This is an Open-Label Study to Assess the Safety and Efficacy of GC012F in Patients With Multiple Sclerosis

NCT07364487 | Withdrawn Early Phase 1

• 1 other identifier: KY2025-1011/D831DN00001
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

This is an open-label,early-stage exploratory clinical study to evaluate the safety and preliminary efficacy of GC012F CAR T cell injection in Multiple Sclerosis subjects.

Conditions

Multiple Sclerosis

Keywords

Multiple Sclerosis; CAR-T cell therapy

Outcome Measures

Primary Outcomes (3)

• Dose-Limiting Toxicity (DLT) Rate

  DLT is defined as an AE that occurs within 28 days of GC012F CAR-T product reinfusion.DLT will be evaluated according to NCI-CTCAE V5.0 criteria

  28 days

• Adverse Events （AEs）Rate

  Proportion of subjects experiencing AE within 15 years after infusion of GC012F CAR-T cell injection.

  Up to 15 years from treatment discontinuation

• Iimmunoglobulins (Ig) levels in peripheral blood

  Observe blood the highest Quantification of the immunoglobulins (Ig)

  Up to 15 years from treatment discontinuation

Secondary Outcomes (19)

• Peak blood and CSF concentration(Cmax)(Pharmacokinetic evaluation indicators)

  Up to 36 months from treatment discontinuation

• GC012F CAR gene copy number in peripheral blood and cerebrospinal fluid (CSF)(Pharmacodynamic evaluation indicators,)

  Up to 36 months from treatment discontinuation

• Changes in the concentration of soluble B-cell maturation antigen (BCMA) in peripheral blood

  Up to 36 months from treatment discontinuation

• Levels of Interleukins (IL-2, IL-6, IL-8, IL-10)

  Up to 84 days from treatment discontinuation

• Levels of Interferons-γ (IFN-γ)

  Up to 84 days from treatment discontinuation

Study Arms (1)

GC012F CAR-T Cell Injection

EXPERIMENTAL

GC012F CAR-T Cell Injection Arm

Drug: GC012F CAR-T Cell Injection

Interventions

GC012F CAR-T Cell Injection DRUG

A single dose group is planned for the CAR-T cell infusion dose is administrated for each subject.Single IV infusion.

GC012F CAR-T Cell Injection

Eligibility Criteria

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

You may qualify if:

• Subjects or their legal representatives voluntarily sign a written informed consent form and are willing and able to comply with the procedures of this study;
• Age 18 to 75 years (inclusive) at the time of signing the ICF, regardless of gender;
• Women of childbearing potential must (women who have had a hysterectomy or have been postmenopausal for at least 2 years are not considered to be of potential childbearing potential):
• a) At screening, the result of serum β human chorionic gonadotropin pregnancy test is negative;
• b) Agree to refrain from breastfeeding for the duration of study participation until at least 2 years after GC012F injection infusion or until 2 consecutive flow cytometry tests show no more CAR-T cells (whichever occurs later);
• \. Male subjects with sexual partners and female subjects of potential childbearing potential agree to use highly effective contraceptive methods (e.g.: birth control pills, intrauterine devices, or condoms) from screening until then GC012F injection is no longer present for at least 2 years after infusion or until 2 consecutive flow cytometry tests show no more In CAR-T cells (whichever occurs later). Male subjects must agree to use a condom during sexual contact with a pregnant woman or a woman of childbearing potential for at least 2 years after GC012F injection infusion, even after a successful vasectomy;
• \. The venous access required for collection can be established, and there are no contraindications to leukocyte collection;
• The laboratory test results at screening must meet the following criteria:
• Organ and bone marrow function:
• a)Absolute neutrophil count ≥ 1.0 × 10\^9/L (no growth factor is given for supportive care within 7 days prior to testing);
• b)Absolute lymphocyte count ≥ 1.0×10\^9/L;
• c)Hemoglobin ≥ 80 g/L (no red blood cell transfusion is given within 7 days prior to testing);
• d)Platelet count ≥ 50×10\^9/L (no blood transfusion is given within 7 days prior to testing);
• e)Serum IgG ≥ 500 mg/dL;
• f)Activated partial thromboplastin time ≤ 1.5 × upper limit of normal (ULN), prothrombin time (PT) ≤ 1.5 × ULN;

You may not qualify if:

• Have received any investigational drug treatment within 3 months prior to screening or within 5 half-lives (whichever is longer);
• Fungal, bacterial, viral, or other infection not controlled and/or requiring hospitalization or intravenous antimicrobial therapy within 4 weeks prior to screening. Uncomplicated urinary tract infection and uncomplicated bacterial pharyngitis are permitted if responding to current therapy;
• Active tuberculosis or latent tuberculosis that has not been treated appropriately prior to screening;
• History of severe hypersensitivity or allergy;
• Primary immunodeficiency;
• Impaired cardiac function or clinically significant cardiac disease;
• History of serious respiratory diseases or current serious respiratory diseases, including moderate or severe or above asthma or chronic obstructive pulmonary disease, interstitial lung disease, or pulmonary fibrosis;
• History of severe respiratory disease or current severe respiratory disease;
• Current or history of cirrhosis;
• History of Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus within the past 2 years, and the need for continuous use of systemic immunosuppressants/systemic disease-modifying drugs;
• Any active malignancy or history of malignancy within 5 years prior to screening. The following are exceptions: early-stage tumors that have undergone radical treatment (carcinoma in situ or stage I tumors, non-ulcerative primary melanoma with a depth \< 1 mm and no lymph node involvement), basal cell carcinoma of the skin, squamous cell carcinoma of the skin, thyroid carcinoma in situ or early-stage thyroid cancer that has undergone radical treatment, cervical carcinoma in situ, or breast cancer in situ that has undergone potentially radical treatment;
• Those who have clinically significant bleeding symptoms or definite haemorrhagic diathesis within 6 months prior to screening;
• Arterial or venous thrombotic events such as cerebrovascular disorders (including cerebral hemorrhage, cerebral infarction, etc), deep venous thrombosis, and/or pulmonary embolism within 6 months prior to screening;
• Hematologic disorders: History of cytopenia consistent with myelodysplastic syndrome; history of sickle-cell anemia or other hemoglobinopathies;
• Severe underlying medical conditions, such as:

Sponsors & Collaborators

• Huashan Hospital: lead

Study Sites (1)

Huashan Hospital Affiliated to Fudan University

Shanghai, Shanghai Municipality, 200040, China

Location

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: In this study, a single dose group is planned for the CAR-T cell infusion dose.Among them, 6 subjects will be enrolled in the DLT observation phase, and 9 subjects will be enrolled in the expansion phase.

Study Record Dates

• First Submitted: January 13, 2026
• First Posted: January 23, 2026
• Study Start: January 26, 2026
• Primary Completion: March 25, 2026
• Study Completion: March 25, 2026
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)