A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

Launched by REGENERON PHARMACEUTICALS · Nov 12, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a new combination treatment using two experimental drugs called pozelimab and cemdisiran for adults with a condition known as paroxysmal nocturnal hemoglobinuria (PNH). The goal of the study is to find out how safe and effective this combination is compared to two existing treatments, ravulizumab and eculizumab. Researchers will look at how well the new treatment works, any potential side effects, and how the body responds to the drugs.

To participate in this study, individuals must be adults diagnosed with PNH and currently experiencing symptoms. Key eligibility criteria include having a specific blood test confirming PNH and meeting certain health requirements. Participants can expect regular visits for monitoring and will need to receive certain vaccinations before starting the treatment. This study aims to provide valuable information that could help improve treatment options for people with PNH.

Gender

ALL

Eligibility criteria

• Key Inclusion Criteria:
• • 1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
• • 2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
• • 3. LDH level ≥2 × ULN at the screening visit
• • 4. Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol
• Key Exclusion Criteria:
• • 1. Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
• • 2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
• • 3. Body weight \<40 kilograms at screening visit
• • 4. Planned use of any complement inhibitor therapy other than study drugs during the treatment period
• • 5. Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol.
• • 6. Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B).
• • 7. Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab \[Cohort A\] or eculizumab \[Cohort B\] prescribing information, where available, or national guidelines/local practice, or if necessary when administration of the first dose of the quadrivalent meningococcal vaccine \[serotype ACWY\] or the second dose of the serotype B meningococcal vaccine \[when available\] is less than 2 weeks prior to study treatment initiation) as described in the protocol
• • 8. 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
• • 9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
• • Note: Other protocol-defined Inclusion/ Exclusion Criteria apply