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Purpose:
The primary objective of the study is to evaluate the effect on hemolysis and red blood cells (RBC) transfusions over a 26-week treatment period of pozelimab and cemdisiran combination treatment versus ravulizumab treatment in patients with active Paroxysmal Nocturnal Hemoglobinuria (PNH) who are complement inhibitor treatment-naive or have not recently received complement inhibitor therapy.
Status:
Recruiting
Bone Marrow Disease(s):
Associated Drug(s):
Phase:
Phase 3
Gender:
Female
Male
Age Group:
18 years and older
Accepts Healthy Volunteers:
No
Details on clinicaltrials.gov:
Inclusion Criteria:
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes as described in the protocol
- Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol, or history of RBC transfusion due to PNH within 3 months of the screening visit
- LDH level ≥2 × ULN at the screening visit
Exclusion Criteria:
- Prior treatment with a eculizumab within 3 months prior to screening visit, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening.
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
- Body weight <40 kilograms at screening visit
- Planned use of any complement inhibitor therapy other than study drugs during the treatment period
- Not meeting meningococcal vaccination requirements for ravulizumab according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit
- Any contraindication for receiving Neisseria meningitidis vaccination
- Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab prescribing information, where available, or national guidelines/local practice or if necessary when vaccination is less than 2 weeks from study treatment initiation)
- Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
- Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
Note: Other protocol-defined inclusion/ exclusion criteria apply