Universal STAR-T Cell Injection in Generalized Myasthenia Gravis

ConditionGeneralized Myasthenia Gravis

Trial statusNot yet recruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age18-65

SponsorDaishi Tian

About this trial

This is a Phase I, single-arm, open-label, dose-escalation and dose-expansion study.

This is an exploratory clinical study of universal STAR-T cell injection in patients with refractory generalized myasthenia gravis (GMG). Approximately 10-24 participants aged 18-65 years (inclusive) with the condition are planned to be enrolled.

The primary objective is to evaluate the safety, tolerability, preliminary efficacy, pharmacokinetic/pharmacodynamic (PK/PD) profile, and immunogenicity of universal STAR-T cell injection. The starting dose is 1.5E6 STAR+ T cells/kg, administered as a single intravenous infusion.Based on safety, PK results, and preliminary efficacy data obtained from the initial dose cohorts, a recommended dose will be selected for subsequent dose-expansion studies to further systematically evaluate the safety and efficacy of universal STAR-T cell injection.

This study includes the screening period (from D-28 to D-6), the pre-clearance treatment and rest observation period (from D-5 to D-1), the cell infusion and main study endpoint observation period (from D0 to W12 after infusion), and the follow-up period (from W12 after infusion to W104).

The study is being conducted at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology.

Eligibility criteria

Qualifiers

Age 18-65 years (inclusive), gender (no gender restriction);

Previously diagnosed with Generalized Myasthenia Gravis (GMG), meeting the 2020 MGFA diagnostic criteria, with MG-ADL total score ≥6 and ocular-related subscore <50% of the total score, positive relevant antibodies, MGFA classification Grade II-IV, and having received at least 2 kinds of immunosuppressants or biological agents for standardized treatment;

MG-ADL total score increased by ≥2 points, and no single ocular item increased by >1 point;

QMGS total score increased by ≥3 points, or ≥2 non-ocular items each increased by ≥1 point;

Disqualifiers

Have used immunomodulatory or immunosuppressive drugs with therapeutic effects on the disease within 5 half-lives before enrollment, or biologics within 4 weeks (except for those who have received rituximab, with the last use of rituximab <3 months prior [B-cell reconstitution is excluded]);

Have a history of severe drug allergy or allergic constitution;

Have uncontrolled or requiring treatment for fungal, bacterial, or viral infections;

Have active tumor lesions at screening;

Trial design

Treatments tested in this trial

Universal STAR-T Cell

Treatment groups

10 Participants

are divided into 1 treatment group

Sponsors and collaborators

Daishi Tian

Lead sponsor

Tongji Hospital

Sponsor institution

China Immunotech (Beijing) Biotechnology Co., Ltd.

Collaborator

Source ClinicalTrials.gov