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

Primary Purpose

Paroxysmal Nocturnal Hemoglobinuria

Status
Available
Phase
Locations
Study Type
Expanded Access
Intervention
Pozelimab
Cemdisiran
Sponsored by
Regeneron Pharmaceuticals
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Paroxysmal Nocturnal Hemoglobinuria

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All Sexes

Key Inclusion Criteria: 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 Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission 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 as appropriate Key Exclusion Criteria: Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician 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

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    August 31, 2023
    Last Updated
    August 31, 2023
    Sponsor
    Regeneron Pharmaceuticals
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    1. Study Identification

    Unique Protocol Identification Number
    NCT06028594
    Brief Title
    Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
    Official Title
    An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2023
    Overall Recruitment Status
    Available
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    Regeneron Pharmaceuticals

    4. Oversight

    5. 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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Paroxysmal Nocturnal Hemoglobinuria

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    Pozelimab
    Other Intervention Name(s)
    REGN3918
    Intervention Description
    Subcutaneous (SC) administration
    Intervention Type
    Drug
    Intervention Name(s)
    Cemdisiran
    Other Intervention Name(s)
    ALN-CC5
    Intervention Description
    Subcutaneous (SC) administration

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    18 Years
    Eligibility Criteria
    Key Inclusion Criteria: 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 Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's permission 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 as appropriate Key Exclusion Criteria: Significant history or concerns of non-compliance that could impact the patient's safety per the treating physician 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
    Central Contact Person:
    First Name & Middle Initial & Last Name or Official Title & Degree
    Requests for compassionate use must be initiated by a treating physician Physicians with questions about access can contact Clinigen
    Phone
    +44(0) 1932 824 100
    Email
    medicineaccess@clinigengroup.com

    12. IPD Sharing Statement

    Learn more about this trial

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

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