NCT06028594
No Longer Available
Not Applicable
An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria
ConditionsParoxysmal Nocturnal Hemoglobinuria
Overview
- Phase
- Not Applicable
- Status
- No Longer Available
- Sponsor
- Regeneron Pharmaceuticals
Overview
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.
Study Design
- Study Type
- Expanded Access
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
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
Investigators
Similar Trials
Unknown
Not Applicable
Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing EnteropathyCD55-Deficient Protein-Losing EnteropathyNCT06003881Regeneron Pharmaceuticals
Available
Not Applicable
Expanded Access Program (EAP) Designed to Provide Access to Botensilimab and Balstilimab Before Regulatory ApprovalColorectal Cancer (CRC)Pancreatic CancerNon-Small Cell Lung Cancer (NSCLC)Hepatocellular Carcinoma (HCC)MelanomaSarcomaOvarian CancerProstate CancerNCT06751524Agenus Inc.
No Longer Available
Not Applicable
Expanded Access Protocol for Cemiplimab in Metastatic or Locally Advanced Cutaneous Squamous Cell CarcinomaCutaneous Squamous Cell CarcinomaNCT03492489Regeneron Pharmaceuticals
Approved For Marketing
Not Applicable
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With InhibitorsHemophilia ANCT03154437Genentech, Inc.
No Longer Available
Not Applicable
Expanded Access for MarizomibSolid TumorNCT04644107Celgene