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Updated May 2026 · ClinicalTrials.gov

RECRUITINGPhase 3INTERVENTIONAL

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

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

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) is a Phase 3 interventional studying Generalized Myasthenia Gravis, sponsored by Novartis Pharmaceuticals. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

What Stage of Research Is This?

Phase 3 trials confirm efficacy and safety in large patient groups (often 300–3,000+) and form the evidence base for an FDA approval submission. For Generalized Myasthenia Gravis, Phase 3 studies typically randomize participants between the investigational treatment and either a placebo or current standard of care. A successful Phase 3 result is the threshold most treatments need to clear before regulatory approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 146 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Generalized Myasthenia Gravis subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Who May Qualify: - 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. ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

Iptacopan

Hard gelatin capsule

OTHER

Matching Placebo

Hard gelatin capsule

Locations (20)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

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

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT06517758), the sponsor (Novartis Pharmaceuticals), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT06517758 clinical trial studying?

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. The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT06517758?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT06517758?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT06517758. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT06517758. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.