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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

This trial does not accept healthy volunteers

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

Design model

Single group

Treatments tested in this trial

  • Universal STAR-T Cell

    Drug

    Subjects will receive infusion of Universal STAR-T Cells at the starting dose of 1.5E6 STAR+T cells/kg.

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: Universal STAR-T CellExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Type, severity, and frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs).

Characterization of treatment-emergent adverse events (TEAEs) graded by NCI-CTCAE v6.0, including laboratory abnormalities, vital sign changes, and infusion-related reactions.

Time frame
AEs observation will be follow-up for 24 weeks. The observation period is extended to 104 weeks.
2

Incidence of Dose-Limiting Toxicities (DLTs).

To assess the safety and tolerability of \[Drug Name\] and determine the Maximum Tolerated Dose (MTD) or Recommended Phase 2 Dose (RP2D). DLTs are defined according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) v6.0.

Time frame
Within 28 days after infusion

Secondary outcomes

1

Change in Myasthenia Gravis Quantitative Scale (QMG) or Myasthenia Gravis Activities of Daily Living (MG-ADL) Scores.

Evaluation of preliminary efficacy based on the Myasthenia Gravis Quantitative Scale (QMG) and Myasthenia Gravis Activities of Daily Living (MG-ADL) scale. Response is defined as a ≥5-point reduction from baseline in either the QMG score or the MG-ADL score. Both scales are standardized patient-reported outcome measures.

Time frame
The efficacy endpoint evaluation for 104 weeks.
2

Maximum Plasma Concentration of Universal STAR-T Cells (Cmax)

To evaluate the maximum observed plasma concentration of Universal STAR-T Cells .

Time frame
Up to 24 weeks (Core Analysis Period); Extended observation up to 104 weeks.
3

Time to Reach Maximum Plasma Concentration (Tmax) of Universal STAR-T Cells.

To evaluate the time to reach the maximum observed plasma concentration of Universal STAR-T Cells.

Time frame
Up to 24 weeks (Core Analysis Period); Extended observation up to 104 weeks.
4

Area Under the Plasma Concentration-Time Curve (AUC) of Universal STAR-T Cells.

To evaluate the total systemic exposure to Universal STAR-T Cells over time.

Time frame
Up to 24 weeks (Core Analysis Period); Extended observation up to 104 weeks.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Daishi Tian

Lead sponsor

Tongji Hospital

Sponsor institution

China Immunotech (Beijing) Biotechnology Co., Ltd.

Collaborator

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: