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Evaluate the Safety and Efficacy of CAR-T Cells in the Treatment of Refractory Myasthenia Gravis

Sponsor
Zhejiang University (Other)
Overall Status
Recruiting
CT.gov ID
NCT05828225
Collaborator
(none)
9
Enrollment
1
Location
1
Arm
35.7
Anticipated Duration (Months)
0.3
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is a single-arm, open-label, single-center, phase I study. The primary objective is to evaluate the safety of CD19 CAR-T therapy for patients with refractory myasthenia gravis, and to evaluate the pharmacokinetics of CD19 CAR-T in patients.

Condition or Disease Intervention/Treatment Phase
  • Drug: CD19 CAR-T cells injection
 Phase 1

Study Design

Study Type:
Interventional
Anticipated Enrollment :
9 participants
Allocation:
N/A
Intervention Model:
Single Group Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Refractory Myasthenia Gravis
Anticipated Study Start Date :
Apr 30, 2023
Anticipated Primary Completion Date :
Apr 20, 2026
Anticipated Study Completion Date :
Apr 20, 2026

Arms and Interventions

Arm Intervention/Treatment
Experimental: Treatment Group

Myasthenia Gravis

Drug: CD19 CAR-T cells injection
CD19 CAR-T in the Treatment of Refractory Myasthenia Gravis

Outcome Measures

Primary Outcome Measures

  1. Dose-limiting toxicity (DLT) [Baseline up to 28 days after CD19 CAR T-cells infusion]

    Adverse events assessed according to NCI-CTCAE v5.0 criteria

  2. Incidence of treatment-emergent adverse events (TEAEs) [Up to 90 days after CD19 CAR T-cells infusion]

    Incidence of treatment-emergent adverse events [Safety and Tolerability]

  3. Maximum tolerable dose [From date of initial treatment to Day 28 post CD19 CAR-T infusion.]

    Maximum tolerable dose

Secondary Outcome Measures

  1. Changes in serum AchR antibody titer [days 7, 14, 21, 28 and 90]

    Changes in serum AchR antibody titer

  2. MG-activities of daily living profile (MG-ADL) [Baseline up to 28 days after CD19 CAR T-cells infusion]

    MG-ADL Myasthenia Gravis Daily Activity Scale

  3. MG-activities of daily living profile (QMG) [Baseline up to 28 days after CD19 CAR T-cells infusion]

    Quantitative Score for Myasthenia Gravis

  4. Myasthenia Gravis Composite Scale (MGC) [Baseline up to 28 days after CD19 CAR T-cells infusion]

    Myasthenia Gravis Composite Scale

  5. MG-QOL15 scale [Baseline up to 28 days after CD19 CAR T-cells infusion]

    MG-QOL15 Quality of Life Scale for Myasthenia Gravis (15 items)

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
Inclusion Criteria:
    1. Age ≥18 and gender unlimited；
    1. Confirmed as refractory myasthenia gravis with AchR antibody positive and accord one of the following three conditions

  1. Repeated electrical stimulation suggests neuromuscular conduction deficits；

  2. Tensilon test and neostigmine test positive;

  3. The doctor judged that the symptoms of MG improved after treatment with oral cholinease inhibitors;

    1. Consistent with the clinical classification of MGFA myasthenia gravis IIa-IVb (including IIa，IIb，IIIa， IIIb， IVa，IVb)
    1. The baseline MG-ADL score ≥5 and the musculi oculi related score< 50 ；
    1. Baseline QMG score>11;
    1. Regular treatment with poor efficacy and/or lack of effective treatment means that there is still recurrence or exacerbation after treatment with conventional hormones, immunosuppressants (such as azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A, cyclophosphamide, etc.) or rituximab；
    1. The estimated survival time is more than 12 weeks;
    1. Women of childbearing age who have negative urine pregnancy test before medication administration and agree to take effective contraceptive measures during the trial period until the last follow-up.
Exclusion Criteria:
    1. Epilepsy history or other central nervous system disease;
    1. During the screening visit, the patient's thymectomy was less than 12 months or thymectomy was necessary during the study period or thymic radiation therapy ;
    1. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
    1. Pregnant (or lactating) women;
    1. Patients with severe active infections;
    1. Active infection of hepatitis B virus or hepatitis C virus;
    1. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
    1. Those who have used any gene therapy products before;
    1. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
    1. Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0 mg /dl;
    1. Those who suffer from other uncontrolled diseases are not suitable to join the study;
    1. HIV infection;
    1. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.

Contacts and Locations

Locations

Site City State Country Postal Code
1 The first affiliated hospital of medical college of zhejiang university Hangzhou Zhejiang China 310000

Sponsors and Collaborators

  • Zhejiang University

Investigators

  • Principal Investigator: He Huang, MD, Zhejiang University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
He Huang, Clinical Professor, Zhejiang University
ClinicalTrials.gov Identifier:
NCT05828225
Other Study ID Numbers:
  • TXB2023006
First Posted:
Apr 25, 2023
Last Update Posted:
Apr 25, 2023
Last Verified:
Apr 1, 2023
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Myasthenia Gravis
Muscle Weakness

Study Results

No Results Posted as of Apr 25, 2023