A Phase 3 double blind, placebo controlled study of generalized Myasthenia Gravis
Research type
Research Study
Full title
A randomized, double-blind, placebo-controlled phase III study to evaluate the efficacy, safety, and tolerability of iptacopan in patients with generalized Myasthenia Gravis (gMG), followed by an open label extension phase.
IRAS ID
1008989
Contact name
Ilaria Zuliani
Contact email
Sponsor organisation
Novartis Pharma AG
Eudract number
2023-507064-39
Clinicaltrials.gov Identifier
Research summary
The purpose of this Phase 3, double-blind, randomised, placebo-controlled, multicentre study is to find out whether iptacopan treatment in patients with generalised Myasthenia Gravis, who are on stable standard of care treatment is safe, well tolerated and effective at improving participants daily functioning and quality of life.
Generalized Myasthenia gravis is a rare autoimmune neuromuscular condition where antibodies damage the signals between nerves and muscles, causing muscle weakness and tiredness. It is hoped that iptacopan will work to inhibit these antibodies without the use of immunosuppressant therapies, hence improving participants quality of life.
The study is split into two parts. A Core part and an Extension part.
Core Part includes a screening period, treatment period of 6 months where participants will receive either iptacopan or placebo and two safety follow-up visits. Maximum length of the Core Part of the study is up to 9 months. Participants and their study doctor will not know which treatment they are assigned to except in an emergency.
During the treatment period, participants will undergo health assessments and complete questionnaires to assess their activities of daily living and quality of life.
Upon completion of the Core Part, participants who are eligible will continue onto an open label extension part for up to 24 months with two safety follow up visits. All participants will receive iptacopan during the open label extension part and will be assessed for long term safety and efficacy.
Approximately 146 participants from 10 countries will participate in this study.
The pharmaceutical company sponsoring this study is Novartis AG Pharma.REC name
East of England - Cambridge East Research Ethics Committee
REC reference
24/EE/0060
Date of REC Opinion
2 May 2024
REC opinion
Further Information Favourable Opinion