[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"paroxysmal-nocturnal-hemoglobinuria\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:paroxysmal-nocturnal-hemoglobinuria":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,23,0,[8,43,65,87,109,139,152,179,213,239,265,286,313,334,355,372,398,419,438,462,483,504,531],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":31,"lastUpdatePostDateStruct":32,"startDateStruct":35,"completionDateStruct":37,"leadSponsor":39,"locationsCount":42},"100054215","phase-3-a-study-in-adult-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-to-evaluate-how-safe-long-term-treatment-with-pozelimab--cemdisiran-combination-therapy-is-and-how-well-it-works-100054215",false,"NCT05744921","A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works","An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria","ACCESS-EXT","Key Inclusion Criteria:\n\nPatients Entering from the Parent Study\n\n1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021\\[NCT05133531\\]), including the post-Open-label treatment period (OLTP) transition period, if applicable.\n2. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol.\n\nPatients Entering with C5 polymorphism\n\n1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol\n2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes\n3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol\n4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit\n5. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol\n\nKey Exclusion Criteria:\n\nPatients Entering from the Parent Study\n\n1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and\u002For safety of the patient\n2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study\n\nPatients Entering with C5 polymorphism\n\n1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary\n2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant\n3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol\n4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening\n5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol\n6. Known hereditary complement deficiency\n7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases\n8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol\n\nNote: Other protocol-defined Inclusion\u002F Exclusion Criteria apply","ALL","18 Years",{"count":20,"type":21},202,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as \"study drugs\" in this section.\n\nThis study is looking at several other research questions, including:\n\n* How effective is the pozelimab + cemdisiran combination?\n* What side effects may happen from taking the study drugs?\n* How much of each study drug is in the blood at different times?\n* Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)",[27],"Paroxysmal Nocturnal Hemoglobinuria",[29],"PNH","RECRUITING","2026-07-10",{"date":33,"type":34},"2026-07-13","ACTUAL",{"date":36,"type":34},"2023-03-07",{"date":38,"type":21},"2029-02-26",{"name":40,"class":41},"Regeneron Pharmaceuticals","INDUSTRY",45,{"id":44,"slug":45,"hasResults":11,"nctId":46,"briefTitle":47,"officialTitle":48,"acronym":4,"eligibilityCriteria":49,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":50,"targetDuration":4,"studyType":22,"phases":52,"briefSummary":53,"conditions":54,"keywords":55,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":56,"lastUpdatePostDateStruct":57,"startDateStruct":59,"completionDateStruct":61,"leadSponsor":63,"locationsCount":64},"100604840","phase-3-a-study-to-evaluate-how-pozelimab--cemdisiran-combination-therapy-works-in-adult-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-whose-current-treatment-is-not-working-efficiently-100604840","NCT07154745","A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently","A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy","Key Inclusion Criteria:\n\n1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing\n2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months\n3. LDH persistently \\> 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis\n4. At least 2 screening LDH values from different visits as described in the protocol\n5. Willing and able to comply with clinic\u002Fremote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study\n\nKey Exclusion Criteria:\n\n1. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants\n2. Body weight \\\u003C40 kilograms at screening visit\n3. Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol\n4. Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol\n5. Known hereditary complement deficiency\n\nNote: Other protocol-defined Inclusion\u002F Exclusion Criteria apply",{"count":51,"type":21},35,[24],"This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as \"study drugs\". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH).\n\nThe aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab\u002Feculizumab biosimilar, ravulizumab or crovalimab.\n\nThe study is looking at several other research questions, including:\n\n* What side effects may happen from taking the study drugs?\n* How much of the study drugs are in the blood at different times?\n* Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)",[27],[29],"2026-07-01",{"date":58,"type":34},"2026-07-02",{"date":60,"type":34},"2026-06-11",{"date":62,"type":21},"2031-12-04",{"name":40,"class":41},16,{"id":66,"slug":67,"hasResults":11,"nctId":68,"briefTitle":69,"officialTitle":70,"acronym":71,"eligibilityCriteria":72,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":73,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":76,"conditions":77,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":78,"lastUpdatePostDateStruct":79,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":86},"100498916","a-real-world-effectiveness-study-of-pegcetacoplan-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100498916","NCT05776472","A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Single Arm, Long-term, Multicentre Observational Study to Evaluate Effectiveness of Pegcetacoplan Under Real World Conditions in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","COMPLETE","Inclusion Criteria:\n\n* Patients ≥18 years of age with a documented PNH diagnosis.\n* Patient started routine treatment with pegcetacoplan for PNH up to 12 months before enrollment or prescribed pegcetacoplan at enrollment. Decision to initiate treatment shall be made by the treating physician and independently from the decision to include the patient in the study.\n* Patient is willing and able to provide written informed consent to participate in the study in a manner approved by the Institutional Review Board\u002FIndependent Ethics Committee and local regulations.\n\nExclusion Criteria:\n\n* Enrollment in a concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to the start of the current pegcetacoplan treatment.\n* Initiated current treatment with pegcetacoplan in an interventional study.",{"count":74,"type":21},200,"OBSERVATIONAL","This is a 36-month, long-term, multicenter, observational study designed to describe the real world effectiveness of pegcetacoplan in patients with PNH. Patients meeting the eligibility criteria will be enrolled in the study and followed prospectively for approximately 36 months. Patient data will be collected from start of pegcetacoplan treatment to end of follow-up. Retrospective data on pegcetacoplan will be captured from the time of pegcetacoplan treatment initiation. Pegcetacoplan treatment data will be collected for a minimum of approximately 36 months and up to a maximum of approximately 72 months, including retrospective period depending on when the patient started pegcetacoplan treatment. After pegcetacoplan treatment discontinuation, patients will remain in the study for 8 weeks to capture any AEs. The scope of the study is to collect both retrospective and prospective data. Baseline is defined as start of pegcetacoplan treatment. The main part of the study will be prospective,collecting data on effectiveness, safety (all AEs), patient- and clinician-reported outcomes and health care resource use.",[27],"2026-06-30",{"date":56,"type":34},{"date":81,"type":34},"2023-06-26",{"date":83,"type":21},"2029-08-21",{"name":85,"class":41},"Swedish Orphan Biovitrum",87,{"id":88,"slug":89,"hasResults":11,"nctId":90,"briefTitle":91,"officialTitle":92,"acronym":4,"eligibilityCriteria":93,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":94,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":96,"conditions":97,"keywords":98,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":102,"completionDateStruct":104,"leadSponsor":106,"locationsCount":108},"100628093","danicopan-pms-in-korea-100628093","NCT07457151","Danicopan PMS in Korea","Danicopan Regulatory Post-Marketing Study in Korea","Inclusion Criteria:\n\n1. Patients eligible for and treated with Danicpan as add-on therapy to a C5 inhibitor (Eculizumab or Ravulizumab) in patient with PNH in Korea\n2. Provision of a signed and dated written informed consent by the patient or their legally acceptable representative\n\nExclusion Criteria:\n\n1. Participation in any concurrent interventional trials during the period of study drug treatment\n2. Other off-label indications according to the approved label in Korea",{"count":95,"type":21},8,"As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical practice settings. This study is designed to assess the known safety profile or identify previously unsuspected adverse reactions and to evaluate the effectiveness of Danicopan under conditions of routine daily medical practice in Korea.",[27],[29],"2026-06-26",{"date":101,"type":34},"2026-06-29",{"date":103,"type":34},"2026-04-10",{"date":105,"type":21},"2028-06-27",{"name":107,"class":41},"AstraZeneca",2,{"id":110,"slug":111,"hasResults":11,"nctId":112,"briefTitle":113,"officialTitle":114,"acronym":4,"eligibilityCriteria":115,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":116,"enrollmentInfo":117,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":119,"conditions":120,"keywords":122,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":130,"lastUpdatePostDateStruct":131,"startDateStruct":132,"completionDateStruct":134,"leadSponsor":136,"locationsCount":138},"100624940","a-study-of-iptacopan-in-korean-patients-with-paroxysmal-nocturnal-hemoglobinuria-or-c3-glomerulopathy-100624940","NCT07416162","A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy","A Post-marketing Surveillance of Fabhalta® (Iptacopan) in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) or C3 Glomerulopathy (C3G)","Inclusion criteria:\n\n1. Patients aged 18 years or older who have been diagnosed with PNH or C3G.\n2. Patients who have received vaccination in accordance with the approved Korean labeling prior to initiating treatment with iptacopan.\n3. Patients who are being treated or will be treated with iptacopan in accordance with the approved Korean labeling.\n4. Patients who have voluntarily provided consent for study participation (written informed consent).\n\nExclusion criteria:\n\n1. Patients who fall under the contraindications for iptacopan administration according to approved Korean labeling.\n2. Patients for whom iptacopan administration is deemed inappropriate based on the investigator's judgment.","99 Years",{"count":118,"type":21},21,"This is a post-marketing surveillance study conducted as part of the Risk Management Plan (RMP) for South Korea, to evaluate the safety and effectiveness of iptacopan in real-world clinical settings for the treatment of either PNH or C3G in Korean patients. Prospective data will be collected from patient medical records to address the objectives for all eligible populations.",[27,121],"C3 Glomerulopathy",[123,124,125,126,127,128,129,27,121],"Iptacopan","Factor B inhibitor","Post-Marketing Surveillance","Complement inhibition","Hemolysis","Proteinuria","Dense Deposit Disease","2026-06-25",{"date":99,"type":34},{"date":133,"type":34},"2026-06-15",{"date":135,"type":21},"2029-02-01",{"name":137,"class":41},"Novartis Pharmaceuticals",3,{"id":140,"slug":4,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":141,"targetDuration":4,"studyType":22,"phases":142,"briefSummary":25,"conditions":143,"keywords":144,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":145,"lastUpdatePostDateStruct":146,"startDateStruct":148,"completionDateStruct":149,"leadSponsor":150,"locationsCount":151},"100496491",{"count":20,"type":21},[24],[27],[29],"2026-06-16",{"date":147,"type":34},"2026-06-18",{"date":36,"type":34},{"date":38,"type":21},{"name":40,"class":41},44,{"id":153,"slug":154,"hasResults":11,"nctId":155,"briefTitle":156,"officialTitle":157,"acronym":4,"eligibilityCriteria":158,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":4,"targetDuration":4,"studyType":159,"phases":4,"briefSummary":160,"conditions":161,"keywords":163,"overallStatus":172,"whyStopped":4,"lastUpdateSubmitDate":173,"lastUpdatePostDateStruct":174,"startDateStruct":4,"completionDateStruct":4,"leadSponsor":176,"locationsCount":178},"100514779","danicopan-early-access-program-100514779","NCT05982938","Danicopan Early Access Program","Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)","Key Inclusion Criteria\n\n* Male or female, aged 18 years and older\n* Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5 g\u002FdL) with absolute reticulocyte count ≥ 120 × 109\u002FL.\n* Currently receiving treatment with SOLIRIS® or ULTOMIRIS® for at least 6 months prior to initiation of danicopan treatment.\n* Platelet count ≥ 30,000\u002FµL without the need for platelet transfusions.\n* Absolute neutrophil counts ≥ 500\u002FµL.\n* Vaccinated against N meningitidis.\n\nKey Exclusion Criteria\n\n* Participants who are nursing or pregnant (or women who are planning to become pregnant during treatment with danicopan).\n* Laboratory abnormalities at screening, as follows:\n* Alanine aminotransferase (ALT) \\> 2 × upper limit of normal (ULN)\n* Direct bilirubin \\> 2 × ULN, with the exception of:\n\n  * participants with increased bilirubin due to EVH in the opinion of the Treating Physician or\n  * participants with documented Gilbert's syndrome\n* Active bacterial or viral infection, a body temperature \\> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to initiating danicopan treatment.","EXPANDED_ACCESS","This is an EAP, designed to provide early access to danicopan for participants with PNH experiencing clinically significant EVH who have failed, not tolerated, or are unable to receive other approved treatments; in the Treating Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.",[27,29,162],"Extravascular Hemolysis",[164,29,165,166,167,168,169,170,171],"paroxysmal nocturnal hemoglobinuria","component 5 inhibitors","C5i","intravascular hemolysis","IVH","anemia","EVH","extravascular hemolysis","AVAILABLE","2026-06-03",{"date":175,"type":34},"2026-06-04",{"name":177,"class":41},"Alexion Pharmaceuticals, Inc.",15,{"id":180,"slug":181,"hasResults":11,"nctId":182,"briefTitle":183,"officialTitle":183,"acronym":4,"eligibilityCriteria":184,"healthyVolunteers":11,"sex":17,"minAge":185,"maxAge":186,"enrollmentInfo":187,"targetDuration":4,"studyType":22,"phases":189,"briefSummary":191,"conditions":192,"keywords":196,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":203,"lastUpdatePostDateStruct":204,"startDateStruct":205,"completionDateStruct":207,"leadSponsor":209,"locationsCount":212},"100547786","phase-2-mt2023-20-hematopoietic-cell-transplant-with-reduced-intensity-conditioning-and-post-transplant-cyclophosphamide-for-severe-aplastic-anemia-and-other-forms-of-acquired-bone-marrow-failure-100547786","NCT06412497","MT2023-20: Hematopoietic Cell Transplant With Reduced Intensity Conditioning and Post-transplant Cyclophosphamide for Severe Aplastic Anemia and Other Forms of Acquired Bone Marrow Failure.","Inclusion Criteria:\n\n* Idiopathic Severe Aplastic Anemia (SAA), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL AND bone marrow cellularity \\\u003C50% (with \\\u003C 30% residual hematopoietic cells)\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%), without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic SAA with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Paroxysmal Nocturnal Hemoglobinuria (PNH), including AA-PNH overlap syndrome, acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT), characterized by one of the following:\n\n  1. Refractory cytopenia(s), with 1+ of the following:\n\n     1. Platelets \\\u003C20,000\u002FuL or transfusion dependent\n     2. Absolute neutrophil count \\\u003C500\u002FuL without hematopoietic growth factor support\n     3. Absolute reticulocyte count \\\u003C60,000\u002FuL or red cell transfusion dependent AND Bone marrow evidence of 1 to 3-lineage aplasia OR peripheral blood PNH clone \\>\u002F= 10%\n  2. Early myelodysplastic features (bone marrow (BM) blasts \\\u003C5%) without history of MDS\u002FAML pre-treatment.\n  3. Idiopathic PNH, aPRCA, or aAT with post-HCT graft failure (blood\u002Fmarrow donor chimerism \\\u003C5%) requiring a 2nd allogeneic HCT\n* Adequate organ function within 30 days of conditioning regimen\n\nExclusion Criteria:\n\n* Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 7 days of the start of treatment\n* Uncontrolled infection\n* Evidence of moderate or severe portal fibrosis or cirrhosis on biopsy\n* Known allergy to any of the study components\n* Prior radiation therapy deemed excessive by radiation therapist for proposed low dose TBI exposure on this protocol\n* Diagnosis of an inherited bone marrow failure disorder such as Fanconi anemia, Telomere biology disorder, or Schwachman-Diamond syndrome, unless reviewed by the principal investigator and deemed appropriate for this approach (e.g. GATA2 deficiency)\n* Advanced myelodysplastic syndrome (MDS; BM blasts \\>5%) or acute myeloid leukemia\n* Psychiatric illness\u002Fsocial situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements\n* Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study","0 Years","75 Years",{"count":188,"type":21},60,[190],"PHASE2","A phase II trial of a reduced intensity conditioned (RIC) allogeneic hematopoietic cell transplant (HCT) with post-transplant cyclophosphamide (PTCy) for idiopathic severe aplastic anemia (SAA), paroxysmal nocturnal hemoglobinuria (PNH), acquired pure red cell aplasia (aPRCA), or acquired amegakaryocytic thrombocytopenia (aAT) utilizing population pharmacokinetic (popPK)-guided individual dosing of pre-transplant conditioning and differential dosing of low dose total body irradiation based on age, presence of myelodysplasia and\u002For clonal hematopoiesis.",[193,194,195,27],"Severe Aplastic Anemia","Acquired Amegakaryocytic Thrombocytopenia","Acquired Pure Red Cell Aplasia",[197,198,199,200,201,202],"HCT","RIC","SAA","PTCy","aAT","aPRCA","2026-06-02",{"date":173,"type":34},{"date":206,"type":34},"2024-06-05",{"date":208,"type":21},"2036-05-01",{"name":210,"class":211},"Masonic Cancer Center, University of Minnesota","OTHER",1,{"id":214,"slug":215,"hasResults":11,"nctId":216,"briefTitle":217,"officialTitle":218,"acronym":219,"eligibilityCriteria":220,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":221,"enrollmentInfo":222,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":224,"conditions":225,"keywords":226,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":230,"lastUpdatePostDateStruct":231,"startDateStruct":233,"completionDateStruct":235,"leadSponsor":237,"locationsCount":238},"100587692","a-multi-center-ambispective-cohort-study-to-evaluate-the-impact-of-iptacopan-for-adult-patients-with-pnh-in-china-100587692","NCT06931691","A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China","A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan on Disease Management, Treatment-Related Outcomes and Healthcare Resource Utilization for Adult Patients With Paroxysmal Nocturnal Hemoglobinuria in China","PNH-NIS","Inclusion criteria For Cohort 1,\n\nPatient who meets all the following criteria can be included in this study:\n\n1. Age ≥ 18 years at the time of signing the ICF;\n2. Patient with a documented diagnosis of PNH;\n3. Patient who has never received complement inhibitor therapy;\n4. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;\n5. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation; If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.\n6. Patient who has signed the ICF.\n\nFor Cohort 2,\n\nPatient who meets all the following criteria can be included in this study:\n\n1. Age ≥ 18 years at the time of signing the ICF;\n2. Patient with a documented diagnosis of PNH; Patients who have been receiving stable treatment with C5 complement inhibitors for at least three months prior to enrollment;\n3. Patient who is initiating iptacopan therapy; Patient who is initiating iptacopan therapy must start the first dose within 60 days of signing the ICF;\n4. Documented vaccination against Neisseria meningitidis and Streptococcus pneumoniae and the date of vaccination must be at least 2 weeks prior to the date of iptacopan initiation;\n5. If an urgent prescription for Iptacopan is needed, it is recommended that the antibiotic be used continuously according to the drug label until 14 days after the vaccination is completed, and that the vaccination be completed as soon as possible.\n6. Patient who has signed the ICF. Exclusion criteria\n\nFor Cohort 1 and Cohort 2, patients who meet any of the following criteria will meet the exclusion criteria for this study:\n\n1. Participating in an interventional PNH clinical study;\n2. Have an active systemic bacterial, viral (incl. COVID-19) or fungal infection within 14 days prior to first dose;\n3. Documented with a history of recurrent invasive infections, e.g. active systemic bacterial, viral or fungal infection within 14 days prior to first dose;\n4. Documented with a history of HIV infection;\n5. Women who are pregnant or breastfeeding or intending to conceive during the study period;\n6. Existence of bone marrow failure (reticulocytes \\\u003C 100 × 109\u002FL, platelets \\\u003C 30 × 109\u002FL, and neutrophils \\\u003C 0.5 × 109\u002FL) determined by the investigator;\n7. Other conditions that are not suitable for participating in the study, in the judgment of the investigator.","80 Years",{"count":223,"type":21},80,"The implementation of new standards for the management of PNH and the use of iptacopan in patients with PNH are expected to change the treatment landscape and improve the overall prognosis of patients.\n\nBased on these backgrounds, we plan to conduct a real-world study of iptacopan to further evaluate its impact on treatment-related outcomes, disease management, and healthcare resource utilization in Chinese patients with PNH.",[27],[227,228,27,229],"Phase IV","Adult Patients","LNP023","2026-05-28",{"date":232,"type":34},"2026-06-01",{"date":234,"type":34},"2025-06-10",{"date":236,"type":21},"2027-04-21",{"name":137,"class":41},14,{"id":240,"slug":241,"hasResults":11,"nctId":242,"briefTitle":243,"officialTitle":244,"acronym":4,"eligibilityCriteria":245,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":186,"enrollmentInfo":246,"targetDuration":4,"studyType":22,"phases":248,"briefSummary":250,"conditions":251,"keywords":252,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":256,"lastUpdatePostDateStruct":257,"startDateStruct":259,"completionDateStruct":261,"leadSponsor":263,"locationsCount":212},"100629139","phase-1-study-of-safety-and-efficacy-of-hs-10542-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-100629139","NCT07470762","Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria","A Phase IB\u002FII,Open Label Study to Assess Efficacy, and Safety, of HS-10542 in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) With Signs of Active Hemolysis","Inclusion Criteria:\n\n1. Men or women aged more than or equal to (≥) 18 years, and less than (≤) 75 years.\n2. It was confirmed to be PNH during screening, and the clone size of red blood cells or\u002Fand granulocytes or\u002Fand monocytes was detected by flow cytopy ≥10%\n3. Stable use of C5 complement inhibitor ikuzumab\u002Fcovalimab for the first 6 months of random treatment\n4. Have at least one blood transfusion record within the last 4 months, or sustain a hemoglobin level below 100g\u002FL the last 4 months prior to screening.\n5. The average hemoglobin level from two tests conducted by the laboratory at the time of screening is less than 100 g\u002FL, or hemoglobin level \\\u003C100g\u002FL before transfusion.\n6. LDH \\> 1.5 x Upper Limit of Normal (ULN) at the time of screening\n7. Inoccution of Neisseris meningitis and Streptococcus pneumoniae vaccine at least 2 weeks before the first administration of HS-10542;\n8. if HS-10542 treatment must begin less than 2 weeks after vaccination, preventive antibiotic treatment must begin at least 2 weeks after vaccination.\n9. Male and female subjects with fertility must agree to adopt efficient contraceptive measures with their partners within 60\u002F120 days from the signing of the informed consent form to the last administration,\n10. Male subjects who are infertile (such as those who have undergone effective sterilization surgery) must take additional efficient contraceptive measures when it is uncertain whether they have sperm，\n\nExclusion Criteria:\n\n1. Known or suspected hereditary or acquired complement deficiency\n2. Currently active primary or secondary immunodeficiency\n3. History of infection with pod bacteria (such as Neisseris meningitis, Streptococcus pneumoniae, etc.)\n4. Patients with laboratory evidence of bone marrow failure (reticulocytes \\\u003C100x109\u002FL; platelets \\\u003C30x109\u002FL; neutrophils \\\u003C0.5x109\u002FL);\n5. Presence of a bone marrow failure disorder (e.g., aplastic anemia, myelodysplastic syndrome, myelofibrosis)\n6. Presence of active anemia unrelated to PNH, such as renal anemia or anemia due to blood loss.\n7. There is or is suspected of systemic active bacteria, virus or fungal infection 2 weeks before the first administration of HS-10542 (according to the researcher's judgment)\n8. During screening, there are advanced heart disease (such as NYHA level IV),\n9. unstable thrombosis events that may exist for other causes,\n10. Abnormal ECG: The absolute value of QTcF (QT interval corrected by Fridericia 's formula \\> 450 msec for males and \\> 470 msec for females; or other clinically significant abnormalities as judged by the investigator.\n11. Major surgery within 3 months prior to the first dose. \\*Note: See Appendix for definitions of Grade 3\u002F4 surgeries.\n12. Known active infection requiring systemic therapy\n13. Diagnosed malignant tumors in the past 5 years\n14. Those who have a history of splenectomy or History of bone marrow\u002Fhematopoietic stem cells or solid organ transplantation\n15. Severe or poorly controlled hypertension\n16. poorly controlled diabetes\n17. Those who are suspected of being allergic to experimental drugs or any ingredient in experimental drugs\n18. Use any of the following drugs, unless there is a stable treatment plan before screening: a) erythropoietin (ESA), hypoxic-inducing factor proaminoyl hydroxylase inhibitor (HIF-PHI) or immunosuppressant for at least 8 weeks b) Systemic use of glucocorticoids (≤15 mg\u002Fday Prednisone or equivalent doses of glucocorticoids) at least 4 weeks c) Vitamin K antagonists (such as warfarin) have a stable international standardized ratio (INR) at least 4 weeks d) Low molecular weight heparin, oral anticoagulants such as aspirin, rvaroxaban, apifloxaban, etc. at least 4 weeks e) Iron supplements , vitamin B12, folic acid or androgen for at least 4 weeks\n19. Except for C5 complement inhibitors (including but not limited to ecucizumab and covalizumab), the situation of participating in other clinical trials or using other study drugs or approved therapies for experimental use before screening, and the trial drug is still within 5 half-lives or 2 weeks\n20. Participants who have previously received B-factor inhibitor treatment, with a treatment duration of no more than one week and having stopped taking the drug for more than five half-lives before screening, may not be excluded\n21. During screening, there are serious concurrent diseases, such as severe kidney disease (such as eGFR\\\u003C30 mL\u002Fmin\u002F1.73 m2, dialysis),\n22. ALT\u002FALP\\>3×ULN,\n23. Screening positive blood pregnancy test and breastfeeding women at the time of the visit,",{"count":247,"type":21},50,[249,190],"PHASE1","This was a phase 1b\u002F2,open label, multi-center study to assess efficacy and safety of HS-10542 in adulte patients with paroxysmal nocturnal hemoglobinuria (PNH) with signs of active hemolysis.",[27],[253,254,255],"HS-10542","paroxysmal nocturnal hemoglobinuria（PNH）","CFB","2026-04-09",{"date":258,"type":34},"2026-04-13",{"date":260,"type":34},"2026-02-14",{"date":262,"type":21},"2029-07-31",{"name":264,"class":41},"Jiangsu Hansoh Pharmaceutical Co., Ltd.",{"id":266,"slug":267,"hasResults":11,"nctId":268,"briefTitle":269,"officialTitle":270,"acronym":4,"eligibilityCriteria":271,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":272,"targetDuration":4,"studyType":22,"phases":274,"briefSummary":275,"conditions":276,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":278,"lastUpdatePostDateStruct":279,"startDateStruct":280,"completionDateStruct":282,"leadSponsor":284,"locationsCount":4},"100488934","phase-2-study-of-nm8074-in-adult-pnh-patients-with-inadequate-response-to-soliris-100488934","NCT05646563","Study of NM8074 in Adult PNH Patients With Inadequate Response to Soliris","A Phase II, Open-Label Study of NM8074 in Soliris®-Treated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n* Patients ≥ 18 years (males and females), weight ≥ 45 kg at the time of consent.\n* Confirmation of PNH diagnosis by flow cytometry evaluation white blood cells (WBCs), with neutrophil, granulocyte and\u002For monocyte clone size of ≥10%.\n* Evidence of ongoing hemolysis.\n* ≥1 pRBC transfusion within 12 months prior to screening.\n* Anemia (Hemoglobin ≤10.5 g\u002FdL).\n* Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening.\n* Treatment with Soliris\n* All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics.\n* Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule.\n\nExclusion Criteria:\n\n* Subjects currently or previously under other complement inhibitor treatments other than Soliris less than 3 months prior to study Day 1\n* History of bone marrow, hematopoietic stem cell, or solid organ transplantation\n* History of splenectomy\n* Participation in any other investigational drug trial within 5 elimination half-lives of enrollment, or within 30 days, whichever is longer\n* Participants with known or suspected hereditary or acquired complement deficiency\n* History of currently active primary or secondary immunodeficiency\n* Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections\n* Has a known history of meningococcal disease or N. meningitidis infection\n* Patients on immunosuppressive agents or systemic corticosteroids less than 8 weeks prior to dosing\n* Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study.\n* Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (CKD stage 4, dialysis)\n* Pregnant, planning to become pregnant, or nursing female subjects. Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 week after stopping the investigational drug.\n* Females who have a positive pregnancy test result at Screening or on Day 1.\n* Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to not donate sperm for the duration of the study.",{"count":273,"type":21},12,[190],"This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 in PNH patients undergoing complement-inhibitor therapy with Soliris.",[27],"NOT_YET_RECRUITING","2026-04-07",{"date":258,"type":34},{"date":281,"type":21},"2027-01",{"date":283,"type":21},"2030-08",{"name":285,"class":41},"NovelMed Therapeutics",{"id":287,"slug":288,"hasResults":11,"nctId":289,"briefTitle":290,"officialTitle":291,"acronym":4,"eligibilityCriteria":292,"healthyVolunteers":11,"sex":17,"minAge":293,"maxAge":294,"enrollmentInfo":295,"targetDuration":4,"studyType":22,"phases":297,"briefSummary":298,"conditions":299,"keywords":300,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":304,"lastUpdatePostDateStruct":305,"startDateStruct":307,"completionDateStruct":309,"leadSponsor":311,"locationsCount":312},"100550586","phase-3-study-of-danicopan-as-add-on-treatment-to-ravulizumab-or-eculizumab-in-pediatric-participants-with-pnh-who-have-clinically-significant-extravascular-hemolysis-100550586","NCT06449001","Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis","A Phase 3 Open-Label Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Significant Extravascular Hemolysis","Inclusion Criteria:\n\n* Confirmed diagnosis of PNH.\n* CS-EVH defined by: Anemia: Hgb ≤ 11.0 g\u002FdL, and absolute reticulocyte count ≥ 100 × 109\u002FL\n* Treated with ravulizumab or eculizumab for at least 12 weeks immediately preceding Day 1, the dose received should be stable during this period, and there should be no anticipated changes in dosage or interval during the first 12 weeks of this study.\n* all participants must be vaccinated against meningococcal infection from serogroups A, C, W, and Y and serogroup B within 3 years prior to, or at least 14 days prior to Day 1\n* vaccinated against Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae\n\nExclusion Criteria:\n\n* Platelet count \\\u003C 30000\u002FμL or there is a need for platelet transfusions.\n* ANC \\\u003C 500\u002FμL.\n* Clinically significant laboratory abnormalities related to liver function, including:\n\n  * ALT \\> 2 × ULN or ALT \\> 3 × ULN for participants with documented liver iron overload defined by serum ferritin values ≥ 500 ng\u002FmL.\n  * Direct bilirubin \\> 2 × ULN, unless, in the Investigator's opinion, is due to hemolysis or Gilbert's syndrome based on medical history.\n* Current evidence of biliary cholestasis.\n* Known aplastic anemia or other bone marrow failure that requires HSCT or other therapies, including anti-thymocyte globulin and immunosuppressants unless the dosage of immunosuppressant has been stable for at least 12 weeks before Day 1 and is expected to remain stable through Week 12.\n* History of a major organ transplant (eg, heart, lung, kidney, liver) or HSCT.\n* Known or suspected complement deficiency.\n* Active bacterial or viral infection, a body temperature \\> 38°C on 2 consecutive daily measures, evidence of other infection, or history of any febrile illness within 14 days prior to first study intervention administration.","12 Years","17 Years",{"count":296,"type":21},6,[24],"The primary objective of this study is to evaluate efficacy of danicopan as add-on treatment to ravulizumab or eculizumab as assessed by hemoglobin (Hgb) change from Baseline at Week 12 in pediatric participants with paroxysmal nocturnal hemoglobinuria (PNH) and clinically significant extravascular hemolysis (CS-EVH).",[27,29,162],[27,29,162,301,302,303],"Ravulizumab","Eculizumab","Danicopan","2025-12-19",{"date":306,"type":34},"2025-12-23",{"date":308,"type":34},"2025-08-11",{"date":310,"type":21},"2028-03-10",{"name":177,"class":41},4,{"id":314,"slug":315,"hasResults":11,"nctId":316,"briefTitle":317,"officialTitle":318,"acronym":4,"eligibilityCriteria":319,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":320,"enrollmentInfo":321,"targetDuration":4,"studyType":22,"phases":323,"briefSummary":324,"conditions":325,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":326,"lastUpdatePostDateStruct":327,"startDateStruct":329,"completionDateStruct":330,"leadSponsor":332,"locationsCount":212},"100613405","phase-2-efficacy-safety-and-pharmacokinetics-of-lp-005-injection-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-pnh-100613405","NCT07266155","Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","A Phase Ⅱ Extension Clinical Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n1. Patients have fully understood the trial, have voluntarily agreed to participate in this clinical trial, and have signed a written Informed Consent Form (ICF).\n2. Patients who have completed the treatment of the Phase Ⅱ clinical study of LP-005 Injection and are assessed by the investigator as eligible for continued treatment with LP-005 Injection.\n3. Patients who have received Neisseria meningitidis vaccine and Streptococcus pneumoniae vaccine in accordance with the requirements of previous studies; if the vaccine protection period does not cover the treatment duration of this study, patients must agree to receive booster vaccination in a timely manner in accordance with the vaccine administration guidelines and the requirements of local vaccination institutions.\n4. Females and males of childbearing potential (including male subjects with female partners) must agree to use effective contraceptive measures from the start of the trial until 3 months after the end of the trial.\n\nExclusion Criteria:\n\n1. Patients who have not completed the treatment of the Phase II clinical trial (P10-LP005-02) of the study drug.\n2. Patients who have completed the Phase II treatment phase but are unwilling to continue receiving the study drug treatment.\n3. Patients for whom the investigator does not recommend the continued use of LP-005 Injection after comprehensive assessment.\n4. Patients who did not participate in the Phase II clinical trial (P10-LP005-02) of the study drug.","65 Years",{"count":322,"type":21},30,[190],"This is an extension study for patients who have completed a prior P10-LP005-02 clinical study. The aim of this study is to evaluate the long-term safety, efficacy, and pharmacokinetics of LP-005 injection in adult patients with paroxysmal nocturnal hemoglobinuria (PNH).",[27],"2025-11-24",{"date":328,"type":34},"2025-12-05",{"date":328,"type":21},{"date":331,"type":21},"2028-05-01",{"name":333,"class":41},"Longbio Pharma",{"id":335,"slug":336,"hasResults":11,"nctId":337,"briefTitle":338,"officialTitle":339,"acronym":4,"eligibilityCriteria":340,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":341,"targetDuration":4,"studyType":22,"phases":343,"briefSummary":344,"conditions":345,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":346,"lastUpdatePostDateStruct":347,"startDateStruct":349,"completionDateStruct":351,"leadSponsor":353,"locationsCount":212},"100606615","phase-3-a-phase-iii-study-of-ntq5082-capsules-in-the-treatment-of-paroxysmal-nocturnal-hemoglobinuria-patients-100606615","NCT07177859","A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients","A Multicenter, Randomized, Open-label, Active-controlled Phase III Clinical Trial Evaluating the Efficacy and Safety of NTQ5082 Capsules in the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria","Inclusion Criteria:\n\n\\- 1) Age ≥ 18 years, regardless of gender. 2) Subjects diagnosed with PNH according to the PNH diagnostic criteria in the Chinese \"Guidelines for the Diagnosis and Treatment of Rare Diseases\" (2019 edition), with PNH erythrocyte and\u002For granulocyte clone levels \\> 10% within 6 months prior to screening or during the screening period.\n\n3\\) PNH patients who have not previously received complement inhibitor therapy. 4) Laboratory test (central laboratory) hemoglobin meets one of the following conditions: (1) The average hemoglobin value during screening period V1 and V2 (interval ≥ 7 days) is \\\u003C100g\u002FL; (2) If the subject receives red blood cell transfusion for PNH-related anemia during the screening period, the hemoglobin value at V1 must be \\\u003C100g\u002FL; 5) Blood lactate dehydrogenase (LDH) (central laboratory) is \\>1.5×upper limit of normal (ULN) during screening period V1 and V2 (interval ≥ 7 days);\n\nExclusion Criteria:\n\n* 1\\) During the screening period, laboratory (local laboratory) results show bone marrow failure (reticulocyte count \\\u003C100×109\u002FL, or platelet count \\\u003C30×109\u002FL \\[must not have received platelet transfusion within 7 days prior to the laboratory test\\], or neutrophil count \\\u003C0.5×109\u002FL \\[must not have received short-acting granulocyte colony-stimulating factor within 14 days or long-acting granulocyte colony-stimulating factor within 28 days prior to the laboratory test\\]).\n\n  2\\) During the screening period, laboratory (local laboratory) results show alanine aminotransferase (ALT), alkaline phosphatase (ALP), or gamma-glutamyltransferase (GGT) \\>3×ULN and the investigator determines that the patient is not suitable for study participation.\n\n  3\\) Patients were receiving any of the following medications before screening, and the duration of treatment at a stable dose of the drug: (1) systemic glucocorticoids for less than 4 weeks; or prednisone or equivalent dose of glucocorticoids at a dose of \\>15 mg\u002Fday; (2) iron, vitamin B12, folic acid, or androgens for less than 4 weeks; (3) vitamin K antagonists (such as warfarin) with a stable international normalized ratio (INR) for less than 4 weeks; (4) low molecular weight heparin, oral anticoagulants (such as aspirin, rivaroxaban, edoxaban, apixaban) for less than 4 weeks; (5) erythropoietin (ESA), hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHI), or immunosuppressants (including but not limited to cyclosporine, tacrolimus, mycophenolate mofetil or mycophenolic acid, cyclophosphamide, methotrexate, etc.) for less than 8 weeks.\n\n  4\\) Previous history of bone marrow\u002Fhematopoietic stem cell or solid organ transplantation (such as heart, lung, kidney, liver). 5) Patients with a history of splenectomy or planned surgery during the trial.\n\n  6\\) Patients with a history of recurrent invasive infection with encapsulated bacteria (such as meningococci, Streptococcus pneumoniae, etc.), a history of systemic anti-tuberculosis treatment or current tuberculosis infection, or a history of active systemic bacterial, viral, or fungal infection within 14 days prior to the first administration of the investigational drug (defined as D1) (as determined by the investigator).",{"count":342,"type":21},78,[24],"NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, randomized, open-label, active-controlled Phase III clinical trial designed to evaluate the efficacy and safety of NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal hemoglobinuria.",[27],"2025-09-10",{"date":348,"type":34},"2025-09-17",{"date":350,"type":21},"2025-11",{"date":352,"type":21},"2026-11",{"name":354,"class":41},"Nanjing Chia-tai Tianqing Pharmaceutical",{"id":356,"slug":357,"hasResults":11,"nctId":358,"briefTitle":359,"officialTitle":360,"acronym":4,"eligibilityCriteria":361,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":362,"targetDuration":4,"studyType":22,"phases":363,"briefSummary":364,"conditions":365,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":346,"lastUpdatePostDateStruct":366,"startDateStruct":367,"completionDateStruct":369,"leadSponsor":371,"locationsCount":212},"100606616","phase-3-a-long-term-efficacy-and-safety-of-ntq5082-capsules-100606616","NCT07177872","A Long-term Efficacy and Safety of NTQ5082 Capsules","A Multicenter, Open-label Study Evaluating the Long-term Efficacy and Safety of NTQ5082 Capsules in the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria","Inclusion Criteria:\n\n* 1\\) Age ≥ 18 years, regardless of gender. 2) Subjects who have previously received and completed NTQ5082 study treatment and, as determined by the investigator, have benefited from treatment and are likely to benefit from continued NTQ5082 treatment, or subjects in the control group receiving eculizumab.\n\n  3\\) Subjects who have received ACYW135 group meningococcal and pneumococcal vaccines as required by the previous protocol, and whose vaccine protection period, as determined by the investigator, falls within the treatment duration of this study.\n\n  4\\) Subjects who agree to use at least one effective contraceptive method during sexual intercourse with their partner from the time they sign the informed consent form until 4 weeks after the last dose, refrain from cryopreservation of sperm or eggs, and refrain from sperm or egg donation.\n\n  5\\) Subjects who agree to sign the informed consent form and commit to abide by all study regulations.\n\nExclusion Criteria:\n\n* 1\\) Previous history of bone marrow\u002Fhematopoietic stem cell or solid organ transplant (e.g., heart, lung, kidney, liver).\n\n  2\\) Previous history of splenectomy. 3) Previous history of recurrent invasive infection with encapsulated bacteria (e.g., meningococci, Streptococcus pneumoniae), previous history of systemic anti-tuberculosis treatment, or current tuberculosis infection.\n\n  4\\) History of malignancy within 5 years prior to screening, excluding cured localized basal cell carcinoma of the skin, squamous cell carcinoma of the skin, papillary thyroid carcinoma, or carcinoma in situ of the cervix.\n\n  5\\) Known or suspected hereditary complement deficiency or primary or severe secondary immunodeficiency. 6) Patients with a history of clinically significant kidney, heart, liver, or lung disease who are deemed unsuitable for participation by the investigator, including but not limited to severe kidney disease (e.g., eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m²), advanced heart disease (e.g., NYHA class IV), or severe lung disease (e.g., severe pulmonary hypertension (WHO class IV).\n\n  7\\) Patients suspected of being allergic to the study drug or any component of the study drug.\n\n  8\\) Pregnant or lactating female subjects. 9) Other conditions deemed unsuitable for participation by the investigator.",{"count":342,"type":21},[24],"NTQ5082 capsules are a small molecule CFB inhibitor. This study is a multicenter, open-label study evaluating the long-term efficacy and safety of NTQ5082 capsules in the treatment of patients with paroxysmal nocturnal hemoglobinuria",[27],{"date":348,"type":34},{"date":368,"type":21},"2026-06",{"date":370,"type":21},"2028-06",{"name":354,"class":41},{"id":373,"slug":374,"hasResults":11,"nctId":375,"briefTitle":376,"officialTitle":377,"acronym":4,"eligibilityCriteria":378,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":116,"enrollmentInfo":379,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":381,"conditions":382,"keywords":383,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":391,"lastUpdatePostDateStruct":392,"startDateStruct":394,"completionDateStruct":396,"leadSponsor":397,"locationsCount":212},"100547719","home-reported-outcomes-in-pnh-100547719","NCT06411626","Home Reported Outcomes in PNH","Home Reported Outcomes in PNH: A Mobile App-Based, Prospective, Observational Program to Evaluate Disease Burden and Treatment Patterns in Paroxysmal Nocturnal Hemoglobinuria in the US","Inclusion Criteria:\n\nStudy participants eligible for inclusion in this study must meet all of the following criteria:\n\n* Aged 18 or older\n* US-based with a proficient understanding of and ability to read the English language\n* Any patient with a diagnosis of PNH, regardless of symptom or treatment history\n\nExclusion Criteria:\n\nStudy participants who do not fit all inclusion criteria listed above are unable to participate in this study. Outside of required inclusion criteria, there are no other exclusion criteria in order to meet the exploratory nature of the primary endpoint.",{"count":380,"type":21},128,"The study aims to longitudinally capture the full spectrum of symptoms, treatment utilization, and overall Health-Related Quality of Life (HRQoL) experienced by PNH patients. By primarily utilizing home reported outcomes (HRO) data on symptom burden and treatment usage, supplemented with patient-reported outcome (PRO) measures, the study seeks to establish a new real-world data (RWD) source to understand symptom variability and HRQoL among PNH patients, including those receiving orally administered iptacopan.",[27],[384,29,385,386,387,388,389,390],"Paroxysmal nocturnal hemoglobinuria","home reported outcomes","HRO","patient-reported outcome","PRO","real-world data","iptacopan","2025-08-26",{"date":393,"type":34},"2025-08-27",{"date":395,"type":34},"2024-06-11",{"date":133,"type":21},{"name":137,"class":41},{"id":399,"slug":400,"hasResults":11,"nctId":401,"briefTitle":402,"officialTitle":403,"acronym":4,"eligibilityCriteria":404,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":186,"enrollmentInfo":405,"targetDuration":4,"studyType":22,"phases":407,"briefSummary":408,"conditions":409,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":410,"lastUpdatePostDateStruct":411,"startDateStruct":413,"completionDateStruct":415,"leadSponsor":417,"locationsCount":212},"100587752","phase-3-study-of-safety-and-efficacy-of-my008211a-in-patients-with-residual-anemia-despite-anti-c5-antibody-treatment-100587752","NCT06932471","Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment","A Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of MY008211A Tablets in Patients With PNH and Residual Anemia, Despite Treatment With Anti-C5 Antibody.","Inclusion Criteria:\n\n1. 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%.\n2. Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still \\\u003C 100 g\u002FL.\n3. The average hemoglobin level of at least two tests in 4 months before screening \\\u003C 100 g\u002FL.\n4. The average hemoglobin level of two tests in the central laboratory during screening \\\u003C 100 g\u002FL.\n5. 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\n1. Patients with reticulocytes \\\u003C100x10\\^9\u002FL; platelets \\\u003C30x10\\^9\u002FL; neutrophils \\\u003C0.5x10\\^9\u002FL.\n2. History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.\n3. Known or suspected hereditary complement deficiency.\n4. Previous bone marrow or hematopoietic stem cell transplantation.\n5. Previous splenectomy.\n6. 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":406,"type":21},20,[24],"The main purpose of this study is to evaluate the efficacy of MY008211A in PNH patients with residual anemia despite treatment with anti-C5 antibody.",[27],"2025-05-05",{"date":412,"type":34},"2025-05-09",{"date":414,"type":34},"2024-08-30",{"date":416,"type":21},"2025-12-30",{"name":418,"class":41},"Wuhan Createrna Science and Technology Co., Ltd",{"id":420,"slug":421,"hasResults":11,"nctId":422,"briefTitle":423,"officialTitle":424,"acronym":4,"eligibilityCriteria":425,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":426,"targetDuration":4,"studyType":22,"phases":427,"briefSummary":428,"conditions":429,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":430,"lastUpdatePostDateStruct":431,"startDateStruct":433,"completionDateStruct":435,"leadSponsor":437,"locationsCount":4},"100488931","phase-2-study-of-efficacy-and-safety-of-nm8074-in-adult-pnh-patients-who-are-naive-to-complement-inhibitor-therapy-100488931","NCT05646524","Study of Efficacy and Safety of NM8074 in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy","A Phase II, Open-Label Study of NM8074 in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n* Patients ≥ 18 years (males and females), weight ≥ 45 kg at the time of consent\n* Confirmation of PNH diagnosis by flow cytometry evaluation of white blood cells (WBCs), with neutrophil, granulocyte and\u002For monocyte clone size of ≥10%\n* Evidence of ongoing hemolysis\n* ≥1 packed red blood cell (pRBC) transfusion within 12 months prior to screening\n* Anemia (Hemoglobin ≤10.5 g\u002FdL)\n* Lactate dehydrogenase (LDH) level ≥ 1.5 times the upper limit of normal (xULN) during Screening\n* All patients must be vaccinated prior to dosing with MenACWY Menactra® polysaccharide diphtheria toxoid conjugate vaccination against Neisseria meningitidis serogroups A, C, Y, and W-135 and MenB meningococcal serogroup B vaccine (Bexsero®). If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics\n* Willing and able to understand and complete informed consent procedures, including signing and dating the informed consent form (ICF), and comply with the study visit schedule\n\nExclusion Criteria:\n\n* History of bone marrow, hematopoietic stem cell, or solid organ transplantation\n* History of splenectomy\n* Participation in any other investigational drug trial within 5 elimination half-lives of enrollment, or within 30 days, whichever is longer\n* Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1\n* Participants with known or suspected hereditary or acquired complement deficiency\n* History of currently active primary or secondary immunodeficiency\n* Currently active systemic infection or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections\n* Has a known history of meningococcal disease or N. meningitidis infection\n* Patients on immunosuppressive agents or systemic corticosteroids less than 8 weeks prior to dosing\n* Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study\n* Severe concurrent co-morbidities not amenable to active treatment, e.g., patients with severe kidney disease (chronic kidney disease (CKD) stage 4, dialysis)\n* Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1\n* Pregnant, planning to become pregnant, or nursing female subjects. Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for 1 week after stopping the investigational drug\n* Females who have a positive pregnancy test result at Screening or on Day 1\n* Male patients and partners of child-bearing potential must agree to use contraceptives and male patients must agree to refrain from donating sperm for the duration of the study",{"count":273,"type":21},[190],"This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).",[27],"2025-03-06",{"date":432,"type":34},"2025-03-11",{"date":434,"type":21},"2026-04",{"date":436,"type":21},"2028-09",{"name":285,"class":41},{"id":439,"slug":440,"hasResults":11,"nctId":441,"briefTitle":442,"officialTitle":443,"acronym":4,"eligibilityCriteria":444,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":445,"targetDuration":447,"studyType":75,"phases":4,"briefSummary":448,"conditions":449,"keywords":450,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":453,"lastUpdatePostDateStruct":454,"startDateStruct":456,"completionDateStruct":458,"leadSponsor":460,"locationsCount":212},"100497332","global-pnh-patient-registry-100497332","NCT05755867","Global PNH Patient Registry","Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry","Inclusion Criteria:\n\n\\- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. PNH is defined as a genetic mutation in the PIG-A gene.\n\nIndividuals must be willing to provide informed consent. Participants can be:\n\n* legal adult participants who are able to provide their own consent;\n* children and adults unable to provide their own consent, for whom consent must be provided by a Legally Authorized Representative (LAR) who is a legal adult.\n* Individuals must have at least periodic access to the internet and be able to comply with web-based study procedures and data collections\n\nExclusion Criteria:\n\n* Individuals not able to read and understand English.",{"count":446,"type":21},500,"10 Years","The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following:\n\n* Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH.\n* Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials).\n* Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics.\n* Assist the PNH community with the development of recommendations and standards of care.\n* Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.",[27],[29,451,452,27],"Registry","Patient Registry","2025-02-18",{"date":455,"type":34},"2025-02-20",{"date":457,"type":34},"2021-05-06",{"date":459,"type":21},"2031-05-06",{"name":461,"class":211},"Aplastic Anemia and MDS International Foundation",{"id":463,"slug":464,"hasResults":11,"nctId":465,"briefTitle":466,"officialTitle":467,"acronym":4,"eligibilityCriteria":468,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":469,"targetDuration":4,"studyType":22,"phases":471,"briefSummary":472,"conditions":473,"keywords":4,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":474,"lastUpdatePostDateStruct":475,"startDateStruct":477,"completionDateStruct":479,"leadSponsor":481,"locationsCount":212},"100573387","phase-2-long-term-safety-and-tolerability-of-hsk39297-in-patients-with-paroxysmal-nocturnal-hemoglobinuria-100573387","NCT06745622","Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria","A Multicenter, Open-label Study to Evaluate the Long-term Safety, Tolerability and Efficacy of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria（PNH）","Inclusion Criteria:\n\n1. Patients With PNH who have previously received and completed HSK39297 treatment , and According to the researchers' judgment, the benefits of treatment outweigh the risks and may benefit from continued treatment with HSK39297;\n2. Understand the study procedures and methods, voluntarily participate in this trial.\n\nExclusion Criteria:\n\n1. Hereditary or acquired complement deficiency;\n2. Active primary or secondary immunodeficiency;\n3. History of splenectomy, bone marrow\u002F hematopoietic stem cell or solid organ transplants;\n4. History of recurrent invasive infections caused by encapsulated organisms( e.g. meningococcus or pneumococcus) or Mycobacterium tuberculosis;\n5. History of serious comorbidities that have been determined to be unsuitable for participation in the study;\n6. Pregnant or Lactating women.",{"count":470,"type":21},47,[190],"This is a multicenter, open-label study to evaluate the long-term safety, tolerability and efficacy of HSK39297. Adult patients with PNH who had previously received and completed HSK39297 study treatment will be included. Eligible subjects can maintain HSK39297 treatment until the end of the study.",[27],"2024-12-17",{"date":476,"type":34},"2024-12-20",{"date":478,"type":21},"2025-01-03",{"date":480,"type":21},"2027-06",{"name":482,"class":41},"Haisco Pharmaceutical Group Co., Ltd.",{"id":484,"slug":485,"hasResults":11,"nctId":486,"briefTitle":487,"officialTitle":488,"acronym":4,"eligibilityCriteria":489,"healthyVolunteers":490,"sex":17,"minAge":18,"maxAge":491,"enrollmentInfo":492,"targetDuration":4,"studyType":22,"phases":493,"briefSummary":494,"conditions":495,"keywords":4,"overallStatus":277,"whyStopped":4,"lastUpdateSubmitDate":496,"lastUpdatePostDateStruct":497,"startDateStruct":499,"completionDateStruct":501,"leadSponsor":503,"locationsCount":212},"100557849","phase-1-to-evaluate-the-effect-of-single-oral-dose-of-my008211a-tablets-on-qtc-interval-in-healthy-subjects-100557849","NCT06543459","To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects","A Single-center, Randomized, Double-blind, Single Oral Dose, Placebo-controlled Study to Evaluate the Effect of MY008211A Tablets on QTc Interval in Healthy Chinese Adult Subjects","Inclusion Criteria:\n\n1. Volunteers must be fully informed of this study and the content, process of the study and possible adverse events related to experimental drug will be fully understood, and voluntarily signed a written Informed Consent Form (ICF);\n2. 18≤ age ≤ 45 years old, male or female Chinese adult volunteers;\n3. Body weight: ≥50 kg for male, ≥45 kg for female; body mass index (BMI): 19.0-26.0 kg\u002Fm2 (inclusive) at screening;\n4. Volunteers should be able to communicate well with the investigator, understand and comply with the requirements of the study.\n\nExclusion Criteria:\n\n1. The investigator judges that there are other disease or medical conditions that are clinical significant or may prevent the volunteer from following the study protocol and completing the study, abnormal including but not limited to central nervous system, cardiovascular system, digestive system, respiratory system, urinary system, hematological system, immune system, mental system, endocrine and metabolic system;\n2. Volunteers with chronic or active gastrointestinal diseases such as esophageal disease, gastritis, gastric ulcer, enteritis, active gastrointestinal bleeding, or gastrointestinal surgery within the past three years and judged by the investigator to have clinical significance at present;\n3. Volunteers with torsades de pointes (TDP) or other risk factors for malignant arrhythmias, or short QT syndrome, long QT syndrome, sudden unexplained death or drowning in youth (≤ 40 years old), or a family history of a first-degree relative (i.e., biological parent, sibling, or child) of sudden infant death syndrome, or other heart disease that the investigators determined was not suitable for inclusion;\n4. Volunteers with hyperkalemia, hypokalemia, hypermagnesia, hypomagnesemia, hypercalcemia or hypocalcemia and judged by the investigator to have clinical significance;\n5. History of known or suspected immunodeficiency (e.g., history of frequent recurrent infections), inherited or acquired complement deficiency；\n6. Volunteers who underwent surgery within 6 months pre-dose, which judged by the investigator to affect the absorption, distribution, metabolism, and excretion of the experimental drug(e.g. cholecystectomy, except appendicitis surgery); Surgical procedures within 4 weeks pre-dose or planned to undergo a surgical procedure during the trial;\n7. Volunteers who had a clear history of capsular microbial infection within 6 months before screening; Including but not limited to: Streptococcus pneumoniae, Bacillus anthracis, Salmonella, Salmonella typhi, Klebsiella pneumoniae, Pseudomonas aeruginosa, Bacteroides fragilis, Neisseria meningitidis, Haemophilus influenzae, Legionella pneumophila infection history;\n8. Volunteers with previous or current history of TB infection or with positive lymphobacteria culture + interferon test；\n9. Active systemic bacterial, viral, or fungal infection within 14 days pre-dose;\n10. Fever (≥ 38 ℃) within 7 days pre-dose;\n11. Volunteers with a history of clinically significant drug allergy or allergic disease (such as asthma, urticaria, eczematous dermatitis, etc.), or a possible or clear allergy to the experimental drug (including similar drugs) or any excipients thereof as judged by the investigator;\n12. Volunteers with clinical significant examination abnormalities which judged by the investigator such as vital signs, physical examination, routine laboratory tests (can reviewable, including: blood routine, reticulocyte count, procalcitonin + myoglobin, blood biochemical + hypersensitive C-reactive protein, urine routine, coagulation function + plasma D-Dimer determination), chest X-ray, abdominal ultrasound at screening or baseline；\n13. Volunteers with 12-ECG examination and reviewable result such as QTcF≥450 ms or PR interval ≥200ms or QRS wave complex ≥120ms or clinical significant abnormality ECG which judged by the investigator at screening or baseline or pre-dose；\n14. Volunteers who administrated any other clinical study drug or enrolled in any Interventional clinical trial within 3 months before screening；\n15. Volunteers who donated blood or lost blood (≥ 400mL) within 3 months pre-dose, received a blood transfusion or use of blood products within 4 weeks pre-dose, or intended to donate blood or blood components during or within 3 months after study;\n16. Volunteers who had taken any medicine or OTC medicine or Chinese herbal medicine or food supplement (including vitamins, health foods, etc.) other non-drug therapeutic factors that affect drug absorption, distribution, metabolism and excretion;\n17. Volunteers who received a vaccine or live attenuated vaccine within 14 days pre-dose, or who plan to receive a vaccine during the study;\n18. Volunteers with difficulty in blood collection or cannot tolerate intravenous puncture;\n19. Volunteers with a history of drug use or substance abuse or with positive urine drug abuse screening test;\n20. Volunteers with any positive result of virological test;\n21. Volunteers who drink more than 14 units a week (one unit is defined as 360mL of beer or 45mL of liquor or 150mL of wine) within 3 months before screening or with positive result of alcohol breath test or unwillingness to stop drinking alcohol or any alcohol-based product during the study；\n22. Volunteers who smoke more than 5 cigarettes per day(or use a significant amount of nicotine products) within 3 months before screening or will be unable to stop using any tobacco products during the study or with positive result of urine nicotine test；\n23. Volunteers who habitual consumption of grapefruit juice or excessive amounts of tea, coffee and\u002For caffeinated beverages and will be unable to stop use during the study, or consume any food or drink containing chocolate, caffeine, or rich in xanthines within 48h pre-dose；\n24. Volunteers who have special requirements for diet and cannot abide by the uniform diet；\n25. Volunteers (or their partners) who plan to be pregnant or donate sperm or eggs during the study to 3 months after the end of the study, or who are unwilling to take one or more non-drug contraceptive measures (such as complete abstinence, condoms, contraceptive rings, surgical sterilization, etc.);\n26. Volunteers of pregnant or lactating women; or having unprotected sex within 2 weeks pre-dose; or oral contraceptive use within 30 days or long-acting estrogen or progestin injectable or implant use within 6 months pre-dose; or with positive blood pregnancy test pre-dose;\n27. Volunteers have other reasons for not fit for participating in the study as judged by the investigator.",true,"45 Years",{"count":64,"type":21},[249],"A Concentration-QT Interval Correction (C-QTc) study of MY008211A Tablets in Healthy Subjects",[27],"2024-08-06",{"date":498,"type":34},"2024-08-09",{"date":500,"type":21},"2024-08-10",{"date":502,"type":21},"2024-12-13",{"name":418,"class":41},{"id":505,"slug":506,"hasResults":11,"nctId":507,"briefTitle":508,"officialTitle":508,"acronym":4,"eligibilityCriteria":509,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":510,"targetDuration":4,"studyType":75,"phases":4,"briefSummary":512,"conditions":513,"keywords":514,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":522,"lastUpdatePostDateStruct":523,"startDateStruct":525,"completionDateStruct":527,"leadSponsor":529,"locationsCount":212},"100556409","the-international-pnh-interest-group-pnh-registry-100556409","NCT06524726","The International PNH Interest Group PNH Registry","Inclusion Criteria:\n\n* Patients with PNH confirmed by flow cytometry.\n* Patient and\u002For parent\u002Flegally authorized representative provide written informed consent\u002Fassent to participate in the registry in a manner approved by the Institutional Review Board\u002FIndependent Ethics Committee and local regulations.\n\nExclusion Criteria:\n\n* Participating in an interventional PNH clinical trial. Note: A patient included in the registry, who enrolls in an interventional PNH clinical trial during the course of the registry, will be kept in the registry but data collection will be paused in the registry during their involvement in the clinical trial\u002Fextension study. Data collection in the registry will continue after patient involvement in the clinical trial\u002Fextension study has ended or trial protocol mandated data collection ceases.",{"count":511,"type":21},2000,"The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and health-resource utilization (HRU) on all enrolled patients, as well as long term safety data.",[27],[29,451,452,27,515,516,517,518,519,520,521],"Anemia, Hemolytic","Anemia","Hematologic Diseases","Myelodysplastic Syndromes","Bone Marrow Diseases","Hemoglobinuria","Hemoglobinuria, Paroxysmal","2024-07-23",{"date":524,"type":34},"2024-07-29",{"date":526,"type":34},"2024-05-10",{"date":528,"type":21},"2029-05-10",{"name":530,"class":211},"International PNH Interest Group",{"id":532,"slug":533,"hasResults":11,"nctId":534,"briefTitle":535,"officialTitle":536,"acronym":4,"eligibilityCriteria":537,"healthyVolunteers":490,"sex":17,"minAge":18,"maxAge":116,"enrollmentInfo":538,"targetDuration":4,"studyType":22,"phases":540,"briefSummary":541,"conditions":542,"keywords":543,"overallStatus":30,"whyStopped":4,"lastUpdateSubmitDate":544,"lastUpdatePostDateStruct":545,"startDateStruct":547,"completionDateStruct":549,"leadSponsor":551,"locationsCount":553},"100539062","phase-2-long-term-safety-tolerability-and-efficacy-of-oms906-in-paroxysmal-nocturnal-hemoglobinuria-100539062","NCT06298955","Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria","An Open-Label Study to Evaluate the Long-Term Safety, Tolerability and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)","Inclusion Criteria:\n\n1. Have completed the last dosing visit of the prior OMS906 PNH study.\n2. Female patients of child bearing potential must have a negative result from a highly sensitive urine pregnancy test prior to each dose of OMS906.\n3. Females must use highly effective birth control to prevent pregnancy during the clinical trial and for 20 weeks following their last dose of study drug.\n4. Males must use highly effective birth control with a female partner to prevent pregnancy during the clinical trial and for 20 weeks after last dose of study drug.\n5. Have current vaccination status for Neisseria meningitidis, Streptococcus pneumonia and Hemophilus influenza and agree to maintain vaccination throughout the study.\n6. Have provided informed consent\n\nExclusion Criteria:\n\n1. Platelet count \\\u003C30,000\u002FµL or absolute neutrophil count \\\u003C500 cells\u002FµL at the start of the Evaluation Period.\n2. Elevation of liver function tests, defined as total bilirubin \\> 2 x ULN, direct bilirubin \\> 1.5 x ULN, and elevated transaminases (alanine or aspartate aminotransferase), \\> 2 X ULN unless due to PNH-related hemolysis.\n3. History of any severe hypersensitivity reactions to other monoclonal antibodies or excipients included in the OMS906 preparation.\n4. Patients with unresolved serious infections caused by encapsulated bacteria including H. influenzae, S. pneumoniae and N. meningitidis.\n5. Pregnant, planning to become pregnant, or nursing female patients.\n6. History of any significant medical, neurologic, or psychiatric disorder that in the opinion of the investigator would make the patient unsuitable for participation in the long-term extension.\n7. Unable or unwilling to comply with the requirements of the study.",{"count":539,"type":21},25,[190],"The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg\u002Fkg IV administration at 8-week intervals in patients with PNH.",[27],[29],"2024-03-01",{"date":546,"type":34},"2024-03-07",{"date":548,"type":34},"2024-02-19",{"date":550,"type":21},"2027-04",{"name":552,"class":41},"Omeros Corporation",5]