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Clinical Trials in the USA / NCT06517758
Active, not recruiting Phase 3

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

NCT06517758 · tracked via the Priya Life Science USA tracker
Sponsor
Novartis Pharmaceuticals
Phase
Phase 3
Started
2024-07-31
Last updated
2026-08-12

Condition(s) studied

Generalized Myasthenia Gravis

Investigational drug(s) / intervention(s)

IptacopanMatching Placebo

Iptacopan: Hard gelatin capsule

Matching Placebo: Hard gelatin capsule

Study 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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
85 Years
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (116)

FacilityCityRegionStatus
Honor Health Research Institute Scottsdale Arizona
Fullerton Neuro and Headache Ctr Fullerton California
California Pacific Medical Center Sacramento California
Augusta University Georgia Augusta Georgia
University of Chicago Medical Centr Chicago Illinois
Prairie Heart Institute Springfield Illinois
Mid Atlantic Epilepsy and Sleep Ctr Bethesda Maryland
Massachusetts General Hospital Boston Massachusetts
Henry Ford Health System Detroit Michigan
Duke University Medical Center Durham North Carolina
Penn Presbyterian Medical Center Philadelphia Pennsylvania
Vanderbilt University Medical CenterX Nashville Tennessee
Nerve and Muscle Center of Texas Houston Texas
Novartis Investigative Site CABA Buenos Aires
Novartis Investigative Site Córdoba Córdoba Province
Novartis Investigative Site Rosario Santa Fe Province
Novartis Investigative Site Buenos Aires Argentina
Novartis Investigative Site Caba Argentina
Novartis Investigative Site Caba Argentina
Novartis Investigative Site Capital Federal Argentina
Novartis Investigative Site Córdoba Argentina
Novartis Investigative Site Camperdown Sydney
Novartis Investigative Site Porto Alegre Rio Grande do Sul
Novartis Investigative Site Porto Alegre Rio Grande do Sul
Novartis Investigative Site Joinville Santa Catarina
Novartis Investigative Site Bahia Brazil
Novartis Investigative Site São Paulo Brazil
Novartis Investigative Site Hefei Anhui
Novartis Investigative Site Guangzhou Guangdong
Novartis Investigative Site Shenzhen Guangdong
Novartis Investigative Site Shijiazhuang Hebei
Novartis Investigative Site Changsha Hunan
Novartis Investigative Site Suzhou Jiangsu
Novartis Investigative Site Xi'an Shaanxi
Novartis Investigative Site Xianyang Shaanxi
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

+ 76 more sites — see the full list on the official registry below.

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06517758 on ClinicalTrials.gov ↗ ← All trials in the USA