A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal Hemoglobinuria

Phase 1

• Conditions: Paroxysmal nocturnal hemoglobinuria (PNH); MedDRA version: 21.1Level: LLTClassification code 10055629Term: Paroxysmal nocturnal hemoglobinuriaSystem Organ Class: 100000004857; Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2021-004931-10-PL
• Lead Sponsor: Regeneron Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2022-11-16
• Last Update Posted: 21 August 2023

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 202

Inclusion Criteria

Patients Entering from the Parent Study
1. Patients with PNH who have completed treatment, including the
corresponding post OLTP transition period in the parent study (R3918-
PNH-2021[NCT05133531]), if applicable.
Patients Entering with C5 polymorphism
1. Patients with PNH who have a documented C5 polymorphism
rendering them refractory to eculizumab or ravulizumab (eg,
p.Arg885His, p.Arg885Cys), as described in the protocol
2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing
as described in the protocol
3. Active disease, as defined by the presence of 1 or more PNH-related
sign or symptom as described in the protocol
4. LDH level =2 × upper limit of normal (ULN) at the screening visit

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 172
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

Exclusion Criteria

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