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

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ClinicalTrials.gov Identifier: NCT06028594

Expanded Access Status : Available

First Posted : September 8, 2023

Last Update Posted : September 8, 2023

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

Regeneron Pharmaceuticals

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
Paroxysmal Nocturnal Hemoglobinuria	Drug: Pozelimab Drug: Cemdisiran

Study Design

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

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Paroxysmal nocturnal hemoglobinuria

Genetic and Rare Diseases Information Center resources: Paroxysmal Nocturnal Hemoglobinuria Myelodysplastic Syndromes

U.S. FDA: Expanded Access (Compassionate Use)

U.S. FDA Resources

Interventions

Intervention Details:

Drug: Pozelimab
Subcutaneous (SC) administration

Other Name: REGN3918
Drug: Cemdisiran
Subcutaneous (SC) administration

Other Name: ALN-CC5

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:	All

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

Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06028594

Contacts

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Contact: Requests for compassionate use must be initiated by a treating physician Physicians with questions about access can contact Clinigen	+44(0) 1932 824 100	medicineaccess@clinigengroup.com

Sponsors and Collaborators

Regeneron Pharmaceuticals

More Information

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Responsible Party:	Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier:	NCT06028594
Other Study ID Numbers:	R3918-PNH-2238
First Posted:	September 8, 2023 Key Record Dates
Last Update Posted:	September 8, 2023
Last Verified:	August 2023

Additional relevant MeSH terms:

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Hemoglobinuria Hemoglobinuria, Paroxysmal Proteinuria Urination Disorders Urologic Diseases Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Urogenital Diseases	Male Urogenital Diseases Urological Manifestations Anemia, Hemolytic Anemia Hematologic Diseases Myelodysplastic Syndromes Bone Marrow Diseases

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