A RANDOMIZED, OPEN-LABEL, C5 INHIBITOR-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY

Phase 3

• Conditions: D599 Acquired haemolytic anaemia, unspecified; Acquired haemolytic anaemia, unspecified; D599

• Registration Number: PER-036-23
• Lead Sponsor: REGENERON PHARMACEUTICALS, INC.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-24
• Last Update Posted: 20 August 2024

Recruitment & Eligibility

• Status: Without startig enrollment
• Sex: All
• Target Recruitment: 0

Inclusion Criteria

1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes described in the protocol

2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms described in the protocol

3. LDH level =2 × upper limit of normal (ULN) at the screening visit

Exclusion Criteria

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

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

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

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
DH<br> NAME OF THE RESULT: Cohort A: Percent change in lactate dehydrogenase (LDH)<br> PERIOD OF TIME WHERE TE MEASUREMENT WILL BE CONDUCTED AND WHICH WILL ALLOW OBTAINING THE<br> PRIMARY RESULT: Baseline to week 26 inclusive ;LDH<br> NAME OF THE RESULT: Cohort B: Maintenance of adequate control of hemolysis, defined as <br>LDH =1.5 × ULN<br> PERIOD OF TIME WHERE TE MEASUREMENT WILL BE CONDUCTED AND WHICH WILL ALLOW OBTAINING THE<br> PRIMARY RESULT: Week 8 through week 26 ;RBC<br> NAME OF THE RESULT: Cohort B: Transfusion avoidance ((not requiring a red blood cell (RBC)<br>transfusion per the protocol)<br> PERIOD OF TIME WHERE TE MEASUREMENT WILL BE CONDUCTED AND WHICH WILL ALLOW OBTAINING THE<br> PRIMARY RESULT: Day 1 through week 26