[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"paroxysmal-nocturnal-hemoglobinuria-pnh\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:paroxysmal-nocturnal-hemoglobinuria-pnh":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,15,0,[8,45,79,106,134,158,192,218,248,286,309,331,352,391,411],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100325717","phase-2-haplo-identical-transplantation-for-severe-aplastic-anemia-hypo-plastic-mds-and-pnh-using-peripheral-blood-stem-cells-and-post-transplant-cyclophosphamide-for-gvhd-prophylaxis-100325717",false,"NCT03520647","Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis","* INCLUSION CRITERIA - RECIPIENT:\n* Diagnosed with severe aplastic anemia with bone marrow cellularity \\\u003C30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and\u002For neutropenia (absolute neutrophil count less than or equal to 1000 cells\u002F microL or for patients receiving granulocyte transfusions, absolute neutrophil count less than or equal to 1000 cells\u002FmicroL before beginning granulocyte transfusions).\n\nOR\n\n--History of severe aplastic anemia transformed to MDS. that meet the following criteria: a) International Prognostic Scoring System (IPSS) risk category of INT-1 or greater, b) \\\u003C5% myeloblasts and \\\u003C30% of cellularity in the bone marrow on screening morphologic analysis.\n\nOR\n\n* PNH that is either refractory to treatment with eculizumab\u002Fravulizumab or occurs in patients who don t have access to treatment with eculizumab associated with either a) life- threatening thrombosis and\u002For b) cytopenia associated with transfusion dependence and\u002For c) recurrent and debilitating hemolytic crisis.\n\n  * Subjects with severe aplastic anemia, hypoplastic MDS or PNH with associated bone marrow failure syndromes who have intolerance of or failure to respond to immunosuppressive therapy. This also includes patients who have failed immunosuppressive therapy with ATG and cyclosporine or therapy with cyclosporine combined with eltrombopag in those who are intolerant of or do not have access to treatment with ATG.\n  * Availability of at least one HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) to serve as a stem cell donor for the allogeneic transplant.\n  * Availability of a backup stem cell source in the event of graft rejection:\n* at least one additional haploidentical related alternative donor (i.e. HLA- haploidentical related donor (i.e. \\>= 5\u002F10 HLA match: HLA-A, B, C, DR, and DQ loci) or \\>= 9\u002F10 HLA matched unrelated donor who is available to serve as a stem cell donor for a salvage allogeneic transplant in the event that the haplo-transplant has been rejected.\n* umbilical cord blood unit\u002Fs that can be used for a salvage cord blood transplant in the event that the haplo-transplant has been rejected\n\n  * The patient does not have any HLA antibodies detectable against any of the mismatched HLA alleles expressed by the haplo-donor.\n  * Ages 4-60 years inclusive.\n  * Ability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to subjects aged 4-17 years with formal consent being obtained from parents or legal guardian.\n\nEXCLUSION CRITERIA - RECIPIENT (ANY OF THE FOLLOWING):\n\n* Availability of an HLA identical (12\u002F12) matched related or unrelated donor who is available within optimal timeline and suitable considering graft source and established donor selection factors (e.g. age, sex, viral exposure, ABO compatibility, pregnancy status, etc) per PI discretion.\n* The patient is deemed to be a candidate for a 12\u002F12 HLA matched unrelated stem cell transplant (availability of a donor and resources required for such a transplant).\n* ECOG performance status of 2 or more.\n* Major anticipated illness or organ failure incompatible with survival from transplant.\n* Current pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control or practice abstinence to refrain from pregnancy, if of childbearing potential for one year.\n* HIV positive.\n* Diagnosis of Fanconi s anemia (by chromosome breakage study).\n* Diffusion capacity of carbon monoxide (DLCO) \\\u003C40% using DLCO corrected for Hgb or lung volumes (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO assessed).\n* Left ventricular ejection fraction \\\u003C40% (evaluated by ECHO)\n* Transaminases \\> 5x upper limit of normal.\n* Direct bilirubin \\>3 mg\u002Fdl.\n* Creatinine clearance \\\u003C 50 cc\u002Fmin\u002FBSAm\\^2 by 24-hour urine collection adjusted by body surface area.\n* Serum creatinine \\> 2.5 mg\u002Fdl\n* Presence of an active infection not adequately responding to appropriate therapy.\n* History of a malignant disease liable to relapse or progress within 5 years.\n\nINCLUSION CRITERIA - DONOR:\n\nDonor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood for research. Donors will be evaluated in accordance with existing Standard NIH Policies and Procedures for determination of eligibility and suitability for clinical donation. Note that participation in this study is offered to all 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 - RELATED DONOR:\n\nNone","ALL","4 Years","75 Years",{"count":19,"type":20},56,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","Background:\n\nSevere aplastic anemia (SAA), and myelodysplastic syndrome (MDS), and paroxysmal nocturnal hemoglobinuria\n\n(PNH) cause serious blood problems. Stem cell transplants using bone marrow or blood plus chemotherapy can help. Researchers want to see if using peripheral blood stem cells (PBSCs) rather than bone marrow cells works too. PBSCs are easier to collect and have more cells that help transplants.\n\nObjectives:\n\nTo see how safely and effectively SAA, MDS and PNH are treated using peripheral blood hematopoietic stem cells from a family member plus chemotherapy.\n\nEligibility:\n\nRecipients ages 4-60 with SAA, MDS or PNH and their relative donors ages 4-75\n\nDesign:\n\nRecipients will have:\n\n* Blood, urine, heart, and lung tests\n* Scans\n* Bone marrow sample\n\nRecipients will need a caregiver for several months. They may make fertility plans and a power of attorney.\n\nDonors will have blood and tissue tests, then injections to boost stem cells for 5-7 days.\n\nDonors will have blood collected from a tube in an arm or leg vein. A machine will separate stem cells and maybe white blood cells. The rest of the blood will be returned into the other arm or leg.\n\nIn the hospital for about 1 month, recipients will have:\n\n* Central line inserted in the neck or chest\n* Medicines for side effects\n* Chemotherapy over 8 days and radiation 1 time\n* Stem cell transplant over 4 hours\n\nUp to 6 months after transplant, recipients will stay near NIH for weekly physical exams and blood tests.\n\nAt day 180, recipients will go home. They will have tests at their doctor s office and NIH several times over 5 years.",[26,27,28],"Severe Aplastic Anemia (SAA)","Hypo-Plastic Myelodysplastic Syndrome (MDS)","Paroxysmal Nocturnal Hemoglobinuria (PNH)",[30,31],"Haploidentical CD34+cells","Non-Myeloablative","RECRUITING","2026-07-01",{"date":35,"type":36},"2026-07-02","ACTUAL",{"date":38,"type":36},"2019-02-19",{"date":40,"type":20},"2028-06-01",{"name":42,"class":43},"National Heart, Lung, and Blood Institute (NHLBI)","NIH",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":15,"minAge":52,"maxAge":53,"enrollmentInfo":54,"targetDuration":4,"studyType":21,"phases":56,"briefSummary":57,"conditions":58,"keywords":60,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":55},"100431732","phase-2-a-study-of-pegcetacoplan-in-pediatric-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100431732","NCT04901936","A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","An Open Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria","Inclusion Criteria:\n\n* Are 12-17 years old at the time of screening\n* Weigh at least 20 kg (approx. 44 lbs)\n* Have the diagnosis of PNH, confirmed by high-sensitivity flow cytometry (granulocyte or monocyte clone \\>10%)\n* EITHER:\n\n  * Not being treated with an approved complement inhibitor (eculizumab or ravulizumab) prior to start of pegcetacoplan dosing, AND have hemolytic anemia. Hemolytic anemia is defined as hemoglobin (Hb) less than the lower limit of normal (Hb \\\u003C LLN) and LDH \\>1.5 times the upper limit of normal (ULN); OR\n  * Currently receiving treatment with an approved complement inhibitor (eculizumab or ravulizumab) AND have evidence of ongoing anemia. Ongoing anemia is defined as Hb \\\u003C LLN and ARC \\> ULN\n* Have a platelet count \\>75,000\u002Fmm3 and an absolute neutrophil count \\>1000\u002Fmm3\n\nExclusion Criteria:\n\n* Are an adult, 18 years of age or older, with PNH\n* Known or suspected hereditary fructose intolerance (HFI)\n* History of hereditary complement deficiency, bone marrow transplant, or meningococcal disease (meningitis, bacteremia or septicemia)\n* Females who are pregnant or breastfeeding","12 Years","17 Years",{"count":55,"type":20},12,[23],"The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal nocturnal hemoglobinuria (PNH).",[28,59],"Paroxysmal Hemoglobinuria",[61,62,63,64,65,66,67,68],"Paroxysmal Nocturnal Hemoglobinuria","PNH","Pediatric","Adolescent","Apellis","Anemia, Hemolytic","Pegcetacoplan","APL-2","2026-06-23",{"date":71,"type":36},"2026-06-26",{"date":73,"type":36},"2021-02-04",{"date":75,"type":20},"2028-12",{"name":77,"class":78},"Apellis Pharmaceuticals, Inc.","INDUSTRY",{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":84,"acronym":85,"eligibilityCriteria":86,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":4,"enrollmentInfo":88,"targetDuration":4,"studyType":90,"phases":4,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":97,"lastUpdatePostDateStruct":98,"startDateStruct":100,"completionDateStruct":102,"leadSponsor":104,"locationsCount":55},"100610565","real-care-real-world-effectiveness-of-iptacopan-in-italian-patients-with-paroxysmal-nocturnal-hemoglobinuria-100610565","NCT07229235","REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria","REAL-CARE: Real-world Effectiveness of iptacopAn in itaLian Patients With Paroxysmal noCturnAl HemoglobinuRia: an Observational Study","REAL-CARE","Inclusion criteria:\n\n1. signed informed consent: patient must provide written informed before any study assessment is collected; in case of deceased patients, informed consent is not required as per Art. 110, comma 1 of Legislative Decree n° 196, dated 30 June 2003 as amended by D.lgs. 101\u002F2018 and the \"Autorizzazione generale al trattamento dei dati personali effettuato per scopi di ricerca scientifica\" n° 9\u002F2016\n2. Male and female,\n3. ≥ 18 years of age,\n4. documented diagnosis of PNH,\n5. followed in Italian clinical sites,\n6. on treatment with iptacopan prescribed as per routine medical care (i.e. patients naïve to treatment for whom the decision to start this treatment has already been made on the basis of clinical practice and according to SmPC and AIFA criteria and regardless of inclusion in this study or patients already treated under the Managed Access Program (MAP))\n\nExclusion criteria:\n\n1.Concomitant participation in an interventional clinical study related to PNH or its treatment.\n\nNo other inclusion\u002Fexclusion criteria apply other than the requirements stated in the Summary of Product Characteristics (SmPC).","18 Years",{"count":89,"type":20},50,"OBSERVATIONAL","This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.\n\nThe primary objective of the REAL-CARE study is to is to assess the long-term hematological response following iptacopan initiation. This will be assessed through the absolute change in hemoglobin (Hb) levels at 12 months post-initiation, and the proportion of patients who remain free from red blood cell (RBC) transfusions, prescribed as per local requirement and based on Investigator's judgment, from Day 14 through Month 12 after starting iptacopan.",[28],[85,94,95,62,96],"ptacopan","paroxysmal nocturnal hemoglobinuria","NIS","2026-06-22",{"date":99,"type":36},"2026-06-25",{"date":101,"type":36},"2026-01-13",{"date":103,"type":20},"2028-08-31",{"name":105,"class":78},"Novartis Pharmaceuticals",{"id":107,"slug":108,"hasResults":11,"nctId":109,"briefTitle":110,"officialTitle":111,"acronym":4,"eligibilityCriteria":112,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":4,"enrollmentInfo":113,"targetDuration":4,"studyType":21,"phases":115,"briefSummary":117,"conditions":118,"keywords":120,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":133},"100607349","phase-1-a-first-in-human-safety-and-efficacy-study-of-aln-cfb-a-small-interfering-rna-sirna-targeting-complement-factor-b-in-adult-participants-with-paroxysmal-nocturnal-hemoglobinuria-with-persistent-anemia-on-a-c5-inhibitor-100607349","NCT07187401","A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor","A Randomized, Double-Blind, Placebo-Controlled, First-In-Human Study of the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of ALN-CFB, an Investigational siRNA Therapeutic Against Complement Factor B, in Participants With Paroxysmal Nocturnal Hemoglobinuria and Persistent Anemia on Approved C5-Inhibitor Therapy","Key Inclusion Criteria:\n\n1. Has been diagnosed with PNH confirmed by a history of high flow cytometry from prior testing\n2. Treated with a stable dose of C5 inhibitor (eculizumab or approved eculizumab biosimilar, ravulizumab, or crovalimab) for at least 24 weeks prior to screening visit, as described in the protocol\n3. Has hemoglobin ≤10.5 g\u002FdL at screening visit 1, with evidence of anemia prior to this visit, as described in the protocol\n4. Has peripheral blood reticulocyte count of ≥100 x 10\\^9\u002FL at screening visit 1\n\nKey Exclusion Criteria:\n\n1. Has history of bone marrow transplantation or receipt of an organ transplant\n2. Has history of meningococcal infection or similar recurrent infections by other encapsulated bacterial organisms\n3. Has any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period\n4. Has laboratory evidence of bone marrow failure, as described in the protocol\n5. Have recent, unstable medical conditions, not related to PNH or PNH-related complications, as described in the protocol\n\nNOTE: Other Protocol Defined Inclusion \u002F Exclusion Criteria Apply",{"count":114,"type":20},24,[116,23],"PHASE1","This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor (\"C5-inhibitor\") and continue to have anemia (low red blood cell count).\n\nThe aim of the study is to see how tolerable ALN-CFB is compared to placebo. A placebo looks like the study drug but does not contain any drug.\n\nThe study is looking at several other research questions, including:\n\n* What side effects may happen from taking ALN-CFB\n* How much ALN-CFB is in the blood at different times\n* How much Complement Factor B (CFB) protein levels in the blood are affected by ALN-CFB",[28,119],"Persistent Anemia",[121,122,123],"C5 inhibitor","Complement Factor B (CFB) protein","Red Blood Cells","2026-06-17",{"date":126,"type":36},"2026-06-18",{"date":128,"type":36},"2026-02-11",{"date":130,"type":20},"2031-07-15",{"name":132,"class":78},"Regeneron Pharmaceuticals",8,{"id":135,"slug":136,"hasResults":11,"nctId":137,"briefTitle":138,"officialTitle":139,"acronym":62,"eligibilityCriteria":140,"healthyVolunteers":141,"sex":15,"minAge":87,"maxAge":4,"enrollmentInfo":142,"targetDuration":4,"studyType":21,"phases":143,"briefSummary":144,"conditions":145,"keywords":146,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":148,"lastUpdatePostDateStruct":149,"startDateStruct":151,"completionDateStruct":153,"leadSponsor":155,"locationsCount":157},"100506586","phase-1-safety-tolerability-pk-and-pd-of-adx-038-in-healthy-participants-and-paroxysmal-nocturnal-hemoglobinuria-pnh-patients-100506586","NCT05876312","Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients","A Phase 1, Randomized, Double Blind, Placebo-Controlled, Single Ascending Dose Study in Healthy Participants Followed by a Phase 2a Open Label Study in Participants With PNH and Residual Anemia to Evaluate the Safety, Tolerability, PK and PD of ADX-038","Phase 1 Key Inclusion Criteria\n\n* 18 to 55 years of age\n* Participants who are healthy as determined by medical evaluation\n* History of recent meningococcal, pneumococcal and Haemophilus influenzae type B vaccinations or willing to be vaccinated\n* Screening tests negative for illicit drug, nicotine, and alcohol use\n\nPhase 1 Key Exclusion Criteria\n\n* History of any significant medical conditions, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia without evidence of recurrence within the prior 3 months\n* Any viral, bacterial, parasitic, or fungal infection within the prior 30 days\n* Frequent respiratory, nasopharyngeal or ear infections (more than 5 infections per year)\n* History of environmental exposure or sick contact that increase the risk of meningococcal, pneumococcal and\u002For Haemophilus influenza type B infections\n* Complement deficiency or immunodeficiency syndrome\n* Major surgery or significant traumatic injury within the prior 3 months\n* History of anaphylaxis or hypersensitivity reactions\n* History of penicillin allergy\n* History of splenectomy\n* History of alcohol abuse or illicit drug use\n* Donated plasma within the prior 7 days\n* Donated blood or loss more than 400 milliliters of blood (excluding blood volume drawn at screening) within the prior 90 days\n* Screening estimated creatinine clearance of less than 60 milliliters per minute\n* Screening hematology, serum chemistry, or coagulation parameters that are outside the normal range\n* Screening vital signs that are abnormal per protocol specification\n* Screening electrocardiogram findings that are clinically significant\n* Pregnant or lactating females\n* Use of prescription (except for contraceptives and study-related prophylactic antibiotics) or over-the counter medications (except for paracetamol or ibuprofen) or vitamins\u002Fsupplements within the prior 7 days\n* Use of medications that may reduce the effectiveness of hormonal contraceptives within the prior 28 days\n* Use of an investigational therapeutics within the prior 30 days or within the expected washout (at least 5 half-lives)\n* Unwilling or unable to adhere to study-related prophylactic antibiotics requirements\n\nPhase 2a Key Inclusion Criteria\n\n* at least 18 years of age\n* Diagnosis of paroxysmal nocturnal hemoglobinuria based on documented clone size\n* Hemoglobin concentration of less than 12 gram per deciliter\n* History of recent meningococcal, pneumococcal and Haemophilus influenzae type b vaccinations or willing to be vaccinated\n* On a stable anti-C5 regimen for greater than or equal to 12 weeks prior to Day 1\n\nPhase 2a Key Exclusion Criteria\n\n* Any viral, bacterial, parasitic, or fungal infection within the prior 14 days\n* HIV, active hepatitis C or hepatitis B infection\n* History of meningococcal or tuberculosis infection\n* History of malignancy in the past 5 years, except for completely excised non-melanoma skin cancer or low grade cervical intraepithelial neoplasia with no evidence of recurrence within the prior 3 months\n* Complement deficiency syndrome\n* History of hematopoietic stem cell transplantation\n* History of splenectomy\n* Inflammatory bowel disease, systemic lupus erythematosus, rheumatoid arthritis, or chronic liver disease\n* Clinically significant and uncontrolled medical conditions including, but not limited to, thromboembolic disease, acute coronary syndrome, and diabetes\n* Pregnant or lactating females\n* Use of an investigational therapeutics within the prior 30 days or within the expected washout period (at lest 5 half-lives)\n* Abstain from alcohol consumption for 48 hrs before day of dosing and restrict to no more than an average of 14 standard drinks per week",true,{"count":89,"type":20},[116,23],"The first-in-human Phase 1\u002FPhase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal nocturnal hemoglobinuria (PNH).",[28],[147,62],"siRNA","2026-06-12",{"date":150,"type":36},"2026-06-16",{"date":152,"type":36},"2023-08-07",{"date":154,"type":20},"2028-07-31",{"name":156,"class":78},"ADARx Pharmaceuticals, Inc.",4,{"id":159,"slug":160,"hasResults":11,"nctId":161,"briefTitle":162,"officialTitle":163,"acronym":4,"eligibilityCriteria":164,"healthyVolunteers":11,"sex":165,"minAge":4,"maxAge":4,"enrollmentInfo":166,"targetDuration":168,"studyType":90,"phases":4,"briefSummary":169,"conditions":170,"keywords":176,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":182,"lastUpdatePostDateStruct":183,"startDateStruct":185,"completionDateStruct":187,"leadSponsor":189,"locationsCount":191},"100540115","study-of-ultomiris-ravulizumab-safety-in-pregnancy-100540115","NCT06312644","Study of Ultomiris® (Ravulizumab) Safety in Pregnancy","Observational Study of Ultomiris® (Ravulizumab) Safety in Pregnancy","Inclusion Criteria:\n\n* Female participant must have a medically confirmed qualifying pregnancy (prospectively or retrospectively identified).\n* Participant informed consent (written or e-consent per local regulations or ethics committee requirements) must be obtained prior to the participant's enrollment. If the participant is a minor, consent must be obtained from the parent or legal guardian, with assent from the minor (as locally appropriate).\n* Willing to provide contact information for the participant.\n* Willing to authorize HCP(s) to release maternal and infant medical information to the study, upon request, if applicable to current local regulations.\n* Diagnosed with an indication for which Ultomiris is approved, based on HCP or medical records.\n* Exposed to Ultomiris at any point during the defined exposure window based on HCP or medical record documentation. (If exact exposure dates are unknown, the reporter must be able to specify or estimate trimester or timing of exposure \\[prior to conception as LMP+14 days, or during breastfeeding\\].)\n* Use of Ultomiris per local product information (i.e., United States Prescribing Information \\[USPI\\] or summary of product characteristics \\[SmPC\\])\n\nExclusion Criteria:\n\n* Participants who are unable to provide consent or assent (as locally appropriate) (e.g., diagnosed with severe psychiatric conditions or severe intellectual disabilities) will be excluded from this study","FEMALE",{"count":167,"type":20},75,"21 Months","The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of selected fetal\u002Fneonatal\u002Finfant outcomes in utero, at birth, and through 1 year of age after exposure in utero or via breastmilk.",[171,172,28,173,174,175],"Ultomiris-exposed Pregnant\u002F Postpartum","Pregnancy","Atypical Hemolytic Uremic Syndrome (aHUS)","Generalized Myasthenia Gravis (gMG)","Neuromyelitis Optica Spectrum Disorder (NMOSD)",[177,178,62,179,180,181],"Ultomiris","pregnancy","aHUS","gMG","NMOSD","2026-04-15",{"date":184,"type":36},"2026-04-16",{"date":186,"type":36},"2024-12-16",{"date":188,"type":20},"2034-07-11",{"name":190,"class":78},"Alexion Pharmaceuticals, Inc.",7,{"id":193,"slug":194,"hasResults":11,"nctId":195,"briefTitle":196,"officialTitle":197,"acronym":4,"eligibilityCriteria":198,"healthyVolunteers":11,"sex":15,"minAge":199,"maxAge":87,"enrollmentInfo":200,"targetDuration":4,"studyType":21,"phases":201,"briefSummary":203,"conditions":204,"keywords":205,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":210,"lastUpdatePostDateStruct":211,"startDateStruct":212,"completionDateStruct":214,"leadSponsor":216,"locationsCount":217},"100587944","phase-3-study-to-assess-the-pharmacokinetics-safety-and-tolerability-of-iptacopan-in-pediatric-pnh-patients-100587944","NCT06934967","Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients","An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to \u003C18 Years of Age","Inclusion Criteria:\n\n* Male and female participants 2 to \\\u003C 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes\u002Fmonocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.\n* Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.\n* Patients who are anti-C5 treatment naive: mean hemoglobin level \\\u003C 10 g\u002FdL confirmed by central laboratory assessment during screening.\n* Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) \\> 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.\n* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.\n* Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.\n\nExclusion Criteria:\n\n* History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.\n* Known or suspected hereditary complement deficiency at screening.\n* History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).\n* Patients with laboratory evidence of bone marrow failure (reticulocytes \\\u003C 100 x 10 to the ninth\u002FL; platelets \\\u003C 30 × 10 to the ninth\u002FL; neutrophils \\\u003C 0.5 × 10 to the ninth\u002FL).\n* Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.\n* Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","2 Years",{"count":55,"type":20},[202],"PHASE3","The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \\\u003C 18 years of age.",[28],[206,207,208,209],"Iptacopan,","PNH,","hemoglobin,","anemia","2026-04-13",{"date":184,"type":36},{"date":213,"type":36},"2025-10-28",{"date":215,"type":20},"2031-12-19",{"name":105,"class":78},13,{"id":219,"slug":220,"hasResults":11,"nctId":221,"briefTitle":222,"officialTitle":223,"acronym":4,"eligibilityCriteria":224,"healthyVolunteers":141,"sex":15,"minAge":87,"maxAge":225,"enrollmentInfo":226,"targetDuration":4,"studyType":21,"phases":228,"briefSummary":229,"conditions":230,"keywords":233,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":239,"lastUpdatePostDateStruct":240,"startDateStruct":242,"completionDateStruct":244,"leadSponsor":246,"locationsCount":44},"100628526","phase-1-a-phase-i-clinical-trial-to-evaluate-cms-d017-following-single-and-multiple-doses-in-healthy-participants-100628526","NCT07462780","A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants","A Randomized, Double-blind, Placebo-controlled, Dose-escalation Phase I Study to Evaluate the Safety, Tolerability, PK and PD Characteristics of CMS-D017 Following Single and Multiple Administrations in Healthy Participants","Inclusion Criteria:\n\n1. Voluntarily participates in this study and signs the informed consent form.\n2. Able to communicate well with the investigator and understands and complies with all requirements and restrictions of this study, and is able to complete the study in accordance with the protocol.\n3. Aged 18-55 years (inclusive, as of the day of signing the informed consent form), male or female.\n4. Body Mass Index (BMI) between 19.0 and 26.0 kg\u002Fm² (inclusive) at screening, with females weighing ≥ 45.0 kg and males weighing ≥ 50.0 kg.\n5. Participants (and their partners) with reproductive capacity must have no plans for pregnancy, egg donation, or sperm donation from the date of signing the informed consent form until 3 months after the last dose of the study drug, and must comply with contraceptive requirements (see Appendix 1), agreeing to use at least one highly effective non-hormonal contraceptive method.\n\nExclusion Criteria:\n\nAllergy History\n\n1. History of severe allergies, including food allergies, or allergy to the study drug or its components.\n\n   Medical History\u002FConditions\n2. Significant history or clinical manifestations of cardiovascular, respiratory, digestive, urogenital, hematologic, endocrine and metabolic, rheumatic, immunologic, neuropsychiatric, or musculoskeletal diseases requiring medication and\u002For other treatments (including dietary restrictions and physical therapy), as deemed unsuitable for participation in this study by the investigator.\n3. Any condition that may affect drug absorption, including but not limited to: malabsorption syndrome, inflammatory bowel disease, celiac disease, gastrectomy, cholecystectomy, bowel resection (except appendectomy).\n4. History of meningococcal infection or first-degree relatives with history of meningococcal infection.\n5. Active infection or acute disease state (e.g., fever, nausea, vomiting, or diarrhea) within 2 weeks prior to screening.\n6. Current history of tuberculosis infection; or positive tuberculosis (TB) test result. Note: If the TB test result is indeterminate, one repeat test is allowed.\n7. History of severe trauma or surgery within 8 weeks prior to screening, or planned surgery during the study period.\n8. History or current presence of the following cardiac risk factors:\n\n   Torsades de pointes or risk factors (e.g., hypokalemia, hypomagnesemia, use of drugs causing delayed cardiac repolarization) History of cardiac arrest, syncope, heart failure; myocardial infarction, angina; valvular heart disease; cardiomyopathy or family history; clinically significant arrhythmias (e.g., sick sinus syndrome, atrioventricular conduction block, Adams-Stokes syndrome, Brugada syndrome or family history, long QT syndrome, atrial flutter, atrial fibrillation, supraventricular tachycardia, ventricular tachycardia).\n\n   Prior\u002FConcomitant Treatments\n9. Participation in any other clinical study involving drugs or medical devices within 3 months prior to screening, or planned participation in such studies during this study, or within 5 half-lives of that drug (whichever is longer).\n10. Vaccination within 4 weeks prior to screening or planned vaccination during the study or within 1 month after last dose (participants vaccinated against meningococcal and pneumococcal infections may be included if vaccination was completed at least 2 weeks before dosing).\n11. Use of known CYP2C8 or CYP3A inducers or inhibitors, or P-gp inhibitors within 4 weeks prior to dosing (see Appendix 2).\n12. Use of any prescription or over-the-counter drugs (including herbal medicines, vitamins, minerals, and dietary supplements) within 2 weeks or at least 5 half-lives prior to dosing, whichever is longer.\n\n    Substance Use, Alcohol, Tobacco, or Nicotine, Dietary\u002FExercise Restrictions\n13. History of drug abuse within 6 months prior to screening, or positive result for any drug abuse test.\n14. Alcohol consumption exceeding 14 units per week within 3 months prior to screening (1 unit = 360 mL beer, 150 mL wine, or 45 mL spirits), or positive breath alcohol test, or inability to abstain from alcohol during the study.\n15. Average smoking of more than 5 cigarettes per day within 3 months prior to screening, or inability to stop using any tobacco products during the study.\n16. Inability to abstain from grapefruit or grapefruit-related citrus fruits or juices (e.g., pomelo) within 7 days prior to dosing and during the study.\n17. Consumption of caffeine-containing products (e.g., coffee, tea, cola, other caffeinated beverages, or chocolate) within 3 days prior to dosing, or refusal to avoid such products throughout the study.\n18. Engagement in strenuous exercise or physical activity within 3 days prior to dosing, or refusal to avoid such activities throughout the study.\n\n    Examinations and Assessments\n19. Corrected QT interval (using Fridericia's formula, QTcF = QT\u002F(RR\\^0.33)) \\> 450 msec in males or females.\n20. Abnormal findings in physical examination, vital signs, safety laboratory tests, 12-lead ECG, or other auxiliary tests (chest X-ray, abdominal ultrasound) deemed clinically significant by the investigator.\n21. Positive test results for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCVAb), syphilis antibody (TPAb), or human immunodeficiency virus antibody (HIV Ab).\n\n    Other\n22. Pregnant or lactating females.\n23. Special dietary requirements or inability to comply with standardized diet.\n24. Difficulty with venous blood sampling (e.g., history of needle or blood phobia), or poor venous condition as deemed unsuitable for enrollment by the investigator.\n25. Donation or loss of ≥ 400 mL blood within 3 months prior to screening, or receipt of blood transfusion or blood products; or planned blood or blood component donation during the study.\n26. Other conditions deemed unsuitable for participation in this study by the investigator.","55 Years",{"count":227,"type":20},88,[116],"This study is a first-in-human (FIH) trial of CMS-D017 conducted in healthy Chinese adult participants, consisting of two parts: Part 1-a single ascending dose (SAD) study (referred to as Part 1 SAD), and Part 2-a multiple ascending dose (MAD) study (referred to as Part 2 MAD). The study aims to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) characteristics of CMS-D017 capsules following single and multiple oral administrations in healthy Chinese adult participants.\n\nBoth parts of the study are designed as randomized, double-blind, placebo-controlled, sequential cohort trials. Part 1 SAD plans to include 6 dose cohorts, with 8 participants per cohort (6 receiving CMS-D017 and 2 receiving placebo), for a total of 48 participants. Part 2 MAD plans to include 4 dose cohorts, with 10 participants per cohort (8 receiving CMS-D017 and 2 receiving placebo), for a total of 40 participants.",[231,232],"Paroxysmal Nocturnal Hemoglobinuria, PNH","Complement-mediated Kidney Disease",[234,235,236,237,238],"CMS-D017","Phase I trial in healthy participants","single and multiple ascending dose studies","pharmacokinetics","pharmacodynamics","2026-04-02",{"date":241,"type":36},"2026-04-08",{"date":243,"type":36},"2026-03-05",{"date":245,"type":20},"2026-12",{"name":247,"class":78},"Shenzhen Kangzhe Biotechnology Co., Ltd.",{"id":249,"slug":250,"hasResults":11,"nctId":251,"briefTitle":252,"officialTitle":253,"acronym":254,"eligibilityCriteria":255,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":256,"targetDuration":4,"studyType":90,"phases":4,"briefSummary":258,"conditions":259,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":276,"lastUpdatePostDateStruct":277,"startDateStruct":279,"completionDateStruct":281,"leadSponsor":283,"locationsCount":44},"100412026","national-longitudinal-cohort-of-hematological-diseases-100412026","NCT04645199","National Longitudinal Cohort of Hematological Diseases","National Longitudinal Cohort of Hematological Diseases (NICHE)","NICHE","Inclusion Criteria:\n\n* Patients who were diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders or received bone marrow transplantation in the investigating hospitals from January 1, 2020.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Alcohol and drug addictions affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",{"count":257,"type":20},2300,"Background Hematological diseases are disorders of the blood and hematopoietic organs. The current hematological cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in hematology. The purpose of this study is to analyze the incidence and risk factors of major blood diseases, the treatment methods, prognosis and medical expenses of these patients in China.\n\nMethod The study will include patients diagnosed with acute myeloid leukemia, multiple myeloma, hemophilia, aplastic anemia, leukemia, myelodysplastic syndrome, lymphoma, bleeding disorders, autoimmune hemolytic anemia, large granular lymphocyte leukemia, essential thrombocythemia, blood infection or received bone marrow transplantation in the investigating hospitals from January 1, 2020, and collect basic information, diagnostic and treatment information, prognosis information, as well as medical expense information from medical records. In its current form, the NICHE registry incorporates historical data (collected from 2000) and is systematically collecting prospective data in two phases with broadening reach, and prospectively follow-up to collect the prognosis information.",[260,261,262,263,264,265,266,267,268,269,270,271,272,273,274,275,231],"Multiple Myeloma","Acute Myeloid Leukemia","Hemophilia","Hemophilia A","Hemophilia B","Myelodysplastic Syndrome","MDS","Lymphoma","Leukemia","Aplastic Anemia","Bleeding Disorder","Bone Marrow Transplantation","Blood Disease Infection","Autoimmune Hemolytic Anemia, AIHA","Essential Thrombocythemia, ET","Large Granular Lymphocyte Leukemia, LGLL","2026-02-09",{"date":278,"type":36},"2026-02-12",{"date":280,"type":36},"2020-12-01",{"date":282,"type":20},"2030-12-01",{"name":284,"class":285},"Institute of Hematology & Blood Diseases Hospital, China","OTHER",{"id":287,"slug":288,"hasResults":11,"nctId":289,"briefTitle":290,"officialTitle":291,"acronym":4,"eligibilityCriteria":292,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":293,"enrollmentInfo":294,"targetDuration":4,"studyType":21,"phases":296,"briefSummary":297,"conditions":298,"keywords":4,"overallStatus":299,"whyStopped":4,"lastUpdateSubmitDate":300,"lastUpdatePostDateStruct":301,"startDateStruct":303,"completionDateStruct":305,"leadSponsor":307,"locationsCount":4},"100622721","phase-2-sln12140-in-adult-participants-with-paroxysmal-nocturnal-hemoglobinuria-pnh-in-china-100622721","NCT07387302","SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China","A Phase II Clinical Study Evaluating SLN12140 in Complement Inhibitor-Naïve Adult Subjects With Paroxysmal Nocturnal Hemoglobinuria","Inclusion Criteria:\n\n* Adult complement inhibitor naïve PNH patients (age\\>=18), which is confirmed by flow cytometry evaluation\n* Must be vaccinated against meningococcal vaccine and pneumococcal vaccine\n\nExclusion Criteria:\n\n* Significant bone marrow failure\n* Meningitidis infection or unresolved meningococcal disease\n* Other significant systemic diseases that might have impact on efficacy and safety assessment","80 Years",{"count":295,"type":20},10,[23],"The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults. It will also learn about the safety, pharmacokinetic characteristics, and dosing of drug SLN12140.\n\nThe study is divided into four phases: screening period, core treatment period, extended dosing period, and follow-up period, and includes two cohorts (Cohorts 1-2), with each cohort enrolling at least 5 treatment-naïve adult PNH subjects for complement inhibitor therapy.",[28],"NOT_YET_RECRUITING","2026-01-27",{"date":302,"type":36},"2026-02-04",{"date":304,"type":20},"2026-02-10",{"date":306,"type":20},"2027-12-31",{"name":308,"class":78},"Linno Pharmaceuticals, Inc.",{"id":310,"slug":311,"hasResults":11,"nctId":312,"briefTitle":313,"officialTitle":313,"acronym":4,"eligibilityCriteria":314,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":315,"phases":4,"briefSummary":316,"conditions":317,"keywords":319,"overallStatus":326,"whyStopped":4,"lastUpdateSubmitDate":327,"lastUpdatePostDateStruct":328,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":330,"locationsCount":4},"100456347","managed-access-programs-for-lnp023-iptacopan-100456347","NCT05222412","Managed Access Programs for LNP023, Iptacopan","Inclusion Criteria:\n\n* An independent request should be received from a licensed physician (in some instances from Health Authorities, Institutions or Governments).\n* The patient has a serious or life-threatening disease or condition and no comparable or satisfactory alternative therapy is available for diagnosis, monitoring or treatment; patient is not medically eligible for available treatment alternatives or has exhausted all available treatment options.\n* The patient is not eligible or able to enroll in a Novartis clinical trial or continue participation in a Novartis clinical trial.\n* There is a potential patient benefit to justify the potential risk of the treatment use, and the potential risk is not unreasonable in the context of the disease or condition to be treated.\n* The patient must meet any other medical criteria established by the medical experts responsible for the product or by the Health Authority in a country (as applicable).\n* Provision of the product will not interfere with the initiation, conduct or completion of a Novartis clinical trial or overall development program.\n* Managed access provision is allowed per local laws\u002Fregulations.","EXPANDED_ACCESS","The purpose of this registration form is to list all Managed Access Programs (MAPs) related to LNP023, Iptacopan",[318,28],"C3 Glomerulopathy (C3G)",[320,321,322,323,324,325],"C3 glomerulopathy (C3G)","Paroxysmal nocturnal hemoglobinuria (PNH)","MAP","Manage access program","Iptacopan","LNP023","AVAILABLE","2025-12-01",{"date":329,"type":36},"2025-12-05",{"name":105,"class":78},{"id":332,"slug":333,"hasResults":11,"nctId":334,"briefTitle":335,"officialTitle":336,"acronym":4,"eligibilityCriteria":337,"healthyVolunteers":141,"sex":15,"minAge":87,"maxAge":338,"enrollmentInfo":339,"targetDuration":4,"studyType":21,"phases":340,"briefSummary":341,"conditions":342,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":343,"lastUpdatePostDateStruct":344,"startDateStruct":346,"completionDateStruct":348,"leadSponsor":350,"locationsCount":44},"100604651","phase-1-investigation-of-pharmacokineticssafetyand-pharmacodynamics-of-hsk39297-in-subjects-with-hepatic-impairment-100604651","NCT07152288","Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment","A Single-dose, Open-label, Phase I Study Comparing the Pharmacokinetics, Safety, and Pharmacodynamics of HSK39297 in Subjects With Mild and Moderate Hepatic Impairment and Normal Hepatic Function","Inclusion Criteria:\n\n1. Ability to understand the study procedures and methods, participate voluntarily and be able to complete the study according to the protocol requirements, and sign the informed consent form (ICF) in writing;\n2. Aged 18-70 years old on the date of signing the ICF (including the threshold), both male and female;\n3. At the time of screening, male subjects weighing no less than 50 kg and female subjects weighing no less than 45 kg; body mass index (BMI): 18\\~32 kg\u002Fm2 (including the threshold);\n4. Normal or abnormal physical examination, 12-ECG, vital signs, chest frontal and lateral radiographs\u002FCT, abdominal ultrasound, and laboratory tests (blood routine, blood biochemistry, urine routine, coagulation function, etc.) in the screening and baseline periods were not clinically significant.\n5. The demographic means of subjects in the normal liver function group (Group C) at screening must meet the following matching criteria:\n\n   1. BMI matched to the hepatic impairment group (Group A + Group B) with a mean value ± 15%;\n   2. Age-matched to the hepatic impairment group (Group A + Group B), mean ± 10 years;\n   3. Sex-matched to liver impairment group (Group A + Group B), mean value ± 1 case;\n6. Glomerular filtration rate (eGFR) ≥75 mL\u002Fmin\u002F1.73m2 calculated using the Chronic Kidney Disease Epidemiology Collaborative Study Group (CKD-EPI) formula;\n7. Subjects with childbearing potential must agree to have no plans for childbearing and voluntarily use highly effective contraception with their partner from the time of signing the ICF until 1 month after administration of the test drug, and to avoid sperm\u002Fegg donation. Female subjects of childbearing potential must have a negative serum pregnancy test at both screening and baseline and not be breastfeeding.\n\n   For subjects with hepatic impairment, the following inclusion criteria must also be met:\n8. Not on medication within 4 weeks prior to screening, or have at least 4 weeks of stable medication for hepatic impairment and\u002For other co-morbidities requiring long-term treatment;\n9. Child-Pugh classification of Class A or B (without the use of albumin within 14 days), which is chronic liver injury caused by previous primary liver diseases, including but not limited to non-alcoholic steatohepatitis, viral hepatitis (hepatitis B, hepatitis C), etc.\n\nExclusion Criteria:\n\n1. Smoked an average of more than 5 cigarettes per day in the past 3 months or those who cannot comply with the prohibition of smoking during the trial;\n2. Known or suspected (as judged by the researcher) immunodeficiency diseases or hereditary complement deficiencies;\n3. Allergic to two or more allergens, or in the judgment of the investigator, may be allergic to the study drug or its components;\n4. Screen those who have a history of heavy drinking in the past 3 months (with an average daily alcohol consumption of \\> 2 units of alcohol (1 unit = 285 mL of beer, or 30 mL of spirits, or 100 mL of wine)) or those with a positive breath alcohol test;\n5. Disease or medical condition that, in the judgment of the investigator, may interfere with the absorption, distribution, metabolism, and excretion of the drug or that may reduce compliance;\n6. History of capsular bacterial infection in the past; including but not limited to Neisseria meningitidis, Streptococcus pneumoniae, Haemophilus influenzae type b, etc;\n7. Severe trauma or undergone surgery within 2 weeks prior to screening, or plan to undergo surgery during the trial period;\n8. Participated in a clinical trial of any other drug or medical device within 3 months prior to screening or plan to do so during the study period, or still within 5 half-lives of the drug prior to screening (whichever is longer);\n9. A previous diagnosis of malignant tumors (excluding radically resected basal cell carcinoma of the skin, papillary thyroid carcinoma, squamous cell carcinoma of the skin, or carcinoma in situ of the cervix);\n10. History of drug or substance abuse; or a positive urine drug test at screening;\n11. Donated or lost ≥ 400 mL of blood within 30 days, or received a blood transfusion prior to screening;\n12. QTcF (males) \\> 480 ms at screening, or other 12-ECG abnormalities judged by the investigator to be clinically significant or unsuitable for participation;\n13. Difficulty in swallowing, collecting blood intravenously, or physically unable to tolerate blood collection, or not expected to complete the entire trial follow-up;\n14. Vaccined within 2 weeks prior to dosing or plan to receive the vaccine during the study;\n15. Positive result in the screening of any of the following indicators: hepatitis B surface antigen, hepatitis C antibody, HIV antibody, or syphilis antibody (hepatitis B surface antigen and hepatitis C antibody can be positive in subjects in the liver insufficiency group).\n16. Any physical or psychological disease or condition that, in the judgment of the investigator, is likely to increase the risk of the trial, interfere with compliance with the protocol and ability to complete the trial.\n\n    Additional exclusion criteria for subjects with hepatic impairment:\n17. History of any serious disease other than the primary disease of the liver itself, or a history of and\u002For abnormal clinically significant clinical laboratory tests that, in the opinion of the investigator, may affect the results of the test, including, but not limited to, a history of circulatory, endocrine, neurological, gastrointestinal, urologic, or hematologic, immunologic, psychiatric, and metabolic disorders;\n18. Liver failure, or cirrhosis with combination of hepatic encephalopathy (West-Haven Level 2 and above), hepatocellular carcinoma (except for those with Barcelona Clinic Liver Cancer (BCLC) stage 0 or those who have achieved radical treatment results), ruptured variceal hemorrhage of the esophagogastric fundus vein, and other complications that, in the judgment of the investigator, make the subject unsuitable for participation in the study;\n19. On systemic hormones (except subjects on stable use of ≤ 5 mg\u002Fday of prednisone or equivalent doses of glucocorticoids) or other immunosuppressive agents within 4 weeks prior to screening, or more than 4 weeks from their last immunosuppressive therapy at screening but are still within the drug's efficacy period (as assessed by the investigator);\n20. Alpha-fetoprotein \\> 20 ng\u002FmL; hemoglobin (Hb) ≤ 70 g\u002FL; alanine aminotransferase (ALT) or mentholatum aminotransferase (AST) ≥ 5 times the upper limit of normal (ULN); severe active bleeding or a platelet level ≤30×10⁹\u002FL (except for those judged by the investigator to be in a stable state);\n21. History of liver transplantation.","70 Years",{"count":114,"type":20},[116],"The study is being conducted to compare the pharmacokinetics, safety, and pharmacodynamics of HSK39297 in subjects with mild to moderate hepatic impairment and normal hepatic function",[28],"2025-08-26",{"date":345,"type":36},"2025-09-03",{"date":347,"type":36},"2025-05-29",{"date":349,"type":20},"2025-11",{"name":351,"class":78},"Haisco Pharmaceutical Group Co., Ltd.",{"id":353,"slug":354,"hasResults":11,"nctId":355,"briefTitle":356,"officialTitle":357,"acronym":358,"eligibilityCriteria":359,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":17,"enrollmentInfo":360,"targetDuration":4,"studyType":90,"phases":4,"briefSummary":362,"conditions":363,"keywords":371,"overallStatus":299,"whyStopped":4,"lastUpdateSubmitDate":382,"lastUpdatePostDateStruct":383,"startDateStruct":385,"completionDateStruct":387,"leadSponsor":389,"locationsCount":4},"100601248","hematological-disorders-in-ehpvo-patients-100601248","NCT07108023","Hematological Disorders in EHPVO Patients","A Prospective Study of the Spectrum of Haematological Disorders in Patients With Extrahepatic Portal Vein Obstruction","EHPVO-HEM","Inclusion Criteria:\n\n* Age 18 years or older . Diagnosis of extra hepatic portal vein obstruction based on imaging ( Doppler, CT , MRI ) preserved liver function . Available complete medical records including CBC , LFTs and coagulation profile .\n\nExclusion Criteria:\n\n* Patients with cirrhosis or intrahepatic portal hypertension • Incomplete or missing medical records • Patients with known hematologic malignancies or undergoing chemotherapy",{"count":361,"type":20},115,"This study focuses on patients who have a condition called extrahepatic portal vein obstruction (EHPVO), where a blood clot blocks the portal vein outside the liver. This blockage can cause problems like an enlarged spleen, bleeding from swollen veins in the digestive system, and low blood cell counts. Many of these patients may have hidden blood disorders that increase the risk of clotting, such as myeloproliferative neoplasms (MPNs), antiphospholipid syndrome (APS), or paroxysmal nocturnal hemoglobinuria (PNH). This study will collect and analyze blood test results-such as complete blood count (CBC), liver function tests (LFTs), and clotting tests-from patients with EHPVO. The aim is to find patterns that may suggest an underlying blood disorder, even if the patient doesn't show obvious symptoms.By understanding these patterns early, doctors may be able to diagnose and treat the root causes of clotting in these patients more accurately, helping prevent complications and improve outcomes.",[364,365,366,367,28,368,369,370],"Extrahepatic Portal Vein Obstruction (EHPVO)","Thrombophilia","Myeloproliferative Neoplasms (MPN)","Antiphospholipid Syndrome (APS)","Thrombocytopenia","Anemia","Leukopenia",[372,373,374,375,376,377,378,379,380,381],"Portal vein thrombosis","EHPVO","Hypersplenism","Hematological disorders","Coagulation profile","Liver function tests","CBC","JAK2 mutation","Thrombosis in MPN","Cross-sectional study","2025-07-31",{"date":384,"type":36},"2025-08-06",{"date":386,"type":20},"2025-08",{"date":388,"type":20},"2025-12",{"name":390,"class":285},"Rahab Nady",{"id":392,"slug":393,"hasResults":11,"nctId":394,"briefTitle":395,"officialTitle":396,"acronym":4,"eligibilityCriteria":397,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":17,"enrollmentInfo":398,"targetDuration":4,"studyType":21,"phases":400,"briefSummary":401,"conditions":402,"keywords":4,"overallStatus":299,"whyStopped":4,"lastUpdateSubmitDate":403,"lastUpdatePostDateStruct":404,"startDateStruct":406,"completionDateStruct":407,"leadSponsor":409,"locationsCount":4},"100526413","phase-2-study-of-safety-and-efficacy-of-my008211a-in-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100526413","NCT06134414","Study of Safety and Efficacy of MY008211A in in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Multi-center, Randomized, Parallel, Open-label Clinical Phase II Study, to Evaluate the Efficacy and Safety of MY008211A in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients With Signs of Active Hemolysis","Inclusion Criteria:\n\n* Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg\u002Fm2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.\n* Mean hemoglobin level \\\u003C100 g\u002FL.\n* LDH \\> 1.5 x Upper Limit of Normal (ULN).\n* Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.\n\nExclusion Criteria:\n\n* Patients with reticulocytes \\\u003C100x10\\^9\u002FL; platelets \\\u003C30x10\\^9\u002FL; neutrophils \\\u003C0.5x10\\^9\u002FL.\n* Were using a complement inhibitor before the first administration of MY008211A tablets or had discontinued a previous complement inhibitor for less than five half-lives or 120 days, whichever was the longest.\n* History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.\n* Known or suspected hereditary complement deficiency.\n* Previous bone marrow or hematopoietic stem cell transplantation.\n* Previous splenectomy.\n* A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.",{"count":399,"type":20},40,[23],"The main purpose of this study is to evaluate the efficacy of MY008211A in adult patients with PNH, showing signs of active hemolysis.",[28],"2025-07-27",{"date":405,"type":36},"2025-07-29",{"date":388,"type":20},{"date":408,"type":20},"2027-12",{"name":410,"class":78},"Wuhan Createrna Science and Technology Co., Ltd",{"id":412,"slug":413,"hasResults":11,"nctId":414,"briefTitle":415,"officialTitle":416,"acronym":4,"eligibilityCriteria":417,"healthyVolunteers":11,"sex":15,"minAge":87,"maxAge":4,"enrollmentInfo":418,"targetDuration":4,"studyType":21,"phases":420,"briefSummary":421,"conditions":422,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":423,"lastUpdatePostDateStruct":424,"startDateStruct":426,"completionDateStruct":428,"leadSponsor":430,"locationsCount":44},"100587863","phase-2-long-term-safety-and-tolerability-of-my008211a-tablets-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-100587863","NCT06933914","Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria","An Open Label, Multicenter Study to Evaluate the Long-term Safety and Tolerability of MY008211A Tablets in Patients With PNH Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n1. Patients who have previously received and completed MY008211A study treatment, and are judged by the investigator to have treatment benefit and may benefit from continued treatment of MY008211A.\n2. Prior vaccinations against Neisseria meningitidis, Streptococcus pneumoniae and Haemophilus influenzae infections.\n\nExclusion Criteria:\n\n1. History of recurrent invasive infections caused by encapsulated organisms, e.g. meningococcus or pneumococcus.\n2. Known or suspected hereditary complement deficiency.\n3. Any comorbidity or medical condition (including but not limited to any active systemic bacterial, viral or fungal infection or malignancy) that, in the opinion of the investigator, could put the subject at increased risk or potentially confound study data.",{"count":419,"type":20},120,[23,202],"This is a multicenter, single-arm, open-label study to characterize long-term safety and tolerability of MY008211A tablets and to provide access to MY008211A tablets to patients with PNH who have completed Phase 2 or 3 studies with MY008211A tablets.",[28],"2025-04-11",{"date":425,"type":36},"2025-04-18",{"date":427,"type":36},"2024-11-30",{"date":429,"type":20},"2026-12-30",{"name":410,"class":78}]