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NCT06517758

PHASE3

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

Sponsor: Novartis Pharmaceuticals

View on ClinicalTrials.gov

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.

Official title: 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

Key Details

Gender

All

Age Range

18 Years - 85 Years

Study Type

INTERVENTIONAL

Enrollment

146

Start Date

2024-07-31

Completion Date

2032-05-27

Last Updated

2026-03-30

Healthy Volunteers

Conditions

Generalized Myasthenia Gravis

Interventions

DRUG

Iptacopan

Hard gelatin capsule

OTHER

Matching Placebo

Hard gelatin capsule

Inclusion Criteria: * Adult patients with generalized Myasthenia Gravis (age 18-85 years) at screening * Positive serology testing for AChR+ antibody at screening * Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need 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 test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride) * 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 receiving at least one of the following treatments for gMG for ≥ 6 months prior to baseline; * One or more NSISTs or * plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or * an approved FcRN antagonist approved for gMG; or * rituximab or * other approved gMG disease modifying therapies excluding complement inhibitors. * Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster was required, the vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated at the start of study treatment and continued until at least 2 weeks after vaccination or booster was completed. Note: For US sites participating in Study CLNP023Q12301, the completion of the meningococcal vaccination or booster is required for patients with gMG prior to initiating study treatment, irrespective of prophylactic antibiotic use. 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); Active Hepatitis C Virus (HCV); * 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 from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of study treatment and an additional one week following cessation of study treatment. Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., hormonal profile confirming menopause and/or age-appropriate history of vasomotor symptoms). * 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

Locations (110)

Honor Health Research Institute

Scottsdale, Arizona, United States

Fullerton Neuro and Headache Ctr

Fullerton, California, United States

SC3 Research Pasadena

Pasadena, California, United States

California Pacific Medical Center

Sacramento, California, United States

Neurology Offices Of South Florida

Boca Raton, Florida, United States

Superior Associates in Research LLC

Hialeah, Florida, United States

Augusta University Georgia

Augusta, Georgia, United States

Hawaii Pacific Neuroscience LLC

Honolulu, Hawaii, United States

University of Chicago Medical Centr

Chicago, Illinois, United States

Prairie Heart Institute

Springfield, Illinois, United States

Mid Atlantic Epilepsy and Sleep Ctr

Bethesda, Maryland, United States

Duke University Medical Center

Durham, North Carolina, United States

Neuroscience Research Ctr

Canton, Ohio, United States

Ohio State University Medical Center

Columbus, Ohio, United States

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania, United States

Vanderbilt University Medical CenterX

Nashville, Tennessee, United States

Nerve and Muscle Center of Texas

Houston, Texas, United States

Central TX Neuro Consultants P A

Round Rock, Texas, United States

Center for Neurological Disorders G

Greenfield, Wisconsin, United States

Novartis Investigative Site

Buenos Aires, Argentina

Novartis Investigative Site

São Paulo, Brazil

Novartis Investigative Site

Hefei, Anhui, China

Novartis Investigative Site

Guangzhou, Guangdong, China

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Shenzhen, Guangdong, China

Novartis Investigative Site

Shijiazhuang, Hebei, China

Novartis Investigative Site

Changsha, Hunan, China

Novartis Investigative Site

Suzhou, Jiangsu, China

Novartis Investigative Site

Nanchang, Jiangxi, China

Novartis Investigative Site

Xi'an, Shaanxi, China

Novartis Investigative Site

Xianyang, Shaanxi, China

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

Novartis Investigative Site

Copenhagen, Denmark

Novartis Investigative Site

Limoges, Haute Vienne, France

Novartis Investigative Site

Garches, France

Novartis Investigative Site

Nice, France

Novartis Investigative Site

Paris, France

Novartis Investigative Site

Paris, France

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Würzburg, Bavaria, Germany

Novartis Investigative Site

Frankfurt am Main, Hesse, Germany

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Bochum, Germany

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Athens, Greece

Novartis Investigative Site

Chaïdári, Greece

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Larissa, Greece

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Pátrai, Greece

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Thessaloniki, Greece

Novartis Investigative Site

Haifa, Israel

Novartis Investigative Site

Jerusalem, Israel

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Bologna, BO, Italy

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Florence, FI, Italy

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Genova, GE, Italy

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Milan, MI, Italy

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Palermo, PA, Italy

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Palermo, PA, Italy

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Roma, RM, Italy

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Roma, RM, Italy

Novartis Investigative Site

Roma, RM, Italy

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Orbassano, TO, Italy

Novartis Investigative Site

Naples, Italy

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Narita, Chiba, Japan

Novartis Investigative Site

Sapporo, Hokkaido, Japan

Novartis Investigative Site

Nishinomiya, Hyōgo, Japan

Novartis Investigative Site

Hanamaki, Iwate, Japan

Novartis Investigative Site

Sendai, Miyagi, Japan

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Suita, Osaka, Japan

Novartis Investigative Site

Higashi-Matsuyama, Saitama, Japan

Novartis Investigative Site

Shinjuku Ku, Tokyo, Japan

Novartis Investigative Site

Chiba, Japan

Novartis Investigative Site

Fukuoka, Japan

Novartis Investigative Site

Fukushima, Japan

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Hiroshima, Japan

Novartis Investigative Site

Lublin, Lublin Voivodeship, Poland

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Warsaw, Masovian Voivodeship, Poland

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Krakow, POL, Poland

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Katowice, Silesian Voivodeship, Poland

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Bydgoszcz, Poland

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Katowice, Poland

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Krakow, Poland

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Lublin, Poland

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Poznan, Poland

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Rzeszów, Poland

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Warsaw, Poland

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Lisbon, Portugal

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Lisbon, Portugal

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Porto, Portugal

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Vila Nova de Gaia, Portugal

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Belgrade, Serbia

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Niš, Serbia

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Daegu, South Korea

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Gwangju, South Korea

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Seoul, South Korea

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Seoul, South Korea

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Santiago Compostela, A Coruna, Spain

Novartis Investigative Site

L'Hospitalet de Llobregat, Barcelona, Spain

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Alicante, Spain

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Barcelona, Spain

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Lleida, Spain

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Madrid, Spain

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Málaga, Spain

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Valencia, Spain

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Inverness, Invernesshire, United Kingdom

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Ilford, London, United Kingdom

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Swinton, Manchester, United Kingdom

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Birmingham, West Midlands, United Kingdom

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Liverpool, United Kingdom

Novartis Investigative Site

London, United Kingdom

Novartis Investigative Site

Southampton, United Kingdom

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (110)