A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Vor Biopharma Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

25 Feb 2025 → ongoing

Decision date (initial)

2024-11-11

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Remegen Co. Ltd.

External identifiers

EU CT number

2024-512126-29-00

ClinicalTrials.gov

NCT06456580

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy, Others, Pharmacodynamic, Pharmacokinetic

To evaluate the efficacy of telitacicept versus placebo in patients with generalized myasthenia gravis (gMG)

Secondary objectives 1

1. To further evaluate the efficacy of telitacicept versus placebo in patients with gMG. To evaluate safety and tolerability of telitacicept in patients with gMG

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Study design 2 periods

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. Patients must provide signed informed consent to participate in the study and agreed to compliant with the study procedure.
2. 2. Male or female patient aged ≥18 years at screening.
3. 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.
4. 4. Patients have positive antibodies against AChR or MuSK at screening.
5. 5. MG‐ADL score ≥6 points at screening and baseline with ocular-related score <50% of the total score.
6. 6. QMG score ≥ 8 points, and ≥ 4 items score at least 2 points at screening and baseline
7. 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
8. 8. Patients agree to use highly effective contraception
9. 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 16

1. 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.
2. 2. Patients have abnormal laboratory parameters at screening
3. 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.
4. 4. Patients have received intravenous immunoglobulin or plasma exchange therapy ≤4 weeks before randomization.
5. 5. Patients have received live or attenuated vaccine ≤4 weeks prior to screening or during the study.
6. 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).
7. 7. Patients have acute or chronic infection prior to randomization.
8. 8. Patients receiving treatment for any chronic infection (eg, tuberculosis, pneumocystis, cytomegalovirus, herpes simplex virus, herpes zoster, atypical mycobacterium, etc).
9. 9. Patients have thymoma within 5 years or have received thymectomy ≤6 months prior to screening.
10. 10. Patients have current or history of primary immunodeficiency.
11. 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.
12. 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.
13. 13. Patients have a known allergy to human biological products or any of the listed excipients.
14. 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.
15. 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).
16. 16. Women who are currently breastfeeding or intend to breastfeed during the study period.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

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

Secondary endpoints 5

1. • Change from baseline in MG Quality of Life scale (MG-QOL15r) at Week 24
2. • Proportion of patients with a decrease of ≥2 points from baseline in MG-ADL score at Week 24
3. • Proportion of patients with a decrease of ≥3 points from baseline in QMG score at Week 24
4. • Proportion of patients who achieved minimal symptomatic expression (MSE, defined as having MG-ADL score of 0 or 1) at Week 24
5. Change from baseline in Quantitative Myasthenia Gravis (QMG) score at Week 24

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Vor Biopharma Inc.

Sponsor organisation

Vor Biopharma Inc.

Address

500 Boylston Street Suite 1350

City

Boston

Postcode

02116-3740

Country

United States

Scientific contact point

Organisation

Remegen Co. Ltd.

Contact name

Vor Biopharma Inc.

Public contact point

Organisation

Remegen Co. Ltd.

Contact name

Vor Biopharma Inc.

Third parties 7

Locations

8 EU/EEA countries · 40 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 114 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications