Actively Recruiting
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
Led by Novartis Pharmaceuticals · Updated on 2026-05-27
146
Participants Needed
124
Research Sites
264 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the efficacy, safety, and tolerability of iptacopan in adults aged 18 to 85 years with generalized Myasthenia Gravis who are positive for AChR antibodies and are on stable standard-of-care treatment. This randomized, double-blind, placebo-controlled, multicenter Phase III study aims to compare iptacopan with placebo over six months, followed by a long-term open-label extension to further assess treatment effects and safety. Participants will be randomly assigned in equal groups to receive either oral iptacopan or a matching placebo daily for six months while continuing their standard care. After this double-blind phase, all participants are offered open-label iptacopan treatment for up to 60 months. Safety follow-up assessments occur 7 and 30 days after the last study treatment dose. During the study, participants will attend regular visits to assess changes in their Myasthenia Gravis symptoms using several scales, including the MG-ADL score, QMG, MGC, and quality of life questionnaires. Researchers will monitor adverse events and corticosteroid use throughout both the core and extension phases. The total study participation can last up to about five years, including the initial treatment, extension, and follow-up periods.
CONDITIONS
Brief Title
A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 85 years with generalized Myasthenia Gravis at screening
- Positive antibody test for AChR+ at screening
- MGFA Class II-IV generalized Myasthenia Gravis and unlikely to need a respirator during the study
- Diagnosis confirmed by at least one test: abnormal neuromuscular transmission, positive acetylcholinesterase inhibitor test, or symptom improvement on oral acetylcholinesterase inhibitors
- Baseline MG-ADL score of 6 or higher with at least 50% from non-ocular symptoms
- On stable treatment for at least 6 months with one or more NSISTs, plasmapheresis, intravenous immunoglobulin, approved FcRN antagonist, rituximab, or other approved disease-modifying therapies excluding complement inhibitors
- Vaccinated against Neisseria meningitidis and Streptococcus pneumoniae before starting study treatment, or receiving appropriate prophylactic antibiotics if needed
You will not qualify if you...
- Treatment with intravenous immunoglobulin or plasma exchange in past month
- Treatment with rituximab in past 6 months
- Treatment with eculizumab in past 2 months or ravulizumab/complement inhibitors in past 3 months
- Treatment with efgartigimod or other anti-FcRn therapies in past 3 months
- Thymectomy within past 6 months or planned during the trial
- Active or uncontrolled infections including Hepatitis B, Hepatitis C, or HIV with AIDS-defining conditions or low CD4 count
- Pregnant or lactating women, or those planning pregnancy
- Women of childbearing potential not using effective contraception during and one week after treatment
- Active systemic infections or major infections requiring hospitalization or injectable therapy within 14 days prior to treatment
- History of recurrent invasive infections from encapsulated organisms
- Fever of 38°C (100.4°F) or higher within 7 days before treatment
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 months
Participants receive either Iptacopan or placebo orally during a 6-month double-blind treatment period.
Visits during the 6-month treatment period (visit frequency as per protocol)
Duration - Up to 60 months
Participants receive open-label Iptacopan for up to 60 months following the double-blind period.
Visits during the extension period as scheduled by the study
Duration - Approximately 1 month
Participants undergo safety follow-up assessments after the last administration of study treatment.
2 visits: one 7 days and one 30 days after last treatment
Trial Site Locations
Total: 124 locations
1
Honor Health Research Institute
Scottsdale, Arizona, United States, 85258
Actively Recruiting
2
Fullerton Neuro and Headache Ctr
Fullerton, California, United States, 92835
Actively Recruiting
3
SC3 Research Pasadena
Pasadena, California, United States, 91105
Actively Recruiting
4
California Pacific Medical Center
Sacramento, California, United States, 94115
Actively Recruiting
5
Neurology Offices Of South Florida
Boca Raton, Florida, United States, 33428
Withdrawn
6
Superior Associates in Research LLC
Hialeah, Florida, United States, 33012
Withdrawn
7
Augusta University Georgia
Augusta, Georgia, United States, 30912
Actively Recruiting
8
Hawaii Pacific Neuroscience LLC
Honolulu, Hawaii, United States, 96817
Withdrawn
9
University of Chicago Medical Centr
Chicago, Illinois, United States, 60637
Actively Recruiting
10
Prairie Heart Institute
Springfield, Illinois, United States, 62769
Actively Recruiting
11
Mid Atlantic Epilepsy and Sleep Ctr
Bethesda, Maryland, United States, 20817-1807
Actively Recruiting
12
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Actively Recruiting
13
Henry Ford Health System
Detroit, Michigan, United States, 48202
Actively Recruiting
14
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
15
Neuroscience Research Ctr
Canton, Ohio, United States, 44718
Withdrawn
16
Ohio State University Medical Center
Columbus, Ohio, United States, 43210
Withdrawn
17
Penn Presbyterian Medical Center
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
18
Vanderbilt University Medical CenterX
Nashville, Tennessee, United States, 37221
Actively Recruiting
19
Nerve and Muscle Center of Texas
Houston, Texas, United States, 77030
Actively Recruiting
20
Central TX Neuro Consultants P A
Round Rock, Texas, United States, 78681
Withdrawn
21
Center for Neurological Disorders G
Greenfield, Wisconsin, United States, 53228-1321
Actively Recruiting
22
Novartis Investigative Site
CABA, Buenos Aires, Argentina, C1428AQK
Actively Recruiting
23
Novartis Investigative Site
Buenos Aires, Argentina, C1012AAR
Actively Recruiting
24
Novartis Investigative Site
Córdoba, Argentina, X5004AOA
Actively Recruiting
25
Novartis Investigative Site
Córdoba, Argentina, X5004CDT
Actively Recruiting
26
Novartis Investigative Site
Camperdown, Sydney, Australia, NSW 2050
Actively Recruiting
27
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
28
Novartis Investigative Site
Porto Alegre, Rio Grande do Sul, Brazil, 90560-030
Actively Recruiting
29
Novartis Investigative Site
Joinville, Santa Catarina, Brazil, 89202-165
Actively Recruiting
30
Novartis Investigative Site
São Paulo, Brazil, 04038-002
Actively Recruiting
31
Novartis Investigative Site
Hefei, Anhui, China, 230001
Actively Recruiting
32
Novartis Investigative Site
Guangzhou, Guangdong, China, 510515
Actively Recruiting
33
Novartis Investigative Site
Shenzhen, Guangdong, China, 518053
Actively Recruiting
34
Novartis Investigative Site
Shijiazhuang, Hebei, China, 50030
Actively Recruiting
35
Novartis Investigative Site
Changsha, Hunan, China, 410008
Actively Recruiting
36
Novartis Investigative Site
Suzhou, Jiangsu, China, 215004
Actively Recruiting
37
Novartis Investigative Site
Nanchang, Jiangxi, China, 330006
Actively Recruiting
38
Novartis Investigative Site
Xi'an, Shaanxi, China, 710075
Actively Recruiting
39
Novartis Investigative Site
Xianyang, Shaanxi, China, 712000
Actively Recruiting
40
Novartis Investigative Site
Beijing, China, 065001
Actively Recruiting
41
Novartis Investigative Site
Beijing, China, 100034
Actively Recruiting
42
Novartis Investigative Site
Beijing, China, 100730
Actively Recruiting
43
Novartis Investigative Site
Fujian, China, 350001
Actively Recruiting
44
Novartis Investigative Site
Jinan, China, 250012
Actively Recruiting
45
Novartis Investigative Site
Copenhagen, Denmark, DK-2100
Actively Recruiting
46
Novartis Investigative Site
Limoges, Haute Vienne, France, 87000
Actively Recruiting
47
Novartis Investigative Site
Garches, France, 92380
Actively Recruiting
48
Novartis Investigative Site
Nice, France, 06001
Actively Recruiting
49
Novartis Investigative Site
Paris, France, 75013
Actively Recruiting
50
Novartis Investigative Site
Paris, France, 75940
Actively Recruiting
51
Novartis Investigative Site
Munich, Bavaria, Germany, 81675
Actively Recruiting
52
Novartis Investigative Site
Regensburg, Bavaria, Germany, 93053
Actively Recruiting
53
Novartis Investigative Site
Würzburg, Bavaria, Germany, 97070
Actively Recruiting
54
Novartis Investigative Site
Frankfurt am Main, Hesse, Germany, 60590
Actively Recruiting
55
Novartis Investigative Site
Bochum, Germany, 44789
Actively Recruiting
56
Novartis Investigative Site
Bochum, Germany, 44791
Actively Recruiting
57
Novartis Investigative Site
Essen, Germany, 45147
Actively Recruiting
58
Novartis Investigative Site
Athens, Greece, 115 28
Actively Recruiting
59
Novartis Investigative Site
Chaïdári, Greece, 124 62
Actively Recruiting
60
Novartis Investigative Site
Larissa, Greece, 411 10
Actively Recruiting
61
Novartis Investigative Site
Pátrai, Greece, 265 04
Withdrawn
62
Novartis Investigative Site
Thessaloniki, Greece, 54636
Actively Recruiting
63
Novartis Investigative Site
Haifa, Israel, 3109601
Actively Recruiting
64
Novartis Investigative Site
Jerusalem, Israel, 9112001
Actively Recruiting
65
Novartis Investigative Site
Bologna, BO, Italy, 40139
Actively Recruiting
66
Novartis Investigative Site
Florence, FI, Italy, 50134
Actively Recruiting
67
Novartis Investigative Site
Genova, GE, Italy, 16132
Actively Recruiting
68
Novartis Investigative Site
Milan, MI, Italy, 20133
Actively Recruiting
69
Novartis Investigative Site
Palermo, PA, Italy, 90127
Actively Recruiting
70
Novartis Investigative Site
Palermo, PA, Italy, 90146
Actively Recruiting
71
Novartis Investigative Site
Roma, RM, Italy, 00133
Actively Recruiting
72
Novartis Investigative Site
Roma, RM, Italy, 00135
Actively Recruiting
73
Novartis Investigative Site
Roma, RM, Italy, 00189
Actively Recruiting
74
Novartis Investigative Site
Orbassano, TO, Italy, 10043
Actively Recruiting
75
Novartis Investigative Site
Naples, Italy, 80131
Actively Recruiting
76
Novartis Investigative Site
Narita, Chiba, Japan, 286-8520
Actively Recruiting
77
Novartis Investigative Site
Sapporo, Hokkaido, Japan, 0630005
Actively Recruiting
78
Novartis Investigative Site
Nishinomiya, Hyōgo, Japan, 6638501
Actively Recruiting
79
Novartis Investigative Site
Hanamaki, Iwate, Japan, 0250082
Actively Recruiting
80
Novartis Investigative Site
Sendai, Miyagi, Japan, 9838520
Actively Recruiting
81
Novartis Investigative Site
Suita, Osaka, Japan, 565-0871
Actively Recruiting
82
Novartis Investigative Site
Higashi-Matsuyama, Saitama, Japan, 355-0005
Actively Recruiting
83
Novartis Investigative Site
Shinjuku Ku, Tokyo, Japan, 160-0023
Actively Recruiting
84
Novartis Investigative Site
Chiba, Japan, 2608677
Actively Recruiting
85
Novartis Investigative Site
Fukuoka, Japan, 8128582
Actively Recruiting
86
Novartis Investigative Site
Fukushima, Japan, 9601295
Actively Recruiting
87
Novartis Investigative Site
Hiroshima, Japan, 7348551
Actively Recruiting
88
Novartis Investigative Site
Lublin, Lublin Voivodeship, Poland, 20-064
Actively Recruiting
89
Novartis Investigative Site
Warsaw, Masovian Voivodeship, Poland, 02-676
Actively Recruiting
90
Novartis Investigative Site
Krakow, POL, Poland, 31-505
Actively Recruiting
91
Novartis Investigative Site
Katowice, Silesian Voivodeship, Poland, 40-650
Actively Recruiting
92
Novartis Investigative Site
Bydgoszcz, Poland, 85-065
Actively Recruiting
93
Novartis Investigative Site
Katowice, Poland, 40-689
Actively Recruiting
94
Novartis Investigative Site
Krakow, Poland, 31-870
Actively Recruiting
95
Novartis Investigative Site
Lublin, Poland, 20-410
Actively Recruiting
96
Novartis Investigative Site
Poznan, Poland, 61-731
Actively Recruiting
97
Novartis Investigative Site
Rzeszów, Poland, 35-055
Actively Recruiting
98
Novartis Investigative Site
Warsaw, Poland, 01-684
Actively Recruiting
99
Novartis Investigative Site
Lisbon, Portugal, 1349-019
Actively Recruiting
100
Novartis Investigative Site
Lisbon, Portugal, 1649-035
Actively Recruiting
101
Novartis Investigative Site
Porto, Portugal, 4099-001
Actively Recruiting
102
Novartis Investigative Site
Vila Nova de Gaia, Portugal, 4434 502
Actively Recruiting
103
Novartis Investigative Site
Belgrade, Serbia, 11000
Actively Recruiting
104
Novartis Investigative Site
Niš, Serbia, 18108
Actively Recruiting
105
Novartis Investigative Site
Daegu, South Korea, 41404
Actively Recruiting
106
Novartis Investigative Site
Gwangju, South Korea, 61469
Actively Recruiting
107
Novartis Investigative Site
Seoul, South Korea, 03722
Actively Recruiting
108
Novartis Investigative Site
Seoul, South Korea, 04763
Actively Recruiting
109
Novartis Investigative Site
Santiago Compostela, A Coruna, Spain, 15706
Actively Recruiting
110
Novartis Investigative Site
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
Actively Recruiting
111
Novartis Investigative Site
Alicante, Spain, 03010
Actively Recruiting
112
Novartis Investigative Site
Barcelona, Spain, 08036
Actively Recruiting
113
Novartis Investigative Site
Lleida, Spain, 25198
Actively Recruiting
114
Novartis Investigative Site
Madrid, Spain, 28006
Actively Recruiting
115
Novartis Investigative Site
Málaga, Spain, 29010
Actively Recruiting
116
Novartis Investigative Site
Valencia, Spain, 46026
Actively Recruiting
117
Novartis Investigative Site
Inverness, Invernesshire, United Kingdom, IV2 3RE
Actively Recruiting
118
Novartis Investigative Site
Ilford, London, United Kingdom, IG1 4HP
Withdrawn
119
Novartis Investigative Site
Swinton, Manchester, United Kingdom, M27 8FF
Actively Recruiting
120
Novartis Investigative Site
Birmingham, West Midlands, United Kingdom, B15 2TH
Actively Recruiting
121
Novartis Investigative Site
Liverpool, United Kingdom, L9 7LT
Withdrawn
122
Novartis Investigative Site
London, United Kingdom, NW1 2BU
Actively Recruiting
123
Novartis Investigative Site
London, United Kingdom, SW17 0QT
Actively Recruiting
124
Novartis Investigative Site
Southampton, United Kingdom, SO16 6YD
Actively Recruiting
Research Team
N
Novartis Pharmaceuticals
N
Novartis Pharmaceuticals
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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