A Study of HY001N in the Treatment of Relapsed or Refractory Neurological Autoimmune Diseases

NCT07265206 | Not Yet Recruiting Early Phase 1

• 1 other identifier: HY001N001
• Study Type: interventional
• Target: 15
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Neurological Autoimmune Diseases

Keywords

CAR-T; HY001N; neurological autoimmune diseases

Outcome Measures

Primary Outcomes (2)

• Number of participants with dose-limiting toxicities (DLTs)

  Up to 28 days after infusion

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

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

  up to Month 36

Secondary Outcomes (10)

• 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

Study Arms (1)

Single dose of HY001N

EXPERIMENTAL

Biological: HY001N cell injection

Interventions

HY001N cell injection BIOLOGICAL

A conditioning chemotherapy regimen of fludarabine and cyclophosphamide will be administered followed by investigational treatment, HY001N.

Single dose of HY001N

Eligibility Criteria

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

You may qualify if:

• 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;
• The MG-ADL score is ≥6 points, and the eye muscle score is less than 50% of the total score;
• There is one of the following poor control conditions in the previous treatment: i. After at least two conventional immunotherapy drugs (including hormone and non-hormone immunosuppressants) have been used for ≥1 year without treatment failure, that is, despite receiving IST treatment, ADL remains persistently impaired (persistent weakness, crisis or inability to tolerate IST); ii. At least one IST treatment has failed, and long-term PE or IVIg treatment is required to control symptoms, that is, PE or IVIg treatment for muscle weakness needs to be carried out regularly for at least two cycles in the past 12 months.
• Chronic inflammatory demyelinating polyradiculopathy (CIDP): Meet the 2021 EAN/PNS diagnostic criteria (progressive or recurrent type), and have a corrected INCAT disability scale total score of 2-9 (with 2 points coming from leg disability), and meet the following requirements:
• Standardized use of at least one first-line therapy (cortisol hormone therapy, γ -globulin or plasma exchange therapy) for more than 3 months with poor symptom control;
• Inability to tolerate cortisol hormone, gamma globulin and plasma exchange due to side effects or other circumstances;

You may not qualify if:

• 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.
• 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:
• Left ventricular ejection fraction (LVEF) ≤45%;
• Persistent hypertension (≥160/100 MMHG) that remains uncontrolled despite standardized treatment
• New York Heart Association (NYHA) Grade III or IV congestive heart failure or active heart disease;
• Severe arrhythmias requiring treatment (excluding atrial fibrillation and paroxysmal supraventricular tachycardia);
• The QTcB interval is ≥450ms for males and ≥470ms for females (QTcB=QT/RR1/2);
• Had myocardial infarction, bypass surgery or stent surgery within 6 months prior to the study;

Sponsors & Collaborators

• Juventas Cell Therapy Ltd.: lead
• Beijing Tiantan Hospital: collaborator

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 17, 2025
• First Posted: December 4, 2025
• Study Start: December 30, 2025
• Primary Completion (Estimated): December 30, 2026
• Study Completion (Estimated): December 30, 2028
• Last Updated: December 4, 2025

Record last verified: 2025-11