A C5 Inhibitor-controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

Phase 3

• Conditions: Health Condition 1: D595- Paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli]

• Registration Number: CTRI/2023/11/059377
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Yet Recruiting
• Sex: Not specified
• Target Recruitment: 0

Inclusion Criteria

1.Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes 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 greater than or equal to 2 into ULN at the screening visit

4.Other protocol defined Inclusion Criteria apply

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 less than 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 for ravulizumab (Cohort A) or eculizumab (Cohort B) according to the current local prescribing information (where available) and at a minimum documentation of meningococcal vaccination within 5 years prior to screening visit

6.Any contraindication for receiving Neisseria meningitidis vaccination

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 vaccination is less than 2 weeks from study treatment initiation)

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

10.Other protocol defined Exclusion Criteria apply

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
- Transfusion avoidance (Not requiring a red blood cell (RBC) transfusion per the protocol) <br/ ><br>- Maintenance of adequate control of hemolysis (LDH less than or equal to 1.5 × ULN)Timepoint: - Post-baseline Day 1 through week 26 <br/ ><br>- From week 8 through week 26, inclusive <br/ ><br>