Instructions
If you are interested in learning more about your possible participation in this clinical trial, please complete the form. Your information will be forwarded directly to the sponsoring company.
Status:
Recruiting
Associated Drug(s):
Phase:
Phase 3
Gender:
Female
Male
Age Group:
18 years and older
Accepts Healthy Volunteers:
No
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