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NCT Number: NCT06485232

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

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.

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Key information

Conditions

Neuromyelitis Optica Spectrum Disorders Autoimmune Diseases Autoimmune Diseases of the Nervous System Chronic Disease Chronic Inflammatory Demyelinating Polyradiculoneuropathy Cranial Nerve Diseases Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Disease Attributes Eye Diseases Immune System Diseases Multiple Sclerosis Myasthenia Gravis Myasthenia Gravis, Generalized Myelitis, Transverse Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neuromuscular Diseases Neuromuscular Junction Diseases Neuromyelitis Optica Optic Nerve Diseases Optic Neuritis Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System Pathologic Processes Pathological Conditions, Signs and Symptoms Peripheral Nervous System Diseases Polyneuropathies Polyradiculoneuropathy Polyradiculoneuropathy, Chronic Inflammatory Demyelinating

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Early Phase 1

Primary location

Xuanwu Hospital, Capital Medical University

Beijing, Beijing Municipality, 100053, China

Location contact

Junwei Hao, MD; PhD

CONTACT

[email protected]

01083198277

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Universal BCMA CAR-T

Drug

Universal BCMA CAR-T

Universal CD19 CAR-T

Drug

Universal CD19 CAR-T

Universal BCMA CAR-T; Universal CD19 CAR-T

Drug

Universal BCMA CAR-T; Universal CD19 CAR-T

Primary outcomes

  1. Incidence of dose-limiting toxicities(DLTs)

    Time frame: First 28 days after infusion

    Incidence of dose-limiting toxicities(DLTs)

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

    Time frame: Up to 12 months after infusion

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

Secondary outcomes

  1. Concentrations of UCAR-T cells

    Time frame: 3 months

    The concentration of BCMA UCAR T cells and CD19 UCAR-T cells in peripheral blood after infusion

  2. B cell levels in peripheral blood

    Time frame: 3 months

    The change of B cell levels in peripheral blood after infusion

  3. Changes of pathogenic antibody titers after infusion

    Time frame: 1, 3, 6 ,12months

    The changes of pathogenic antibody titers in peripheral blood or cerebrospinal fluid.

  4. NMOSD: Annualized relapse rate

    Time frame: 6, 12months

    ARR is defined as the number of relapses divided by the total participant-years after infusion

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

    Time frame: 1, 3, 6, 12months

    MG-ADL scale assesses the impact of gMG on daily functions by measuring 8 signs or symptoms that are commonly affected in gMG. Each item is measured on a 4-point scale, where a score of 0 represents normal function and a score of 3 represents the loss of ability to perform that function. Total scores range from 0 to 24 points, with a higher score showing more severe gMG.

  6. CIDP:Changes of Inflammatory Neuropathy Cause and Treatment (INCAT) Score after infusion.

    Time frame: 1, 3, 6, 12months

    The INCAT score assesses the functionality of the arms and legs by giving a 0-5 score for arms and legs, with 0 representing no disability and 5 representing no arm function or inability to stand/walk.

  7. MS: Changes of the number of Gd-enhancing T1 Lesions

    Time frame: 6, 12months

    The changes of enhancing Lesions as detected by brain Magnetic Resonance Imaging (MRI)

  8. MS: Changes of the number of Number of New or Enlarging T2 Lesions

    Time frame: 6, 12months

    Number of new/enlarging T2 lesions on last available MRI scan compared to baseline.

Study contacts

Contact information is provided by the study sponsor or research team.

Junwei Hao, MD;PhD

CONTACT

[email protected]

01083198277

Sponsors and collaborators

Lead sponsor

Xuanwu Hospital, Beijing

Other

Collaborators

  • Bioray Laboratories

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2026
Study completion
2027
First posted
Jul 3, 2024
Registry last updated
Jan 1, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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