Clinical trials: PNH - Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal Nocturnal Hemoglobinuria (PNH)
SLN12140 in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) in China
The goal of this clinical trial is to learn if drug SLN12140 works to treat Complement Inhibitor-Naïve Subjects with Paroxysmal Nocturnal Hemoglobinuria in adults.
- Trial name
- NCT07387302
- Testing
- SLN12140
- Volunteers
- 10 people
- Sponsor
- Linno Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH
This study will be the initial exploration of pegcetacoplan in patients with PNH. The assessments of the safety, tolerability, PK, and PD following administration of single and multiples doses of pegcetacoplan will guide…
- Trial name
- NCT02264639
- Testing
- Pegcetacoplan
- Where
- 7 sites in United States
- Volunteers
- 9 people
- Sponsor
- Apellis Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors
A study designed to evaluate the non-inferiority of crovalimab compared with eculizumab in participants with PNH who have not been previously treated with complement inhibitor therapy.
- Trial name
- COMMODORE 2 (NCT04434092)
- Testing
- Crovalimab, Eculizumab
- Where
- 63 sites in Argentina, Brazil and 21 more
- Volunteers
- 210 people
- Sponsor
- Hoffmann-La Roche
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Danicopan in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The purpose of this study was to determine the safety and efficacy of ACH-0144471 (also known as danicopan and ALXN2040) in currently untreated participants with PNH.
- Trial name
- NCT03053102
- Testing
- Danicopan
- Where
- 5 sites in Italy, New Zealand and 2 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
Home Reported Outcomes in PNH
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.
- Trial name
- NCT06411626
- Testing
- PNH-relevant therapies
- Where
- United States
- Volunteers
- 128 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
Prospective Observational Study of Long-term Pathogenic Treatment of Elizaria®
This is prospective observational study of long-term pathogenic treatment of Elizaria® in patients with paroxysmal nocturnal hemoglobinuria.
- Trial name
- NAP (NCT04671810)
- Testing
- Elizaria®
- Where
- 8 sites in Russia
- Volunteers
- 44 people
- Sponsor
- AO GENERIUM
Paroxysmal Nocturnal Hemoglobinuria
Pozelimab and Cemdisiran Combination Therapy in Adult Participants With Paroxysmal Nocturnal Hemoglobinuria Who Switch From Eculizumab Therapy
The primary objective of the study is to evaluate the safety and tolerability of pozelimab and cemdisiran combination therapy in participants with PNH who switch from eculizumab therapy The secondary objectives of the study are…
- Trial name
- NCT04888507
- Testing
- Pozelimab, Cemdisiran
- Where
- United Kingdom
- Volunteers
- 6 people
- Sponsor
- Regeneron Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy
The purpose of this Phase 3 study was to determine whether iptacopan is efficacious and safe for the treatment of Paroxysmal nocturnal hemoglobinuria (PNH) patients who were naïve to complement inhibitor therapy.
- Trial name
- APPOINT-PNH (NCT04820530)
- Testing
- Iptacopan (LNP023)
- Where
- 12 sites in China, France and 6 more
- Volunteers
- 40 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Real-world, Multi-center, Prospective, Observational Study for PNH in China
As a rare disease listed in the First Catalogue of Rare Diseases in China (National Health Commission of the People's Republic of China, 2019), PNH is poorly studied in China subse-quently leading to the inadequate elucidation of…
- Trial name
- ReWoPNH (NCT06154512)
- Where
- 40 sites in China
- Volunteers
- 724 people
- Sponsor
- AstraZeneca
Paroxysmal Nocturnal Hemoglobinuria
Efficacy, Safety, Pharmacokinetics and Pharmacodynamics Study, Assessing Multiple LNP023 Doses in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
This was a Phase II randomized, open-label, multicenter, efficacy, safety, pharmacokinetic and pharmacodynamic study assessing four iptacopan doses in adult Paroxysmal nocturnal hemoglobinuria (PNH) patients with active hemolysis…
- Trial name
- NCT03896152
- Testing
- LNP023
- Where
- 5 sites in Malaysia, Singapore and 2 more
- Volunteers
- 13 people
- Sponsor
- Novartis Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria
A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
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…
- Trial name
- NCT07177859
- Testing
- NTQ5082 capsules 200 mg, Eculizumab Injection
- Where
- China
- Volunteers
- 78 people
- Sponsor
- Nanjing Chia-tai Tianqing Pharmaceutical
Paroxysmal Nocturnal Hemoglobinuria
Efficacy and Safety of HRS-5965 in Patients With PNH Who Are Still Anemia After Anti-C5 Antibody Treatment
This trial is a multi-center, single-arm, open-label phase III clinical trial. A total of approximately 35 patients with paroxysmal nocturnal hemoglobinuria who remained anemic despite stable use of C5 complement inhibitor…
- Trial name
- NCT06715943
- Testing
- HRS-5965 capsule
- Where
- 2 sites in China
- Volunteers
- 39 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria, Natural History Study
Natural History Study of Adult Patients With Paroxysmal Nocturnal Hemoglobinuria of High-risk Hemolysis in China
This is a single-center observational study conducted in adult patients with paroxysmal nocturnal hemoglobinuria of high-risk hemolysis.
- Trial name
- NCT05125341
- Where
- China
- Volunteers
- 67 people
- Sponsor
- CARE Pharma Shanghai Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Clinical Trial of BCD-148 and Soliris® for the Treatment of Patients With Paroxysmal Nocturnal Hemoglobinuria
This clinical study is a randomized, open-label, international, multi-center, comparative study of efficacy and safety of BCD-148 and Soliris® in PNH patients.
- Trial name
- NCT04060264
- Testing
- BCD-148, Soliris
- Where
- Russia
- Volunteers
- 28 people
- Sponsor
- Biocad
Ultomiris-exposed Pregnant/ Postpartum, Pregnancy
Study of Ultomiris® (Ravulizumab) Safety in Pregnancy
The primary objective of this study is to describe the frequency and characteristics of pregnancy outcomes and maternal complications among participants exposed to Ultomiris and to describe the frequency and characteristics of…
- Conditions
- Ultomiris-exposed Pregnant/ Postpartum, Pregnancy, Paroxysmal Nocturnal Hemoglobinuria (PNH), Atypical Hemolytic Uremic Syndrome (aHUS)
- Trial name
- NCT06312644
- Testing
- Ultomiris
- Where
- 7 sites in United States, Australia and 5 more
- Volunteers
- 75 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria, PNH
Open-label, Multiple Ascending Dose Study of Ravulizumab (ALXN1210) in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary purpose of this study is to evaluate the safety, tolerability, and efficacy of multiple intravenous (IV) doses of ravulizumab administered to complement inhibitor treatment-naïve participants with PNH.
- Trial name
- NCT02605993
- Testing
- Ravulizumab
- Where
- 16 sites in Canada, France and 5 more
- Volunteers
- 26 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study to Evaluate the Efficacy and Safety of Oral HRS-5965 in Adult Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
A study of the efficacy and safety of HRS-5965 capsules compared to eculizumab for 24 weeks in patients with PNH.
- Trial name
- NCT06593938
- Testing
- HRS-5965 capsule, Eculizumab Injection
- Where
- 2 sites in China
- Volunteers
- 76 people
- Sponsor
- Chengdu Suncadia Medicine Co., Ltd.
Paroxysmal Nocturnal Hemoglobinuria
Dose Escalation of BCX10013 in Participants with Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a multicenter, open-label, intra-subject, dose escalation study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and therapeutic potential of BCX10013 in participants with PNH.
- Trial name
- NCT06100900
- Testing
- BCX10013
- Where
- 4 sites in Malaysia, South Africa
- Volunteers
- 8 people
- Sponsor
- BioCryst Pharmaceuticals
Hemoglobinuria, Paroxysmal
Eculizumab Pharmacokinetics/Pharmacodynamics Study in Pediatric/Adolescent Paroxysmal Nocturnal Hemoglobinuria (PNH)
The primary objective of this study was to evaluate the pharmacokinetic (PK) and pharmacodynamic (PD) parameter estimates of eculizumab to confirm the dose regimens for pediatric and adolescent participants with PNH.
- Trial name
- NCT00867932
- Testing
- Eculizumab
- Where
- 3 sites in United States
- Volunteers
- 7 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria
This is a multicenter, open-label study to evaluate the long-term safety and efficacy of XH-S003 capsules in PNH patients. About 26 PNH patients who had received and completed XH-S003 study treatment will be enrolled to take…
- Trial name
- XH-S003-II-102 (NCT07657052)
- Testing
- XH-S003 Capsule
- Where
- China
- Volunteers
- 26 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.