An Exploratory Clinical Study on the Safety and Efficacy of HY001N Cell Injection in the Treatment of Relapsed or Refractory Autoimmune Neurological Diseases

Juventas Cell Therapy Ltd.0 sites15 target enrollmentDecember 30, 2025

InterventionsHY001N cell injection

Overview

Phase: Early Phase 1
Intervention: HY001N cell injection
Conditions: Not specified
Sponsor: Juventas Cell Therapy Ltd.
Enrollment: 15
Primary Endpoint: Number of participants with dose-limiting toxicities (DLTs)
Status: Not yet recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Similar Trials

Overview

Brief Summary

This study is a single-arm, open-label, non-randomized clinical trial aimed at evaluating the safety, tolerability and preliminary efficacy of HY001N cell injection in the treatment of patients with relapsed/refractory Neurological Autoimmune Diseases.

Registry

clinicaltrials.gov

Start Date

December 30, 2025

End Date

December 30, 2028

Last Updated

4 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Juventas Cell Therapy Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Patients who are willing to sign the informed consent form;
•Aged 18-75 years, male or female;
•At screening, subjects with relapsed/refractory antibody-mediated inflammatory neurological diseases without effective treatment, the expression of CD19+ B cells in peripheral blood and meeting the special criteria for different indications include:
•Neuromyelitis optica spectrum disorder (NMOSD): Comply with the diagnostic criteria for NMOSD of the International panel for NMO diagnosis (IPND) in 2015 and meet the following requirements:
•aquaporin 4 (AQP4) -IgG positive
•At least one immunosuppressant has been used for more than one year and the symptoms have not been well controlled;
•EDSS score ≤8 points;
•There must be at least two relapses within 24 months prior to screening or at least one relapse within the past 12 months, and the relapses should be stable for at least 4 weeks.
•Myasthenia gravis (MG): MG subjects who meet the MGFA classification II-IV as defined by the 2020 American Myasthenia Gravis Foundation (MGFA) diagnostic criteria and satisfy the following requirements:
•AChR-Ab serum positive or MuSK antibody positive;

Exclusion Criteria

•Current medical conditions or neurological disorders that may affect the effectiveness assessment, such as dementia, schizophrenia, bipolar disorder, major depressive disorder, history of multiple traumatic brain injury, current alcohol/drug abuse or dependence, or alcohol/drug dependence within the past two years.
•Pregnancy or breastfeeding;
•Have received organ or hematopoietic stem cell transplantation in the past;
•There has been a history of new thrombosis or organ infarction within the past six months;
•Patients diagnosed with active connective tissue diseases and requiring non-hormonal immunosuppressants/modulators for treatment;
•Combined with active infections (such as sepsis, bacteremia, mycosis, uncontrolled pulmonary infection and active tuberculosis, etc.);
•Positive for hepatitis B surface antigen (HBsAg) and/or hepatitis Be antigen (HBeAg); Positive hepatitis Be antibody (HBe-Ab) and/or hepatitis B core antibody (HBc-Ab), and HBV-DNA copy number greater than the measurable lower limit; Positive for hepatitis C (HCV) antibody Positive for human immunodeficiency virus (HIV) antibody; Those who test positive for syphilis (TP); The copy numbers of EBV-DNA and CMV-DNA are greater than the measurable lower limit.
•Having undergone major surgery that was evaluated by the researcher as unsuitable for inclusion within 4 weeks prior to screening;
•Other malignant tumors that have occurred or are currently present within the five years prior to screening are excluded, except for those with negligible risk of metastasis or death and curable tumors, such as well-treated cervical carcinoma in situ and basal cell carcinoma of the skin.
•The patient's heart meets any of the following conditions:

Arms & Interventions

Single dose of HY001N

Intervention: HY001N cell injection

Outcomes

Primary Outcomes

Number of participants with dose-limiting toxicities (DLTs)

Time Frame: Up to 28 days after infusion

Number of participants with adverse events (AEs)/serious adverse events (SAEs)

Time Frame: up to Month 36

AEs and SAEs: The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included.

Secondary Outcomes

Number of participants with disability improvement confirmed per EDSS(up to Month 36)
Annualized relapse rate(Up to 36 months)
Change from baseline in magnetic resonance imaging (MRI) metrics(up to Month 36)
Number of participants with confirmed disability progression per Expanded Disability Status Scale (EDSS)(up to Month 6)
Number of participants with at least 3 points improvement in Myasthenia Gravis activities of daily living (MG-ADL) score(up to Month 36)
Number of participants with at least 3 point improvement in quantitative Myasthenia Gravis (QMG) score(up to Month 36)
Number of participants who have obtained evidence of clinical improvement (ECI)(up to Month 36)
Change in antibody titer(up to Month 36)
Time of maximum observed blood concentration (Tmax)(up to Month 36)
Maximum observed blood concentration (Cmax)(up to Month 36)