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Clinical trials test new treatments โ€” sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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30 recruiting trials

for PNH - Paroxysmal Nocturnal Hemoglobinuria

Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT06933914Phase 2, Phase 31 site๐Ÿ“ Tianjin Wuhan Createrna Science and Technology Co., Ltd ยท Tests: MY008211A tablets Global PNH Patient Registry NCT05755867n/a1 site๐Ÿ“ Bethesda Aplastic Anemia and MDS International Foundation Home Reported Outcomes in PNH NCT06411626n/a1 site๐Ÿ“ East Hanover Novartis Pharmaceuticals ยท Tests: PNH-relevant therapies Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients NCT06934967Phase 315 sites๐Ÿ“ Atlanta๐Ÿ“ New Brunswick๐Ÿ“ Philadelphia Novartis Pharmaceuticals ยท Tests: LNP023 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. NCT06412497Phase 21 site๐Ÿ“ Minneapolis Masonic Cancer Center, University of Minnesota ยท Tests: Rituximab, Rabbit ATG, Cyclophosphamide, Fludarabine, Total Body Irradiation Enhancing the Clinical Management of CMF Through a National Pediatric and Adult Network Based on Biological and Genetic Data NCT07877727n/a4 sites๐Ÿ“ Avellino๐Ÿ“ Genova๐Ÿ“ Naples Istituto Giannina Gaslini Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT07470762Phase 1, Phase 21 site๐Ÿ“ Hangzhou Jiangsu Hansoh Pharmaceutical Co., Ltd. ยท Tests: HS-10542, HS-10542, HS-10542 REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria NCT07229235n/a12 sites๐Ÿ“ Alessandria๐Ÿ“ Avellino๐Ÿ“ Brescia Novartis Pharmaceuticals A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants NCT07462780Phase 11 site๐Ÿ“ Beijing Shenzhen Kangzhe Biotechnology Co., Ltd. ยท Tests: CMS-D017 Capsule, CMS-D017 Placebo Capsule, CMS-D017 Capsule, CMS-D017 Placebo Capsule Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy NCT06932744Phase 32 sites๐Ÿ“ Beijing๐Ÿ“ Tianjin Wuhan Createrna Science and Technology Co., Ltd ยท Tests: MY008211A tablets, Eculizumab Injection Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis NCT03520647Phase 21 site๐Ÿ“ Bethesda National Heart, Lung, and Blood Institute (NHLBI) ยท Tests: Cyclophosphamide, Peripheral Blood Stem Cells Danicopan PMS in Korea NCT07457151n/a4 sites๐Ÿ“ Busan๐Ÿ“ Hwasun-gun๐Ÿ“ Seoul AstraZeneca To Evaluate Safety, Tolerability, PK/PD & Food Effect of Single/Multiple Oral TUL321 Capsules in Healthy Participants. NCT07760467Phase 11 site๐Ÿ“ Changsha Zhuhai United Laboratories Co., Ltd. ยท Tests: Part1: TUL321, Part1: Placebo, Part2: TUL321, Part2: Placebo 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 NCT07187401Phase 1, Phase 28 sites๐Ÿ“ Toronto๐Ÿ“ Suwon๐Ÿ“ Seoul Regeneron Pharmaceuticals ยท Tests: ALN-CFB, Placebo Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment NCT06932471Phase 31 site๐Ÿ“ Beijing Wuhan Createrna Science and Technology Co., Ltd ยท Tests: MY008211A tablets 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 NCT05744921Phase 348 sites๐Ÿ“ Sรฃo Paulo๐Ÿ“ Toronto๐Ÿ“ Medellรญn Regeneron Pharmaceuticals ยท Tests: Pozelimab, Cemdisiran Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment NCT07152288Phase 11 site๐Ÿ“ Beijing Haisco Pharmaceutical Group Co., Ltd. ยท Tests: HSK39297 tablets, HSK39297 tablets, HSK39297 tablets Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis NCT06449001Phase 34 sites๐Ÿ“ Saskatoon๐Ÿ“ Paris๐Ÿ“ Leeds Alexion Pharmaceuticals, Inc. ยท Tests: Danicopan Study of Ultomirisยฎ (Ravulizumab) Safety in Pregnancy NCT06312644n/a7 sites๐Ÿ“ Boston๐Ÿ“ Melbourne๐Ÿ“ Paris Alexion Pharmaceuticals, Inc. ยท Tests: Ultomiris National Longitudinal Cohort of Hematological Diseases NCT04645199n/a1 site๐Ÿ“ Tianjin Institute of Hematology & Blood Diseases Hospital, China Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria NCT06745622Phase 21 site๐Ÿ“ Nanjing Haisco Pharmaceutical Group Co., Ltd. ยท Tests: HSK39297 tablets A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT06978699Phase 21 site๐Ÿ“ Tianjin S-INFINITY Pharmaceuticals Co., Ltd ยท Tests: XH-S003 Capsule A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT04901936Phase 212 sites๐Ÿ“ Atlanta๐Ÿ“ Prague๐Ÿ“ Paris Apellis Pharmaceuticals, Inc. ยท Tests: Pegcetacoplan Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients NCT05876312Phase 1, Phase 24 sites๐Ÿ“ Brisbane๐Ÿ“ Melbourne๐Ÿ“ Parkville ADARx Pharmaceuticals, Inc. ยท Tests: ADX-038, Placebo A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China NCT06931691n/a14 sites๐Ÿ“ Hefei๐Ÿ“ Guangzhou๐Ÿ“ Guangzhou Novartis Pharmaceuticals ยท Tests: LNP023 The International PNH Interest Group PNH Registry NCT06524726n/a1 site๐Ÿ“ Altamonte Springs International PNH Interest Group Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells NCT01174108Phase 22 sites๐Ÿ“ Baltimore๐Ÿ“ Bethesda National Heart, Lung, and Blood Institute (NHLBI) ยท Tests: Miltenyi CD34 Reagent System, Donor derived G-CSF mobilized PBC 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 NCT07154745Phase 320 sites๐Ÿ“ Ribeirรฃo Preto๐Ÿ“ Santo Andrรฉ๐Ÿ“ Sรฃo Paulo Regeneron Pharmaceuticals ยท Tests: Pozelimab, Cemdisiran A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) NCT07256301Phase 12 sites๐Ÿ“ Tianjin๐Ÿ“ Hangzhou Shanghai Lanyi Therapeutics Co., Ltd. ยท Tests: EA5 A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy NCT07416162n/a3 sites๐Ÿ“ Jeollanam๐Ÿ“ Seoul๐Ÿ“ Seoul Novartis Pharmaceuticals
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