and should continue to receive luspatercept treatment, OR\n   2. Assigned to placebo arm in the parent protocol (at the time of unblinding or in follow-up) and should cross over to luspatercept treatment, OR\n   3. Assigned to the Follow-up Phase of the parent protocol, previously treated with luspatercept or placebo in the parent protocol who shall continue into LTPTFU phase in the rollover study until the follow-up commitments are met (unless requirements are met as per parent protocol to crossover to luspatercept treatment).\n4. Participant understands and voluntarily signs an informed consent document prior to any study-related assessments or procedures being conducted.\n5. Participant demonstrates compliance, as assessed by the investigator, with the parent study protocol requirements.\n6. Applies to on treatment Participants only- females of childbearing potential (FCBP) defined as a sexually mature woman who:\n\n1\\) has achieved menarche at some point, 2) has not undergone a hysterectomy or bilateral oophorectomy, or 3) has not been naturally postmenopausal (amenorrhea following cancer therapy or amenorrhea due to other medical reasons does not rule out childbearing potential) for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must:\n\n1. Have two negative pregnancy tests as verified by the investigator prior to starting study therapy. She must agree to ongoing pregnancy testing during the course of the study, and after end of study therapy. This applies even if the participant practices true abstinence from heterosexual contact.\n2. Agrees to use, and be able to comply with highly effective, contraception without interruption, 35 days prior to starting investigational product (IP), during the study therapy (including dose interruptions), and for 84 days after discontinuation of study therapy.\n\n   7\\. Applies to on treatment participants only- Male participants must:\n\na. Agrees to use a condom during sexual contact with a pregnant female or a female of childbearing potential while participating in the study, during dose interruptions and for at least 84 days following investigational product discontinuation even if he has undergone a successful vasectomy.\n\nExclusion Criteria:\n\nThe presence of any of the following will exclude a participant from enrollment:\n\n1. Applies to on treatment participants only- Concomitant use of any medications\u002Fprocedures that are prohibited in the parent luspatercept protocol.\n2. Participant has met one or more criteria for study discontinuation as stipulated in the parent luspatercept protocol.\n3. Applies to on treatment participants only- More than 26 days between last luspatercept dose in the parent protocol and first dose into ACE-536-LTFU-001 protocol unless dose delay or dose discontinuation criteria met.\n4. Applies to on treatment participants only- Pregnant or breastfeeding females.\n5. Participant has any significant medical condition, laboratory abnormality, psychiatric illness, or is considered vulnerable by local regulations (eg, imprisoned or institutionalized) that would prevent the subject from participating in the study.\n6. Participant has any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he\u002Fshe were to participate in the study.\n7. Participant has any condition that confounds the ability to interpret data from the study.","18 Years",{"count":70,"type":21},665,[72],"PHASE3","A Phase 3b, open-label, single-arm, rollover study to evaluate the long-term safety of luspatercept, to the following participants:\n\n* Participants receiving luspatercept on a parent protocol at the time of their transition to the rollover study, who tolerate the protocol-prescribed regimen in the parent trial and, in the opinion of the investigator, may derive clinical benefit from continuing treatment with luspatercept\n* Participants in the follow-up phase previously treated with luspatercept or placebo in the parent protocol will continue into long-term post-treatment follow-up in the rollover study until the follow-up commitments are met\n* The study design is divided into the Transition Phase, Treatment Phase and Follow-up Phase. Participants will enter transition phase and depending on their background will enter either the treatment phase or the Long-term Post-treatment Follow-up (LTPTFU) phase\n* Transition Phase is defined as one Enrollment visit\n* Treatment Phase: For participants in luspatercept treatment the dose and schedule of luspatercept in this study will be the same as the last dose and schedule in the parent luspatercept study. This does not apply to participants that are in long-term follow-up from the parent protocol\n* Follow-up Phase includes:\n\n  \\- 42 Day Safety Follow-up Visit\n* During the Safety Follow up, the participants will be followed for 42 days after the last dose of luspatercept, for the assessment of safety-related parameters and adverse event (AE) reporting\n\n  \\- Long-term Post-treatment Follow-up (LTPTFU) Phase\n* Participants will be followed for overall survival every 6 months for at least 5 years from first dose of luspatercept in the parent protocol, or 3 years of post-treatment from last dose, whichever occurs later, or until death, withdrawal of consent, study termination, or until a subject is lost to follow-up. Participants will also be monitored for progression to AML or any malignancies\u002Fpre-malignancies. New anticancer or disease related therapies should be collected at the same time schedule\n\nParticipants transitioning from a parent luspatercept study in post-treatment follow-up (safety or LTPTFU) will continue from the same equivalent point in this rollover study.\n\nThe ACE-536-LTFU-001 rollover study will be terminated, and relevant participants will discontinue from the study when all participants fulfill 5 years on the study, including treatment and follow-up.",[75,29,76],"Myelodysplastic Syndromes (MDS)","Myeloproliferative Neoplasm(MPN)-Associated Myelofibrosis",[78,79,80,29,81],"ACE-536","Luspatercept","MDS","Myeloproliferative neoplasm (MPN)-associated myelofibrosis","2026-06-30",{"date":54,"type":38},{"date":85,"type":38},"2019-08-12",{"date":87,"type":21},"2028-05-12",{"name":89,"class":90},"Celgene","INDUSTRY",143,{"id":93,"slug":94,"hasResults":11,"nctId":95,"briefTitle":96,"officialTitle":97,"acronym":4,"eligibilityCriteria":98,"healthyVolunteers":11,"sex":16,"minAge":68,"maxAge":99,"enrollmentInfo":100,"targetDuration":4,"studyType":22,"phases":102,"briefSummary":103,"conditions":104,"keywords":105,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":46},"100544122","phase-1-als20-101-lentiviral-gene-therapy-for-beta-thalassemia-100544122","NCT06364774","ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia","Phase 1\u002F2 Study Evaluating the Safety and Efficacy of Gene Therapy Employing Lentiviral Vector ALS20-transduced Hematopoietic Progenitor Cells in Subjects With Transfusion-dependent-thalassemia","Inclusion Criteria:\n\n1. Age 18 to \\\u003C 40 years at the time of consent\n2. Diagnosis of transfusion dependent beta thalassemia (β0 β0, β+β0, β+β+, βEβ0, βEβ+,β0 or β+ \u002FβA + alpha triplication(s)). Transfusion-dependent is defined as a history of receiving at least 120 mL\u002Fkg\u002Fyear packed red blood cells or at least 8 transfusions per year in the past two years. The first 2 subjects enrolled must have a non- β0 β0 genotype.\n3. Genetic confirmation of α and β thalassemia diagnosis (β0β0, β+β0, β+β+, βEβ0, βEβ+, β0 or β+ \u002FβA + alpha triplication(s)) by a Clinical Laboratory Improvement Amendments (CLIA) certified laboratory is required.\n4. Clinically stable, Karnofsky score at least 70, and eligible to undergo Hematopoietic Stem Cell Transplantation (HSCT).\n5. Female subjects of childbearing potential must agree to use acceptable method(s) of contraception from consent through at least 6 months after CHOP-ALS20 infusion\n6. Male subjects of reproductive capacity must agree to use effective contraception from start of mobilization through at least 6 months after CHOP-ALS20 infusion\n7. All potential treatment options including allogeneic HSCT (HLA-matched related, HLA-matched unrelated, and haploidentical) as well as FDA approved gene therapy options have been thoroughly discussed with the independent hematologist and\u002For transplant physician and subject agrees to proceed with this clinical trial.\n\nExclusion Criteria:\n\n1. Prior receipt of HSCT or gene therapy\n2. More than one alpha globin gene deletions\u002Fmutations.\n3. Any prior or current malignancy (excluding adequately treated basal or squamous cell carcinoma of the skin)\n4. Known cancer predisposition syndrome\n5. Positive for HIV-1, HIV-2, Human T Cell Lymphotropic Virus-1,2 (HTLV-1, HTLV-2) or active hepatitis B or active hepatitis C infection\n6. Clinically significant active bacterial, viral (including COVID-19 and influenza), fungal, or parasitic infection (temporary exclusion)\n7. Clinically significant bleeding disorder\n8. Evidence of cardiac dysfunction (left ventricular ejection fraction \\\u003C50% or shortening fraction \\\u003C27%) or clinically significant arrhythmia\n9. Evidence of advanced liver disease (ALT \\>5x the upper limit of normal (ULN), prothrombin time \\>1.5 x ULN, direct bilirubin \\> 3x ULN) not attributable to iron chelation therapy, or evidence of bridging fibrosis on liver biopsy or fibrosis stage of F3 or higher by magnetic resonance elastography (MRE) if obtained as part of clinical care\n10. Liver R2 or R2 MRI or liver biopsy with liver iron concentration 15 mg\u002Fg dw (temporary exclusion)\n11. Diffusion capacity of the lungs for carbon monoxide (DLco) \\\u003C50% of predicted (corrected for Hb)\n12. Pulse oximetry in room air \\\u003C92%\n13. Evidence of renal dysfunction (creatinine \\>1.5x ULN or Glomerular Filtration Rate (GFR) \\\u003C70 ml\u002Fmin\u002F1.73 m2 based on cystatin C\u002Fcreatinine equation)\n14. Cardiac T2 MRI \\\u003C 10 ms\n15. Platelet count \\\u003C100,000\u002FmcL or absolute neutrophil count \\\u003C1000\u002FmcL except if attributed to benign ethnic neutropenia\n16. Unable to receive red cell transfusion (significant allo\u002Fauto immunization)\n17. Uncontrolled systemic hypertension\n18. Uncontrolled seizure disorder\n19. Diagnosis of a significant psychiatric disorder that could seriously impede the ability to participate in the study as determined by the investigator\n20. Immediate family member with a known or suspected Familial Cancer Syndrome\n21. Contraindication to anesthesia\n22. For female subjects, pregnancy or breastfeeding\n23. Participation in another clinical trial of an investigational drug within 30 days or 5 drug half-lives, whichever is longer, of screening (temporary exclusion)\n24. Any other condition that would render the subject ineligible for mobilization\u002Fapheresis and\u002For HSCT as determined by the investigator","40 Years",{"count":101,"type":21},12,[24,25],"The main goal of this study is to find out if the blood disorder called transfusion-dependent beta thalassemia can be safely treated by modifying blood stem cells. This is done by collecting blood stem cells from the subject, modifying those cells, adding a healthy beta globin gene, and then giving them back to the subject. It is hoped that these modified cells will decrease the need for blood transfusions. The gene modified blood stem cells are called CHOP-ALS20 (\"study drug\"). This experimental gene therapy has not been tried on human beings before and is not FDA approved.",[31],[106,107,108,109],"Thalassemia","Beta Thalassemia","gene therapy","beta globin gene","2026-04-13",{"date":112,"type":38},"2026-04-15",{"date":114,"type":38},"2025-04-14",{"date":116,"type":21},"2027-12-31",{"name":118,"class":119},"Children's Hospital of Philadelphia","OTHER",{"id":121,"slug":122,"hasResults":11,"nctId":123,"briefTitle":124,"officialTitle":124,"acronym":4,"eligibilityCriteria":125,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":126,"targetDuration":4,"studyType":128,"phases":4,"briefSummary":129,"conditions":130,"keywords":4,"overallStatus":133,"whyStopped":4,"lastUpdateSubmitDate":134,"lastUpdatePostDateStruct":135,"startDateStruct":137,"completionDateStruct":138,"leadSponsor":140,"locationsCount":4},"100629923","evaluation-of-micro-rna-155-expression-in-relation-to-alloantibody-formation-in-transfusion-dependent-patients-100629923","NCT07480980","Evaluation of Micro RNA-155 Expression in Relation to Alloantibody Formation in Transfusion-Dependent Patients","Inclusion Criteria:\n\n1- Confirmed transfusion-dependent . 2-Regular RBC transfusion history 3- Age ≥5 years\n\n\\-\n\nExclusion Criteria:\n\n* 1- Autoimmune diseases 2- Active infections or inflammatory conditions. 3- Immunosuppressive therapy Ý",{"count":127,"type":21},100,"OBSERVATIONAL","Transfusion-dependent patients, particularly those with β-thalassemia major, require lifelong regular red blood cell (RBC) transfusions to maintain adequate hemoglobin levels and prevent severe anemia. Although transfusion therapy significantly improves survival and quality of life, it is associated with several immunological complications, the most important of which is red cell alloimmunization. Alloimmunization occurs when the recipients immune system recognizes foreign antigens on donor RBCs and produces alloantibodies against them, which may lead to hemolytic transfusion reactions, difficulty in finding compatible blood and increased transfusion requirements (1). The incidence of RBC alloimmunization in transfusion-dependent patients varies widely but remains a major clinical challenge in transfusion medicine (2).\n\nRecent advances in molecular hematology have highlighted the importance of microRNAs (miRNAs) in regulating immune responses and hematopoiesis. MicroRNAs are small non-coding RNA molecules that regulate gene expression at the post-transcriptional level and play a key role in both innate and adaptive immunity (3). Among them, microRNA-155 (miR-155) has emerged as a critical regulator of inflammatory pathways, antigen presentation, and lymphocyte activation. It modulates immune cell differentiation and cytokine production, thereby influencing immune responses to foreign antigens (4, 5).\n\nIn patients with β-thalassemia, miR-155 is also implicated in erythropoiesis and ineffective red cell production, suggesting its involvement in both hematologic and immunologic pathways of the disease. Increased expression of miR-155 has been reported in thalassemic erythroid cells and is associated with altered erythroblast proliferation and differentiation (6).\n\nImportantly, recent studies suggest that miR-155 may contribute to the development of alloimmunization in transfusion-dependent patients.",[131,132,107],"Red Cell Alloantibodies","Blood Transfusion-Dependent","NOT_YET_RECRUITING","2026-03-31",{"date":136,"type":38},"2026-04-06",{"date":54,"type":21},{"date":139,"type":21},"2028-07-01",{"name":141,"class":119},"Assiut University",{"id":143,"slug":144,"hasResults":11,"nctId":145,"briefTitle":146,"officialTitle":147,"acronym":4,"eligibilityCriteria":148,"healthyVolunteers":11,"sex":16,"minAge":149,"maxAge":150,"enrollmentInfo":151,"targetDuration":4,"studyType":22,"phases":153,"briefSummary":154,"conditions":155,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":160,"lastUpdatePostDateStruct":161,"startDateStruct":163,"completionDateStruct":165,"leadSponsor":167,"locationsCount":169},"100475948","phase-3-evaluation-of-efficacy-and-safety-of-a-single-dose-of-ctx001-in-participants-with-transfusion-dependent--thalassemia-and-severe-sickle-cell-disease-100475948","NCT05477563","Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease","A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Transfusion-Dependent β-Thalassemia or Severe Sickle Cell Disease","Key Inclusion Criteria:\n\n* Participants with TDT and SCD:\n* Eligible for autologous stem cell transplant as per investigator's judgment.\n* Participants with TDT:\n* Diagnosis of TDT as defined by:\n* Documented homozygous β-thalassemia or compound heterozygous β-thalassemia including β-thalassemia\u002Fhemoglobin E (HbE). Participants can be enrolled based on historical data, but a confirmation of the genotype using the study central laboratory will be required before busulfan conditioning\n* History of at least 100 milliliter (mL)\u002Fkilograms (kg)\u002Fyear or 10 units\u002Fyear of packed red blood cells (RBC) transfusions in the prior 2 years before signing the consent or the last rescreening for patients going through re-screening\n* Participants with SCD:\n* Diagnosis of severe SCD as defined by:\n* Documented SCD genotypes\n* History of at least two severe VOCs events per year for the previous two years prior to enrollment\n\nKey Exclusion Criteria:\n\n* Participants with TDT and SCD:\n* A willing and healthy 10\u002F10 human leukocyte antigen (HLA)-matched related donor is available per investigator's judgement\n* Prior hematopoietic stem cell transplant (HSCT)\n* Clinically significant and active bacterial, viral, fungal, or parasitic infection as determined by the investigator\n* Participants with TDT:\n* Participants with associated α-thalassemia and \\>1 alpha deletion, or alpha multiplications\n* Participants with sickle cell β-thalassemia variant\n* Participants with SCD:\n* History of untreated moyamoya syndrome or presence of moyamoya syndrome at screening\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.","12 Years","35 Years",{"count":152,"type":21},26,[72],"This is a single-dose, open-label study in participants with transfusion-dependent β-thalassemia (TDT) or severe sickle cell disease (SCD). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells (hHSPCs) using CTX001.",[31,106,156,157,158,28,159],"Hematologic Diseases","Genetic Diseases, Inborn","Hemoglobinopathies","Sickle Cell Anemia","2026-03-18",{"date":162,"type":38},"2026-03-23",{"date":164,"type":38},"2022-08-02",{"date":166,"type":21},"2027-06-09",{"name":168,"class":90},"Vertex Pharmaceuticals Incorporated",6,{"id":171,"slug":172,"hasResults":11,"nctId":173,"briefTitle":174,"officialTitle":175,"acronym":4,"eligibilityCriteria":176,"healthyVolunteers":11,"sex":16,"minAge":177,"maxAge":99,"enrollmentInfo":178,"targetDuration":4,"studyType":22,"phases":180,"briefSummary":181,"conditions":182,"keywords":185,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":191,"lastUpdatePostDateStruct":192,"startDateStruct":194,"completionDateStruct":196,"leadSponsor":198,"locationsCount":46},"100565884","phase-1-hematopoietic-stem-cell-bcl11a-enhancer-gene-editing-for-severe--hemoglobinopathies-100565884","NCT06647979","Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies","An Adaptive Design Basket Trial of Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies","Inclusion Criteria:\n\n1. Diagnosis of either a) sickle cell disease with genotype HbSS, HbS\u002FB0 thalassemia, HbSD, or HbSO, or b) transfusion-dependent β-thalassemia\n2. Age 13-40 years.\n3. Clinically severe disease, defined as: For sickle cell disease, the presence of one or more of the following clinical complications: i) Minimum of two episodes of acute chest syndrome (ACS) in the 2 years before study entry. ii) History of three or more episodes of severe pain events requiring a visit to a medical facility and treatment with parenteral opioids in the 2 years before study entry. For β-thalassemia patients: i) At least 100 mL\u002Fkg\u002Fyear or 10 units\u002Fyear of blood transfusions, on an annualized basis for the two years preceding enrollment.\n4. Adequate hematologic parameters including:\n\n   1. White blood cell (WBC) count within the range of 2.5 - 25.0 x 109 \u002FL\n   2. Platelet count within the range of 150 - 700 x 109 \u002FL\n5. Adequate organ function and performance status:\n\n   1. Karnofsky performance status ≥70%\n   2. Serum creatinine \\\u003C\u002F=1.5 times the upper limit of normal for age, and calculated creatinine clearance or GFR \\\u003C\u002F= 60 mL\u002Fmin\u002F1.73 m2.\n   3. Direct bilirubin ≤ 2.0 mg\u002FdL\n   4. DLCO (corrected for hemoglobin), FEV1, FVC \\>50% of predicted\n   5. Left ventricular ejection fraction \\>40% or shortening fraction \\>25%\n6. For sickle cell patients: Failure of hydroxyurea therapy due to lack of clinical improvement, inability to tolerate due to side effects (e.g., myelosuppression, gastrointestinal symptoms, or hepatic enzyme elevations) or not clinically indicated (such as in a patient on a chronic transfusion regimen). Clinical criteria (per above) must be met despite taking hydroxyurea for greater than or equal to 6 months, unless not indicated or not tolerated. Patients taking hydroxyurea who still meet all inclusion criteria are eligible for the trial. Hydroxyurea should be discontinued when transfusions prior to gene therapy begin.\n7. Confirmed sickle cell disease or β-thalassemia diagnosis by molecular genetic testing.\n8. No HLA genotypically-identical related appropriate bone marrow donor available.\n9. Parental\u002Fguardian\u002Fpatient signed informed consent.\n10. Willingness to return for follow-up for 15 years.\n\nExclusion Criteria:\n\n1. Subjects who have concomitant condition or illness including, but not limited to:\n\n   1. Uncontrolled infection, such as current febrile illness, infection requiring parenteral antibiotics, or systemic fungal infection.\n   2. Active malignancy.\n   3. Active complication of underlying hemoglobinopathy that would place the patient at unacceptable risk for participation, in the judgment of the Investigators.\n   4. Major surgery in the past 30 days.\n   5. Medical\u002Fpsychiatric illness\u002Fsocial situations that would limit compliance with study requirements as determined by the treating physician.\n2. Contraindication to administration of conditioning medication (busulfan).\n3. Subjects who have undergone allogeneic or autologous hematopoietic stem cell transplant previously.\n4. Either or both of the following findings on screening bone marrow aspirate\u002Fbiopsy: a) diagnosis of myelodysplastic syndrome (MDS) based on morphology and\u002For cytogenetics (based on WHO definitions) or b) pathogenic mutation in any gene on the Rapid Heme Panel (RHP), a next-generation targeted sequencing clinical assay for hematologic malignancy associated mutations.\n5. For SCD patients:\n\n   1. Severe cerebral vasculopathy (defined by occlusion or stenosis in the circle of Willis; or presence of Moyamoya disease)\n   2. Receiving a chronic transfusion regimen for primary or secondary stroke prophylaxis. (Note: patients with a history of abnormal transcranial Doppler (TCD) who have transitioned from transfusions to hydroxyurea for stroke prophylaxis are also not eligible for the study. Most recent TCD must be within one year of screening for patients up to 16 years old.)\n   3. History of overt stroke or any neurologic event lasting \\> 24 hours. (Note: patients with imaging evidence of silent stroke but not on a chronic transfusion regimen are not excluded.)\n6. Severe iron overload that is deemed to be grounds for exclusion based on the opinion of the Principal Investigator.\n7. Known positive HIV serology or HIV nucleic acid testing, or positive serology for HCV, HBV, or HTLV.\n8. Known acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on prior biopsy.\n9. Receipt of an investigational study drug or procedure within 90 days of study enrollment.\n10. Pregnancy, or breastfeeding in a postpartum female, or absence of adequate contraception for fertile subjects. Females of child-bearing potential must agree to use a medically acceptable method of birth control such as oral contraceptive, intrauterine device, barrier and spermicide, or contraceptive implant\u002Finjection from Screening through at least 6 months after drug product infusion. Male subjects must agree to use effective contraception (including condoms) from Screening through at least 6 months after drug product infusion.\n11. An assessment by the Investigators that the subject will not comply with the study procedures outlined in the study protocol, or that, as determined by the investigators and\u002For transplant physician, the subject has any other condition rendering the subject ineligible for HSCT or other study procedures.\n12. Patients carrying at least one cytosine (C) alternate allele at the SNP site rs114518452, chr2:210530659-210530659 (GRCh38\u002Fhg38), where guanine (G) is the reference allele.","13 Years",{"count":179,"type":21},10,[24],"A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly DNA) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new genetic material in order to fix or replace a diseased gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of Graft-Versus-Host Disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. The method used to fix or replace a diseased gene is called gene editing. A person's own cells are edited using a specialized biological medicine that has been formulated for use in human beings.\n\nFetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. Investigators have recently discovered a gene called BCL11A that is very important in the control of fetal hemoglobin expression. Increasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, and therefore potentially cure the condition.",[28,183,31,184],"Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)","Transfusion Dependent Beta-Thalassaemia",[108,186,187,188,189,190],"stem cell transplant","bone marrow transplant","autologous hematopoietic stem cell transplant","gene editing","hematopoietic stem cell transplant","2026-01-31",{"date":193,"type":38},"2026-02-03",{"date":195,"type":38},"2025-12-01",{"date":197,"type":21},"2030-12",{"name":199,"class":119},"Daniel Bauer",{"id":201,"slug":202,"hasResults":11,"nctId":203,"briefTitle":204,"officialTitle":205,"acronym":4,"eligibilityCriteria":206,"healthyVolunteers":11,"sex":16,"minAge":207,"maxAge":208,"enrollmentInfo":209,"targetDuration":4,"studyType":22,"phases":211,"briefSummary":212,"conditions":213,"keywords":214,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":217,"lastUpdatePostDateStruct":218,"startDateStruct":220,"completionDateStruct":222,"leadSponsor":224,"locationsCount":152},"100373538","phase-2-study-of-safety--pk-of-luspatercept-ace-536-in-pediatric-participants-with-beta--thalassemia-100373538","NCT04143724","Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia","A Phase 2a Study to Evaluate the Safety and Pharmacokinetics of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia","Inclusion Criteria\n\n* Participants must be 6 years to \\\u003C 18 years of age at the time of signing the informed consent form (ICF)\u002Finformed assent form (IAF).\n* Participants (and when applicable, parent\u002Flegal representative) must understand and voluntarily sign an ICF\u002FIAF prior to conducting any study-related assessments\u002Fprocedures.\n* Participants (and when applicable, parent\u002Flegal representative) is willing and able to adhere to the study visit schedule and other protocol requirements.\n* Participants must have documented diagnosis of β-thalassemia or Hemoglobin E\u002Fβ-thalassemia.\n* Transfusion dependence (TD):\n\n  a. TD participant i. Participant is regularly transfused, defined as: ≥ 4 RBC transfusion events in the 24 weeks prior to enrollment with no transfusion-free period ≥ 42 days during that period.\n\nNote: For the purpose of the study, transfusions administered over 2 or 3 consecutive days are considered as part of a single transfusion event. Participant must have a history of regular transfusions for at least 2 years.\n\nb. NTD participant (ex-US sites only) i. Participant must have received \\\u003C 4 RBC transfusion events in the 24 weeks prior to enrollment.\n\nii. Participant must not be on a regular transfusion program and must be RBC transfusion-free for at least 8 weeks prior to enrollment.\n\niii. Participant must have mean baseline hemoglobin ≤ 10 g\u002FdL, based on a minimum of 2 measurements ≥ 1 week apart within 4 weeks prior to enrollment; hemoglobin values within 21 days post- transfusion will be excluded.\n\n* Participants have Karnofsky (age ≥16 years) or Lansky (age \\\u003C 16 years) performance status score ≥ 50 at screening.\n* Female children of childbearing potential (FCCBP), individuals of childbearing potential (IOCBP), and male (as assigned at birth) participants that have reached puberty (and when applicable, parent\u002Flegal representative) must agree to undergo physician-approved reproductive education and discuss the side effects of the study therapy on reproduction.\n* Female children of childbearing potential, defined as females who have achieved menarche and\u002For breast development in Tanner Stage 2 or greater and have not undergone a hysterectomy or bilateral oophorectomy and individuals of childbearing potential (IOCBP) defined as a sexually mature woman who has achieved menarche at some point, has not undergone a hysterectomy or bilateral oophorectomy and has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months) must meet the following conditions below (Note: Secondary amenorrhea from any cause does not rule out childbearing potential):\n* Medically supervised serum pregnancy tests with a sensitivity of at least 25 mIU\u002FmL must be conducted in Female children of childbearing potential (FCCBP)\u002F individuals of childbearing potential (IOCBP), including those who commit to complete abstinence. Female children of childbearing potential\u002F individuals of childbearing potential (IOCBP) must have 2 negative pregnancy tests as verified by the Investigator prior to starting study therapy (one of these tests should be performed by central laboratory). Female children of childbearing potential\u002F individuals of childbearing potential (IOCBP) must agree to ongoing pregnancy testing during the course of the study at the End of Treatment (EOT) visit and at the 9-week Safety Follow-up visit.\n* Female participants must, as appropriate to age and at the discretion of the site Investigator, either commit to true abstinence\\* from heterosexual contact (which must be reviewed on a monthly basis) or agree to use, and be able to comply with, effective\\*\\* contraception without interruption, 28 days prior to starting IP, during the study therapy (including dose interruptions), and for 12 weeks (approximately 5 times the mean terminal t1\u002F2 of luspatercept based on multiple-dose PK data) after discontinuation of study therapy.\n* Male (as assigned at birth) participants, as appropriate to age and the discretion of the study physician:\n\n  * Must practice true abstinence\\* (which must be reviewed on a monthly basis) or agree to use a synthetic or latex condom during sexual contact with a pregnant female or a Female children of childbearing potential (FCCBP)\u002F IOCBP while participating in the study, during dose interruptions and for at least 12 weeks (approximately 5 times the mean terminal t1\u002F2 of luspatercept based on multiple-dose PK data) following IP discontinuation, even if he has undergone a successful vasectomy.\n\n    * True abstinence is acceptable when this is in line with the preferred and usual lifestyle of the participant. \\[Periodic abstinence (eg, calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.\\] \\*\\* Agreement to use highly effective methods of contraception that alone or in combination result in a failure rate of a Pearl index of less than 1% per year when used consistently and correctly throughout the course of the study. Such methods include: Combined (estrogen and progesterone\u002Fprogestin containing) hormonal contraception: Oral; Intravaginal; Transdermal; Progestogen\u002Fprogestin only hormonal contraception associated with inhibition of ovulation: Oral; Injectable hormonal contraception; Implantable hormonal contraception; Placement of an intrauterine device (IUD); Placement of an intrauterine hormone-releasing system (IUS); Bilateral tubal occlusion; Vasectomized partner; Sexual Abstinence.\n\nExclusion Criteria\n\n* Participant has a diagnosis of Hemoglobin S\u002Fβ-thalassemia or alpha (α)-thalassemia (eg, Hemoglobin H); β-thalassemia combined with α-thalassemia is allowed.\n* Participant has of active hepatitis C (HCV) infection, as demonstrated by a positive HCF-ribonucleic acid (RNS) test of sufficient sensitivity, or active infectious hepatitis B (as demonstrated by the presence of hepatitis B surface antigen (HBsAG) and\u002For hepatitis B virus (HBV)-deoxyribonucleic acid (DNA) positive, or known positive human immunodeficiency virus (HIV).\n\nNote: Participants receiving antiviral therapies should have 2 negative HCV-RNA tests 3 months apart before ICF\u002FIAF signature, ie, one test at the end of the antiviral therapy and the second test 3 months following the first test.\n\n* Participant has deep vein thrombosis (DVT), stroke, or other thromboembolic event(s) (except clogged indwelling catheter) requiring medical intervention ≤ 24 weeks prior to enrollment.\n* Participant has platelet count \\> 1000 x 109\u002FL.\n* Participant has treatment with another investigational drug or device ≤ 28 days prior to enrollment.\n* Participant has prior exposure to sotatercept (ACE-011) or luspatercept (ACE-536).\n* Participant underwent or is scheduled for HSCT or gene therapy.\n* Participant use of iron chelation therapy (ICT), if initiated ≤ 8 weeks prior to enrollment (allowed if initiated \\> 8 weeks before or during treatment).\n* Participant received treatment with hydroxyurea immunomodulatory drugs IMiDs (such as thalidomide), other fetal Hb (HbF) inducers or erythropoiesis-stimulating agents (ESAs) ≤ 12 weeks prior to enrollment for NTD participants and ≤ 24 weeks for TD participants.\n* Participant is pregnant or breastfeeding female or plan to get pregnant during the study.\n* Participant has uncontrolled hypertension. Controlled hypertension for this protocol is considered: blood pressure value corresponding to ≤ Grade 1 according to NCI CTCAE version 5.0 with or without pharmacological treatment.\n* Participant has major organ damage, including:\n\n  1. Symptomatic splenomegaly\n  2. Liver disease with alanine aminotransferase (ALT)\u002Faspartate aminotransferase (AST) \\> 3X the upper limit of normal (ULN) for age\n  3. Heart disease, heart failure as classified by the New York Heart Association (NYHA) classification 3 or higher, or significant arrhythmia requiring treatment, or recent myocardial infarction within 6 months of enrollment\n  4. Lung disease, including pulmonary fibrosis or pulmonary hypertension of Grade ≥ 3 according to NCI-CTCAE version 5.0.\n  5. Renal insufficiency defined as:\n* A serum creatinine based on age\u002Fgender based on threshold derived from Schwartz formula for estimating GFR utilizing child length and stature data published by the Centers for Disease Control.\n* Participant has proteinuria ≥ Grade 3 according to NCI CTCAE version 5.0 (which is equivalent to a urine protein\u002Fcreatinine ratio \\> 215 mg\u002Fmmol of creatinine), or a urine albumin\u002Fcreatinine ratio \\> 129 mg\u002Fmmol of creatinine.\n* Participant use of high dose long-term therapy with systemic glucocorticoids ≤ 12 weeks prior to enrollment (physiologic replacement therapy for adrenal insufficiency is allowed). Low-dose long-term (defined as ≤ 0.2 mg\u002Fkg\u002Fday or ≤ 10 mg\u002Fday of prednisone equivalent), short treatment (eg, for prevention or treatment of transfusion reactions) inhaled, intranasal and topical corticosteroids are allowed.\n* Participant has history of severe allergic or anaphylactic reactions or hypersensitivity to recombinant proteins or excipients in the IP (refer to the IB).\n* Participant use of cytotoxic agents, immunosuppressants ≤ 28 days prior to enrollment (ie, antithymocite globulin (ATG) or cyclosporine).\n* Participant has history of malignancy with the exception of:\n\n  1. Curatively resected nonmelanoma skin cancer.\n  2. Curatively treated carcinoma in situ.\n  3. Other solid tumor with no known active disease in the opinion of the Investigator.\n* Participant who has extramedullary hematopoiesis (EMH) complications or requires treatment to control the growth of EMH masse(s) during the screening period.\n* Participants with any medical or psychiatric condition that in the opinion of the investigator would put the participant at unacceptable risk of participating in the study or may impact interpretation of the study results.\n* Participants who use herbs or food supplements (eg: Chinese traditional medicine), if, per investigator's judgment, likely to impact the safety and efficacy assessment, for 24 weeks before initiating the study treatment for TD participants, and 12 weeks for NTD participants.","6 Years","17 Years",{"count":210,"type":21},99,[25],"This is a Phase 2a study to evaluate the safety and pharmacokinetics (PK) of luspatercept in pediatric participants with β-thalassemia.\n\nThe study will be conducted in 2 parts for both transfusion-dependent (TD) and non-transfusion-dependent (NTD) β-thalassemia participants: TD Part A will be in adolescent participants aged 12 to \\\u003C18 years with two dose escalation cohorts, followed by a dose expansion cohorts. NTD Part A will be conducted in the same age group participants as TD Part A with dose confirmation and expansion cohorts. After Part A TD participants have completed at least one year of treatment, all available safety data from Part A adolescent participants will be evaluated before initiating TD and NTD Part B in the age group from 6 to \\\u003C12 years old. Part B will consist of two dose escalation cohorts for TD and two dose escalation cohorts for NTD.\n\nUpon completion of the Treatment Period, participants of any cohort who are benefiting from the study treatment, will be offered the opportunity to continue luspatercept treatment in the Long-term Treatment Period for up to 5 years from their first dose.\n\nParticipants who discontinue study treatment at any time will continue in the Posttreatment Follow-up Period for at least 5 years from their first dose of luspatercept, or 3 years from their last dose, whichever occurs later, or until they withdraw consent\u002Fassent, are lost to follow-up, or the End of Trial, whichever occurs first.",[31],[78,79,215,31,216],"Pharmacokinetics","Red Blood Cell Transfusion","2026-01-02",{"date":219,"type":38},"2026-01-06",{"date":221,"type":38},"2019-11-07",{"date":223,"type":21},"2035-06-11",{"name":89,"class":90},{"id":226,"slug":227,"hasResults":11,"nctId":228,"briefTitle":229,"officialTitle":230,"acronym":4,"eligibilityCriteria":231,"healthyVolunteers":11,"sex":16,"minAge":232,"maxAge":208,"enrollmentInfo":233,"targetDuration":4,"studyType":22,"phases":235,"briefSummary":237,"conditions":238,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":239,"lastUpdatePostDateStruct":240,"startDateStruct":242,"completionDateStruct":244,"leadSponsor":246,"locationsCount":46},"100552940","a-long-term-follow-up-study-in-participants-who-received-cs-101-100552940","NCT06479616","A Long-term Follow-up Study in Participants Who Received CS-101","A Long-term Follow-up Study Evaluating the Safety and Efficacy of Subjects With β-thalassemia Treated With Base-edited Autologous Hematopoietic Stem Cell (CS-101) Transplantation","Inclusion Criteria:\n\n* Participants (or his or her legally appointed and authorized representative or guardian) must sign and date informed consent form (ICF) and, where applicable, an assent form\n* Participants must have received CS-101 infusion in last IIT study\n\nExclusion Criteria:\n\n* There are no exclusion criteria","3 Years",{"count":234,"type":21},5,[236],"NA","This is a study to evaluate the long-term safety and efficacy of CS-101 in participants who received CS-101 in study CS -101-03 (NCT06065189)",[31],"2025-09-04",{"date":241,"type":38},"2025-09-10",{"date":243,"type":38},"2024-06-18",{"date":245,"type":21},"2027-06",{"name":247,"class":119},"Children's Hospital of Fudan University",{"id":249,"slug":250,"hasResults":11,"nctId":251,"briefTitle":252,"officialTitle":253,"acronym":254,"eligibilityCriteria":255,"healthyVolunteers":11,"sex":16,"minAge":68,"maxAge":256,"enrollmentInfo":257,"targetDuration":4,"studyType":22,"phases":259,"briefSummary":260,"conditions":261,"keywords":262,"overallStatus":133,"whyStopped":4,"lastUpdateSubmitDate":265,"lastUpdatePostDateStruct":266,"startDateStruct":268,"completionDateStruct":270,"leadSponsor":272,"locationsCount":46},"100605069","phase-3-evaluating-the-effect-of-n-acetyl-cysteine-and-alpha-lipoic-acid-in-patients-with-beta-thalassemia-100605069","NCT07157722","Evaluating the Effect of N-Acetyl Cysteine and Alpha Lipoic Acid in Patients With Beta Thalassemia","The Potential Role of N-Acetyl Cysteine or Alpha-Lipoic Acid as Adjuvant Therapies in the Treatment of Patients With Beta Thalassemia","NAC\u002FALA","Inclusion Criteria:\n\n* Patients with beta-thalassemia who will receive conventional thalassemia management.\n* Both genders.\n* Age ≥ 18 years old.\n\nExclusion Criteria:\n\n* Patients with familial hypercholesterolemia or history of premature atherosclerosis.\n* Patients with a prior history of significant cardiovascular diseases, such as coronary artery disease, myocardial infarction, or stroke.\n* Patients with severe renal dysfunction.\n* Patients with severe hepatic dysfunction.\n* Patients with diabetes.\n* Patients who will be non-compliant with the prescribed therapy.\n* Patients with other hemoglobinopathies.\n* Pregnant women.\n* Obese patients.\n* Patients who will receive antioxidant or anti-inflammatory medications.\n* Patients with inflammatory diseases, such as Systemic lupus erythematous, rheumatoid arthritis and inflammatory bowel disease.\n* Patients with oxidative stress related diseases, such as Alzheimer, Parkinson, COPD and cancer.","70 Years",{"count":258,"type":21},66,[72],"The current study is to investigate the potential roles of N-acetyl cysteine and Alpha-lipoic acid in patients with beta-thalassemia.",[107],[263,264],"NAC","ALA","2025-08-28",{"date":267,"type":38},"2025-09-05",{"date":269,"type":21},"2025-08-30",{"date":271,"type":21},"2026-01-30",{"name":273,"class":119},"Tanta University",{"id":275,"slug":276,"hasResults":11,"nctId":277,"briefTitle":278,"officialTitle":279,"acronym":4,"eligibilityCriteria":280,"healthyVolunteers":11,"sex":16,"minAge":281,"maxAge":282,"enrollmentInfo":283,"targetDuration":4,"studyType":22,"phases":285,"briefSummary":286,"conditions":287,"keywords":295,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":299,"lastUpdatePostDateStruct":300,"startDateStruct":302,"completionDateStruct":304,"leadSponsor":305,"locationsCount":46},"100370178","phase-2-allosct-for-malignant-and-non-malignant-hematologic-diseases-utilizing-alphabeta-t-cell-and-cd19-b-cell-depletion-100370178","NCT04099966","AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion","Allogeneic Stem Cell Transplantation for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha\u002FBeta T Cell and CD19+ B Cell Depletion - NYMC 588","Inclusion Criteria:\n\n1. ALL:ALL high risk including one or more of the following: (t(9;22) or 11q23 chromosomal abnormality, primary induction failure (\\\u003C15% blasts at time of registration), mixed phenotype acute leukemia (MPAL), persistent MRD (\\\u003C0.01% by flow or persistent abnormal karyotype detected by cytogenetics) or hypodiploidy (44 chromosomes)) in first remission ' ALL in second remission and beyond;\n2. AML: History of AML induction\u002Freinduction Failure (\\\u003C15% blasts at time of registration); AML in CR1 with poor cytogenetics (i.e. 12p, 5a, -7, FLT3 mutation\u002Fduplication, t(9;11) and others); AML with persistent minimal residual disease (MRD) in CR1(\\\u003C0.01% on flow or persistent abnormal karyotype detected by cytogenetics); AML CR2 or beyond; AML in refractory relapse but ≤15% bone marrow leukemia blasts; Therapy-related AML\n3. High Risk Myelodysplastic syndrome (MDS) 4 Lymphoma: Hodgkin (HL) or Non-Hodgkin (NHL): HL or NHL in induction failure; HL or NHL in PR1 or PR2 ; HL or NHL in CR2 or subsequent remission\n\n5\\. Bone marrow failure syndromes: Kostmann syndrome refractory or intolerant to granulocyte colony-33stimulating factor; Diamond-Blackfan anemia refractory or intolerant to corticosteroids and\u002For cyclosporine'; amegakaryocytic thrombocytopenia 6. Sickle Cell Disease (Homozygous Hemoglobin S Disease, or Hemoglobin S β 0\u002F+ thalassemia, or Hemoglobin SC Disease) 7. age 0-30 years 8. adequate organ function\n\nExclusion Criteria:\n\n1. Females who are pregnant or breast-feeding are not eligible.\n2. Patients with documented uncontrolled infection at the time of study entry are not eligible.\n3. Karnofsky\u002FLansky (age appropriate) Performance Score \\\u003C60\n4. Demonstrated lack of compliance with medical care\n5. Patients who have received allogeneic HSCT within 6 months, unless being done as a boost.\n6. Patients with active \\\u003CGrade 2 GVHD.","1 Day","30 Years",{"count":284,"type":21},20,[25],"Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α\u002Fβ CD3+\u002FCD19+ cell depletion. All other treatment is standard of care.",[288,289,290,291,292,293,294,28,31],"Acute Leukemia","Severe Aplastic Anemia","Non-hodgkin Lymphoma","Hodgkin Lymphoma","Kostmann","Diamond Blackfan Anemia","Amegakaryocytic Thrombocytopenia",[296,297,298],"allogeneic stem cell transplantation","t-cell depletion","alpha beta cell depletion","2025-08-07",{"date":301,"type":38},"2025-08-08",{"date":303,"type":38},"2021-04-01",{"date":116,"type":21},{"name":306,"class":119},"Mitchell Cairo",{"id":308,"slug":309,"hasResults":11,"nctId":310,"briefTitle":311,"officialTitle":312,"acronym":4,"eligibilityCriteria":313,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":314,"targetDuration":316,"studyType":128,"phases":4,"briefSummary":317,"conditions":318,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":319,"lastUpdatePostDateStruct":320,"startDateStruct":322,"completionDateStruct":324,"leadSponsor":326,"locationsCount":169},"100536952","a-study-of-participants-with--thalassemia-treated-with-betibeglogene-autotemcel-100536952","NCT06271512","A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel","A Safety and Effectiveness Registry Study of Patients with Β-Thalassemia Treated with Betibeglogene Autotemcel (the Glostar Registry)","Inclusion Criteria:\n\n* Participant must be treated with beti-cel in the post marketing setting at a center in the US that participates in the Registry.\n* Participant must sign an informed consent and\u002For assent prior to enrollment as required under applicable laws and regulations.\n* Participant must have signed an informed consent and\u002For assent permitting data to be shared with Center for International Blood and Marrow Transplant Research (CIBMTR).\n* Participant must be followed by a hematologist based in the US.\n\nExclusion Criteria:\n\n\\- There are no exclusion criteria for Registry participation.",{"count":315,"type":21},150,"15 Years","The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.",[31],"2025-02-10",{"date":321,"type":38},"2025-02-11",{"date":323,"type":38},"2024-01-23",{"date":325,"type":21},"2043-12",{"name":327,"class":90},"Genetix Biotherapeutics Inc.",{"id":329,"slug":330,"hasResults":11,"nctId":331,"briefTitle":332,"officialTitle":333,"acronym":4,"eligibilityCriteria":334,"healthyVolunteers":11,"sex":16,"minAge":68,"maxAge":18,"enrollmentInfo":335,"targetDuration":4,"studyType":22,"phases":337,"briefSummary":338,"conditions":339,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":340,"lastUpdatePostDateStruct":341,"startDateStruct":343,"completionDateStruct":345,"leadSponsor":347,"locationsCount":349},"100575475","phase-1-a-multiple-ascending-dose-study-of-9mw3011-in-patients-with-non-transfusion-dependent--thalassemia-100575475","NCT06772766","A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia","A Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Non-transfusion-dependent β- Thalassemia","Key Inclusion Criteria:\n\n1. Male and female subjects aged 18 to 65 years (inclusive)\n2. Subject must have a documented genetic diagnosis of β-thalassemia or hemoglobin E\u002F β-thalassemia\n3. Subjects must meet the criteria for non-transfusion-dependent thalassemia\n4. Subjects must have a baseline hemoglobin level between 70-100 g\u002FL(inclusive), based on 2 consecutive measurements taken at least 1 week apart within 4 weeks before randomization\n5. Subjects must have evidence of iron overload during screening\n6. Subject must have performance status: Eastern Cooperative Oncology Group (ECOG) performance score of 0 to 1\n7. Subjects must fully understand the study procedures and methods, voluntarily participate in the trial, and sign an informed consent form\n\nKey Exclusion Criteria:\n\n1. Subjects diagnosed with alpha-thalassemia\n2. Subjects diagnosed with HbS\u002Fbeta-thalassemia or transfusion-dependent beta-thalassemia\n3. Subjects exhibit severe iron overload at the time of screening\n4. In addition to thalassemia, subjects have any other forms of anemia and hematological disorders that the investigator assesses may compromise safety or influence study outcomes\n5. Combined with any significant systemic diseases or psychiatric disorders\n6. Subjects have New York Heart Association (NYHA) Class III-IV heart failure and other cardiovascular diseases within 6 months prior to screening or currently present\n7. During the screening or baseline period, subjects exhibiting a QTcF interval of ≥450ms for males and ≥470ms for females on a 12-lead electrocardiogram (ECG), or presenting an abnormal 12-lead ECG with clinical significance\n8. Uncontrolled hypertension before screening\n9. A history of malignant neoplasm occurring within the last five years\n10. Severe infection requiring hospitalization or intravenous antimicrobial therapy, or uncontrolled systemic bacterial, fungal, or viral active infection\n11. Subject have received concomitant treatment that was not permitted by the protocol\n12. Subjects whose hematological parameters did not meet the inclusion criteria during screening\n13. Subjects with a history of substance abuse, as well as those who yield positive results on substance abuse screening\n14. Subjects who are unable to undergo MRI scans\n15. Pregnant or lactating women\n16. Subjects presenting any other factors deemed unsuitable for participation assessed by the investigator",{"count":336,"type":21},40,[24],"This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and immunogenicity of 9MW3011 in patients with non-transfusion-dependent β- thalassemia .",[31],"2025-01-09",{"date":342,"type":38},"2025-01-14",{"date":344,"type":38},"2024-12-30",{"date":346,"type":21},"2026-10",{"name":348,"class":90},"Mabwell (Shanghai) Bioscience Co., Ltd.",2,{"id":351,"slug":352,"hasResults":11,"nctId":353,"briefTitle":354,"officialTitle":354,"acronym":4,"eligibilityCriteria":355,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":356,"targetDuration":4,"studyType":128,"phases":4,"briefSummary":358,"conditions":359,"keywords":375,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":377,"lastUpdatePostDateStruct":378,"startDateStruct":380,"completionDateStruct":382,"leadSponsor":384,"locationsCount":46},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":357,"type":21},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[360,31,361,362,363,364,365,366,367,368,369,370,371,372,28,373,374],"Alpha-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Pompe Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[376],"rare diseases","2024-11-12",{"date":379,"type":38},"2024-11-14",{"date":381,"type":38},"2024-06-10",{"date":383,"type":21},"2026-06-10",{"name":385,"class":90},"xCures",{"id":387,"slug":388,"hasResults":11,"nctId":389,"briefTitle":390,"officialTitle":391,"acronym":4,"eligibilityCriteria":392,"healthyVolunteers":11,"sex":16,"minAge":393,"maxAge":4,"enrollmentInfo":394,"targetDuration":4,"studyType":128,"phases":4,"briefSummary":396,"conditions":397,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":399,"lastUpdatePostDateStruct":400,"startDateStruct":402,"completionDateStruct":404,"leadSponsor":406,"locationsCount":349},"100521766","a-post-marketing-surveillance-study-to-assess-safety-of-luspatercept-in-korean-patients-with-myelodysplastic-syndrome-or--thalassemia-100521766","NCT06073860","A Post-Marketing Surveillance Study to Assess Safety of Luspatercept in Korean Patients With Myelodysplastic Syndrome or β-thalassemia","Reblozyl® (Luspatercept) Post-Marketing Surveillance in Korean Patients With Myelodysplastic Syndrome or Beta Thalassemia","Inclusion Criteria:\n\n* Adult participants 19 years of age or older\n* Participants who will be treated with luspatercept according to the approved label in the Republic of Korea\n* Participants who sign the informed consent form\n\nExclusion Criteria:\n\n* Participants who are prescribed luspatercept for therapeutic indications not approved in the Republic of Korea\n* Participants for whom luspatercept is contraindicated as clarified in the Korean prescribing information approved by the Ministry of Food and Drug Safety","19 Years",{"count":395,"type":21},104,"The purpose of this observational study is to assess the real-world safety of luspatercept in Korean participants with myelodysplastic syndrome (MDS) or beta thalassemia. Investigators will enroll participants who will begin treatment with at least 1 dose of luspatercept.",[398,107],"Myelodysplastic Syndrome","2024-11-05",{"date":401,"type":38},"2024-11-06",{"date":403,"type":38},"2024-03-25",{"date":405,"type":21},"2027-12-21",{"name":407,"class":90},"Bristol-Myers Squibb",{"id":409,"slug":410,"hasResults":11,"nctId":411,"briefTitle":412,"officialTitle":412,"acronym":4,"eligibilityCriteria":413,"healthyVolunteers":11,"sex":16,"minAge":414,"maxAge":68,"enrollmentInfo":415,"targetDuration":4,"studyType":22,"phases":417,"briefSummary":418,"conditions":419,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":420,"lastUpdatePostDateStruct":421,"startDateStruct":423,"completionDateStruct":425,"leadSponsor":427,"locationsCount":46},"100559806","adherence-of-beta-thalssemia-patients-to-oral-chelation-therapy-100559806","NCT06568926","Adherence of Beta Thalssemia Patients to Oral Chelation Therapy","Inclusion Criteria:\n\n1. confirmed diagnosis of beta thalassemia major or intermedia,\n2. Age between 2-18 years,\n3. Receiving regular blood transfusions every 2-5 weeks\n4. Prescribed oral iron chelation therapy with deferasirox for at least 1 year prior to enrollment.\n\nExclusion Criteria:\n\n1. Age less than 2 years and more than 18 years\n2. Any cause of blood transfusion other than beta Thalassemia\n3. Patients on deferoxamine","2 Years",{"count":416,"type":21},200,[236],"The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity. The degree of anemia relies on several genetic and environmental factors and determines the need for regular transfusion therapy. It is now common practice to classify patients as having transfusion dependent β-thalassemia (TDT) or non-transfusion-dependent β-thalassemia (NTDT). Regarding geographical distribution of β-thalassemia, it prevails more in countries on the Mediterranean, South east of Asia and in the east of Europe. In Egypt, it is the most common cause of chronic blood loss: One thousand cases are recorded annually for every 1.5 million live births the disease prevalence is equal to1000 cases per 1.5 million live births (Ghazala et al., 2021).\n\nThe only curative treatment for thalassemia currently is a bone marrow transplant. However, it is restricted to only a few patients due to the non-availability of an HLA-matched donor and high cost. Thus, most patients receive regular blood transfusions accompanied by iron chelation therapy (ICT) as the standard of care. The ideal management of a patient with transfusion-dependent thalassemia (TDT) requires a multidisciplinary therapeutic approach. The main iron chelating agents include deferoxamine (DFO), deferiprone (DFP), and deferasirox (DFX). Due to poor oral bioavailibility, DFO is the only chelator that must be administered subcutaneously or intravenously up to once a day; DFP and DFX may be administered orally up to three times a day. The known side effects associated with each chelator include infusion reactions in DFO, gastrointestinal distress, agranulocytosis in DFP, and transaminitis in DFP and DFX.",[31],"2024-08-21",{"date":422,"type":38},"2024-08-23",{"date":424,"type":38},"2024-07-14",{"date":426,"type":21},"2025-07-14",{"name":428,"class":119},"Sohag University",{"id":430,"slug":431,"hasResults":11,"nctId":432,"briefTitle":433,"officialTitle":433,"acronym":434,"eligibilityCriteria":435,"healthyVolunteers":436,"sex":16,"minAge":68,"maxAge":4,"enrollmentInfo":437,"targetDuration":4,"studyType":128,"phases":4,"briefSummary":439,"conditions":440,"keywords":446,"overallStatus":133,"whyStopped":4,"lastUpdateSubmitDate":455,"lastUpdatePostDateStruct":456,"startDateStruct":458,"completionDateStruct":460,"leadSponsor":462,"locationsCount":46},"100555454","plasticizer-exposure-and-its-consequences-on-health-100555454","NCT06512298","Plasticizer Exposure and Its Consequences on Health","PEACH","TDTM:\n\n* Inclusion: Adult, homozygous ß-Thalassemia Major, transfusion dependency.\n* Exclusion: Plastic implants, chronic infectious disease, pregnancy.\n\nThalassemia Intermedia\u002FMinor:\n\n* Inclusion: Adult, homozygous\u002Fheterozygous ß-Thalassemia Intermedia\u002FMinor\n* Exclusion: Plastic implants, chronic infectious disease, pregnancy, transfusion dependency\n\nHealthy adults:\n\n* Inclusion: Adult\n* Exclusion: Chronic disease, plastic implants, infectious disease, pregnancy, anemia, medication-, drug-, or alcohol-abuse\n\nGlioma patients:\n\n* Inclusion: Adult, high\u002Flow-grade glioma, tumor larger then 3cm, resection with access to the ventricular system\n* Exclusion: Large intraventricular hemorrhage, plastic implants, infectious disease\n\nICU patients:\n\n* Inclusion: Adult, brain hemorrhage, external CSF drain from ventricle\n* Exclusion: intraventricular hemorrhage, plastic implants, ECMO, hemodialysis, infectious disease\n\nPatients undergoing diagnostic lumbar-puncture:\n\n* Inclusion: Adult, diagnostic CSF sampling, outpatient setting\n* Exclusion: Ventricular hemorrhage, plastic implants, infectious disease",true,{"count":438,"type":21},120,"Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects on human health. Evidence suggests that these substances might disrupt various biological functions such as the immune system, the balance of gut bacteria, hormone regulation, and brain processes. While some studies have linked plasticizer exposure to health issues, definitive data from human studies are still lacking.\n\nThe PEACH study aims to bridge these knowledge gaps by investigating how plasticizers affect human health. The study focuses on understanding how these chemicals are absorbed, distributed, and accumulated in the body across different groups of patients. The investigators are particularly interested in how plasticizers influence gut microbiota and the functionality of immune cells, as well as their effects on neurotransmitters involved in brain function.\n\nA combination of patient data, systems biology, and laboratory models will be used to thoroughly assess the biological impacts of plasticizers. Advanced techniques such as mass spectrometry will aid in studying toxicokinetic properties, sequencing technologies will be used to examine immune effects, and radiouptake assays will be employed to explore interactions with neurotransmitter transport. This comprehensive methodology will provide new insights into the effects of both short-term and long-term exposure to plasticizers.\n\nThe PEACH study introduces innovative methods to the field, aiming to create a robust model for understanding how plasticizer compounds behave in the human body. It employs state-of-the-art techniques to assess the dynamics of these chemicals, marking a significant advancement in environmental health research.",[29,441,442,443,444,445],"Glioma","Healthy Controls","Transfusion-dependent Beta-Thalassemia","Intracranial Hemorrhages","Toxicity",[447,448,449,450,451,452,215,453,454],"Plasticizer","Transfusion","Systems Biology","Immunology","Microbiome","Neuropharmacology","Pharmacodynamics","Exposomics","2024-07-22",{"date":457,"type":38},"2024-07-24",{"date":459,"type":21},"2024-10-01",{"date":461,"type":21},"2027-09-30",{"name":463,"class":119},"Medical University of Vienna"]