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New hope for muscle weakness: drug trial targets myasthenia gravis

NCT ID NCT06456580

Disease control Sponsor: Vor Biopharma Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a drug called telitacicept in 180 adults with generalized myasthenia gravis, a condition causing muscle weakness. Participants receive either the drug or a placebo for 24 weeks to see if it improves daily activities and muscle strength. The goal is to control the disease, not cure it.

Why investors are watching

Vor Biopharma, a very small publicly traded company, is running a phase 3 trial of the drug telaticept in 180 people with generalized myasthenia gravis, a chronic muscle weakness condition. For a micro-cap company, this late-stage readout is a major event because a positive result could lead to regulatory approval and its first commercial product. A negative result would likely be a serious setback for the company's future.

If it works: If the trial shows telaticept safely improves symptoms better than placebo, Vor Biopharma could gain a marketable treatment for a disease with few options. That outcome could attract partners or funding to support the drug's launch.

If it fails: Phase 3 trials often fail, and a negative result would likely end telaticept's development and leave Vor Biopharma without a lead candidate. Delays in enrollment or data readout could also strain the company's limited resources.

AI-written from the trial record. Speculative, and not investment advice.

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 180 people

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

Started

Jul 2024

Expected to finish

Jan 2029

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 years and older

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.

Key Inclusion Criteria: 1. Male or female patient aged ≥18 years at screening. 2. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV. 3. Patients have positive antibodies against AChR or MuSK at screening. 4. MG-ADL score ≥6 points at screening and baseline with ocular-related score \<50% of the total score. 5. QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline. Key Exclusion Criteria: 1. Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk. 2. Patients having acute or chronic infection. 3. Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening. Patients with thymoma diagnosed 3-5 years prior to screening may be eligible if thymoma was at a localized stage and definitively treated with complete surgical resection. 4. Patients having current or history of primary immunodeficiency. 5. Patients having history of malignancy within the last 5 years. 6. Patient having prior or continuing diagnosis of serious cardiovascular, liver, kidney, respiratory system, endocrine or hematologic disease.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    111 sites in 14 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Affiliated Hospital of ZunYi Medical University

    RECRUITING

    Zunyi, Guizhou, 563000, China

  • Allied Biomedical Research Institute (ABRI)

    RECRUITING

    Miami, Florida, 33155, United States

  • AnHui Provincial Hospital

    RECRUITING

    Hefei, Anhui, 230000, China

  • Aversi Clinic - Central Branch

    RECRUITING

    Tbilisi, 0160, Georgia

  • Azienda Ospedaliera - Universitaria Sant' Andrea

    RECRUITING

    Rome, Rome, 00189, Italy

  • Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli

    RECRUITING

    Naples, Napoli, 80131, Italy

  • Azienda Ospedaliera Universitaria Federico II

    RECRUITING

    Naples, Napoli, 80131, Italy

  • Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli

    RECRUITING

    Naples, Napoli, 80131, Italy

  • Beijing Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100730, China

  • Center for Neurological Disorders, S.C. - Greenfield - Gamma Therapeutic Center

    RECRUITING

    Greenfield, Wisconsin, 53228, United States

  • Centre Hospitalier Régional de la Citadelle

    RECRUITING

    Liège, Liège, 4000, Belgium

  • Centrum Medyczne Hope Clinic

    RECRUITING

    Lublin, Lubusz Voivodeship, 20-701, Poland

  • Centrum Medyczne Neuroprot

    RECRUITING

    Warsaw, Masovian Voivodeship, 01-684, Poland

  • Clinirem - Lublin

    RECRUITING

    Lublin, Lublin Voivodeship, 20-064, Poland

  • Concord Repatriation General Hospital

    RECRUITING

    Concord, New South Wales, 2139, Australia

  • Empower Clinical Research

    RECRUITING

    Tigard, Oregon, 97223, United States

  • Fakultni Nemocnice Brno

    RECRUITING

    Brno, South Moravian, 625 00, Czechia

  • Fakultni Nemocnice Ostrava

    RECRUITING

    Ostrava-Poruba, Moravian-Silesian, 708 54, Czechia

  • First Hospital of ShanXi Medical University

    RECRUITING

    Taiyuan, Shanxi, 030000, China

  • Fondazione Istituto San Raffaele - G. Giglio Di Cefalu

    RECRUITING

    Cefalù, Palermo, 90015, Italy

  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS

    RECRUITING

    Rome, Rome, 00168, Italy

  • Fujian Medical University Union Hospital

    RECRUITING

    Fuzhou, Fujian, 350000, China

  • Fundación Scherbovsky

    RECRUITING

    Mendoza, Mendoza Province, M5500AXR, Argentina

  • Gold Coast University Hospital

    RECRUITING

    Southport, Queensland, 4215, Australia

  • Groupe Hospitalier Pellegrin

    RECRUITING

    Bordeaux, Gironde, 33076, France

  • Hokkaido University Hospital

    RECRUITING

    Sapporo, Hokkaido, 060-8648, Japan

  • HonorHealth Neurology - Bob Bové Neuroscience Institute

    RECRUITING

    Scottsdale, Arizona, 85258, United States

  • Hospital General Universitario Dr. Balmis

    RECRUITING

    Alicante, Alicante, 03010, Spain

  • Hospital General de Agudos José María Ramos Mejía

    RECRUITING

    Ciudad Autónoma de Buenos Aires, Buenos Aires, C1221ADC, Argentina

  • Hospital Italiano de Buenos Aires

    RECRUITING

    Ciudad Autónoma de Buenos Aires, Buenos Aires, C1199ABB, Argentina

  • Hospital Sisters Health System Medical

    RECRUITING

    O'Fallon, Illinois, 62269, United States

  • Hospital São Lucas da PUCRS

    RECRUITING

    Porto Alegre, Rio Grande do Sul, 90619-900, Brazil

  • Hospital Universitari Mútua Terrassa

    RECRUITING

    Terrassa, Barcelona, 08221, Spain

  • Hospital Universitari Vall d'Hebron

    RECRUITING

    Barcelona, Barcelona, 08035, Spain

  • Hospital Universitari i Politècnic La Fe

    RECRUITING

    Valencia, València, 46026, Spain

  • Hospital Universitario de Basurto

    RECRUITING

    Bilbao, Biscay, 48013, Spain

  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

    RECRUITING

    Ribeirão Preto, São Paulo, 14051-140, Brazil

  • Hospital for Special Surgery

    RECRUITING

    New York, New York, 10021, United States

  • Houston Methodist Neurological Institute

    RECRUITING

    Houston, Texas, 77030, United States

  • Huashan Hospital of the ShangHai FuDan University Medical College

    RECRUITING

    Shanghai, Shanghai Municipality, 201100, China

  • Hôpital Pasteur

    RECRUITING

    Nice, Alpes-Maritimes, 06001, France

  • INECO Neurociencias Oroño

    RECRUITING

    Rosario, Santa Fe Province, S2000TP, Argentina

  • IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo

    RECRUITING

    San Giovanni Rotondo, Foggia, 71013, Italy

  • Instituto de Neurologia de Curitiba

    RECRUITING

    Curitiba, Paraná, 81210-310, Brazil

  • Istituto Neurologico Casimiro Mondino

    RECRUITING

    Pavia, Pavia, 27100, Italy

  • Jilin University China-Japan Union Hospital

    RECRUITING

    Changchun, Jilin, 130000, China

  • Juntendo University Hospital

    RECRUITING

    Bunkyo-ku, Tokyo, 113-8431, Japan

  • Kobe University Hospital

    RECRUITING

    Kobe, Hyōgo, 650-0017, Japan

  • Krakowska Akademia Neurologii Sp. z o.o.

    RECRUITING

    Krakow, Lesser Poland Voivodeship, 31-505, Poland

  • Kyushu University Hospital

    RECRUITING

    Fukuoka, Fukuoka, 812-8582, Japan

  • MINKSneuro s.r.o. - MUDr. Eduard Minks Ph.D.

    RECRUITING

    Brno, South Moravian, 603 00, Czechia

  • MTZ Powered by Pratia

    RECRUITING

    Warsaw, Masovian Voivodeship, 02-172, Poland

  • Medicover Integrated Clinical Services (MICS) - Centrum Medyczne Bydgoszcz

    RECRUITING

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-065, Poland

  • Medsol Clinical Research Center

    RECRUITING

    Port Charlotte, Florida, 33952, United States

  • Michigan State University Clinical Center

    RECRUITING

    East Lansing, Michigan, 48824, United States

  • Montefiore Medical Center

    RECRUITING

    The Bronx, New York, 10467, United States

  • Mount Sinai Health System

    RECRUITING

    New York, New York, 10029, United States

  • Multi-profile Clinic "New Hospitals"

    RECRUITING

    Tbilisi, 0114, Georgia

  • NHO Sendai Medical Center

    RECRUITING

    Sendai, Miyagi, 983-8520, Japan

  • Nagoya City University Hospital

    RECRUITING

    Suita-shi, Osaka, 564-0013, Japan

  • National Hospital Organization - Nagoya Medical Center

    RECRUITING

    Nagoya, Aichi-ken, 460-0001, Japan

  • National Hospital Organization Niigata National Hospital

    RECRUITING

    Kashiwazaki-Shi, Niigata, 945-8585, Japan

  • National Hospital Organization Utano National Hospital

    RECRUITING

    Kyoto, Kyoto, 616-8255, Japan

  • Neurocentrum Bydgoszcz

    RECRUITING

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-796, Poland

  • Neurologia Śląska Centrum Medyczne

    RECRUITING

    Katowice, Silesian Voivodeship, 40-123, Poland

  • Neurology Center of New England

    RECRUITING

    Foxborough, Massachusetts, 02035, United States

    Contact Email: •••••@•••••

  • Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD

    RECRUITING

    Poznan, Greater Poland Voivodeship, 61-853, Poland

  • NingBo Medical Center LiHuiLi Hospital

    RECRUITING

    Ningbo, Zhejiang, 315100, China

  • Okinawa National Hospital

    RECRUITING

    Ginowan-Shi, Okinawa, 901-2214, Japan

  • Pineo Medical Ecosystem

    RECRUITING

    Tbilisi, 0114, Georgia

  • Pseg Centro de Pesquisa Clinica

    RECRUITING

    São Paulo, São Paulo, 04038-002, Brazil

  • QiLU Hospital of ShanDong University

    RECRUITING

    Jinan, Shangdong, 250000, China

  • SFM Clinical Research

    RECRUITING

    Boca Raton, Florida, 33487, United States

  • STAT Research S.A.

    RECRUITING

    Ciudad Autónoma de Buenos Aires, Buenos Aires, C1023AAB, Argentina

  • Sapporo Medical University Hospital

    RECRUITING

    Sapporo, Hokkaido, 060-8543, Japan

  • Seirei Hamamatsu General Hospital

    RECRUITING

    Hamamatsu, Sizuoka [Shizuoka], 430-8558, Japan

  • Simon Khechinashvili University Hospital

    RECRUITING

    Tbilisi, 0179, Georgia

  • St. Marianna University Hospital

    RECRUITING

    Kawasaki, Kanagawa, 216-8511, Japan

  • The 1 School of Medicine, School of Information and Engineering. The 1 Affiliated Hospital of WMU.

    RECRUITING

    Wenzhou, Zhejiang, 325000, China

  • The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army

    RECRUITING

    Jinan, Shangdong, 250031, China

  • The First Affiliated Hospital of Chongqing Medical University

    RECRUITING

    Chongqing, Chongqing Municipality, 400010, China

  • The First Affiliated Hospital, Sun Yat-Sen University

    RECRUITING

    Guangzhou, Guangdong, 510080, China

  • The First People's Hospital of JiNing Medical University

    RECRUITING

    Jining, Shangdong, 272000, China

  • The Second Affiliated Hospital of PLA Air Force Military Medical University

    RECRUITING

    Xi'an, Shaanxi, 710000, China

  • The Second Hospital & Clinical Medical School. Lan Zhou University

    RECRUITING

    Lanzhou, Gansu, 730000, China

  • Tokushima University Hospital

    RECRUITING

    Tokushima, Tokusima [Tokushima], 770-8503, Japan

  • Tokyo Medical University Hospital

    RECRUITING

    Shinjuku-ku, Tokyo, 160-0023, Japan

  • TongJi Medical College of HUST

    RECRUITING

    Wuhan, Hubei, 430030, China

  • Toronto General Hospital

    NOT_YET_RECRUITING

    Toronto, Ontario, M5G 2C4, Canada

  • Townsville University Hospital

    RECRUITING

    Douglas, Queensland, 4814, Australia

  • Trinity Health - Grand Rapids Hospital

    RECRUITING

    Grand Rapids, Michigan, 49503, United States

  • Twoja Przychodnia Nowosolskie Centrum Medyczne

    RECRUITING

    Nowa Sól, Lubusz Voivodeship, 67-100, Poland

  • UM Health Care - Neurology and Sleep Disorders Clinic

    RECRUITING

    Columbia, Missouri, 65212, United States

  • Universidade Estadual de Campinas

    RECRUITING

    Campinas, São Paulo, 13083-888, Brazil

  • Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg

    RECRUITING

    Leuven, Flemish Brabant, 3000, Belgium

  • University Hospital - London Health Sciences Centre

    RECRUITING

    London, Ontario, N6C 2R5, Canada

  • University of California Irvine

    RECRUITING

    Orange, California, 92868, United States

  • University of California San Francisco (UCSF)

    RECRUITING

    San Francisco, California, 94143, United States

  • University of Cincinnati Health Physicians - Clifton

    RECRUITING

    Cincinnati, Ohio, 45219, United States

  • University of Kansas Medical Center

    RECRUITING

    Kansas City, Kansas, 66160, United States

  • University of Kentucky

    RECRUITING

    Lexington, Kentucky, 40506, United States

  • University of Louisville Physicians

    RECRUITING

    Louisville, Kentucky, 40202, United States

  • University of North Carolina Hospitals - Neurology Clinic

    RECRUITING

    Chapel Hill, North Carolina, 27517, United States

  • University of South Florida Health (USF Health)

    RECRUITING

    Tampa, Florida, 33613, United States

  • University of Southern California

    RECRUITING

    Los Angeles, California, 90033, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

  • Uniwersyteckie Centrum Kliniczne w Gdańsku

    RECRUITING

    Gdansk, Pomeranian Voivodeship, 80-214, Poland

  • Wellstar MCG Health Medical Center

    RECRUITING

    Augusta, Georgia, 30912, United States

  • Xi'An GaoXin Hospital

    RECRUITING

    Xi'an, Shaanxi, 710000, China

  • XiangYa Hospital of Central South University

    RECRUITING

    Changsha, Hunan, 410000, China

  • Yokohama Medical Center

    RECRUITING

    Yokohama, Kanagawa, 245-8575, Japan

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