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Recruiting NCT06456580

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

Phase III Interventional Generalized Myasthenia Gravis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Telitacicept, Placebo.
Who it may be relevant to
Registry conditions: Generalized Myasthenia Gravis. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Argentina, Australia, Belgium, Brazil +9
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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

Detailed description

Myasthenia gravis (MG) is an autoimmune disease in which autoantibodies disrupt the postsynaptic membrane, impairing nerve-to-muscle signal transmission. The predominant manifestation is muscle weakness, which typically worsens with repeated muscle exertion, such that function is usually the best in the morning with more pronounced weakness at the end of the day. A major challenge in MG is the lack of therapies that effectively treat the disease.

Telitacicept is a fully human TACI-Fc fusion protein that targets B-lymphocyte stimulator (BLyS) and A proliferating-inducing ligand (APRIL), neutralizing their interactions with receptors on B cells. The blockage of BLyS and APRIL interaction with their respective cell membrane receptors (transmembrane activator and CAML interactor \[TACI\], B-cell maturation antigen, and BLyS receptors) by telitacicept can inhibit B-cell proliferation and maturation. This suppression at the proximal portion of the immune response could alleviate autoimmune symptoms.

This is a randomized, double-blind, placebo-controlled Phase 3 study with an open-label extension (OLE) to evaluate the efficacy and safety of telitacicept in a global patient population with gMG. The total duration of the study is variable but will include an approximately 4-week screening period, a 24-week double-blind treatment period (Week 0 through Week 24), a 48-week OLE (Week 24 through Week 72), followed by an extended OLE period (E-OLE), and an 8-week end-of-study follow-up period. The E-OLE is variable duration, defined as the period after the 48-week OLE period until telitacicept is approved for MG in the country or the further development in the indication is concluded.

Interventions

  • Biological Telitacicept
    Subcutaneous injection
  • Drug Placebo
    Subcutaneous injection

Primary outcome measures

  • Change from baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score at Week 24 [Time frame: Week 24]
Secondary outcome measures (5)
  • Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24 [Time frame: Week 24]
  • Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24 [Time frame: 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 [Time frame: Week 24]
  • Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24 [Time frame: Week 24]
  • Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24 [Time frame: Week 24]

Eligibility criteria

Inclusion criteria

  • 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 criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 26 centers
  • HonorHealth Neurology - Bob Bové Neuroscience Institute — Scottsdale
  • University of Southern California — Los Angeles
  • University of California Irvine — Orange
  • University of California San Francisco (UCSF) — San Francisco
  • SFM Clinical Research — Boca Raton
  • Allied Biomedical Research Institute (ABRI) — Miami
  • Medsol Clinical Research Center — Port Charlotte
  • University of South Florida Health (USF Health) — Tampa
  • … and 18 more centers
China · 19 centers
  • AnHui Provincial Hospital — Hefei
  • Beijing Hospital — Beijing
  • The First Affiliated Hospital of Chongqing Medical University — Chongqing
  • Fujian Medical University Union Hospital — Fuzhou
  • The Second Hospital & Clinical Medical School. Lan Zhou University — Lanzhou
  • The First Affiliated Hospital, Sun Yat-Sen University — Guangzhou
  • Affiliated Hospital of ZunYi Medical University — Zunyi
  • TongJi Medical College of HUST — Wuhan
  • … and 11 more centers
Japan · 16 centers
  • National Hospital Organization - Nagoya Medical Center — Nagoya
  • … and 15 more centers
Poland · 11 centers

Center list to be confirmed — check the primary protocol.

Italy · 8 centers
  • IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo — San Giovanni Rotondo
  • Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli — Naples
  • Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli — Naples
  • Azienda Ospedaliera Universitaria Federico II — Naples
  • Fondazione Istituto San Raffaele - G. Giglio Di Cefalu — Cefalù
  • Istituto Neurologico Casimiro Mondino — Pavia
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCS — Rome
  • Azienda Ospedaliera - Universitaria Sant' Andrea — Rome
Argentina · 5 centers
  • STAT Research S.A. — Ciudad Autónoma de Buenos Aires
  • Hospital Italiano de Buenos Aires — Ciudad Autónoma de Buenos Aires
  • Hospital General de Agudos José María Ramos Mejía — Ciudad Autónoma de Buenos Aires
  • Fundación Scherbovsky — Mendoza
  • INECO Neurociencias Oroño — Rosario
Brazil · 5 centers
  • Instituto de Neurologia de Curitiba — Curitiba
  • Hospital São Lucas da PUCRS — Porto Alegre
  • Universidade Estadual de Campinas — Campinas
  • Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto — Ribeirão Preto
  • Pseg Centro de Pesquisa Clinica — São Paulo
Spain · 5 centers

Center list to be confirmed — check the primary protocol.

Georgia · 4 centers
  • Multi-profile Clinic "New Hospitals" — Tbilisi
  • Pineo Medical Ecosystem — Tbilisi
  • Aversi Clinic - Central Branch — Tbilisi
  • Simon Khechinashvili University Hospital — Tbilisi
Australia · 3 centers
  • Concord Repatriation General Hospital — Concord
  • Townsville University Hospital — Douglas
  • Gold Coast University Hospital — Southport
Czechia · 3 centers
  • Fakultni Nemocnice Ostrava — Ostrava-Poruba
  • MINKSneuro s.r.o. - MUDr. Eduard Minks Ph.D. — Brno
  • Fakultni Nemocnice Brno — Brno
Belgium · 2 centers
  • Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg — Leuven
  • Centre Hospitalier Régional de la Citadelle — Liège
Canada · 2 centers
  • University Hospital - London Health Sciences Centre — London
  • Toronto General Hospital — Toronto
France · 2 centers
  • Hôpital Pasteur — Nice
  • Groupe Hospitalier Pellegrin — Bordeaux

Identifiers

NCT: NCT06456580 · RC18G006

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗