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NCT06517758PHASE3Recruiting

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

Novartis Pharmaceuticals

Start Date

7/31/2024

Completion Date

5/27/2032

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.

Detailed Description

The study consists of a 6-month double-blind treatment period for the primary efficacy and safety analysis followed by a maximum duration of 60 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.

Eligibility Criteria

Age Range: 18 years to 85 years

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

Interventions

DRUG

Iptacopan

OTHER

Matching Placebo

Interested in This Trial?

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Conditions

Generalized Myasthenia Gravis

Locations

Honor Health Research Institute

Scottsdale, Arizona 85258

United States

Fullerton Neuro and Headache Ctr

Fullerton, California 92835

United States

SC3 Research Pasadena

Pasadena, California 91105

United States

California Pacific Medical Center

Sacramento, California 94115

United States

Neurology Offices Of South Florida

Boca Raton, Florida 33428

United States

Superior Associates in Research LLC

Hialeah, Florida 33012

United States

Augusta University Georgia

Augusta, Georgia 30912

United States

Hawaii Pacific Neuroscience LLC

Honolulu, Hawaii 96817

United States

University of Chicago Medical Centr

Chicago, Illinois 60637

United States

Prairie Heart Institute

Springfield, Illinois 62769

United States

Mid Atlantic Epilepsy and Sleep Ctr

Bethesda, Maryland 20817-1807

United States

Massachusetts General Hospital

Boston, Massachusetts 02114

United States

Henry Ford Health System

Detroit, Michigan 48202

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

Neuroscience Research Ctr

Canton, Ohio 44718

United States

Ohio State University Medical Center

Columbus, Ohio 43210

United States

Penn Presbyterian Medical Center

Philadelphia, Pennsylvania 19104

United States

Vanderbilt University Medical CenterX

Nashville, Tennessee 37221

United States

Nerve and Muscle Center of Texas

Houston, Texas 77030

United States

Central TX Neuro Consultants P A

Round Rock, Texas 78681

United States

Center for Neurological Disorders G

Greenfield, Wisconsin 53228-1321

United States

Novartis Investigative Site

CABA, Buenos Aires C1428AQK

Argentina

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Córdoba, Córdoba Province X5004AOA

Argentina

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Buenos Aires, C1012AAR

Argentina

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Córdoba, X5004CDT

Argentina

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Camperdown, Sydney NSW 2050

Australia

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Porto Alegre, Rio Grande do Sul 90035-903

Brazil

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Porto Alegre, Rio Grande do Sul 90560-030

Brazil

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Joinville, Santa Catarina 89202-165

Brazil

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Bahia, 40290-000

Brazil

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São Paulo, 04038-002

Brazil

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Hefei, Anhui 230001

China

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Guangzhou, Guangdong 510515

China

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Shenzhen, Guangdong 518053

China

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Shijiazhuang, Hebei 50030

China

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Changsha, Hunan 410008

China

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Suzhou, Jiangsu 215004

China

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Nanchang, Jiangxi 330006

China

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Xi'an, Shaanxi 710075

China

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Xianyang, Shaanxi 712000

China

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Beijing, 065001

China

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Beijing, 100034

China

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Beijing, 100730

China

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Fujian, 350001

China

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Jinan, 250012

China

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Copenhagen, DK-2100

Denmark

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Limoges, Haute Vienne 87000

France

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Garches, 92380

France

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Nice, 06001

France

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Paris, 75013

France

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Paris, 75940

France

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Munich, Bavaria 81675

Germany

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Regensburg, Bavaria 93053

Germany

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Würzburg, Bavaria 97070

Germany

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Frankfurt am Main, Hesse 60590

Germany

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Bochum, 44789

Germany

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Bochum, 44791

Germany

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Essen, 45147

Germany

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Athens, 115 28

Greece

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Chaïdári, 124 62

Greece

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Larissa, 411 10

Greece

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Pátrai, 265 04

Greece

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Thessaloniki, 54636

Greece

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Haifa, 3109601

Israel

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Jerusalem, 9112001

Israel

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Bologna, BO 40139

Italy

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Florence, FI 50134

Italy

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Genova, GE 16132

Italy

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Milan, MI 20133

Italy

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Palermo, PA 90127

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Palermo, PA 90146

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Roma, RM 00133

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Roma, RM 00135

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Roma, RM 00189

Italy

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Orbassano, TO 10043

Italy

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Naples, 80131

Italy

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Chiba, Chiba 2608677

Japan

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Narita, Chiba 286-8520

Japan

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Sapporo, Hokkaido 0630005

Japan

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Nishinomiya, Hyōgo 6638501

Japan

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Hanamaki, Iwate 0250082

Japan

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Sendai, Miyagi 9838520

Japan

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Suita, Osaka 565-0871

Japan

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Higashi-Matsuyama, Saitama 355-0005

Japan

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Shinjuku Ku, Tokyo 160-0023

Japan

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Fukuoka, 8128582

Japan

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Fukushima, 9601295

Japan

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Hiroshima, 7348551

Japan

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Lublin, Lublin Voivodeship 20-064

Poland

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Warsaw, Masovian Voivodeship 02-676

Poland

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Krakow, POL 31-505

Poland

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Katowice, Silesian Voivodeship 40-650

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Bydgoszcz, 85-065

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Katowice, 40-689

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Krakow, 31-870

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Lublin, 20-410

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Poznan, 61-731

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Rzeszów, 35-055

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Warsaw, 01-684

Poland

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Lisbon, 1349-019

Portugal

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Lisbon, 1649-035

Portugal

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Porto, 4099-001

Portugal

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Vila Nova de Gaia, 4434 502

Portugal

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Belgrade, 11000

Serbia

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Niš, 18108

Serbia

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Daegu, 41404

South Korea

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Gwangju, 61469

South Korea

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Seoul, 03722

South Korea

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Seoul, 04763

South Korea

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Santiago Compostela, A Coruna 15706

Spain

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L'Hospitalet de Llobregat, Barcelona 08907

Spain

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Alicante, 03010

Spain

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Barcelona, 08036

Spain

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Lleida, 25198

Spain

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Madrid, 28006

Spain

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Málaga, 29010

Spain

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Valencia, 46026

Spain

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Inverness, Invernesshire IV2 3RE

United Kingdom

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Ilford, London IG1 4HP

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Swinton, Manchester M27 8FF

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Birmingham, West Midlands B15 2TH

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Liverpool, L9 7LT

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London, NW1 2BU

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London, SW17 0QT

United Kingdom

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Southampton, SO16 6YD

United Kingdom