Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
NCT06028594 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2025-10-17
Summary
This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in your body to break apart.
Conditions
Interventions
- DRUG
-
Pozelimab
Subcutaneous (SC) administration
- DRUG
-
Cemdisiran
Subcutaneous (SC) administration
Sponsors & Collaborators
- lead INDUSTRY
Eligibility
- Min Age
- 18 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
More Related Trials
-
Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease
NCT06325709 ·Status: RECRUITING ·Phase: PHASE1/PHASE2
-
Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy
NCT06003881 ·Status: APPROVED_FOR_MARKETING
-
Pilot Study of Rituximab for Membranoproliferative Glomerulonephritis
NCT00275613 ·Status: COMPLETED ·Phase: PHASE1
-
GVH 022P: Study Using Anti Tumor Necrosis Factor Antibody (Infliximab) for Treatment of Acute Graft Versus Host Disease
NCT00228839 ·Status: COMPLETED ·Phase: PHASE1
-
A Study of Rituximab Combined With Prednisone for the Initial Treatment of Chronic Graft Versus Host Disease (cGVHD)
NCT01066598 ·Status: TERMINATED ·Phase: PHASE2
-
Infliximab and Basiliximab for Treatment of Steroid Refractory Acute Graft Versus Host Disease
NCT01485055 ·Status: TERMINATED ·Phase: PHASE2
-
Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have an Inadequate Response to Eculizumab
NCT03030183 ·Status: COMPLETED ·Phase: PHASE2
-
Study of Danicopan as Add-on Treatment to Ravulizumab or Eculizumab in Pediatric Participants With PNH Who Have Clinically Significant Extravascular Hemolysis
NCT06449001 ·Status: RECRUITING ·Phase: PHASE3
-
Ruxolitinib Phosphate and Danazol in Treating Anemia in Patients With Myelofibrosis
NCT01732445 ·Status: COMPLETED ·Phase: PHASE2
-
Randomized Switch Study From Hydroxyurea to Ruxolitinib for RELIEF of Polycythemia Vera Symptoms: The Relief Study
NCT01632904 ·Status: COMPLETED ·Phase: PHASE3
-
Phase II, Open Label, Single Arm Study of SAR302503 In Myelofibrosis Patients Previously Treated With Ruxolitinib
NCT01523171 ·Status: COMPLETED ·Phase: PHASE2
-
Ruxolitinib Efficacy and Safety in Patients With HU Resistant or Intolerant Polycythemia Vera vs Best Available Therapy.
NCT02038036 ·Status: COMPLETED ·Phase: PHASE3
-
ALXN1210 (Ravulizumab) Versus Eculizumab in Complement Inhibitor Treatment-Naïve Adult Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH)
NCT02946463 ·Status: COMPLETED ·Phase: PHASE3
-
Rituximab in the Treatment of Patients With Bullous Pemphigoid
NCT00286325 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases.
NCT00278954 ·Status: COMPLETED ·Phase: PHASE3
-
Use of Immune Globulin Plus Rituximab for Desensitization in Highly HLA Sensitized Patients Awaiting Deceased Donor Kidney Transplantation
NCT01178216 ·Status: COMPLETED ·Phase: PHASE1/PHASE2
-
Topical Ruxolitinib for Cutaneous Chronic Graft Versus Host Disease (cGVHD)
NCT03395340 ·Status: TERMINATED ·Phase: PHASE2
-
Ruxolitinib With De-Intensified HLH-94 for the Treatment of Hemophagocytic Lymphohistiocytosis (HLH)
NCT06160791 ·Status: RECRUITING ·Phase: PHASE2
-
BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants With Inadequate Response to C5 Inhibitor Therapy
NCT05116774 ·Status: TERMINATED ·Phase: PHASE2
-
Compassionate Use of Evinacumab
NCT06500598 ·Status: APPROVED_FOR_MARKETING
-
Preventing Intravenous Immunoglobulin-associated Adverse Reactions
NCT00675805 ·Status: COMPLETED ·Phase: NA
-
Intravenous Immunoglobulins in Severe and Refractory Solar Urticaria
NCT01360658 ·Status: COMPLETED ·Phase: PHASE2
-
Efficacy and Safety of Ruxolitinib in the Treatment of Anemic Myelofibrosis Patients.
NCT02966353 ·Status: COMPLETED ·Phase: PHASE2
-
An Extension Clinical Study of BCD-148 for the Treatment of Patients With PNH Previously Treated in Clinical Study No. BCD-148-2/NOCTURN
NCT06987864 ·Status: COMPLETED ·Phase: PHASE3
-
Belimumab for Prevention of Chronic Graft-versus-Host Disease Following Allogeneic Hematopoietic Cell Transplantation
NCT03207958 ·Status: COMPLETED ·Phase: PHASE1