A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
Condition(s) studied
Investigational drug(s) / intervention(s)
Iptacopan: Hard gelatin capsule
Matching Placebo: Hard gelatin capsule
Study 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.
Eligibility
Primary outcome measure(s)
- Change from baseline to Month 6 in Myasthenia Gravis Activity of Daily Living (MG-ADL) total score — Baseline to Month 6
The MG-ADL is an 8 item interviewer led patient reporting scale that assesses MG symptoms and their effects on daily activities. MG-ADL is composed of items related to patient's assessment of functional disability secondary to ocular (2 items), bulbar (3 items), respiratory (1 item), and gross motor or limb (2 items) impairment related to effects from MG. Each item is assessed on a 4-points scale where a score 0 represents normal function and a score 3 represents loss of ability to perform that function. The scores ranges from 0 to 24, with a higher score indicating more disability.
Trial sites (116)
| Facility | City | Region | Status |
|---|---|---|---|
| Honor Health Research Institute | Scottsdale | Arizona | |
| Fullerton Neuro and Headache Ctr | Fullerton | California | |
| California Pacific Medical Center | Sacramento | California | |
| Augusta University Georgia | Augusta | Georgia | |
| University of Chicago Medical Centr | Chicago | Illinois | |
| Prairie Heart Institute | Springfield | Illinois | |
| Mid Atlantic Epilepsy and Sleep Ctr | Bethesda | Maryland | |
| Massachusetts General Hospital | Boston | Massachusetts | |
| Henry Ford Health System | Detroit | Michigan | |
| Duke University Medical Center | Durham | North Carolina | |
| Penn Presbyterian Medical Center | Philadelphia | Pennsylvania | |
| Vanderbilt University Medical CenterX | Nashville | Tennessee | |
| Nerve and Muscle Center of Texas | Houston | Texas | |
| Novartis Investigative Site | CABA | Buenos Aires | |
| Novartis Investigative Site | Córdoba | Córdoba Province | |
| Novartis Investigative Site | Rosario | Santa Fe Province | |
| Novartis Investigative Site | Buenos Aires | Argentina | |
| Novartis Investigative Site | Caba | Argentina | |
| Novartis Investigative Site | Caba | Argentina | |
| Novartis Investigative Site | Capital Federal | Argentina | |
| Novartis Investigative Site | Córdoba | Argentina | |
| Novartis Investigative Site | Camperdown | Sydney | |
| Novartis Investigative Site | Porto Alegre | Rio Grande do Sul | |
| Novartis Investigative Site | Porto Alegre | Rio Grande do Sul | |
| Novartis Investigative Site | Joinville | Santa Catarina | |
| Novartis Investigative Site | Bahia | Brazil | |
| Novartis Investigative Site | São Paulo | Brazil | |
| Novartis Investigative Site | Hefei | Anhui | |
| Novartis Investigative Site | Guangzhou | Guangdong | |
| Novartis Investigative Site | Shenzhen | Guangdong | |
| Novartis Investigative Site | Shijiazhuang | Hebei | |
| Novartis Investigative Site | Changsha | Hunan | |
| Novartis Investigative Site | Suzhou | Jiangsu | |
| Novartis Investigative Site | Xi'an | Shaanxi | |
| Novartis Investigative Site | Xianyang | Shaanxi | |
| Novartis Investigative Site | Beijing | China | |
| Novartis Investigative Site | Beijing | China | |
| Novartis Investigative Site | Beijing | China | |
| Novartis Investigative Site | Fujian | China | |
| Novartis Investigative Site | Jinan | China |
+ 76 more sites — see the full list on the official registry below.
More Novartis Pharmaceuticals trials in Australia
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06517758 on ClinicalTrials.gov ↗ ← All trials in Australia