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Neurological Autoimmune DiseasesEarly Phase 1NCT07265206

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

A Early Phase 1 study testing HY001N cell injection in people with Neurological Autoimmune Diseases, run by Juventas Cell Therapy Ltd..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Neurological Autoimmune Diseases
What is being tested?
HY001N cell injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
No sites listed yet
Study dates
30 Dec 2025to 30 Dec 2028
Phase
Early Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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;
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  • 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;
  • Determined as an active disease (CIDP disease activity status [CDAS] score ≥2), (the disease must show activity and present clinically significant deterioration on at least one CIDP clinical assessment tool (including INCAT, I-RODS or mean grip strength)
  • Multiple Sclerosis (MS): According to the revised McDonald diagnostic criteria in 2017, a clinical diagnosis of MS is made, and the following three points are simultaneously met:
  • Before ICF and in any of the following circumstances: i. Two relapses have been recorded in the past two years. ii. One recurrence was recorded within the past year. iii. The result of Gd enhanced MRI scan was positive within one year before screening. Note: If there is no positive result of Gd enhanced scan recorded in the previous year, the screening MRI scan results can be used.
  • Neurological stability within one month before screening and baseline (including no recurrence of MS at this stage)
  • Autoimmune encephalitis (AE): According to the 2016 International Diagnostic Criteria for Autoimmune Encephalitis, the subject was diagnosed with autoimmune encephalitis and met all of the following requirements:
  • At least one pathogenic antibody, NMDAR or LGI1, is positive;
  • Previously standardized use of corticosteroids, at least one immunosuppressant/modulator, including CD20 monoclonal antibodies with poor symptom control or intolerance;
  • Autoimmune encephalitis occurred within 3 months before screening;
  • Modified Rankin Scale (mRS) score ≥2 or Clinical Assessment Scale for Autoimmune Encephalitis (CASE) score ≥4.
  • Anti-myelin oligodendrocyte glycoprotein immunoglobulin G antibody-related disease (MOGAD): Comply with the diagnostic criteria released by the International MOGAD Expert Group in 2023 and meet all of the following requirements:
  • Serum positive for anti-MOG antibodies
  • There was a history of ≥1 recurrence of MOGAD within 12 months prior to screening, or ≥2 episodes within 24 months prior to screening.
  • Proper organ function, complying with the following criteria :
  • Blood routine: Absolute lymphocyte count (ALC) ≥0.4×109/L, platelet count (PLT) ≥50×109/L, hemoglobin ≥80 g/L;
  • Coagulation function: International Normalized ratio (INR) ≤1.5 times the upper limit of normal (ULN), and activated partial prothrombin time (APTT) ≤1.5 times ULN;
  • Liver function: Serum aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤3 times ULN, total bilirubin ≤1.5 times ULN;
  • Renal function: Serum creatinine ≤1.5×ULN or creatinine clearance rate (Cockcroft Gault formula) ≥60 mL/min;
  • Pulmonary function: Under indoor ventilation conditions, the blood oxygen saturation in the non-oxygen inhalation state is ≥92%; There is no clinically significant pleural effusion.
  • The clinical physician evaluated the patient's condition and allowed the use of glucocorticoids at a dose not exceeding 10mg of prednisone or its equivalent during the study period, and permitted the discontinuation of all immunosuppressants.
  • The subjects whose partners are fertile agree to use effective contraceptive measures throughout the treatment period and for 24 months after the treatment, and during this period, they are not allowed to donate eggs/sperm for assisted reproduction. Female subjects of childbearing age (women who have undergone sterilization surgery or have been menopausal for ≥12 months are not considered fertile) have negative urine pregnancy or blood pregnancy tests during the screening period.

Who cannot join

  • 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.);
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  • 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:
  • 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;
  • Other heart diseases that the researcher judged as unsuitable for enrollment;
  • Have received a live vaccine within 6 weeks before screening.
  • Participate in other interventional clinical studies before cell reinfusion,or receiving treatment of an active investigational drug within 5 half-lives . Those who have received active trial drug treatment, or who intend to participate in another clinical trial or receive treatment other than that stipulated in the protocol throughout the study period.
  • Those who are known to have hypersensitivity reactions to the components of the preparations used in the test;
  • Previously received CAR-T cell therapy;
  • Other conditions that the investigator considers unsuitable for participation in the study.

About this study

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

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.

Being tested
HY001N cell injection
Keywords
CAR-T, HY001N, neurological autoimmune diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HY001N cell injectionBiological

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

Study groups in NCT07265206
Study groupRoleWhat they receive
Single dose of HY001NExperimentalNot described
Receives: Biological: HY001N cell injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of participants with dose-limiting toxicities (DLTs)

    Measured: Up to 28 days after infusion

  2. 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.

    Measured: up to Month 36

Secondary outcomes (10)

  1. Number of participants with disability improvement confirmed per EDSS

    NMOSD、MOGAD

    Measured: up to Month 36

  2. Annualized relapse rate

    NMOSD、MOGAD、MS

    Measured: Up to 36 months

  3. Change from baseline in magnetic resonance imaging (MRI) metrics

    NMOSD、MOGAD：MRI metrics assessed are 1) number of gadolinium-enhancing T1 lesions and 2) total number of new or enlarging hyperintense T2-weigted lesions

    Measured: up to Month 36

  4. Number of participants with confirmed disability progression per Expanded Disability Status Scale (EDSS)

    MS

    Measured: up to Month 6

  5. Number of participants with at least 3 points improvement in Myasthenia Gravis activities of daily living (MG-ADL) score

    MG

    Measured: up to Month 36

  6. Number of participants with at least 3 point improvement in quantitative Myasthenia Gravis (QMG) score

    MG

    Measured: up to Month 36

  7. Number of participants who have obtained evidence of clinical improvement (ECI)

    CIDP

    Measured: up to Month 36

  8. Change in antibody titer

    Measured: up to Month 36

  9. Time of maximum observed blood concentration (Tmax)

    Measured: up to Month 36

  10. Maximum observed blood concentration (Cmax)

    Measured: up to Month 36

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted4 Dec 2025
  2. Study start30 Dec 2025 (estimated)
  3. Primary completion30 Dec 2026 (estimated)
  4. Study completion30 Dec 2028 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07265206
Sponsor's protocol ID
HY001N001
Status last verified
Nov 2025
First submitted
17 Nov 2025
Last updated
4 Dec 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing HY001N cell injection in people with Neurological Autoimmune Diseases, run by Juventas Cell Therapy Ltd..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 35 inclusion and 22 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 4 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants with dose-limiting toxicities (DLTs) (time frame: Up to 28 days after infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07265206.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Dec 2025.

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