Clinical trials: Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria
Long-term Safety and Tolerability of Iptacopan in Patients With Paroxysmal Nocturnal Hemoglobinuria
This study is an open-label, single arm, multicenter, roll-over extension study to characterize long-term safety, tolerability and efficacy of iptacopan and to provide access to iptacopan to patients with PNH who have completed…
- Trial name
- NCT04747613
- Testing
- Iptacopan
- Where
- 60 sites in United States, Brazil and 15 more
- Volunteers
- 208 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients
The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of…
- Trial name
- NCT06934967
- Testing
- LNP023
- Where
- 15 sites in United States, Brazil and 4 more
- Volunteers
- 12 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
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
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs".
- Trial name
- NCT07154745
- Testing
- Pozelimab, Cemdisiran
- Where
- 20 sites in Brazil, Canada and 5 more
- Volunteers
- 35 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Study of the Efficacy and Safety of FXS6837 Capsules Compared to Eculizumab for 24 Weeks in Patients With PNH.
An open label study to evaluate the efficacy and safety of FXS6837 capsules compared to eculizumab in naive PNH patients. About 90 PNH patients who are naive to complement inhibitor therapies will be enrolled to take FXS6837…
- Trial name
- NCT07747090
- Testing
- FXS6837 Capsule, Eculizumab infusion
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Long-Term Safety, Tolerability and Efficacy of OMS906 in Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the long-term safety and tolerability of repeat-dose OMS906 5 mg/kg IV administration at 8-week intervals in patients with PNH.
- Trial name
- NCT06298955
- Testing
- OMS906 study drug
- Where
- 5 sites in Germany, Switzerland and 2 more
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Omeros Corporation
Paroxysmal Nocturnal Hemoglobinuria (PNH), Persistent Anemia
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
This study is researching an experimental drug called ALN-CFB. The study is focused on people with Paroxysmal Nocturnal Hemoglobinuria (PNH) who are currently taking a complement component C5 inhibitor ("C5-inhibitor") and…
- Trial name
- NCT07187401
- Testing
- ALN-CFB
- Where
- 8 sites in Canada, South Korea and 1 more
- Volunteers
- 24 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study is to evaluate the safety, effectiveness, and biological activity (how the investigational medication is processed by the body) of pegcetacoplan in 12-17 year-olds (adolescents) who have paroxysmal…
- Trial name
- NCT04901936
- Testing
- Pegcetacoplan
- Where
- 12 sites in United States, Czechia and 7 more
- Volunteers
- 12 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy
A Study of Iptacopan in Korean Patients With Paroxysmal Nocturnal Hemoglobinuria or C3 Glomerulopathy
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…
- Trial name
- NCT07416162
- Where
- 3 sites in South Korea
- Volunteers
- 21 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
REAL-CARE: Real-world Effectiveness of Iptacopan in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study evaluates iptacopan effectiveness and safety in routine clinical practice, with a focus on hematological response, transfusion avoidance, and patient-reported outcomes.
- Trial name
- REAL-CARE (NCT07229235)
- Where
- 12 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
This is a single-arm long-term extension study that will enroll participants with PNH who have completed participation in Alexion-sponsored clinical studies with danicopan as an add on therapy to a C5i.
- Trial name
- NCT05389449
- Testing
- Danicopan
- Where
- 45 sites in United States, Brazil and 13 more
- Volunteers
- 80 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria
Unrelated And Partially Matched Related Donor PSCT w/ T Cell Receptor (TCR) αβ Depletion for Patients With BMF
This is a single arm pilot study using TCR alpha/beta+ T cell-depleted peripheral blood stem cells (PBSC) from closely matched unrelated donors or partially matched/haploidentical related donors for hematopoietic stem cell…
- Conditions
- Acquired Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Inherited Bone Marrow Failure Syndromes
- Trial name
- NCT03047746
- Testing
- CliniMACs
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Children's Hospital of Philadelphia
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Safety, Tolerability, PK and PD of ADX-038 in Healthy Participants and Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients
The first-in-human Phase 1/Phase 2a study described herein will evaluate the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ADX-038 in both healthy participants (HP) and in patients with paroxysmal…
- Trial name
- PNH (NCT05876312)
- Testing
- ADX-038
- Where
- 4 sites in Australia, United Kingdom
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- ADARx Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adults Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study is to evaluate the efficacy of ravulizumab in adult participants with PNH.
- Trial name
- NCT06578949
- Testing
- Ravulizumab
- Where
- 8 sites in China
- Volunteers
- 18 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Danicopan Early Access Program
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…
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria, PNH, Extravascular Hemolysis
- Trial name
- NCT05982938
- Testing
- Danicopan
- Where
- 15 sites in Italy
- Sponsor
- Alexion Pharmaceuticals, Inc.
Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia
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.
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…
- Conditions
- Severe Aplastic Anemia, Acquired Amegakaryocytic Thrombocytopenia, Acquired Pure Red Cell Aplasia, Paroxysmal Nocturnal Hemoglobinuria
- Trial name
- NCT06412497
- Testing
- Rituximab, Rabbit ATG, Cyclophosphamide
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Paroxysmal Nocturnal Hemoglobinuria
Eculizumab in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
This is a Phase 3b, single-arm, open-label, multicenter study to evaluate the efficacy, safety, pharmacokinetics (PK), pharmacodynamics (PD), and immunogenicity of eculizumab in adult participants with paraxysmal noturnal…
- Trial name
- Soliris (NCT05886244)
- Testing
- Eculizumab
- Where
- 7 sites in China
- Volunteers
- 25 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
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.
- Trial name
- PNH-NIS (NCT06931691)
- Testing
- LNP023
- Where
- 14 sites in China
- Volunteers
- 80 people
- Sponsor
- Novartis Pharmaceuticals
PNH - Paroxysmal Nocturnal Hemoglobinuria
A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, Phase Ib clinical trial designed to evaluate the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of multiple doses of the humanized monoclonal antibody EA5 in adult patients with…
- Trial name
- NCT07256301
- Testing
- EA5
- Where
- 2 sites in China
- Volunteers
- 24 people
- Sponsor
- Shanghai Lanyi Therapeutics Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Study of the Safety and Efficacy of OMS906 in Patients With Paroxysmal Nocturnal Hemoglobinuria
The purpose of this study is to assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
- Trial name
- NCT05889299
- Testing
- OMS906
- Where
- Ukraine
- Volunteers
- 15 people
- Sponsor
- Omeros Corporation
Paroxysmal Nocturnal Hemoglobinuria
Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria
This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical…
- Trial name
- NCT05274633
- Testing
- Ravulizumab
- Where
- 6 sites in Italy
- Volunteers
- 120 people
- Sponsor
- Alexion Pharmaceuticals, Inc.