A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)

NCT06456580 | Recruiting Phase 3 DMC FDA Drug

• 1 other identifier: RC18G006
• Study Type: interventional
• Target: 180
• Locations: 13 countries
• Sites: 84

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.

Conditions

Generalized Myasthenia Gravis

Keywords

Myasthenia Gravis; gMG

Outcome Measures

Primary Outcomes (1)

• Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24

  The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale is scored on a 0 to 3 (0=normal, 3=severe disease) point scale. The total score is the sum of all individual item scores ranging from 0 to 24. Higher scores indicate more severe disability due to MG. A decrease from Baseline score indicates improvement.

  Week 24

Secondary Outcomes (5)

• Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

  Week 24

• Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24

  Week 24

• Proportion of patients with a decrease of ≥2 points from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24

  Week 24

• Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

  Week 24

• Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24

  Week 24

Study Arms (2)

Telitacicept

EXPERIMENTAL

Telitacicept

Biological: Telitacicept

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Placebo DRUG

Subcutaneous injection

Placebo

Telitacicept BIOLOGICAL

Subcutaneous injection

Also known as: RC18, RC18-L

Telitacicept

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female patient aged ≥18 years at screening.
• 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.
• Patients have positive antibodies against AChR or MuSK at screening.
• MG-ADL score ≥6 points at screening and baseline with ocular-related score \<50% of the total score.
• QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline.

You may not qualify if:

• Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk.
• Patients having acute or chronic infection.
• 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.
• Patients having current or history of primary immunodeficiency.
• Patients having history of malignancy within the last 5 years.
• Patient having prior or continuing diagnosis of serious cardiovascular, liver, kidney, respiratory system, endocrine or hematologic disease.

Sponsors & Collaborators

• Vor Biopharma: lead

Study Sites (84)

University of Southern California

Los Angeles, California, 90033, United States

RECRUITING

University of California Irvine

Orange, California, 92868, United States

RECRUITING

University of California San Francisco (UCSF)

San Francisco, California, 94143, United States

RECRUITING

SFM Clinical Research

Boca Raton, Florida, 33487, United States

RECRUITING

Allied Biomedical Research Institute (ABRI)

Miami, Florida, 33155, United States

RECRUITING

Medsol Clinical Research Center

Port Charlotte, Florida, 33952, United States

RECRUITING

Wellstar MCG Health Medical Center

Augusta, Georgia, 30912, United States

RECRUITING

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

RECRUITING

University of Kentucky

Lexington, Kentucky, 40506, United States

RECRUITING

University of Louisville Physicians

Louisville, Kentucky, 40202, United States

RECRUITING

Neurology Center of New England

Foxborough, Massachusetts, 02035, United States

RECRUITING

Michigan State University Clinical Center

East Lansing, Michigan, 48824, United States

RECRUITING

Trinity Health - Grand Rapids Hospital

Grand Rapids, Michigan, 49503, United States

RECRUITING

Mount Sinai Health System

New York, New York, 10029, United States

RECRUITING

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

University of Cincinnati Health Physicians - Clifton

Cincinnati, Ohio, 45219, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Houston Methodist Neurological Institute

Houston, Texas, 77030, United States

RECRUITING

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

Greenfield, Wisconsin, 53228, United States

RECRUITING

STAT Research S.A.

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

RECRUITING

Hospital Italiano de Buenos Aires

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

RECRUITING

Hospital General de Agudos José María Ramos Mejía

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

RECRUITING

INECO Neurociencias Oroño

Rosario, Santa Fe Province, S2000TP, Argentina

RECRUITING

Fundación Scherbovsky

Mendoza, M5500AXR, Argentina

RECRUITING

Concord Repatriation General Hospital

Concord, New South Wales, 2139, Australia

RECRUITING

Townsville University Hospital

Douglas, Queensland, 4814, Australia

RECRUITING

Gold Coast University Hospital

Southport, Queensland, 4215, Australia

RECRUITING

Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg

Leuven, Flemish Brabant, 3000, Belgium

RECRUITING

Instituto de Neurologia de Curitiba

Curitiba, Paraná, 81210-310, Brazil

RECRUITING

Hospital São Lucas da PUCRS

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

RECRUITING

Universidade Estadual de Campinas

Campinas, São Paulo, 13083-888, Brazil

RECRUITING

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

Ribeirão Preto, São Paulo, 14051-140, Brazil

RECRUITING

Pseg Centro de Pesquisa Clinica

São Paulo, 04038-002, Brazil

RECRUITING

University Hospital - London Health Sciences Centre

London, Ontario, N6C 2R5, Canada

RECRUITING

Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

NOT YET RECRUITING

Fakultni Nemocnice Ostrava

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

RECRUITING

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

Brno, South Moravian, 603 00, Czechia

RECRUITING

Fakultni Nemocnice Brno

Brno, South Moravian, 625 00, Czechia

RECRUITING

Hôpital Pasteur

Nice, Alpes-Maritimes, 06001, France

RECRUITING

Groupe Hospitalier Pellegrin

Bordeaux, Gironde, 33076, France

RECRUITING

Multi-profile Clinic "New Hospitals"

Tbilisi, 0114, Georgia

RECRUITING

Pineo Medical Ecosystem

Tbilisi, 0114, Georgia

RECRUITING

Aversi Clinic - Central Branch

Tbilisi, 0160, Georgia

RECRUITING

Simon Khechinashvili University Hospital

Tbilisi, 0179, Georgia

RECRUITING

IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo

San Giovanni Rotondo, Foggia, 71013, Italy

RECRUITING

Fondazione Istituto San Raffaele - G. Giglio Di Cefalu

Cefalù, Palermo, 90015, Italy

RECRUITING

Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli

Naples, 80131, Italy

RECRUITING

Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli

Naples, 80131, Italy

RECRUITING

Azienda Ospedaliera Universitaria Federico II

Naples, 80131, Italy

RECRUITING

Istituto Neurologico Casimiro Mondino

Pavia, 27100, Italy

RECRUITING

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, 00168, Italy

RECRUITING

Azienda Ospedaliera - Universitaria Sant' Andrea

Rome, 00189, Italy

RECRUITING

National Hospital Organization - Nagoya Medical Center

Nagoya, Aichi-ken, 460-0001, Japan

RECRUITING

Sapporo Medical University Hospital

Sapporo, Hokkaido, 060-8543, Japan

RECRUITING

Hokkaido University Hospital

Sapporo, Hokkaido, 060-8648, Japan

RECRUITING

Kobe University Hospital

Kobe, Hyōgo, 650-0017, Japan

RECRUITING

St. Marianna University Hospital

Kawasaki, Kanagawa, 216-8511, Japan

RECRUITING

Yokohama Medical Center

Yokohama, Kanagawa, 245-8575, Japan

RECRUITING

NHO Sendai Medical Center

Sendai, Miyagi, 983-8520, Japan

RECRUITING

National Hospital Organization Niigata National Hospital

Kashiwazaki-Shi, Niigata, 945-8585, Japan

RECRUITING

Okinawa National Hospital

Ginowan-Shi, Okinawa, 901-2214, Japan

RECRUITING

Nagoya City University Hospital

Suita-shi, Osaka, 564-0013, Japan

RECRUITING

Seirei Hamamatsu General Hospital

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

RECRUITING

Tokushima University Hospital

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

RECRUITING

Juntendo University Hospital

Bunkyo-ku, Tokyo, 113-8431, Japan

RECRUITING

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo, 160-0023, Japan

RECRUITING

Kyushu University Hospital

Fukuoka, 812-8582, Japan

RECRUITING

National Hospital Organization Utano National Hospital

Kyoto, 616-8255, Japan

RECRUITING

Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD

Poznan, Greater Poland Voivodeship, 61-853, Poland

RECRUITING

Medicover Integrated Clinical Services (MICS) - Centrum Medyczne Bydgoszcz

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

RECRUITING

Neurocentrum Bydgoszcz

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

RECRUITING

Krakowska Akademia Neurologii Sp. z o.o.

Krakow, Lesser Poland Voivodeship, 31-505, Poland

RECRUITING

Clinirem - Lublin

Lublin, Lublin Voivodeship, 20-064, Poland

RECRUITING

Centrum Medyczne Hope Clinic

Lublin, Lubusz Voivodeship, 20-701, Poland

RECRUITING

Twoja Przychodnia Nowosolskie Centrum Medyczne

Nowa Sól, Lubusz Voivodeship, 67-100, Poland

RECRUITING

Centrum Medyczne Neuroprot

Warsaw, Masovian Voivodeship, 01-684, Poland

RECRUITING

MTZ Powered by Pratia

Warsaw, Masovian Voivodeship, 02-172, Poland

RECRUITING

Uniwersyteckie Centrum Kliniczne w Gdańsku

Gdansk, Pomeranian Voivodeship, 80-214, Poland

RECRUITING

Neurologia Śląska Centrum Medyczne

Katowice, Silesian Voivodeship, 40-123, Poland

RECRUITING

Hospital Universitari Mútua Terrassa

Terrassa, Barcelona, 08221, Spain

RECRUITING

Hospital Universitario de Basurto

Bilbao, Biscay, 48013, Spain

RECRUITING

Hospital General Universitario Dr. Balmis

Alicante, 03010, Spain

RECRUITING

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

RECRUITING

Hospital Universitari i Politècnic La Fe

Valencia, 46026, Spain

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

telitacicept

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Jeremy Sokolove

CONTACT

617-655-6580; Study@vorbio.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 7, 2024
• First Posted: June 13, 2024
• Study Start: July 17, 2024
• Primary Completion (Estimated): May 1, 2027
• Study Completion (Estimated): January 1, 2029
• Last Updated: March 10, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (2); BE (1); FR (2); JP (16); US (19); PL (11); ES (5); GE (4); IT (8); CZ (3); AR (5); AU (3); BR (5)