A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal Hemoglobinuria
- Conditions
- Paroxysmal nocturnal hemoglobinuria (PNH)MedDRA version: 21.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2021-004931-10-GR
- Lead Sponsor
- Regeneron Pharmaceuticals, Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 288
1. Patients with PNH who have completed, without permanent discontinuation, study treatment in at least 1 of the parent studies (R3918-PNH-2021[NCT05133531] and/or R3918-PNH-2022 [NCT05131204]) including the transition period, if applicable.
Other protocol-defined Inclusion Criteria apply
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 242
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 46
1. Significant protocol deviation(s) in the parent study based on the investigator’s judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (eg, repetitive non-compliance with dosing by the patient).
2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study.
Other protocol-defined Exclusion Criteria apply
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method