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New pill could ease daily struggles of myasthenia gravis

NCT ID NCT06517758

Disease control Sponsor: Novartis Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Aug 13, 2026 · Updated 3 times

Summary

This study tests an oral drug called iptacopan in people with generalized myasthenia gravis (gMG) who are already on standard treatment. About 146 adults will receive either iptacopan or a placebo for 6 months to see if it improves their ability to do daily activities and reduces muscle weakness. The goal is to find a new way to better control this chronic autoimmune disease.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
iptacopan
What this could lead to
If it works, iptacopan could offer a new oral treatment option to better control symptoms of generalized myasthenia gravis.
What could go wrong
This is a Phase 3 trial, but it's still experimental. It may not prove more effective than placebo, and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 146 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2024

Expected to finish

May 2032

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 85 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

myasthenia gravis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Generalized myasthenia gravis

Contacts and locations

Locations

  • Augusta University Georgia

    Augusta, Georgia, 30912, United States

  • California Pacific Medical Center

    Sacramento, California, 94115, United States

  • Duke University Medical Center

    Durham, North Carolina, 27710, United States

  • Fullerton Neuro and Headache Ctr

    Fullerton, California, 92835, United States

  • Henry Ford Health System

    Detroit, Michigan, 48202, United States

  • Honor Health Research Institute

    Scottsdale, Arizona, 85258, United States

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Mid Atlantic Epilepsy and Sleep Ctr

    Bethesda, Maryland, 20817-1807, United States

  • Nerve and Muscle Center of Texas

    Houston, Texas, 77030, United States

  • Novartis Investigative Site

    CABA, Buenos Aires, C1428AQK, Argentina

  • Novartis Investigative Site

    Córdoba, Córdoba Province, X5004AOA, Argentina

  • Novartis Investigative Site

    Rosario, Santa Fe Province, 2000, Argentina

  • Novartis Investigative Site

    Buenos Aires, C1012AAR, Argentina

  • Novartis Investigative Site

    Caba, C1055AAF, Argentina

  • Novartis Investigative Site

    Caba, C1182, Argentina

  • Novartis Investigative Site

    Capital Federal, C1023AAB, Argentina

  • Novartis Investigative Site

    Córdoba, X5004CDT, Argentina

  • Novartis Investigative Site

    Camperdown, Sydney, NSW 2050, Australia

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

  • Novartis Investigative Site

    Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

  • Novartis Investigative Site

    Joinville, Santa Catarina, 89202-165, Brazil

  • Novartis Investigative Site

    Bahia, 40290-000, Brazil

  • Novartis Investigative Site

    São Paulo, 04038-002, Brazil

  • Novartis Investigative Site

    Hefei, Anhui, 230001, China

  • Novartis Investigative Site

    Guangzhou, Guangdong, 510515, China

  • Novartis Investigative Site

    Shenzhen, Guangdong, 518053, China

  • Novartis Investigative Site

    Shijiazhuang, Hebei, 50030, China

  • Novartis Investigative Site

    Changsha, Hunan, 410008, China

  • Novartis Investigative Site

    Suzhou, Jiangsu, 215004, China

  • Novartis Investigative Site

    Xi'an, Shaanxi, 710075, China

  • Novartis Investigative Site

    Xianyang, Shaanxi, 712000, China

  • Novartis Investigative Site

    Beijing, 100034, China

  • Novartis Investigative Site

    Beijing, 100070, China

  • Novartis Investigative Site

    Beijing, 100730, China

  • Novartis Investigative Site

    Fujian, 350001, China

  • Novartis Investigative Site

    Jinan, 250012, China

  • Novartis Investigative Site

    Copenhagen, DK-2100, Denmark

  • Novartis Investigative Site

    Limoges, Haute Vienne, 87000, France

  • Novartis Investigative Site

    Garches, 92380, France

  • Novartis Investigative Site

    Nice, 06000, France

  • Novartis Investigative Site

    Paris, 75013, France

  • Novartis Investigative Site

    Paris, 75940, France

  • Novartis Investigative Site

    Munich, Bavaria, 81675, Germany

  • Novartis Investigative Site

    Regensburg, Bavaria, 93053, Germany

  • Novartis Investigative Site

    Würzburg, Bavaria, 97070, Germany

  • Novartis Investigative Site

    Frankfurt am Main, Hesse, 60590, Germany

  • Novartis Investigative Site

    Bochum, 44789, Germany

  • Novartis Investigative Site

    Bochum, 44791, Germany

  • Novartis Investigative Site

    Essen, 45147, Germany

  • Novartis Investigative Site

    Athens, 115 28, Greece

  • Novartis Investigative Site

    Chaïdári, 124 62, Greece

  • Novartis Investigative Site

    Larissa, 411 10, Greece

  • Novartis Investigative Site

    Thessaloniki, 54636, Greece

  • Novartis Investigative Site

    Haifa, 3109601, Israel

  • Novartis Investigative Site

    Jerusalem, 9112001, Israel

  • Novartis Investigative Site

    Bologna, BO, 40139, Italy

  • Novartis Investigative Site

    Florence, FI, 50134, Italy

  • Novartis Investigative Site

    Genova, GE, 16132, Italy

  • Novartis Investigative Site

    Milan, MI, 20133, Italy

  • Novartis Investigative Site

    Palermo, PA, 90127, Italy

  • Novartis Investigative Site

    Palermo, PA, 90146, Italy

  • Novartis Investigative Site

    Roma, RM, 00133, Italy

  • Novartis Investigative Site

    Roma, RM, 00135, Italy

  • Novartis Investigative Site

    Roma, RM, 00189, Italy

  • Novartis Investigative Site

    Orbassano, TO, 10043, Italy

  • Novartis Investigative Site

    Chiba, Chiba, 2608677, Japan

  • Novartis Investigative Site

    Narita, Chiba, 286-8520, Japan

  • Novartis Investigative Site

    Sapporo, Hokkaido, 0630005, Japan

  • Novartis Investigative Site

    Nishinomiya, Hyōgo, 6638501, Japan

  • Novartis Investigative Site

    Hanamaki, Iwate, 0250082, Japan

  • Novartis Investigative Site

    Sendai, Miyagi, 9838520, Japan

  • Novartis Investigative Site

    Suita, Osaka, 5650871, Japan

  • Novartis Investigative Site

    Higashi-Matsuyama, Saitama, 355-0005, Japan

  • Novartis Investigative Site

    Shinjuku Ku, Tokyo, 160-0023, Japan

  • Novartis Investigative Site

    Fukuoka, 8128582, Japan

  • Novartis Investigative Site

    Fukushima, 9601295, Japan

  • Novartis Investigative Site

    Hiroshima, 7348551, Japan

  • Novartis Investigative Site

    Lublin, Lublin Voivodeship, 20-064, Poland

  • Novartis Investigative Site

    Warsaw, Masovian Voivodeship, 02-676, Poland

  • Novartis Investigative Site

    Krakow, POL, 31-505, Poland

  • Novartis Investigative Site

    Katowice, Silesian Voivodeship, 40-650, Poland

  • Novartis Investigative Site

    Bydgoszcz, 85-065, Poland

  • Novartis Investigative Site

    Katowice, 40-689, Poland

  • Novartis Investigative Site

    Krakow, 31-870, Poland

  • Novartis Investigative Site

    Lublin, 20-410, Poland

  • Novartis Investigative Site

    Poznan, 61-731, Poland

  • Novartis Investigative Site

    Rzeszów, 35-055, Poland

  • Novartis Investigative Site

    Warsaw, 01-684, Poland

  • Novartis Investigative Site

    Lisbon, 1349-019, Portugal

  • Novartis Investigative Site

    Lisbon, 1649-035, Portugal

  • Novartis Investigative Site

    Porto, 4099-001, Portugal

  • Novartis Investigative Site

    Vila Nova de Gaia, 4434-502, Portugal

  • Novartis Investigative Site

    Belgrade, 11000, Serbia

  • Novartis Investigative Site

    Niš, 18108, Serbia

  • Novartis Investigative Site

    Daegu, 41404, South Korea

  • Novartis Investigative Site

    Gwangju, 61469, South Korea

  • Novartis Investigative Site

    Seoul, 03722, South Korea

  • Novartis Investigative Site

    Seoul, 04763, South Korea

  • Novartis Investigative Site

    Santiago Compostela, A Coruna, 15706, Spain

  • Novartis Investigative Site

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Novartis Investigative Site

    Alicante, 03010, Spain

  • Novartis Investigative Site

    Barcelona, 08036, Spain

  • Novartis Investigative Site

    Lleida, 25198, Spain

  • Novartis Investigative Site

    Madrid, 28006, Spain

  • Novartis Investigative Site

    Málaga, 29010, Spain

  • Novartis Investigative Site

    Valencia, 46026, Spain

  • Novartis Investigative Site

    Inverness, Invernesshire, IV2 3RE, United Kingdom

  • Novartis Investigative Site

    Swinton, Manchester, M27 8FF, United Kingdom

  • Novartis Investigative Site

    Birmingham, West Midlands, B15 2TH, United Kingdom

  • Novartis Investigative Site

    London, NW1 2BU, United Kingdom

  • Novartis Investigative Site

    London, SW17 0QT, United Kingdom

  • Novartis Investigative Site

    Southampton, SO16 6YD, United Kingdom

  • Penn Presbyterian Medical Center

    Philadelphia, Pennsylvania, 19104, United States

  • Prairie Heart Institute

    Springfield, Illinois, 62769, United States

  • University of Chicago Medical Centr

    Chicago, Illinois, 60637, United States

  • Vanderbilt University Medical CenterX

    Nashville, Tennessee, 37221, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.