Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

NCT ID: NCT06228781

Last Updated: 2025-05-30

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: NA
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2026-12-01
• Study Completion Date: 2029-01-01

Brief Summary

Autologous hematopoietic stem cell transplantation (aHSCT) is the only treatment for refractory autoimmune diseases capable of inducing long-term, drug-free and asymptomatic remission. Over the past two decades, aHSCT has been used to treat inflammatory autoimmune disease of the CNS. Patients with relapsing-remitting multiple sclerosis benefit from aHSCT treatment. However, a certain percentage of patients still experience recurrence 3 or 5 years after transplantation. Therefore, exploration of conditioning regimens will drive therapeutic advances in aHSCT in autoimmune diseases of the CNS.

Conditions

Multiple Sclerosis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: BASIC_SCIENCE
• Blinding Strategy: NONE

Study Groups

Hematopoietic Stem Cell Transplantation

Patients will undergo stem cell transplantation for the treatment of refractory MS

Group Type: EXPERIMENTAL

Autologous haemopoietic stem cell transplantation

Intervention Type: PROCEDURE

Immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT).

Stem cell mobilization with cyclophosphamide 2g/m2 and filgrastim 10 ug/kg/d x 5 day.

Stem cell collection with cobe cpectra stem cell purification with Miltenyi CliniMACS Stem cell transplant conditioning with busulphan 3.2 mg/kg ; fludarabine 30mg/m2 or cladribine 10mg ；cytarabine 1-2g/m2 or idarubicin 8mg/m2；cyclophosphamide 40mg/kg followed by CD34 selected autologous hematopoietic stem cell transplant.

Interventions

Autologous haemopoietic stem cell transplantation

Immuno-ablation and autologous CD34 selected hematopoietic stem cell transplantation (HSCT).

Stem cell mobilization with cyclophosphamide 2g/m2 and filgrastim 10 ug/kg/d x 5 day.

Stem cell collection with cobe cpectra stem cell purification with Miltenyi CliniMACS Stem cell transplant conditioning with busulphan 3.2 mg/kg ; fludarabine 30mg/m2 or cladribine 10mg ；cytarabine 1-2g/m2 or idarubicin 8mg/m2；cyclophosphamide 40mg/kg followed by CD34 selected autologous hematopoietic stem cell transplant.

Intervention Type: PROCEDURE

Eligibility Criteria

Inclusion Criteria

1. Age 18-60 years;

2. Diagnosed multiple sclerosis with relapses or progression and sustained accumulated impairment by a neurologist expert in the field;

3. EDSS score of 3-6 (including 3 and 6);

4. EDSS cerebellar functional score ≥ 3 or EDSS pyramidal functional score ≥3；

5. Evidence of current disease activity;

6. If a patient has previously received a cytotoxic agent (mitoxantrone, cyclophosphamide etc.) they must have normal bone marrow morphology and cytogenetics before being considered eligible for this study ;

7. No evidence of hepatic inflammation or fibrosis;

Exclusion Criteria

1. Patients with evidence of myelodysplasia or other non-autoimmune cytopenia;

2. Patients having received a cytotoxic agent within one month of enrolling in this study;

3. Patient with any active or chronic infection (herpes simplex virus, varicella-zoster virus, cytomegalovirus, EB virus, human immunodeficiency virus, hepatitis virus, syphilis, etc.);

4. Patients having received a cytotoxic agent within one month of enrolling in this study;

5. Patients with a malignant tumor currently or within the last 5 years;

6. Patients with cardiac, renal, pulmonary, hepatic or other organ impairment;

7. Patients whose life expectancy is severely limited by another conditions;

8. Pregnancy or risk of pregnancy;

9. Patients unable to give written informed consent in accordance with research ethics board guidelines.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tianjin Medical University General Hospital

OTHER

Sponsor Role: lead

Responsible Party

Qiang Liu

Professor of Department of Neurology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Qiang Liu, M.D.,Ph.D

Role: PRINCIPAL_INVESTIGATOR

Tianjin Medical University General Hospital

Central Contacts

Qiang Liu, M.D.,Ph.D

Role: CONTACT

qliu@tmu.edu.cn

+86 15022439149

Other Identifiers

IRB2023-YX-233-01

Identifier Type: -

Identifier Source: org_study_id