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

NCT ID: NCT05828225

Last Updated: 2023-04-25

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 9 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-04-30
• Study Completion Date: 2026-04-20

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.

Conditions

Myasthenia Gravis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment Group

Myasthenia Gravis

Group Type: EXPERIMENTAL

CD19 CAR-T cells injection

Intervention Type: DRUG

CD19 CAR-T in the Treatment of Refractory Myasthenia Gravis

Interventions

CD19 CAR-T cells injection

CD19 CAR-T in the Treatment of Refractory Myasthenia Gravis

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 1. Age ≥18 and gender unlimited；
• 2. 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;

• 3. Consistent with the clinical classification of MGFA myasthenia gravis IIa-IVb (including IIa，IIb，IIIa， IIIb， IVa，IVb)
• 4. The baseline MG-ADL score ≥5 and the musculi oculi related score< 50 ；
• 5. Baseline QMG score>11;
• 6. 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；
• 7. The estimated survival time is more than 12 weeks;
• 8. 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;
• 2. 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 ;
• 3. Electrocardiogram shows prolonged QT interval, severe heart diseases such as severe arrhythm ia in the past;
• 4. Pregnant (or lactating) women;
• 5. Patients with severe active infections;
• 6. Active infection of hepatitis B virus or hepatitis C virus;
• 7. Systemic steroids have used in the 4 weeks before participating in the treatment (except recently or currently using inhaled steroids);
• 8. Those who have used any gene therapy products before;
• 9. The proiferation rate is less than 5 times response to CD3/CD28 co-stimulation signal;
• 10. Serum creatinine > 2.5mg/dl or ALT / AST > 3 times ULN or bilirubin > 2.0 mg /dl;
• 11. Those who suffer from other uncontrolled diseases are not suitable to join the study;
• 12. HIV infection;
• 13. Any situation that the researchers believe may increase the risk of patients or interfere with the test results.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Zhejiang University

OTHER

Sponsor Role: lead

Responsible Party

He Huang

Clinical Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

He Huang, MD

Role: PRINCIPAL_INVESTIGATOR

Zhejiang University

Locations

The first affiliated hospital of medical college of zhejiang university

Hangzhou, Zhejiang, China

Site Status: RECRUITING

Countries

China

Central Contacts

He Huang, MD

Role: CONTACT

hehuangyu@126.com

13605714822

Yongxian Hu, MD

Role: CONTACT

huyongxian2000@aliyun.com

+8615957162012

Facility Contacts

He Huang, MD

Role: primary

hehuang@zju.edu.cn

0571-87233772

Yongxian Hu, MD

Role: backup

huyongxian2000@aliyun.com

+8615957162012

Other Identifiers

TXB2023006

Identifier Type: -

Identifier Source: org_study_id