Paroxysmal Nocturnal Hemoglobinuria clinical trials

Explore recruiting paroxysmal nocturnal hemoglobinuria clinical trials.

Recruiting paroxysmal nocturnal hemoglobinuria studies right now

Study details & eligibility ClinicalTrials.gov record

Also recruiting in Europe

Paroxysmal Nocturnal Hemoglobinuria trials registered in the EU Clinical Trials Information System (CTIS). These are outside the "near me" matcher above - open a study for full details.

AN ADAPTIVE PHASE I/II STUDY TO ASSESS SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7112689 IN HEALTHY VOLUNTEERS AND PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

Italy, Germany, Hungary, France

Phase I and Phase II (Integrated)- First administration to humans EU CTIS Study details

A Phase III, Randomized, Open-Label, Active controlled, Multicenter Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not Previously Treated with Complement Inhibitors

Germany, Poland, Spain, Sweden, France, Lithuania +3 more

Therapeutic confirmatory (Phase III) EU CTIS Study details

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

Spain, Netherlands

Therapeutic exploratory (Phase II) EU CTIS Study details

An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients aged 2 to <18 years of age

Netherlands, Germany, France, Spain, Italy

Therapeutic confirmatory (Phase III) EU CTIS Study details

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.

Germany, Italy, Czechia, France, Spain, Netherlands +1 more

Therapeutic confirmatory (Phase III) EU CTIS Study details

A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in PNH Patients with a Sub-optimal Response to the C5 inhibitor, Ravulizumab

Greece, Germany

Human Pharmacology (Phase I)- Other EU CTIS Study details

Paroxysmal Nocturnal Hemoglobinuria trials by city

Paroxysmal Nocturnal Hemoglobinuria clinical trials

Recruiting paroxysmal nocturnal hemoglobinuria studies right now

Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients

Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

Study of Safety and Efficacy of HS-10542 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

A First-in-Human Safety and Efficacy Study of ALN-CFB, a Small Interfering RNA (siRNA) Targeting Complement Factor B, in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria With Persistent Anemia on a C5 Inhibitor

A 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 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

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.

Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria

A Phase I Clinical Trial to Evaluate CMS-D017 Following Single and Multiple Doses in Healthy Participants

A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Also recruiting in Europe

AN ADAPTIVE PHASE I/II STUDY TO ASSESS SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF RO7112689 IN HEALTHY VOLUNTEERS AND PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)

A Phase III, Randomized, Open-Label, Active controlled, Multicenter Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) not Previously Treated with Complement Inhibitors

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

An open-label, single-arm, multicenter, phase 3 study to assess pharmacokinetics, safety and tolerability of iptacopan in pediatric PNH patients aged 2 to <18 years of age

An open label, multicenter roll-over extension program (REP) to characterize the long-term safety and tolerability of iptacopan (LNP023) in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who have completed PNH Phase 2 and Phase 3 studies with iptacopan.

A Phase 2 Proof of Concept Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of OMS906 in PNH Patients with a Sub-optimal Response to the C5 inhibitor, Ravulizumab

Paroxysmal Nocturnal Hemoglobinuria trials by city

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