Paroxysmal Nocturnal Hemoglobinuria Clinical Trial
Official title:
A Phase II, Open-Label Study of NM8074 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
The proposed study will enroll a planned number of 12 treatment naïve PNH patients with amaximum of 18 PNH patients who have been diagnosed with hemolytic anemia and meet the inclusion criteria. There will be 2 cohorts with 6 to 9 patients each. Patients in Cohort 1 will be administered NM8074 at 20 mg/kg intravenously (IV) every 2 weeks over the treatment period. Cohort 2 patients will be administered a dose of 10 mg/kg NM8074 weekly for 4 weeks followed by a 20 mg/kg dose of NM8074 administered via IV every 2 weeks for the remainder of the treatment period. This study will determine if NM8074 will provide the desired inhibition of the alternative pathway (AP). ;
| 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 |