NCT05828225RecruitingPhase 1
A Study on the Safety and Efficacy of Chimeric Antigen Receptor T Cells in the Treatment of Refractory Myasthenia Gravis
Zhejiang University1 site in 1 country9 target enrollmentStarted: April 30, 2023Last updated:
Conditions
Interventions
Drugs
Trial Snapshot
- Phase
- Phase 1
- Status
- Recruiting
- Sponsor
- Enrollment
- 9
- Locations
- 1
- Primary Endpoint
- Dose-limiting toxicity (DLT)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Age ≥18 and gender unlimited；
- •Confirmed as refractory myasthenia gravis with AchR antibody positive and accord one of the following three conditions
- •Repeated electrical stimulation suggests neuromuscular conduction deficits；
- •Tensilon test and neostigmine test positive;
- •The doctor judged that the symptoms of MG improved after treatment with oral cholinease inhibitors;
- •Consistent with the clinical classification of MGFA myasthenia gravis IIa-IVb (including IIa，IIb，IIIa， IIIb， IVa，IVb)
- •The baseline MG-ADL score ≥5 and the musculi oculi related score< 50 ；
- •Baseline QMG score>11;
- •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；
- •The estimated survival time is more than 12 weeks;
- •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
- •Epilepsy history or other central nervous system disease;
- •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 ;
- •Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
- •Pregnant (or lactating) women;
- •Patients with severe active infections;
- •Active infection of hepatitis B virus or hepatitis C virus;
- •Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
- •Those who have used any gene therapy products before;
- •The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
- •Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0 mg /dl;
- •Those who suffer from other uncontrolled diseases are not suitable to join the study;
- •HIV infection;
- •Any situation that the researchers believe may increase the risk of patients or interfere with the test results.
Arms & Interventions
Treatment Group
Experimental
Myasthenia Gravis
Intervention: CD19 CAR-T cells injection (Drug)
Outcomes
Primary Outcomes
Dose-limiting toxicity (DLT)
Time Frame: Baseline up to 28 days after CD19 CAR T-cells infusion
Adverse events assessed according to NCI-CTCAE v5.0 criteria
Maximum tolerable dose
Time Frame: From date of initial treatment to Day 28 post CD19 CAR-T infusion.
Maximum tolerable dose
Incidence of treatment-emergent adverse events (TEAEs)
Time Frame: Up to 90 days after CD19 CAR T-cells infusion
Incidence of treatment-emergent adverse events \[Safety and Tolerability\]
Secondary Outcomes
- Changes in serum AchR antibody titer(days 7, 14, 21, 28 and 90)
- MG-QOL15 scale(Baseline up to 28 days after CD19 CAR T-cells infusion)
- MG-activities of daily living profile (MG-ADL)(Baseline up to 28 days after CD19 CAR T-cells infusion)
- Myasthenia Gravis Composite Scale (MGC)(Baseline up to 28 days after CD19 CAR T-cells infusion)
- MG-activities of daily living profile (QMG)(Baseline up to 28 days after CD19 CAR T-cells infusion)
Investigators
He Huang
Clinical Professor
Zhejiang University
Study Sites (1)
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