Generalized Myasthenia GravisPhase 3NCT06456580
A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)
A Phase 3 study testing Telitacicept in people with Generalized Myasthenia Gravis, run by Vor Biopharma at 120 sites in 15 countries.
Trial summary
The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Generalized Myasthenia Gravis
- What is being tested?
- Telitacicept
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 191 participants
- Where
- 120 sitesin 15 countries
- Study dates
- 17 Jul 2024to Jan 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
120 sites in 15 countries.
120 sites in 15 countries
United States30 sites
Wellstar MCG Health Medical Center
Augusta, Georgia, 30912
SFM Clinical Research
Boca Raton, Florida, 33487
University of North Carolina Hospitals - Neurology Clinic
Chapel Hill, North Carolina, 27517
University of Cincinnati Health Physicians - Clifton
Cincinnati, Ohio, 45219
BayCare Medical Group - Clearwater
Clearwater, Florida, 33759
UM Health Care - Neurology and Sleep Disorders Clinic
Columbia, Missouri, 65212
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Michigan State University Clinical Center
East Lansing, Michigan, 48824
Neurology Rare Disease Center
Flower Mound, Texas, 75028
Neurology Center of New England
Foxborough, Massachusetts, 02035
Trinity Health - Grand Rapids Hospital
Grand Rapids, Michigan, 49503
Center for Neurological Disorders, S.C. - Greenfield - Gamma Therapeutic Center
Greenfield, Wisconsin, 53228
Houston Methodist Neurological Institute
Houston, Texas, 77030
University of Kansas Medical Center
Kansas City, Kansas, 66160
University of Tennessee Medical Center
Knoxville, Tennessee, 37920
University of Kentucky
Lexington, Kentucky, 40506
University of Southern California
Los Angeles, California, 90033
University of Louisville Physicians
Louisville, Kentucky, 40202
Allied Biomedical Research Institute (ABRI)
Miami, Florida, 33155
Robert Wood Johnson University Hospital New Brunswick
New Brunswick, New Jersey, 08901
Lange Neurology
New York, New York, 10022
Mount Sinai Health System
New York, New York, 10029
Hospital Sisters Health System Medical
O'Fallon, Illinois, 62269
University of California Irvine
Orange, California, 92868
Medsol Clinical Research Center
Port Charlotte, Florida, 33952
University of California San Francisco (UCSF)
San Francisco, California, 94143
HonorHealth Neurology - Bob Bové Neuroscience Institute
Scottsdale, Arizona, 85258
University of South Florida Health (USF Health)
Tampa, Florida, 33613
Montefiore Medical Center
The Bronx, New York, 10467
Empower Clinical Research
Tigard, Oregon, 97223
China19 sites
Beijing Hospital
Beijing, Beijing Municipality, 100730
Jilin University China-Japan Union Hospital
Changchun, Jilin, 130000
XiangYa Hospital of Central South University
Changsha, Hunan, 410000
The First Affiliated Hospital of Chongqing Medical University
Chongqing, Chongqing Municipality, 400010
Fujian Medical University Union Hospital
Fuzhou, Fujian, 350000
The First Affiliated Hospital, Sun Yat-Sen University
Guangzhou, Guangdong, 510080
AnHui Provincial Hospital
Hefei, Anhui, 230000
QiLU Hospital of ShanDong University
Jinan, Shangdong, 250000
The 960th Hospital of the Joint Service Support Force of the Chinese People's Liberation Army
Jinan, Shangdong, 250031
The First People's Hospital of JiNing Medical University
Jining, Shangdong, 272000
The Second Hospital & Clinical Medical School. Lan Zhou University
Lanzhou, Gansu, 730000
NingBo Medical Center LiHuiLi Hospital
Ningbo, Zhejiang, 315100
Huashan Hospital of the ShangHai FuDan University Medical College
Shanghai, Shanghai Municipality, 201100
First Hospital of ShanXi Medical University
Taiyuan, Shanxi, 030000
The 1 School of Medicine, School of Information and Engineering. The 1 Affiliated Hospital of WMU.
Wenzhou, Zhejiang, 325000
TongJi Medical College of HUST
Wuhan, Hubei, 430030
The Second Affiliated Hospital of PLA Air Force Military Medical University
Xi'an, Shaanxi, 710000
Xi'An GaoXin Hospital
Xi'an, Shaanxi, 710000
Affiliated Hospital of ZunYi Medical University
Zunyi, Guizhou, 563000
Japan16 sites
Juntendo University Hospital
Bunkyo-ku, Tokyo, 113-8431
Kyushu University Hospital
Fukuoka, 812-8582
Okinawa National Hospital
Ginowan-Shi, Okinawa, 901-2214
Seirei Hamamatsu General Hospital
Hamamatsu, Sizuoka [Shizuoka], 430-8558
National Hospital Organization Niigata National Hospital
Kashiwazaki-Shi, Niigata, 945-8585
St. Marianna University Hospital
Kawasaki, Kanagawa, 216-8511
Kobe University Hospital
Kobe, Hyōgo, 650-0017
National Hospital Organization Utano National Hospital
Kyoto, 616-8255
National Hospital Organization - Nagoya Medical Center
Nagoya, Aichi-ken, 460-0001
Sapporo Medical University Hospital
Sapporo, Hokkaido, 060-8543
Hokkaido University Hospital
Sapporo, Hokkaido, 060-8648
NHO Sendai Medical Center
Sendai, Miyagi, 983-8520
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo, 160-0023
Nagoya City University Hospital
Suita-shi, Osaka, 564-0013
Tokushima University Hospital
Tokushima, Tokusima [Tokushima], 770-8503
Yokohama Medical Center
Yokohama, Kanagawa, 245-8575
Poland11 sites
Medicover Integrated Clinical Services (MICS) - Centrum Medyczne Bydgoszcz
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-065
Neurocentrum Bydgoszcz
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-796
Uniwersyteckie Centrum Kliniczne w Gdańsku
Gdansk, Pomeranian Voivodeship, 80-214
Neurologia Śląska Centrum Medyczne
Katowice, Silesian Voivodeship, 40-123
Krakowska Akademia Neurologii Sp. z o.o.
Krakow, Lesser Poland Voivodeship, 31-505
Clinirem - Lublin
Lublin, Lublin Voivodeship, 20-064
Centrum Medyczne Hope Clinic
Lublin, Lubusz Voivodeship, 20-701
Twoja Przychodnia Nowosolskie Centrum Medyczne
Nowa Sól, Lubusz Voivodeship, 67-100
Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD
Poznan, Greater Poland Voivodeship, 61-853
Centrum Medyczne Neuroprot
Warsaw, Masovian Voivodeship, 01-684
MTZ Powered by Pratia
Warsaw, Masovian Voivodeship, 02-172
Italy8 sites
Fondazione Istituto San Raffaele - G. Giglio Di Cefalu
Cefalù, Palermo, 90015
Azienda Ospedaliera di Rilievo Nazionale Antonio Cardarelli
Naples, 80131
Azienda Ospedaliera Universitaria - Università degli Studi della Campania Luigi Vanvitelli
Naples, 80131
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
Istituto Neurologico Casimiro Mondino
Pavia, 27100
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, 00168
Azienda Ospedaliera - Universitaria Sant' Andrea
Rome, 00189
IRCCS Fondazione Casa Sollievo della Sofferenza SG Rotondo
San Giovanni Rotondo, Foggia, 71013
Bulgaria6 sites
Acibadem City Clinic University Multiprofile Hospital for Active Treatment
Panagyurishte, Pazardzhik, 4500
University Multiprofile Hospital For Active Treatment Dr. Georgi Stranski EAD
Pleven, 5800
University Multiprofile Hospital for Active Treatment Sveti George - Base 2
Plovdiv, 4002
University Multiprofile Hospital for Active Treatment and Emergency Medicine N.I. Pirogov
Sevlievo, Gabrovo, 5400
Multiprofile Hospital for Active Treatment in Neurology and Psychiatry Sveti Naum
Sofia, Sofia-Grad, 1113
University Multiprofile Hospital for Active Treatment Aleksandrovska
Sofia, Sofia-Grad, 1431
Argentina5 sites
STAT Research S.A.
Ciudad Autónoma de Buenos Aires, Buenos Aires, C1023AAB
Hospital Italiano de Buenos Aires
Ciudad Autónoma de Buenos Aires, Buenos Aires, C1199ABB
Hospital General de Agudos José María Ramos Mejía
Ciudad Autónoma de Buenos Aires, Buenos Aires, C1221ADC
Fundación Scherbovsky
Mendoza, M5500AXR
INECO Neurociencias Oroño
Rosario, Santa Fe Province, S2000TP
Brazil5 sites
Universidade Estadual de Campinas
Campinas, São Paulo, 13083-888
Instituto de Neurologia de Curitiba
Curitiba, Paraná, 81210-310
Hospital São Lucas da PUCRS
Porto Alegre, Rio Grande do Sul, 90619-900
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto
Ribeirão Preto, São Paulo, 14051-140
Pseg Centro de Pesquisa Clinica
São Paulo, 04038-002
Spain5 sites
Hospital General Universitario Dr. Balmis
Alicante, 03010
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario de Basurto
Bilbao, Biscay, 48013
Hospital Universitari Mútua Terrassa
Terrassa, Barcelona, 08221
Hospital Universitari i Politècnic La Fe
Valencia, 46026
Georgia4 sites
Multi-profile Clinic "New Hospitals"
Tbilisi, 0114
Pineo Medical Ecosystem
Tbilisi, 0114
Aversi Clinic - Central Branch
Tbilisi, 0160
Simon Khechinashvili University Hospital
Tbilisi, 0179
Australia3 sites
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Townsville University Hospital
Douglas, Queensland, 4814
Gold Coast University Hospital
Southport, Queensland, 4215
Czechia3 sites
MINKSneuro s.r.o. - MUDr. Eduard Minks Ph.D.
Brno, South Moravian, 603 00
Fakultni Nemocnice Brno
Brno, South Moravian, 625 00
Fakultni Nemocnice Ostrava
Ostrava-Poruba, Moravian-Silesian, 708 54
Belgium2 sites
Universitair Ziekenhuis (UZ) Leuven - Campus Gasthuisberg
Leuven, Flemish Brabant, 3000
Centre Hospitalier Régional de la Citadelle
Liège, 4000
France2 sites
Groupe Hospitalier Pellegrin
Bordeaux, Gironde, 33076
Hôpital Pasteur
Nice, Alpes-Maritimes, 06001
Canada1 site
University Hospital - London Health Sciences Centre
London, Ontario, N6C 2R5
About this study
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
The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.
Read the 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.
- Conditions
- Generalized Myasthenia Gravis
- Being tested
- Telitacicept, Placebo
- Keywords
- Myasthenia Gravis, gMG
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TelitaciceptBiological
Also called RC18, RC18-L
Subcutaneous injection
PlaceboDrug
Subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Telitacicept | Experimental | Telitacicept Receives: Biological: Telitacicept |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Week 24
Secondary outcomes (5)
Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24
The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 items. Each item in the scale is scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicate more severe impairment. A decrease from Baseline score indicates improvement.
Measured: Week 24
Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24
The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale is scored on a 0 to 2-point scale (0=Not at all, 1=Somewhat, 2=Very much). The total score is the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicate more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicates improvement.
Measured: 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
Proportion of patients with a decrease of ≥2 points from baseline in 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.
Measured: Week 24
Proportion of patients with a decrease of ≥3 points from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24
Proportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consists of 13 items. Each item in the scale is scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicate more severe impairment. A decrease from Baseline score indicates improvement.
Measured: Week 24
Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24
Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24
Measured: Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jun 2024
- Study start17 Jul 2024
- Primary completionMar 2027 (estimated)
- Study completionJan 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Generalized Myasthenia GravisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 191 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vor Biopharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06456580
- Sponsor's protocol ID
- RC18G006
- Status last verified
- Oct 2026
- First submitted
- 7 Jun 2024
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No