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A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 75 Years With gMG.

NCT ID: NCT06517758

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

146 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-07-31

Study Completion Date

2032-05-27

Brief Summary

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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.

Detailed Description

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The study consists of a 6-month double-blind treatment period for the primary efficacy and safety analysis followed by a 24 month open label extension period. A safety follow up assessment will be performed, one 7 days after the last administration of study treatment and one 30 days after the last administration of study treatment for all participants.

Conditions

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Generalized Myasthenia Gravis

Keywords

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Myasthenia Gravis gMG randomized placebo-controlled double-blind phase III AChR+ SOC treatment iptacopan LNP023

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

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.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
This is a participant, investigator, and sponsor-blinded study. Participants, investigator staff, persons performing the assessments and the Clinical Trial Team will remain blinded to the identity of treatment from the time of randomization until database lock after all participants have completed the double-blind treatment period. The following methods will be used to ensure that blinding is properly maintained:

1. Randomization data are kept strictly confidential until the time of unblinding and will not be accessible by anyone involved in the study
2. The identity of the treatment will be concealed by the use of study treatments that are all identical in packaging, labeling, schedule of administration, appearance, taste, and odor

Study Groups

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Iptacopan

Iptacopan orally for 6 months (double-blind) followed by open-label iptacopan for an additional 24 months

Group Type EXPERIMENTAL

Iptacopan

Intervention Type DRUG

Hard gelatin capsule

Matching Placebo

Placebo orally for 6 months (double-blind) followed by open-label iptacopan for 24 months

Group Type PLACEBO_COMPARATOR

Matching Placebo

Intervention Type OTHER

Hard gelatin capsule

Interventions

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Iptacopan

Hard gelatin capsule

Intervention Type DRUG

Matching Placebo

Hard gelatin capsule

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* 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.

Exclusion Criteria

* Have been treated with intravenous immunoglobulin (IVIG)/plasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti- FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period.
* Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV): serologic panel test results indicative of an active (acute or chronic) infection; Active Hepatitis C Virus (HCV): serology positive for HCV-Ab; Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count

* 200 cells/mm3
* Female participants who are pregnant or lactating, or are intending to become pregnant.
* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using effective methods of contraception during dosing of study treatment.
* Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration.
* History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.
* Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Honor Health Research Institute

Scottsdale, Arizona, United States

Site Status RECRUITING

Fullerton Neuro and Headache Ctr

Fullerton, California, United States

Site Status RECRUITING

SC3 Research Pasadena

Pasadena, California, United States

Site Status RECRUITING

Neurology Offices Of South Florida

Boca Raton, Florida, United States

Site Status WITHDRAWN

Superior Associates in Research LLC

Hialeah, Florida, United States

Site Status RECRUITING

Augusta University Georgia

Augusta, Georgia, United States

Site Status RECRUITING

Hawaii Pacific Neuroscience LLC

Honolulu, Hawaii, United States

Site Status RECRUITING

University of Chicago Medical Centr

Chicago, Illinois, United States

Site Status RECRUITING

Prairie Heart Institute

Springfield, Illinois, United States

Site Status RECRUITING

Mid Atlantic Epilepsy and Sleep Ctr

Bethesda, Maryland, United States

Site Status RECRUITING

Duke University Medical Center

Durham, North Carolina, United States

Site Status RECRUITING

Neuroscience Research Ctr

Canton, Ohio, United States

Site Status RECRUITING

Ohio State University Medical Center

Columbus, Ohio, United States

Site Status RECRUITING

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Vanderbilt University Medical CenterX

Nashville, Tennessee, United States

Site Status RECRUITING

Nerve and Muscle Center of Texas

Houston, Texas, United States

Site Status RECRUITING

Central TX Neuro Consultants P A

Round Rock, Texas, United States

Site Status RECRUITING

Center for Neurological Disorders G

Greenfield, Wisconsin, United States

Site Status RECRUITING

Novartis Investigative Site

Guangzhou, Guangdong, China

Site Status RECRUITING

Novartis Investigative Site

Shenzhen, Guangdong, China

Site Status RECRUITING

Novartis Investigative Site

Shijiazhuang, Hebei, China

Site Status RECRUITING

Novartis Investigative Site

Changsha, Hunan, China

Site Status RECRUITING

Novartis Investigative Site

Suzhou, Jiangsu, China

Site Status RECRUITING

Novartis Investigative Site

Nanchang, Jiangxi, China

Site Status RECRUITING

Novartis Investigative Site

Xi'an, Shaanxi, China

Site Status RECRUITING

Novartis Investigative Site

Xianyang, Shaanxi, China

Site Status RECRUITING

Novartis Investigative Site

Beijing, , China

Site Status RECRUITING

Novartis Investigative Site

Beijing, , China

Site Status RECRUITING

Novartis Investigative Site

Fujian, , China

Site Status RECRUITING

Novartis Investigative Site

Jinan, , China

Site Status RECRUITING

Novartis Investigative Site

Copenhagen, , Denmark

Site Status RECRUITING

Novartis Investigative Site

Athens, , Greece

Site Status RECRUITING

Novartis Investigative Site

Chaïdári, , Greece

Site Status RECRUITING

Novartis Investigative Site

Larissa, , Greece

Site Status RECRUITING

Novartis Investigative Site

Pátrai, , Greece

Site Status RECRUITING

Novartis Investigative Site

Thessaloniki, , Greece

Site Status RECRUITING

Novartis Investigative Site

Haifa, , Israel

Site Status RECRUITING

Novartis Investigative Site

Jerusalem, , Israel

Site Status RECRUITING

Novartis Investigative Site

Bologna, BO, Italy

Site Status RECRUITING

Novartis Investigative Site

Florence, FI, Italy

Site Status RECRUITING

Novartis Investigative Site

Genova, GE, Italy

Site Status RECRUITING

Novartis Investigative Site

Milan, MI, Italy

Site Status RECRUITING

Novartis Investigative Site

Palermo, PA, Italy

Site Status RECRUITING

Novartis Investigative Site

Palermo, PA, Italy

Site Status RECRUITING

Novartis Investigative Site

Roma, RM, Italy

Site Status RECRUITING

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Roma, RM, Italy

Site Status RECRUITING

Novartis Investigative Site

Roma, RM, Italy

Site Status RECRUITING

Novartis Investigative Site

Orbassano, TO, Italy

Site Status RECRUITING

Novartis Investigative Site

Napoli, , Italy

Site Status RECRUITING

Novartis Investigative Site

Chiba, Chiba, Japan

Site Status RECRUITING

Novartis Investigative Site

Narita, Chiba, Japan

Site Status RECRUITING

Novartis Investigative Site

Fukuoka, Fukuoka, Japan

Site Status RECRUITING

Novartis Investigative Site

Fukushima, Fukushima, Japan

Site Status RECRUITING

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Sapporo, Hokkaido, Japan

Site Status RECRUITING

Novartis Investigative Site

Nishinomiya, Hyōgo, Japan

Site Status RECRUITING

Novartis Investigative Site

Hanamaki, Iwate, Japan

Site Status RECRUITING

Novartis Investigative Site

Sendai, Miyagi, Japan

Site Status RECRUITING

Novartis Investigative Site

Suita, Osaka, Japan

Site Status RECRUITING

Novartis Investigative Site

Higashi-Matsuyama, Saitama, Japan

Site Status RECRUITING

Novartis Investigative Site

Shinjuku Ku, Tokyo, Japan

Site Status RECRUITING

Novartis Investigative Site

Hiroshima, , Japan

Site Status RECRUITING

Novartis Investigative Site

Lublin, Lublin Voivodeship, Poland

Site Status RECRUITING

Novartis Investigative Site

Krakow, POL, Poland

Site Status RECRUITING

Novartis Investigative Site

Katowice, Silesian Voivodeship, Poland

Site Status RECRUITING

Novartis Investigative Site

Bydgoszcz, , Poland

Site Status RECRUITING

Novartis Investigative Site

Katowice, , Poland

Site Status RECRUITING

Novartis Investigative Site

Krakow, , Poland

Site Status RECRUITING

Novartis Investigative Site

Lublin, , Poland

Site Status RECRUITING

Novartis Investigative Site

Poznan, , Poland

Site Status RECRUITING

Novartis Investigative Site

Rzeszów, , Poland

Site Status RECRUITING

Novartis Investigative Site

Warsaw, , Poland

Site Status RECRUITING

Novartis Investigative Site

Lisbon, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Lisbon, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Porto, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Vila Nova de Gaia, , Portugal

Site Status RECRUITING

Novartis Investigative Site

Santiago Compostela, A Coruna, Spain

Site Status RECRUITING

Novartis Investigative Site

Málaga, Andalusia, Spain

Site Status RECRUITING

Novartis Investigative Site

L'Hospitalet de Llobregat, Barcelona, Spain

Site Status RECRUITING

Novartis Investigative Site

Alicante, , Spain

Site Status RECRUITING

Novartis Investigative Site

Barcelona, , Spain

Site Status RECRUITING

Novartis Investigative Site

Lleida, , Spain

Site Status RECRUITING

Novartis Investigative Site

Madrid, , Spain

Site Status RECRUITING

Novartis Investigative Site

Valencia, , Spain

Site Status RECRUITING

Novartis Investigative Site

Inverness, Invernesshire, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Ilford, London, United Kingdom

Site Status WITHDRAWN

Novartis Investigative Site

Swinton, Manchester, United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Birmingham, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Liverpool, , United Kingdom

Site Status WITHDRAWN

Novartis Investigative Site

London, , United Kingdom

Site Status RECRUITING

Novartis Investigative Site

Southampton, , United Kingdom

Site Status RECRUITING

Countries

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United States China Denmark Greece Israel Italy Japan Poland Portugal Spain United Kingdom

Central Contacts

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Novartis Pharmaceuticals

Role: CONTACT

Phone: 1-888-669-6682

Email: [email protected]

Novartis Pharmaceuticals

Role: CONTACT

Phone: +41613241111

Facility Contacts

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Kristy Osgood

Role: primary

Alexandra Vasquez

Role: primary

Mario Salinas

Role: primary

Kristy Bouchard

Role: primary

Julianne Lee

Role: primary

Deekshitha Turaka

Role: primary

Tara Rosen

Role: primary

Jessica Franceschelli

Role: primary

Role: primary

Thomas Keenan

Role: primary

Lesley Dugat

Role: primary

Lauren Wade

Role: primary

Emily Barraza

Role: primary

Other Identifiers

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2023-507064-39-00

Identifier Type: OTHER

Identifier Source: secondary_id

CLNP023Q12301

Identifier Type: -

Identifier Source: org_study_id