Clinical trials for Paroxysmal nocturnal hemoglobinuria
41 recruiting or active trials synced from ClinicalTrials.gov.
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Clinical trial records are synced from ClinicalTrials.gov through automated extraction.
Report missing data- RECRUITINGNCT07457151Danicopan PMS in KoreaAstraZeneca
- RECRUITINGPhase 3NCT07154745A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working EfficientlyRegeneron Pharmaceuticals · PI: Clinical Trial Management
- RECRUITINGPhase 1NCT07462780A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy ParticipantsShenzhen Kangzhe Biotechnology Co., Ltd.
- RECRUITINGPHASE1, PHASE2NCT07470762Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal HemoglobinuriaJiangsu Hansoh Pharmaceutical Co., Ltd.
- RECRUITINGPHASE1, PHASE2NCT07187401A 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 InhibitorRegeneron Pharmaceuticals · PI: Clinical Trial Management
- ACTIVE NOT RECRUITINGNCT07413250Assess Long-Term Safety of Danicopan Add-on Therapy in Participants With Paroxysmal Nocturnal Hemoglobinuria: Analysis of IPIG-Registry DataAlexion Pharmaceuticals, Inc.
- RECRUITINGNCT07229235REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal HemoglobinuriaNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGPhase 3NCT06934967Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH PatientsNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- ACTIVE NOT RECRUITINGNCT07413679Long-term Safety of Danicopan: IPIG Registry-based Cohort StudyAlexion Pharmaceuticals, Inc.
- RECRUITINGPhase 3NCT06449001Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular HemolysisAlexion Pharmaceuticals, Inc.
- RECRUITINGNCT06931691A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in ChinaNovartis Pharmaceuticals
- RECRUITINGPhase 1NCT07152288Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic ImpairmentHaisco Pharmaceutical Group Co., Ltd.
- RECRUITINGPhase 2NCT06978699A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)S-INFINITY Pharmaceuticals Co., Ltd
- ACTIVE NOT RECRUITINGNCT06903234Post-authorization Safety Study of Iptacopan in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Using Data From the IPIG PNH RegistryNovartis Pharmaceuticals
- ACTIVE NOT RECRUITINGPhase 2NCT06764303A Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria PatientsNanjing Chia-tai Tianqing Pharmaceutical · PI: fengkui zhang
- RECRUITINGPhase 2NCT06745622Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal HemoglobinuriaHaisco Pharmaceutical Group Co., Ltd.
- RECRUITINGNCT06312644Study of Ultomiris® (Ravulizumab) Safety in PregnancyAlexion Pharmaceuticals, Inc. · PI: Sydney Williams
- RECRUITINGPHASE2, PHASE3NCT06933914Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal HemoglobinuriaWuhan Createrna Science and Technology Co., Ltd
- ACTIVE NOT RECRUITINGPhase 2NCT07212426Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in PNH Patients.Longbio Pharma · PI: Guangsheng He
- RECRUITINGNCT06606314Specified Drug-use Surveillance of Fabhalta CapsulesNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGPhase 3NCT06932744Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor TherapyWuhan Createrna Science and Technology Co., Ltd
- RECRUITINGPhase 3NCT06932471Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody TreatmentWuhan Createrna Science and Technology Co., Ltd
- RECRUITINGNCT06411626Home Reported Outcomes in PNHNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGNCT06524726The International PNH Interest Group PNH RegistryInternational PNH Interest Group · PI: Richard Kelly, MBChB PhD
- RECRUITINGPhase 2NCT06298955Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal HemoglobinuriaOmeros Corporation · PI: William Pullman
- ACTIVE NOT RECRUITINGPhase 2NCT06238544Long-term Safety and Tolerability of HRS-5965 in Patients With Paroxysmal Nocturnal HemoglobinuriaChengdu Suncadia Medicine Co., Ltd.
- RECRUITINGPhase 1NCT07256301A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)Shanghai Lanyi Therapeutics Co., Ltd. · PI: Fengkui Zhang, Dr.
- RECRUITINGPHASE1, PHASE2NCT05876312Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) PatientsADARx Pharmaceuticals, Inc.
- RECRUITINGN/ANCT05776472A Real World Effectiveness Study of Pegcetacoplan in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Swedish Orphan Biovitrum · PI: Study Physician +46 08-697-20 00, medical.info@sobi.com
- RECRUITINGPhase 3NCT05744921A 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 WorksRegeneron Pharmaceuticals · PI: Clinical Trial Management
- ACTIVE NOT RECRUITINGPhase 2NCT05476887To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104Kira Pharmacenticals (US), LLC.
- ACTIVE NOT RECRUITINGPhase 3NCT05389449A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNHAlexion Pharmaceuticals, Inc.
- ACTIVE NOT RECRUITINGPhase 3NCT05133531A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor TreatmentRegeneron Pharmaceuticals · PI: Clinical Trial Management
- ACTIVE NOT RECRUITINGPhase 3NCT04747613Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal HemoglobinuriaNovartis Pharmaceuticals · PI: Novartis Pharmaceuticals
- RECRUITINGN/ANCT05755867Global PNH Patient RegistryAplastic Anemia and MDS International Foundation · PI: Alice Houk, MS
- ACTIVE NOT RECRUITINGPhase 3NCT04654468A Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitionHoffmann-La Roche · PI: Clinical Trials
- RECRUITINGPhase 2NCT04901936A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)Apellis Pharmaceuticals, Inc.
- ACTIVE NOT RECRUITINGPhase 3NCT04434092A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement InhibitorsHoffmann-La Roche · PI: Clinical Trials
- ACTIVE NOT RECRUITINGPhase 3NCT04432584A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement InhibitorsHoffmann-La Roche · PI: Clinical Trials
- ACTIVE NOT RECRUITINGN/ANCT03047746Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMFChildren's Hospital of Philadelphia · PI: Timothy Olson, MD, PhD
- ACTIVE NOT RECRUITINGPHASE1, PHASE2NCT03157635Study to Assess Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of Crovalimab in Healthy Volunteers and Participants With Paroxysmal Nocturnal HemoglobinuriaHoffmann-La Roche · PI: Clinical Trials