RecruitingPhase 3NCT06456580

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

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study With an Open-label Extension Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis


Sponsor

Vor Biopharma

Enrollment

180 participants

Start Date

Jul 17, 2024

Study Type

INTERVENTIONAL

Conditions

Summary

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


Eligibility

Min Age: 18 Years

Inclusion Criteria5

  • 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.

Exclusion Criteria6

  • 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.

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Interventions

BIOLOGICALTelitacicept

Subcutaneous injection

DRUGPlacebo

Subcutaneous injection


Locations(84)

University of Southern California

Los Angeles, California, United States

University of California Irvine

Orange, California, United States

University of California San Francisco (UCSF)

San Francisco, California, United States

SFM Clinical Research

Boca Raton, Florida, United States

Allied Biomedical Research Institute (ABRI)

Miami, Florida, United States

Medsol Clinical Research Center

Port Charlotte, Florida, United States

Wellstar MCG Health Medical Center

Augusta, Georgia, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Kentucky

Lexington, Kentucky, United States

University of Louisville Physicians

Louisville, Kentucky, United States

Neurology Center of New England

Foxborough, Massachusetts, United States

Michigan State University Clinical Center

East Lansing, Michigan, United States

Trinity Health - Grand Rapids Hospital

Grand Rapids, Michigan, United States

Mount Sinai Health System

New York, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

University of Cincinnati Health Physicians - Clifton

Cincinnati, Ohio, United States

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Houston Methodist Neurological Institute

Houston, Texas, United States

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

Greenfield, Wisconsin, United States

STAT Research S.A.

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Hospital Italiano de Buenos Aires

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

Hospital General de Agudos José María Ramos Mejía

Ciudad Autónoma de Buenos Aires, Buenos Aires, Argentina

INECO Neurociencias Oroño

Rosario, Santa Fe Province, Argentina

Fundación Scherbovsky

Mendoza, Argentina

Concord Repatriation General Hospital

Concord, New South Wales, Australia

Townsville University Hospital

Douglas, Queensland, Australia

Gold Coast University Hospital

Southport, Queensland, Australia

Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg

Leuven, Flemish Brabant, Belgium

Instituto de Neurologia de Curitiba

Curitiba, Paraná, Brazil

Hospital São Lucas da PUCRS

Porto Alegre, Rio Grande do Sul, Brazil

Universidade Estadual de Campinas

Campinas, São Paulo, Brazil

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto

Ribeirão Preto, São Paulo, Brazil

Pseg Centro de Pesquisa Clinica

São Paulo, Brazil

University Hospital - London Health Sciences Centre

London, Ontario, Canada

Toronto General Hospital

Toronto, Ontario, Canada

Fakultni Nemocnice Ostrava

Ostrava-Poruba, Moravian-Silesian, Czechia

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

Brno, South Moravian, Czechia

Fakultni Nemocnice Brno

Brno, South Moravian, Czechia

Hôpital Pasteur

Nice, Alpes-Maritimes, France

Groupe Hospitalier Pellegrin

Bordeaux, Gironde, France

Multi-profile Clinic "New Hospitals"

Tbilisi, Georgia

Pineo Medical Ecosystem

Tbilisi, Georgia

Aversi Clinic - Central Branch

Tbilisi, Georgia

Simon Khechinashvili University Hospital

Tbilisi, Georgia

IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo

San Giovanni Rotondo, Foggia, Italy

Fondazione Istituto San Raffaele - G. Giglio Di Cefalu

Cefalù, Palermo, Italy

Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli

Naples, Italy

Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli

Naples, Italy

Azienda Ospedaliera Universitaria Federico II

Naples, Italy

Istituto Neurologico Casimiro Mondino

Pavia, Italy

Fondazione Policlinico Universitario Agostino Gemelli IRCCS

Rome, Italy

Azienda Ospedaliera - Universitaria Sant' Andrea

Rome, Italy

National Hospital Organization - Nagoya Medical Center

Nagoya, Aichi-ken, Japan

Sapporo Medical University Hospital

Sapporo, Hokkaido, Japan

Hokkaido University Hospital

Sapporo, Hokkaido, Japan

Kobe University Hospital

Kobe, Hyōgo, Japan

St. Marianna University Hospital

Kawasaki, Kanagawa, Japan

Yokohama Medical Center

Yokohama, Kanagawa, Japan

NHO Sendai Medical Center

Sendai, Miyagi, Japan

National Hospital Organization Niigata National Hospital

Kashiwazaki-Shi, Niigata, Japan

Okinawa National Hospital

Ginowan-Shi, Okinawa, Japan

Nagoya City University Hospital

Suita-shi, Osaka, Japan

Seirei Hamamatsu General Hospital

Hamamatsu, Sizuoka [Shizuoka], Japan

Tokushima University Hospital

Tokushima, Tokusima [Tokushima], Japan

Juntendo University Hospital

Bunkyo-ku, Tokyo, Japan

Tokyo Medical University Hospital

Shinjuku-ku, Tokyo, Japan

Kyushu University Hospital

Fukuoka, Japan

National Hospital Organization Utano National Hospital

Kyoto, Japan

Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD

Poznan, Greater Poland Voivodeship, Poland

Medicover Integrated Clinical Services (MICS) - Centrum Medyczne Bydgoszcz

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

Neurocentrum Bydgoszcz

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

Krakowska Akademia Neurologii Sp. z o.o.

Krakow, Lesser Poland Voivodeship, Poland

Clinirem - Lublin

Lublin, Lublin Voivodeship, Poland

Centrum Medyczne Hope Clinic

Lublin, Lubusz Voivodeship, Poland

Twoja Przychodnia Nowosolskie Centrum Medyczne

Nowa Sól, Lubusz Voivodeship, Poland

Centrum Medyczne Neuroprot

Warsaw, Masovian Voivodeship, Poland

MTZ Powered by Pratia

Warsaw, Masovian Voivodeship, Poland

Uniwersyteckie Centrum Kliniczne w Gdańsku

Gdansk, Pomeranian Voivodeship, Poland

Neurologia Śląska Centrum Medyczne

Katowice, Silesian Voivodeship, Poland

Hospital Universitari Mútua Terrassa

Terrassa, Barcelona, Spain

Hospital Universitario de Basurto

Bilbao, Biscay, Spain

Hospital General Universitario Dr. Balmis

Alicante, Spain

Hospital Universitari Vall d'Hebron

Barcelona, Spain

Hospital Universitari i Politècnic La Fe

Valencia, Spain

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NCT06456580


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