Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)
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 Physician's opinion, the participant is not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a comparable treatment; and according to the Treating Physician, the benefit of danicopan treatment outweighs the potential risks.
n/a
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT04432584 -
A Study Evaluating The Safety, Pharmacokinetics, and Efficacy Of Crovalimab Versus Eculizumab In Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Currently Treated With Complement Inhibitors
|
Phase 3 | |
Completed |
NCT05828485 -
Effect of Food on Pharmacokinetics of MY008211A Tablets in Healthy Adult Subjects
|
Phase 1 | |
Recruiting |
NCT02179359 -
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
|
N/A | |
Active, not recruiting |
NCT04434092 -
A Phase III Study Evaluating the Efficacy and Safety of Crovalimab Versus Eculizumab in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) Not Previously Treated With Complement Inhibitors.
|
Phase 3 | |
Terminated |
NCT05131204 -
Efficacy and Safety of the Combination of Pozelimab and Cemdisiran Versus Continued Eculizumab or Ravulizumab Treatment in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 3 | |
Recruiting |
NCT01374360 -
Paroxysmal Nocturnal Hemoglobinuria (PNH) Registry
|
||
Active, not recruiting |
NCT05389449 -
A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH
|
Phase 3 | |
Recruiting |
NCT06100900 -
Dose Escalation of BCX10013 in Subjects With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 1 | |
Completed |
NCT01272817 -
Nonmyeloablative Allogeneic Transplant
|
N/A | |
Completed |
NCT06326814 -
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
|
Phase 1 | |
Completed |
NCT04463056 -
Efficacy and Safety of Elizaria® vs. Soliris® in Patients With PNH
|
Phase 3 | |
Recruiting |
NCT05476887 -
To Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of KP104
|
Phase 2 | |
Completed |
NCT01192399 -
Safety and Efficacy Study of Eculizumab in Paroxysmal Nocturnal Hemoglobinuria Patients
|
Phase 2 | |
Active, not recruiting |
NCT06051357 -
Proof of Concept Study to Assess the Efficacy, Safety of HRS-5965 in Patients With Paroxysmal Nocturnal Hemoglobinuria
|
Phase 2 | |
Recruiting |
NCT06154512 -
A Real-world, Multi-center, Prospective, Observational Study for PNH in China
|
||
Completed |
NCT04128943 -
Electronic Patient-reported Outcome Monitoring in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria
|
||
Active, not recruiting |
NCT03329365 -
Paroxysmal Nocturnal Hemoglobinuria in ESUS & ETUS
|
||
Recruiting |
NCT05755867 -
Global PNH Patient Registry
|
||
Completed |
NCT04679103 -
A Safety and Immunogenicity Study in Long-term Treatment of Eculizumab (JSC "GENERIUM", Russian Federation)
|
Phase 3 | |
Completed |
NCT05642585 -
A Study of Single-dose MY008211A in Healthy Adults
|
Phase 1 |