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

Status: Recruiting
Location: See all (87) locations...
Intervention Type: Drug, Other
Study Type: Interventional
Study Phase: Phase 3
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
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:

• Adult patients with generalized Myasthenia Gravis (age 18-75 years)

• Positive serology testing for AChR+ antibody at screening

• Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG and likely not in need v of a respirator for the duration of the study, as judged by the Investigator.

• The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests:

• History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation.

• History of positive edrophonium chloride test

• Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.

• Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms

• Participants not optimally controlled for ≥ 6 months on

• just one NSIST; or

• two or more NSISTs; or

• on frequent (at least quarterly) plasmapheresis, plasma exchange, or intravenous immunoglobulin to control symptoms despite treatment with steroids and NSISTs; or

• one of the following gMG treatments:

• a FcRN antagonist approved for gMG

• rituximab

• other approved gMG therapies excluding complement inhibitors.

• Consistent with all other iptacopan trials, participants will have to be vaccinated against Neisseria meningitidis and Streptococcus pneumoniae. In addition, participants will be vaccinated against Haemophilus influenzae, depending on the local regulations and on the availability of this vaccine in the countries of study conduct. The vaccination will be performed at least 2 weeks prior to first dosing with iptacopan, covering as many serotypes as possible. If iptacopan treatment will start earlier than 2 weeks post vaccination, prophylactic antibiotic treatment must be initiated and administered until 2 weeks post vaccination.

Locations
United States
Arizona
Honor Health Research Institute
RECRUITING
Scottsdale
California
Fullerton Neuro and Headache Ctr
RECRUITING
Fullerton
SC3 Research Pasadena
RECRUITING
Pasadena
Florida
Neurology Offices Of South Florida
WITHDRAWN
Boca Raton
Superior Associates in Research LLC
RECRUITING
Hialeah
Georgia
Augusta University Georgia
RECRUITING
Augusta
Hawaii
Hawaii Pacific Neuroscience LLC
RECRUITING
Honolulu
Illinois
University of Chicago Medical Centr
RECRUITING
Chicago
Prairie Heart Institute
RECRUITING
Springfield
North Carolina
Duke University Medical Center
RECRUITING
Durham
Ohio
Neuroscience Research Ctr
RECRUITING
Canton
Ohio State University Medical Center
RECRUITING
Columbus
Pennsylvania
Penn Presbyterian Medical Center
RECRUITING
Philadelphia
Tennessee
Vanderbilt University Medical CenterX
RECRUITING
Nashville
Texas
Nerve and Muscle Center of Texas
RECRUITING
Houston
Central TX Neuro Consultants P A
RECRUITING
Round Rock
Wisconsin
Center for Neurological Disorders G
RECRUITING
Greenfield
Other Locations
China
Novartis Investigative Site
RECRUITING
Beijing
Novartis Investigative Site
RECRUITING
Beijing
Novartis Investigative Site
RECRUITING
Changsha
Novartis Investigative Site
RECRUITING
Fujian
Novartis Investigative Site
RECRUITING
Guangzhou
Novartis Investigative Site
RECRUITING
Jinan
Novartis Investigative Site
RECRUITING
Nanchang
Novartis Investigative Site
RECRUITING
Shenzhen
Novartis Investigative Site
RECRUITING
Shijiazhuang
Novartis Investigative Site
RECRUITING
Suzhou
Novartis Investigative Site
RECRUITING
Xi'an
Novartis Investigative Site
RECRUITING
Xianyang
Denmark
Novartis Investigative Site
RECRUITING
Orbassano
Greece
Novartis Investigative Site
RECRUITING
Athens
Novartis Investigative Site
RECRUITING
Chaïdári
Novartis Investigative Site
RECRUITING
Larissa
Novartis Investigative Site
RECRUITING
Pátrai
Novartis Investigative Site
RECRUITING
Thessaloniki
Israel
Novartis Investigative Site
RECRUITING
Haifa
Novartis Investigative Site
RECRUITING
Jerusalem
Italy
Novartis Investigative Site
RECRUITING
Bologna
Novartis Investigative Site
RECRUITING
Florence
Novartis Investigative Site
RECRUITING
Genova
Novartis Investigative Site
RECRUITING
Milan
Novartis Investigative Site
RECRUITING
Napoli
Novartis Investigative Site
RECRUITING
Palermo
Novartis Investigative Site
RECRUITING
Palermo
Novartis Investigative Site
RECRUITING
Roma
Novartis Investigative Site
RECRUITING
Roma
Novartis Investigative Site
RECRUITING
Roma
Japan
Novartis Investigative Site
RECRUITING
Chiba
Novartis Investigative Site
RECRUITING
Fukuoka
Novartis Investigative Site
RECRUITING
Fukushima
Novartis Investigative Site
RECRUITING
Hanamaki
Novartis Investigative Site
RECRUITING
Higashi-matsuyama
Novartis Investigative Site
RECRUITING
Hiroshima
Novartis Investigative Site
RECRUITING
Narita
Novartis Investigative Site
RECRUITING
Nishinomiya
Novartis Investigative Site
RECRUITING
Sapporo
Novartis Investigative Site
RECRUITING
Sendai
Novartis Investigative Site
RECRUITING
Shinjuku Ku
Novartis Investigative Site
RECRUITING
Suita
Poland
Novartis Investigative Site
RECRUITING
Bydgoszcz
Novartis Investigative Site
RECRUITING
Katowice
Novartis Investigative Site
RECRUITING
Katowice
Novartis Investigative Site
RECRUITING
Krakow
Novartis Investigative Site
RECRUITING
Krakow
Novartis Investigative Site
RECRUITING
Lublin
Novartis Investigative Site
RECRUITING
Lublin
Novartis Investigative Site
RECRUITING
Rzeszów
Novartis Investigative Site
RECRUITING
Warsaw
Portugal
Novartis Investigative Site
RECRUITING
Lisbon
Novartis Investigative Site
RECRUITING
Lisbon
Novartis Investigative Site
RECRUITING
Porto
Novartis Investigative Site
RECRUITING
Vila Nova De Gaia
Spain
Novartis Investigative Site
RECRUITING
Alicante
Novartis Investigative Site
RECRUITING
Barcelona
Novartis Investigative Site
RECRUITING
L'hospitalet De Llobregat
Novartis Investigative Site
RECRUITING
Lleida
Novartis Investigative Site
RECRUITING
Málaga
Novartis Investigative Site
RECRUITING
Pozuelo De Alarcón
Novartis Investigative Site
RECRUITING
Santiago De Compostela
Novartis Investigative Site
RECRUITING
Valencia
United Kingdom
Novartis Investigative Site
RECRUITING
Birmingham
Novartis Investigative Site
RECRUITING
Ilford
Novartis Investigative Site
RECRUITING
Inverness
Novartis Investigative Site
RECRUITING
Liverpool
Novartis Investigative Site
RECRUITING
London
Novartis Investigative Site
RECRUITING
Southampton
Novartis Investigative Site
RECRUITING
Swinton
Contact Information
Primary
Novartis Pharmaceuticals
novartis.email@novartis.com
1-888-669-6682
Backup
Novartis Pharmaceuticals
+41613241111
Time Frame
Start Date: 2024-07-31
Estimated Completion Date: 2032-05-27
Participants
Target number of participants: 146
Treatments
Experimental: Iptacopan
Iptacopan orally for 6 months (double-blind) followed by open-label iptacopan for an additional 24 months
Placebo_comparator: Matching Placebo
Placebo orally for 6 months (double-blind) followed by open-label iptacopan for 24 months
Related Therapeutic Areas
Sponsors
Leads: Novartis Pharmaceuticals

This content was sourced from clinicaltrials.gov

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