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

Sponsor
Regeneron Pharmaceuticals (Industry)
Overall Status
Available
CT.gov ID
NCT06028594
Collaborator
(none)

Study Details

Study Description

Brief 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.

Condition or Disease Intervention/Treatment Phase

Study Design

Study Type:
Expanded Access
Official Title:
An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

Outcome Measures

Primary Outcome Measures

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    All
    Key Inclusion Criteria:
    1. Patients who have completed either the end of treatment visit of the open-label treatment period or open-label extension period in one of the following parent studies:

    • An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (R3918-PNH-2050 [NCT05744921])

    • A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy (R3918-PNH-2092 [NCT04811716])

    • A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from Eculizumab Therapy (R3918-PNH-20105 [NCT04888507]). Note: In some countries, patients that would normally enter into the R3918-PNH-2050 (NCT05744921) parent study before entering this expanded access program, may be given the opportunity to skip R3918-PNH-2050 (NCT05744921) and go straight into this expanded access program, with Regeneron's permission

    1. Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission

    2. Willing and able to comply with clinic visits and related standard-of-care procedures

    3. With Regeneron's permission, patients who have been enrolled in other PNH studies as appropriate

    Key Exclusion Criteria:

    1. Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician

    2. Any new condition or worsening of an existing condition which, in the opinion of the treating physician, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the program

    NOTE: Other protocol defined inclusion / exclusion criteria apply

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • Regeneron Pharmaceuticals

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Regeneron Pharmaceuticals
    ClinicalTrials.gov Identifier:
    NCT06028594
    Other Study ID Numbers:
    • R3918-PNH-2238
    First Posted:
    Sep 8, 2023
    Last Update Posted:
    Sep 8, 2023
    Last Verified:
    Aug 1, 2023
    Additional relevant MeSH terms:
    Hemoglobinuria
    Hemoglobinuria, Paroxysmal

    Study Results

    No Results Posted as of Sep 8, 2023