Clinical trial • Phase III • Immunology|Neurology

TELITACICEPT for Generalized myasthenia gravis

Phase III trial of TELITACICEPT for Generalized myasthenia gravis.

Overview

Trial Therapeutic Area
Immunology|Neurology
Trial Disease
Generalized myasthenia gravis
Trial Stage
Phase III
Drug Modality
Peptide/protein/enzyme
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
19-07-2024
First CTIS Authorization Date
11-11-2024

Trial design

Randomised, open-label, placebo (contains excipients only and is visually indistinguishable from the telitacicept); randomized 1:1 telitacicept injection versus placebo (dose/schedule not specified in ctis summary).-controlled Phase III trial in Belgium, Czechia, Denmark and others.

Randomised
Yes
Open Label
Yes
Comparator
Placebo (contains excipients only and is visually indistinguishable from the Telitacicept); randomized 1:1 Telitacicept injection versus placebo (dose/schedule not specified in CTIS summary).
Target Sample Size
89

Eligibility

Recruits 89 CTIS record flags 'isVulnerablePopulationSelected' = true. All participants must provide signed informed consent ("1. Patients must provide signed informed consent to participate in the study and agreed to compliant with the study procedure."). Participants are adults (aged ≥18 years). No text describing assent or proxy consent was provided in the available CTIS fields..

Pregnancy Exclusion
16. Women who are currently breastfeeding or intend to breastfeed during the study period.
Vulnerable Population
CTIS record flags 'isVulnerablePopulationSelected' = true. All participants must provide signed informed consent ("1. Patients must provide signed informed consent to participate in the study and agreed to compliant with the study procedure."). Participants are adults (aged ≥18 years). No text describing assent or proxy consent was provided in the available CTIS fields.

Inclusion criteria

  • {"criterion_text":"- 1. Patients must provide signed informed consent to participate in the study and agreed to compliant with the study procedure.\n- 2. Male or female patient aged ≥18 years at screening.\n- 3. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness, eg, predominantly proximal, fatigable, fluctuating in severity, more severe in the evening, and improved with rest) meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV. Patient’s diagnosis should meet at least 1 of the following tests: a. History of abnormal neuromuscular transmission demonstrated by single fiber electromyography or repetitive nerve stimulation, OR b. History of positive edrophonium chloride test, OR c. Improvement in MG signs on oral acetylcholine esterase (AChE) inhibitors as assessed by the treating physician.\n- 4. Patients have positive antibodies against AChR or MuSK at screening.\n- 5. MG‐ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.\n- 6. QMG score ≥8 points, and ≥4 items score at least 2 points at screening and baseline\n- 7. Up to 2 concomitant medications (with the exception of cholinesterase inhibitors) for the treatment of MG are permitted if they meet the stability criteria prior to baseline\n- 8. Patients agree to use highly effective contraception\n- 9. Patients are appropriately vaccinated (e.g., vaccinations for pneumococcus, influenza, and COVID-19) per investigator's clinical judgement considering the patient's risk factors and according to country and local guidelines. Meningococcal vaccination is not required prior to initiating treatment with telitacicept"}

Exclusion criteria

  • {"criterion_text":"- 1. Patients have been diagnosed with any other autoimmune disease(s), eg, rheumatoid arthritis, Sjogren’s syndrome, which can potentially pose a safety or efficacy confounding risk. Note: Patients with abnormal thyroid function at screening will be excluded from the study. However, patients with a documented history of hypothyroidism or hyperthyroidism who have been adequately treated and whose laboratory results are within the normal range at screening can be included.\n- 10. Patients have current or history of primary immunodeficiency.\n- 11. Patients have history of malignancy within the last 5 years, except for adequately treated nonmelanoma skin cancer (eg, basal or squamous cell carcinoma) or carcinoma in situ of the cervix.\n- 12. Patient have prior or continuing diagnosis of serious cardiovascular disease(s) (including severe arrhythmias), liver, kidney, respiratory system, endocrine (eg, poorly controlled type I or type IIdiabetes mellitus, defined as glycosylated hemoglobin A1c [HbA1c] >8%), or hematologic disease(s), or other medical conditions that, in the opinion of the investigator, Medical Monitor, and/or Study Sponsor, could reasonably prevent the patient from safely participating in the study or from compliance to study procedures.\n- 13. Patients have a known allergy to human biological products or any of the listed excipients.\n- 14. Patients are currently dependent on alcohol/drugs (including marijuana) or have a history of alcohol/drug (including marijuana) dependence or addiction that, in the opinion of the investigator, may adversely affect patient safety or prevent patient compliance with study procedures.\n- 15. History of a suicidal attempt within the past 12 months, or current suicidal, intent or behavior during the screening period according to the Columbia suicide severity rating scales (C-SSRS).\n- 16. Women who are currently breastfeeding or intend to breastfeed during the study period.\n- 2. Patients have abnormal laboratory parameters at screening\n- 3. Patients have received prohibited immunosuppressants other than protocol permitted stable concomitant medication (per Inclusion Criterion 7, see protocol Table 7), biologics or other agents with protocol defined time period prior to randomization.\n- 4. Patients have received intravenous immunoglobulin or plasma exchange therapy ≤4 weeks before randomization.\n- 5. Patients have received live or attenuated vaccine ≤4 weeks prior to screening or during the study.\n- 6. Patients have participated in any interventional clinical trial or received an investigational treatment ≤3 months or within a period of 5 times of study drug’s half-life (whichever is longer).\n- 7. Patients have acute or chronic infection prior to randomization.\n- 8. Patients receiving treatment for any chronic infection (eg, tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster, atypical mycobacterium, etc).\n- 9. Patients have thymoma within 5 years or have received thymectomy ≤6 months prior to screening."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) score at Week 24","definition_or_measurement_approach":"Change from baseline in MG-ADL score measured at Week 24 (comparison of telitacicept versus placebo)"}

Secondary endpoints

  • {"endpoint_text":"- •\tChange from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24","definition_or_measurement_approach":"Change from baseline in MG-QOL15r score measured at Week 24"}
  • {"endpoint_text":"- •\tProportion of patients with a decrease of ≥2 points from baseline in MG-ADL score at Week 24","definition_or_measurement_approach":"Proportion of patients achieving ≥2-point decrease in MG-ADL at Week 24 compared with baseline"}
  • {"endpoint_text":"- •\tProportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24","definition_or_measurement_approach":"Proportion of patients achieving ≥3-point decrease in QMG score at Week 24 compared with baseline"}
  • {"endpoint_text":"- •\tProportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24","definition_or_measurement_approach":"Proportion of patients with MG-ADL score of 0 or 1 (MSE) at Week 24"}
  • {"endpoint_text":"- Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24","definition_or_measurement_approach":"Change from baseline in QMG score measured at Week 24"}

Recruitment

Digital Remote Recruitment
True, SCOUT email communication and Scout pre-screening materials are included (e.g., L2_CZ_Other subject material_SCOUT_Email Communication, L2_IT_Other subject material_Scout ICF), and use of eCOA/ePRO vendor (Eresearchtechnology Inc. / Clario) for collection of primary endpoint data.
Planned Sample Size
89
Recruitment Window Months
35
Consent Approach
Participants must provide signed informed consent ("1. Patients must provide signed informed consent..."). Consent materials (SIS-ICF) are provided in multiple country/language versions (examples: Dutch, French, German, Italian, Spanish, Polish, Czech, Danish). Participants are adults (aged ≥18); no assent or proxy-consent procedures are described in the available CTIS documents.

Methods

  • Site-based recruitment with country-specific recruitment procedures (K1 documents) and recruitment brochures/patient letters (K2 documents) — documents exist for Belgium, Czechia, Denmark, France, Italy, Spain, Poland.
  • Scout pre-screening materials and Scout informed consent forms (documents labelled 'SCOUT' and 'L1_*_SIS-ICF_Scout') and SCOUT email communication (L2_CZ_Other subject material_SCOUT_Email Communication) indicating email-based pre-screening/contact.
  • Use of patient-facing study brochures and patient letters in local languages (multiple D4 and K2 patient-facing documents listed per country).

Geography

Total Number Of Sites
33
Total Number Of Participants
97

Belgium

Earliest CTIS Part Ii Submission Date
09-10-2024
Latest Decision Or Authorization Date
29-01-2026
Processing Time Days
477
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
UZ Leuven
Department Name
Neurology & Neuromuscular Reference Center (NMRC)
Contact Person Name
Kristl Claeys
Contact Person Email
Kristl.Claeys@uzleuven.be
Site Name
Centre Hospitalier Regional De La Citadelle
Department Name
Centre de Réference des Maladies Neuromusculaires (CRMN)
Contact Person Name
Stéphanie Delstanche

Czechia

Earliest CTIS Part Ii Submission Date
14-08-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
537
Number Of Sites
3
Number Of Participants
9

Sites

Site Name
Neurologie – MINKSneuro s.r.o.
Contact Person Name
Eduard Minsk
Contact Person Email
eduardminks@seznam.cz
Site Name
Fakultni Nemocnice Brno
Department Name
Neurologická klinika
Contact Person Name
Stanislav Voháňka
Contact Person Email
vohanka.stanislav@fnbrno.cz
Site Name
Fakultni Nemocnice Ostrava
Department Name
Neurologická klinika
Contact Person Name
Martin Sabela
Contact Person Email
martin.sabela@fno.cz

Denmark

Earliest CTIS Part Ii Submission Date
11-10-2024
Latest Decision Or Authorization Date
12-11-2024
Processing Time Days
32
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
Rigshospitalet
Department Name
Neuromuscular Clinic and Research Unit Department 8077
Contact Person Name
Nicolai Preisler
Site Name
Aarhus Universitetshospital
Department Name
Department of Neurology
Contact Person Name
Henning Andersen
Contact Person Email
hennande@rm.dk

France

Earliest CTIS Part Ii Submission Date
09-09-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
512
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Service de Neurologie et Maladies Neuromusculaires and Centre d’investigations cliniques (CIC)
Contact Person Name
Guilhem Solé
Contact Person Email
guilhem.sole@chu-bordeaux.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Central Nervous System Unit of Pr. LEBRUNFRENAY
Contact Person Name
Saskia Bresch
Contact Person Email
bresch.s@chu-nice.fr

Italy

Earliest CTIS Part Ii Submission Date
30-09-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
490
Number Of Sites
8
Number Of Participants
14

Sites

Site Name
Fondazione Istituto Neurologico Nazionale Casimiro Mondino
Department Name
Neuroncology and Neuroinflammation
Contact Person Name
Marco Gastaldi
Contact Person Email
matteo.gastaldi@mondino.it
Site Name
Fondazione Istituto G. Giglio Di Cafalu
Department Name
U.O. Neurologia
Contact Person Name
Luigi Maria Edoardo Grimaldi
Contact Person Email
luigi.grimaldi@hsrgiglio.it
Site Name
Fakultni Nemocnice Brno
Department Name
Neurologia
Contact Person Name
Alvino Bisecco
Contact Person Email
alvino.bisecco@unicampania.it
Site Name
Casa Sollievo Della Sofferenza
Department Name
UOC Neurologia
Contact Person Name
Paolo Emilio Alboini
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
Neurologia
Contact Person Name
Francesco Saccà
Contact Person Email
francesco.sacca@unina.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
UOC Neurologia, Neuromuscolar Disease Centre
Contact Person Name
Stefania Morino
Contact Person Email
s.morino@ospedalesantandrea.it
Site Name
Azienda Ospedaliera Di Rilievo Nazionale Antonio Cardarelli
Department Name
UOC Neurofisiopatologia
Contact Person Name
Francesco Habetswallner
Site Name
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Department Name
UOC Neurologia
Contact Person Name
Raffaele Iorio

Spain

Earliest CTIS Part Ii Submission Date
30-10-2024
Latest Decision Or Authorization Date
03-02-2026
Processing Time Days
461
Number Of Sites
5
Number Of Participants
10

Sites

Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Neurology
Contact Person Name
Maria Teresa Sevilla Mantecon
Contact Person Email
sevilla_ter@gva.es
Site Name
Hospital Universitario Basurto
Department Name
Neurology
Contact Person Name
Solange Kapetanovic Garcia
Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Contact Person Name
Raul Juntas Morales
Contact Person Email
raul.juntas@vallhebron.cat
Site Name
Hospital General Universitario Dr. Balmis
Department Name
Neurology
Contact Person Name
Raquel Hernandez Lorido
Contact Person Email
rakel_her@hotmail.com
Site Name
Fundacio Assistencial De Mutua De Terrassa Fpc
Department Name
Neurology
Contact Person Name
Jerzy Krupinski Bielecki
Contact Person Email
jkrupinski@mutuaterrassa.es

Poland

Earliest CTIS Part Ii Submission Date
14-10-2024
Latest Decision Or Authorization Date
02-02-2026
Processing Time Days
476
Number Of Sites
11
Number Of Participants
50

Sites

Site Name
Ilkowski I Partnerzy sp.p. Lekarzy
Department Name
Niepubliczny Zaklad Opieki Zdrowotnej Neuro-Kard
Contact Person Name
Jan Ilkowski
Site Name
Krakowska Akademia Neurologii Sp. z o.o.
Department Name
-
Contact Person Name
Andrzej Szczudlik
Site Name
Centrum Medyczne Hope Clinic Sebastian Szklener
Department Name
-
Contact Person Name
Sebastian Szklener
Contact Person Email
eduardminks@seznam.cz
Site Name
Mtz Clinical Research Powered By Pratia
Department Name
-
Contact Person Name
Małgorzata Zajda
Contact Person Email
info@pratia.com
Site Name
Twoja Przychodnia Nowosolskie Centrum Medyczne Sp. z o.o.
Department Name
Neurology
Contact Person Name
Agata Kłósek
Contact Person Email
ncm@twojaprzychodnia.com
Site Name
Medicover Integrated Clinical Services Sp. z o.o.
Department Name
MICS CENTRUM MEDYCZNE BYDGOSZCZ
Contact Person Name
Łukasz Rzepiński
Contact Person Email
aleksandra.robak@medicover.com
Site Name
Neurologia Śląska Centrum Medyczne
Department Name
-
Contact Person Name
Marek Śmiłowski
Site Name
Neurocentrum Bydgoszcz Sp. z o.o.
Department Name
-
Contact Person Name
Robert Bonek
Contact Person Email
r.bonek@ncbydgoszcz.pl
Site Name
Clinirem Sp. z o.o.
Department Name
-
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
Uniwersyteckie Centrum Kliniczne
Department Name
Klinika Neurologii Dorosłych
Contact Person Name
Małgorzata Bilińska
Contact Person Email
malbili@gumed.edu.pl
Site Name
Centrum Medyczne Neuroprotect
Department Name
-
Contact Person Name
Mariusz Grudniak
Contact Person Email
marta.biel@neuroprotect.pl

Sponsor

Primary sponsor

Full Name
Vor Biopharma Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
Suvoda LLC
Responsibilities
code 3
Name
Icon Clinical Research Limited
Responsibilities
Clinical and Medical Monitoring; Medical Writing; Site Contract & Budget Negotiation; other sponsor duties (codes 1,2,5,6,8,11,12,15)
Name
Medidata Solutions Inc.
Responsibilities
code 7 (eClinical systems)
Name
Cerba
Responsibilities
Specific antibodies tests (AchR-Ab and MuSK-Ab); lab testing (code 4)
Name
Q Squared Solutions Limited
Responsibilities
Safety and Biomarkers tests; lab testing (code 4)
Name
Eresearchtechnology Inc.
Responsibilities
eCOA/ePRO/Patient Diary vendor (collection of primary endpoint data)
Name
Q Squared Solutions LLC
Responsibilities
PK and Immunogenicity tests; lab testing (code 4)

Third parties

  • {"country":"United States","full_name":"Suvoda LLC","duties_or_roles":"code 3","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"codes: 1,11,12,15 (Clinical and Medical Monitoring; Medical Writing; Site Contract & Budget Negotiation),2,5,6,8","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"code 7","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"France","full_name":"Cerba","duties_or_roles":"15 (Specific antibodies tests (AchR-Ab and MuSK-Ab)), 4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United Kingdom","full_name":"Q Squared Solutions Limited","duties_or_roles":"15 (Safety and Biomarkers tests), 4","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"15 (eCOA/ ePRO/ Patient Diary Vendor (collection of primary endpoint data))","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Q Squared Solutions LLC","duties_or_roles":"15 (PK and Immunogenecity tests), 4","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
Telitacicept injection
Active Substance
TELITACICEPT
Modality
Peptide/protein/enzyme
Routes Of Administration
Injection (solution for injection in pre-filled syringe)
Route
Injection (pre-filled syringe)
Authorisation Status
prodAuthStatus 1
Orphan Designation
Yes
Investigational Product Name
Placebo contains excipients only and is visually indistinguishable from the Telitacicept
Modality
Other
Routes Of Administration
Injection (visually matched to Telitacicept injection)
Route
Injection (matched placebo)

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