Clinical trials: Paroxysmal Hemoglobinuria, Nocturnal, open to volunteers
Paroxysmal Nocturnal Hemoglobinuria
The International PNH Interest Group PNH Registry
The aim of this International PNH Interest Group (IPIG) registry is to develop an international database to prospectively collect data on patients with PNH covering clinical outcomes, patient reported outcomes (PROs), and…
- Trial name
- NCT06524726
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- International PNH Interest Group
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
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
Danicopan PMS in Korea
As part of a post-approval commitment, the Korean health authority requests a study to characterize safety and effectiveness in patients who are treated with Danicopan as an add-on to ravulizumab or eculizumab in normal clinical…
- Trial name
- NCT07457151
- Where
- 4 sites in South Korea
- Volunteers
- 27 people
- Sponsor
- AstraZeneca
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
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
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH).
- Trial name
- ACCESS-EXT (NCT05744921)
- Testing
- Pozelimab, Cemdisiran
- Where
- 50 sites in Brazil, Canada and 19 more
- Volunteers
- 202 people
- Sponsor
- Regeneron 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