NCT05070858

A Study to Test How Safe Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Alone Are and How Well They Work in Adult Patients With Generalized Myasthenia Gravis

Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy and Cemdisiran Monotherapy in Patients With Symptomatic Generalized Myasthenia Gravis

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 288 (actual)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This study is researching the experimental drugs called pozelimab and cemdisiran, and cemdisiran monotherapy. The study is focused on patients with generalized Myasthenia Gravis (gMG). Myasthenia gravis is a disease that causes weakness and fatigue in muscles in the body because the nerves and muscles are not communicating properly. The aim of the study is to see how effective pozelimab and cemdisiran are when used in combination and when pozelimab and cemdisiran are used alone for patients with gMG. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs * How the study drugs work inside the body * How much of the study drugs are in the blood at different times * Whether the body makes antibodies against pozelimab and cemdisiran (which could make the drugs less effective or could lead to side effects)

Detailed description

DBTP- Double Blind Treatment Period (24 weeks) ETP - Extension Treatment Period (28 weeks) OLTP- Open Label Treatment Period (68 weeks) FUP-Off treatment Follow Up Period (52 weeks)

Conditions

• Generalized Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Pozelimab + Cemdisiran	Subcutaneous administration as described in the protocol
DRUG	Cemdisiran	SC administration as described in the protocol
OTHER	Placebo	SC administration as described in the protocol
DRUG	Pozelimab	SC administration as described in the protocol

Timeline

• Start date: 2021-12-14
• Primary completion: 2025-07-08
• Completion: 2028-11-11
• First posted: 2021-10-07
• Last updated: 2026-02-12

Locations

116 sites across 19 countries: United States, Belgium, Brazil, Canada, China, Denmark, France, Georgia, Germany, India, Italy, Japan, Poland, Serbia, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study