Actively Recruiting

Phase 3
Age: 18Years - 85Years
All Genders
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.

Led by Novartis Pharmaceuticals · Updated on 2026-05-14

146

Participants Needed

113

Research Sites

408 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official 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

Age: 18Years - 85Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Adults aged 18 to 85 years with generalized Myasthenia Gravis
  • Positive test for acetylcholine receptor (AChR) antibodies
  • Myasthenia Gravis Foundation of America Class II-IV at screening
  • Diagnosis confirmed by at least one of: abnormal neuromuscular transmission tests, positive response to short-acting acetylcholinesterase inhibitors, or improvement on oral acetylcholinesterase inhibitors
  • Baseline MG-ADL score of 6 or higher, with at least 50% of the score from non-ocular symptoms
  • Receiving stable treatment for gMG for at least 6 months, including NSISTs, plasmapheresis, plasma exchange, intravenous immunoglobulin, FcRN antagonists, rituximab, or other approved disease-modifying therapies excluding complement inhibitors
  • Vaccinated against Neisseria meningitidis and Streptococcus pneumoniae according to local guidelines prior to study treatment
Not Eligible

You will not qualify if you...

  • Treatment with intravenous immunoglobulin or plasma exchange within the past month
  • Treatment with rituximab within the past 6 months
  • Treatment with eculizumab within the past 2 months
  • Treatment with ravulizumab or other complement inhibitors within the past 3 months
  • Treatment with efgartigimod or other anti-FcRn therapies within the past 3 months
  • Thymectomy within the past 6 months or planned thymectomy during the trial
  • Clinically significant active or uncontrolled infections including active Hepatitis B or C, HIV with AIDS-defining condition or low CD4 count
  • Pregnant, lactating, or intending to become pregnant
  • Women of child-bearing potential not using effective contraception during and one week after treatment
  • Active systemic bacterial, viral (including COVID-19), or fungal infection or recent major infection requiring hospitalization or injectable therapy within 14 days prior to study treatment
  • History of recurrent invasive infections from encapsulated organisms
  • Fever of 38°C (100.4°F) or higher within 7 days before study treatment

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 113 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

Duke University Medical Center

Durham, North Carolina, United States, 27710

Actively Recruiting

13

Neuroscience Research Ctr

Canton, Ohio, United States, 44718

Withdrawn

14

Ohio State University Medical Center

Columbus, Ohio, United States, 43210

Withdrawn

15

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

16

Vanderbilt University Medical CenterX

Nashville, Tennessee, United States, 37221

Actively Recruiting

17

Nerve and Muscle Center of Texas

Houston, Texas, United States, 77030

Actively Recruiting

18

Central TX Neuro Consultants P A

Round Rock, Texas, United States, 78681

Actively Recruiting

19

Center for Neurological Disorders G

Greenfield, Wisconsin, United States, 53228-1321

Actively Recruiting

20

Novartis Investigative Site

Buenos Aires, Argentina, C1012AAR

Actively Recruiting

21

Novartis Investigative Site

Córdoba, Argentina, X5004AOA

Actively Recruiting

22

Novartis Investigative Site

Porto Alegre, Rio Grande do Sul, Brazil, 90560-030

Actively Recruiting

23

Novartis Investigative Site

Joinville, Santa Catarina, Brazil, 89202-165

Actively Recruiting

24

Novartis Investigative Site

São Paulo, Brazil, 04038-002

Actively Recruiting

25

Novartis Investigative Site

Hefei, Anhui, China, 230001

Actively Recruiting

26

Novartis Investigative Site

Guangzhou, Guangdong, China, 510515

Actively Recruiting

27

Novartis Investigative Site

Shenzhen, Guangdong, China, 518053

Actively Recruiting

28

Novartis Investigative Site

Shijiazhuang, Hebei, China, 50030

Actively Recruiting

29

Novartis Investigative Site

Changsha, Hunan, China, 410008

Actively Recruiting

30

Novartis Investigative Site

Suzhou, Jiangsu, China, 215004

Actively Recruiting

31

Novartis Investigative Site

Nanchang, Jiangxi, China, 330006

Actively Recruiting

32

Novartis Investigative Site

Xi'an, Shaanxi, China, 710075

Actively Recruiting

33

Novartis Investigative Site

Xianyang, Shaanxi, China, 712000

Actively Recruiting

34

Novartis Investigative Site

Beijing, China, 065001

Actively Recruiting

35

Novartis Investigative Site

Beijing, China, 100034

Actively Recruiting

36

Novartis Investigative Site

Beijing, China, 100730

Actively Recruiting

37

Novartis Investigative Site

Fujian, China, 350001

Actively Recruiting

38

Novartis Investigative Site

Jinan, China, 250012

Actively Recruiting

39

Novartis Investigative Site

Copenhagen, Denmark, DK-2100

Actively Recruiting

40

Novartis Investigative Site

Limoges, Haute Vienne, France, 87000

Actively Recruiting

41

Novartis Investigative Site

Garches, France, 92380

Actively Recruiting

42

Novartis Investigative Site

Nice, France, 06001

Actively Recruiting

43

Novartis Investigative Site

Paris, France, 75013

Actively Recruiting

44

Novartis Investigative Site

Paris, France, 75940

Actively Recruiting

45

Novartis Investigative Site

Würzburg, Bavaria, Germany, 97070

Actively Recruiting

46

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany, 60590

Actively Recruiting

47

Novartis Investigative Site

Bochum, Germany, 44789

Actively Recruiting

48

Novartis Investigative Site

Athens, Greece, 115 28

Actively Recruiting

49

Novartis Investigative Site

Chaïdári, Greece, 124 62

Actively Recruiting

50

Novartis Investigative Site

Larissa, Greece, 411 10

Actively Recruiting

51

Novartis Investigative Site

Pátrai, Greece, 265 04

Actively Recruiting

52

Novartis Investigative Site

Thessaloniki, Greece, 54636

Actively Recruiting

53

Novartis Investigative Site

Haifa, Israel, 3109601

Actively Recruiting

54

Novartis Investigative Site

Jerusalem, Israel, 9112001

Actively Recruiting

55

Novartis Investigative Site

Bologna, BO, Italy, 40139

Actively Recruiting

56

Novartis Investigative Site

Florence, FI, Italy, 50134

Actively Recruiting

57

Novartis Investigative Site

Genova, GE, Italy, 16132

Actively Recruiting

58

Novartis Investigative Site

Milan, MI, Italy, 20133

Actively Recruiting

59

Novartis Investigative Site

Palermo, PA, Italy, 90127

Actively Recruiting

60

Novartis Investigative Site

Palermo, PA, Italy, 90146

Actively Recruiting

61

Novartis Investigative Site

Roma, RM, Italy, 00133

Actively Recruiting

62

Novartis Investigative Site

Roma, RM, Italy, 00135

Actively Recruiting

63

Novartis Investigative Site

Roma, RM, Italy, 00189

Actively Recruiting

64

Novartis Investigative Site

Orbassano, TO, Italy, 10043

Actively Recruiting

65

Novartis Investigative Site

Naples, Italy, 80131

Actively Recruiting

66

Novartis Investigative Site

Narita, Chiba, Japan, 286-8520

Actively Recruiting

67

Novartis Investigative Site

Sapporo, Hokkaido, Japan, 0630005

Actively Recruiting

68

Novartis Investigative Site

Nishinomiya, Hyōgo, Japan, 6638501

Actively Recruiting

69

Novartis Investigative Site

Hanamaki, Iwate, Japan, 0250082

Actively Recruiting

70

Novartis Investigative Site

Sendai, Miyagi, Japan, 9838520

Actively Recruiting

71

Novartis Investigative Site

Suita, Osaka, Japan, 565-0871

Actively Recruiting

72

Novartis Investigative Site

Higashi-Matsuyama, Saitama, Japan, 355-0005

Actively Recruiting

73

Novartis Investigative Site

Shinjuku Ku, Tokyo, Japan, 160-0023

Actively Recruiting

74

Novartis Investigative Site

Chiba, Japan, 2608677

Actively Recruiting

75

Novartis Investigative Site

Fukuoka, Japan, 8128582

Actively Recruiting

76

Novartis Investigative Site

Fukushima, Japan, 9601295

Actively Recruiting

77

Novartis Investigative Site

Hiroshima, Japan, 7348551

Actively Recruiting

78

Novartis Investigative Site

Lublin, Lublin Voivodeship, Poland, 20-064

Actively Recruiting

79

Novartis Investigative Site

Warsaw, Masovian Voivodeship, Poland, 02-676

Actively Recruiting

80

Novartis Investigative Site

Krakow, POL, Poland, 31-505

Actively Recruiting

81

Novartis Investigative Site

Katowice, Silesian Voivodeship, Poland, 40-650

Actively Recruiting

82

Novartis Investigative Site

Bydgoszcz, Poland, 85-065

Actively Recruiting

83

Novartis Investigative Site

Katowice, Poland, 40-689

Actively Recruiting

84

Novartis Investigative Site

Krakow, Poland, 31-870

Actively Recruiting

85

Novartis Investigative Site

Lublin, Poland, 20-410

Actively Recruiting

86

Novartis Investigative Site

Poznan, Poland, 61-731

Actively Recruiting

87

Novartis Investigative Site

Rzeszów, Poland, 35-055

Actively Recruiting

88

Novartis Investigative Site

Warsaw, Poland, 01-684

Actively Recruiting

89

Novartis Investigative Site

Lisbon, Portugal, 1349-019

Actively Recruiting

90

Novartis Investigative Site

Lisbon, Portugal, 1649-035

Actively Recruiting

91

Novartis Investigative Site

Porto, Portugal, 4099-001

Actively Recruiting

92

Novartis Investigative Site

Vila Nova de Gaia, Portugal, 4434 502

Actively Recruiting

93

Novartis Investigative Site

Belgrade, Serbia, 11000

Actively Recruiting

94

Novartis Investigative Site

Niš, Serbia, 18108

Actively Recruiting

95

Novartis Investigative Site

Daegu, South Korea, 41404

Actively Recruiting

96

Novartis Investigative Site

Gwangju, South Korea, 61469

Actively Recruiting

97

Novartis Investigative Site

Seoul, South Korea, 03722

Actively Recruiting

98

Novartis Investigative Site

Seoul, South Korea, 04763

Actively Recruiting

99

Novartis Investigative Site

Santiago Compostela, A Coruna, Spain, 15706

Actively Recruiting

100

Novartis Investigative Site

L'Hospitalet de Llobregat, Barcelona, Spain, 08907

Actively Recruiting

101

Novartis Investigative Site

Alicante, Spain, 03010

Actively Recruiting

102

Novartis Investigative Site

Barcelona, Spain, 08036

Actively Recruiting

103

Novartis Investigative Site

Lleida, Spain, 25198

Actively Recruiting

104

Novartis Investigative Site

Madrid, Spain, 28006

Actively Recruiting

105

Novartis Investigative Site

Málaga, Spain, 29010

Actively Recruiting

106

Novartis Investigative Site

Valencia, Spain, 46026

Actively Recruiting

107

Novartis Investigative Site

Inverness, Invernesshire, United Kingdom, IV2 3RE

Actively Recruiting

108

Novartis Investigative Site

Ilford, London, United Kingdom, IG1 4HP

Withdrawn

109

Novartis Investigative Site

Swinton, Manchester, United Kingdom, M27 8FF

Actively Recruiting

110

Novartis Investigative Site

Birmingham, West Midlands, United Kingdom, B15 2TH

Actively Recruiting

111

Novartis Investigative Site

Liverpool, United Kingdom, L9 7LT

Withdrawn

112

Novartis Investigative Site

London, United Kingdom, SW17 0QT

Actively Recruiting

113

Novartis Investigative Site

Southampton, United Kingdom, SO16 6YD

Actively Recruiting

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Research Team

N

Novartis Pharmaceuticals

CONTACT

N

Novartis Pharmaceuticals

CONTACT

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