A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE)
A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients With Symptomatic Generalized Myasthenia Gravis (NIMBLE)
ClinicalTrials.gov ID: NCT05070858
Sponsor: Regeneron Pharmaceuticals
Information provided by: Regeneron Pharmaceuticals (Responsible Party)
Last Update Posted: 2023-08-07
Brief Summary:
The primary objective is:
To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG).
The secondary objectives of the study are:
-
To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on:
- Clinician-assessed signs of myasthenia gravis (MG) and muscle strength
- Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only).
- Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG
- Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength
- Health related quality of life
- Proportion of patients with minimal MG symptoms
- Patient- and clinician-reported signs and symptoms of MG
- To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy
- To assess the concentration of total pozelimab in serum
- To assess the concentrations of cemdisiran and its metabolites in plasma
- To assess the immunogenicity of pozelimab
- To assess the concentration of total C5 in plasma
- To assess the immunogenicity of cemdisiran
- To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation
Detailed Description:
DBTP- Double blind treatment plan (24 weeks) ETP - Extension treatment plan (28 weeks) OLTP- Open label treatment plan (68 weeks) Off-treatment follow up period (52 weeks)
OFFICIAL TITLE
Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Symptomatic Generalized Myasthenia Gravis
INTERVENTION / TREATMENT
Drug: Pozelimab + Cemdisiran
Drug: Cemdisiran
Other: Placebo
Drug: Pozelimab
| Category | Value |
|---|---|
| Study Start (Actual) | 2021-12-14 |
| Primary Completion (Estimated) | 2024-08-14 |
| Study Completion (Estimated) | 2027-05-01 |
| Enrollment (Estimated) | 235 |
| Study Type | Interventional |
| Phase | Phase 3 |
| Other Study ID Numbers | R3918-MG-2018 2020-003272-41 (EudraCT Number) |