Clinical trial NCT06485232: Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.
Official title: An Exploratory Study on the Safety and Efficacy of Universal CAR-T Cells Targeting BCMA and CD19 in the Treatment of Refractory Autoimmune Diseases of the Nervous System
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: EARLY_PHASE1
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
Lead sponsor: Xuanwu Hospital, Beijing
Sponsor class: OTHER
Collaborators: name: Bioray Laboratories
First posted: 2024-07-03
Enrollment: 25
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Incidence of dose-limiting toxicities(DLTs)
timeFrame: First 28 days after infusion
measure: Incidence of adverse events(AEs) and severe adverse
timeFrame: Up to 12 months after infusion
Secondary outcomes:
measure: Concentrations of UCAR-T cells
timeFrame: 3 months
measure: B cell levels in peripheral blood
timeFrame: 3 months
measure: Changes of pathogenic antibody titers after infusion
timeFrame: 1, 3, 6 ,12months
measure: NMOSD: Annualized relapse rate
timeFrame: 6, 12months
measure: gMG: Changes of Myasthenia Gravis Activities if Daily Living (MG-ADL) Score
timeFrame: 1, 3, 6, 12months
measure: CIDP:Changes of Inflammatory Neuropathy Cause and Treatment (INCAT) Score after infusion.
timeFrame: 1, 3, 6, 12months
measure: MS: Changes of the number of Gd-enhancing T1 Lesions
timeFrame: 6, 12months
measure: MS: Changes of the number of Number of New or Enlarging T2 Lesions
timeFrame: 6, 12months
Other outcomes:
Locations:
city: Beijing
country: China
facility: Xuanwu Hospital, Capital Medical University
state: Beijing
Study officials:
References: