Phase Ib/II Study of SYS6020 in Relapsing/Refractory Multiple Sclerosis

NCT07756268 | Not Yet Recruiting Phase 1

• 1 other identifier: SYS6020-005
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

This trial is an investigator-initiated, single-arm, open-label Phase Ib/II study to observe the safety, tolerability, PK/PD characteristics, immunogenicity, and the efficacy of SYS6020 injection in participants with relapsed/refractory multiple sclerosis. The study plans to enroll participants with progressive or relapsing multiple sclerosis. The recommended dosing regimen is as follows: a single administration dose of 45×10\^6 CAR-T cells/kg (allowing a fluctuation of ±20%), administered once a week for 6 consecutive doses. To ensure participant safety, this study will establish a Safety Monitoring Committee (SMC). A staggered enrollment and dosing strategy will be adopted in the early stage. Two early safety evaluation will be established (after the first 3 enrolled participants complete their first 3 infusions of SYS6020, and after the first 3 enrolled participants complete their first 6 infusions of SYS6020) for comprehensive assessment. The study plans to enroll 10-15 participants. Once the enrollment of 10-15 participants is complete, a comprehensive assessment may be conducted based on the actual progress of the study and combined with existing data. This will fully evaluate the safety, preliminary efficacy, and PK/PD/ADA data of the enrolled participants. If the overall safety of the participants is manageable, preliminary efficacy shows a positive trend, and there are value and necessity for further exploration, expanding the number of participants may be considered (up to a maximum of 25 participants in total).

Conditions

Multiple Sclerosis

Keywords

SYS6020, BCMA, Autologous CAR-T Cell Therapy, mRNA CAR-T, Multiple Sclerosis, Primary Progressive Multiple Sclerosis, Secondary Progressive Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Safety and tolerability

  Incidence, severity, and relationship of adverse events and serious adverse events, and incidence of clinically significant laboratory abnormalities.

  From informed consent through Month 24

Secondary Outcomes (23)

• Time to 12-Week Confirmed Disability Progression (CDP) in Participants with Progressive Multiple Sclerosis

  Month 24

• Annualized Relapse Rate (ARR) in Participants with Relapsing Multiple Sclerosis

  Time Frame: Month 24

• Change from Baseline in EDSS(Expanded Disability Status Scale) Score

  Baseline and Months 1, 3, 6, 9, 12, 18, and 24

• Proportion of Participants with an Improvement of at Least 1.0 Point in EDSS(Expanded Disability Status Scale) Score

  Baseline and Months 1, 3, 6, 9, 12, 18, and 24

• Proportion of Participants Without Confirmed Disability Progression

  Baseline and Months 1, 3, 6, 9, 12, 18, and 24

Study Arms (1)

SYS6020 injection

EXPERIMENTAL

Use this field to provide additional information about the arm, including a description of the intervention(s) administered. Participants will receive a single administration dose of 45×10\^6 CAR-T cells/kg (allowing a fluctuation of ±20%), administered once a week for 6 consecutive doses

Biological: SYS6020 injection

Interventions

SYS6020 injection BIOLOGICAL

SYS6020 injection is an injection of autologous CAR-T cells that have been temporarily transfected with LNP-mRNA targeting BCMA. The eligible participants will receive a single administration dose of 45×10\^6 CAR-T cells/kg (allowing a fluctuation of ±20%), administered once a week for 6 consecutive doses

SYS6020 injection

Eligibility Criteria

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

You may qualify if:

• \. Male or female participants aged 18-65 years at the time of signing informed consent.
• \. Diagnosis of progressive multiple sclerosis (primary progressive MS \[PPMS\] or secondary progressive MS \[SPMS\]) or relapsing multiple sclerosis (RMS) according to the 2024 McDonald criteria.
• \. Inadequate response to at least one disease-modifying therapy (DMT) administered for ≥6 months:
• For progressive MS: evidence of worsening disability, such as an increased Expanded Disability Status Scale (EDSS) score.
• For RMS: at least one of the following:
• i. ≥2 relapses within 2 years before screening; ii. ≥1 relapse within 1 year before screening; or iii. Gadolinium-enhancing lesions on MRI within 1 year before screening. 4. Positive cerebrospinal fluid oligoclonal bands or an elevated immunoglobulin G index, documented previously or during screening.
• \. Typical MS lesions on brain and/or spinal cord MRI, documented previously or during screening.
• \. Screening EDSS score of 3.0-7.0. 7. Adequate baseline organ function, including:
• Absolute lymphocyte count ≥0.3 × 10⁹/L, absolute neutrophil count ≥1.0 × 10⁹/L, platelet count ≥50 × 10⁹/L, and hemoglobin ≥80 g/L, without red blood cell or platelet transfusion or colony-stimulating factor within 7 days before testing;
• Total bilirubin ≤2 × upper limit of normal (ULN), and alanine aminotransferase and aspartate aminotransferase ≤3 × ULN;
• Serum creatinine ≤1.5 × ULN and creatinine clearance ≥40 mL/min by the Cockcroft-Gault formula;
• Activated partial thromboplastin time and international normalized ratio ≤1.5 × ULN;
• Oxygen saturation ≥90% on room air;
• Serum potassium ≥3.0 mmol/L and calcium ≥2.0 mmol/L. 8. Participants of reproductive potential must use reliable contraception during the study and for at least 2 years after the last SYS6020 infusion. Women must not donate oocytes and men must not donate sperm for assisted reproduction during this period. Women of childbearing potential must have a negative serum pregnancy test at screening and a negative serum or urine pregnancy test before leukapheresis.

You may not qualify if:

• \. Uncontrolled significant chronic disease that, in the investigator's opinion, may increase the participant's risk.
• \. Another autoimmune disease requiring systemic treatment, except adequately treated autoimmune thyroid disease with stable treatment and normal thyroid function.
• \. History of primary immunodeficiency, organ transplantation, or hematopoietic stem cell/bone marrow transplantation, or planned transplantation during the study.
• \. Current psychotic disorder. 5. Suicidal ideation within 6 months before informed consent, suicidal behavior within 12 months before informed consent, or a significant suicide risk in the investigator's opinion.
• \. Alcohol or drug abuse/dependence likely to impair study compliance. 7. Stroke, transient ischemic attack, or another active central nervous system disorder unrelated to neuroimmunological disease within 6 months before enrollment.
• \. Significant cardiovascular disease, including:
• Clinically significant ventricular arrhythmia, second- or third-degree atrioventricular block, or another serious rhythm/conduction disorder;
• Resting QT interval corrected using Fridericia's formula \>450 msec for men or \>470 msec for women;
• Acute coronary syndrome, congestive heart failure, or another Grade ≥3 cardiovascular event within 6 months before first administration;
• Left ventricular ejection fraction \<50%;
• Risk factors for QT prolongation or arrhythmia, including heart failure, hypokalemia, congenital long QT syndrome, family history of long QT syndrome, or use of QT-prolonging medications;
• Poorly controlled hypertension, defined as systolic blood pressure ≥160 mmHg and/or diastolic blood pressure ≥100 mmHg.
• \. Major surgery or invasive intervention within 4 weeks before leukapheresis, or planned systemic or local tumor resection during the study.
• \. Grade ≥2 bleeding within 30 days before screening or a need for continuous long-term anticoagulant therapy.
• \. Active malignancy or history of malignancy, except:

Sponsors & Collaborators

• Tongji Hospital: lead
• CSPC Baike (Shandong) Bio-Pharmaceutical Co., Ltd.: collaborator

Study Sites (1)

Tongji Hospital, Tongji Medical College of Hust

Wuhan, Hubei, 430000, China

Location

MeSH Terms

Conditions

Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Daishi Tian, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

• Chuan Qin, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

• Zhouping Tang, MD

  Tongji Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yuhang Cheng

CONTACT

+86-13125197960; chengyh_tj@163.com

Study Design

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

Model Details: This trial is an investigator-initiated, single-arm, open-label Phase Ib/II study. A staggered enrollment and dosing strategy will be adopted in the early stage. Two early safety evaluation will be established (after the first 3 enrolled participants complete their first 3 infusions of SYS6020, and after the first 3 enrolled participants complete their first 6 infusions of SYS6020) for comprehensive assessment.

Study Record Dates

• First Submitted: July 30, 2026
• First Posted: August 10, 2026
• Study Start (Estimated): September 16, 2026
• Primary Completion (Estimated): February 7, 2029
• Study Completion (Estimated): February 7, 2029
• Last Updated: August 10, 2026

Record last verified: 2026-08

Locations

CN (1)