NCT06517758

A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.

A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 146 (estimated)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.

Detailed description

The study consists of a 6-month double-blind treatment period for the primary efficacy and safety analysis followed by a maximum duration of 60 month open label extension period. A safety follow up assessment will be performed, one 7 days after the last administration of study treatment and one 30 days after the last administration of study treatment for all participants.

Conditions

• Generalized Myasthenia Gravis

Interventions

Type	Name	Description
DRUG	Iptacopan	Hard gelatin capsule
OTHER	Matching Placebo	Hard gelatin capsule

Timeline

• Start date: 2024-07-31
• Primary completion: 2027-04-30
• Completion: 2032-05-27
• First posted: 2024-07-24
• Last updated: 2026-03-30

Locations

110 sites across 17 countries: United States, Argentina, Brazil, China, Denmark, France, Germany, Greece, Israel, Italy, Japan, Poland, Portugal, Serbia, South Korea, Spain, United Kingdom

Regulatory

• FDA-regulated drug study