Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.

NCT06485232 | Not Yet Recruiting Early Phase 1

• 1 other identifier: 2024-BRL-302-02
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open label, single-site, dose-escalation study in up to 25 participants with refractory autoimmune diseases of nervous system. This study aims to evaluate the safety and efficacy of the treatment with universal BCMA and CD19 CART.

Conditions

Neuromyelitis Optica Spectrum Disorders; Myasthenia Gravis, Generalized; Multiple Sclerosis; Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Keywords

universal CAR-T; Neuromyelitis Optica Spectrum Disorders; Myasthenia Gravis; Multiple Sclerosis; Chronic Inflammatory Demyelinating Polyradiculoneuropathy

Outcome Measures

Primary Outcomes (2)

• Incidence of dose-limiting toxicities(DLTs)

  Incidence of dose-limiting toxicities(DLTs)

  First 28 days after infusion

• Incidence of adverse events(AEs) and severe adverse

  Incidence of adverse events(AEs) and severe adverse events(SAEs).

  Up to 12 months after infusion

Secondary Outcomes (8)

• Concentrations of UCAR-T cells

  3 months

• B cell levels in peripheral blood

  3 months

• Changes of pathogenic antibody titers after infusion

  1, 3, 6 ,12months

• NMOSD: Annualized relapse rate

  6, 12months

• gMG: Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score

  1, 3, 6, 12months

Study Arms (3)

BCMA CAR-T Group

EXPERIMENTAL

Universal BCMA CAR-T

Drug: Universal BCMA CAR-T

CD19 CAR-T Group

EXPERIMENTAL

Universal CD19 CAR-T

Drug: Universal CD19 CAR-T

BCMA CAR-T + CD19 CAR-T Group

EXPERIMENTAL

Universal BCMA CAR-T; Universal CD19 CAR-T

Drug: Universal BCMA CAR-T; Universal CD19 CAR-T

Interventions

Universal BCMA CAR-T DRUG

Universal BCMA CAR-T

BCMA CAR-T Group

Universal CD19 CAR-T DRUG

Universal CD19 CAR-T

CD19 CAR-T Group

Universal BCMA CAR-T; Universal CD19 CAR-T DRUG

Universal BCMA CAR-T; Universal CD19 CAR-T

BCMA CAR-T + CD19 CAR-T Group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Aged 18-75 years (for MS patients, 18-55 years); both genders eligible.
• Subjects with refractory neurological autoimmune diseases who have failed standard treatment or lack effective treatment, Including neuromyelitis optica spectrum disorders(NMOSD), generalized myasthenia gravis(gMG), chronic inflammatory demyelinating Polyradiculoneuropathy(CIDP) and multiple sclerosis(MS).
• Anticipated survival of ≥ 12 weeks as judged by the researcher.
• Agrees to use double barrier methods, condoms, oral or injectable contraceptives, or intrauterine devices during the study period and for one year after taking the study medication.
• Provides written informed consent.

You may not qualify if:

• History of solid organ transplantation.
• Malignant tumor within the last two years.
• Positive for Hepatitis B surface antigen (HBsAg) or Hepatitis B core antibody (HBcAb), with peripheral blood Hepatitis B virus (HBV) DNA detected as positive; positive for Hepatitis C virus antibodies, with peripheral blood Hepatitis C virus RNA detected as positive; positive for Human Immunodeficiency Virus (HIV) antibodies; positive for Cytomegalovirus (CMV) DNA; positive for syphilis.
• Primary immunodeficiency (congenital or acquired).
• Severe cardiac disease.
• History of psychiatric disorders or history of psychotropic drug abuse, with no history of withdrawal.
• Allergic constitution or a history of severe allergies.
• Pregnant or breastfeeding women.

Sponsors & Collaborators

• Xuanwu Hospital, Beijing: lead
• Bioray Laboratories: collaborator

Study Sites (1)

Xuanwu Hospital, Capital Medical University

Beijing, Beijing Municipality, 100053, China

Location

MeSH Terms

Conditions

Neuromyelitis Optica; Myasthenia Gravis; Multiple Sclerosis; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Condition Hierarchy (Ancestors)

Myelitis, Transverse; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Optic Neuritis; Optic Nerve Diseases; Cranial Nerve Diseases; Demyelinating Diseases; Eye Diseases; Autoimmune Diseases; Immune System Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Polyradiculoneuropathy; Polyneuropathies; Peripheral Nervous System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Central Study Contacts

Junwei Hao, MD;PhD

CONTACT

01083198277; haojunwei@vip.163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2024
• First Posted: July 3, 2024
• Study Start: February 28, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2027
• Last Updated: January 1, 2025

Record last verified: 2024-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)