Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

Phase

N/A

Condition

Red Blood Cell Disorders

Bone Marrow Disorder

White Cell Disorders

Treatment

Pozelimab

Cemdisiran

Clinical Study ID

NCT06028594

R3918-PNH-2238

Ages > 18
All Genders

Study Summary

This program is designed to provide access to pozelimab and cemdisiran and document the long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in your body to break apart.

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

Patients who have completed either the end of treatment visit of the open-labeltreatment period or open-label extension period in one of the following parentstudies:

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, andEfficacy of Pozelimab and Cemdisiran Combination Therapy in Patients withParoxysmal Nocturnal Hemoglobinuria (R3918-PNH-2050 [NCT05744921])
A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, andPharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment inPatients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received PozelimabMonotherapy (R3918-PNH-2092 [NCT04811716])
A Single Arm, Open-Label Study to Assess the Safety, Efficacy, andPharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy inPatients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from EculizumabTherapy (R3918-PNH-20105 [NCT04888507]). Note: In some countries, patients thatwould normally enter into the R3918-PNH-2050 (NCT05744921) parent study beforeentering this expanded access program, may be given the opportunity to skipR3918-PNH-2050 (NCT05744921) and go straight into this expanded access program,with Regeneron's permission

Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron'spermission
Willing and able to comply with clinic visits and related standard-of-care procedures
With Regeneron's permission, patients who have been enrolled in other PNH studies asappropriate

Exclusion

Key Exclusion Criteria:

Significant history or concerns of non-compliance that could impact the patient'ssafety per the treating physician
Any new condition or worsening of an existing condition which, in the opinion of thetreating physician, would make the patient unsuitable for enrollment or couldinterfere with the patient participating in or completing the program NOTE: Other protocol defined inclusion / exclusion criteria apply

Study Design

Pozelimab