Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

NCT06228781 | Not Yet Recruiting

• 1 other identifier: IRB2023-YX-233-01
• Study Type: interventional
• Target: 20
• Locations: 0 countries
• Sites: N/A

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

Outcome Measures

Primary Outcomes (1)

• 3 year MS activity free survival

  The events for the primary outcome are: clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, or sustained progression of EDSS score.

  3 year follow-up post transplant

Secondary Outcomes (6)

• Time to MS treatment failure

  3 years

• Transplant related morbidity

  3 years

• Transplant related mortality

  3 years

• Immune reconstitution following transplant

  3 years

• Hematopoietic reconstitution following transplant

  3 years

Study Arms (1)

Hematopoietic Stem Cell Transplantation

EXPERIMENTAL

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

Procedure: Autologous haemopoietic stem cell transplantation

Interventions

Autologous haemopoietic stem cell transplantation 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.

Hematopoietic Stem Cell Transplantation

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Age 18-60 years;
• Diagnosed multiple sclerosis with relapses or progression and sustained accumulated impairment by a neurologist expert in the field;
• EDSS score of 3-6 (including 3 and 6);
• EDSS cerebellar functional score ≥ 3 or EDSS pyramidal functional score ≥3；
• Evidence of current disease activity;
• 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 ;
• No evidence of hepatic inflammation or fibrosis;

You may not qualify if:

• Patients with evidence of myelodysplasia or other non-autoimmune cytopenia;
• Patients having received a cytotoxic agent within one month of enrolling in this study;
• Patient with any active or chronic infection (herpes simplex virus, varicella-zoster virus, cytomegalovirus, EB virus, human immunodeficiency virus, hepatitis virus, syphilis, etc.);
• Patients having received a cytotoxic agent within one month of enrolling in this study;
• Patients with a malignant tumor currently or within the last 5 years;
• Patients with cardiac, renal, pulmonary, hepatic or other organ impairment;
• Patients whose life expectancy is severely limited by another conditions;
• Pregnancy or risk of pregnancy;
• Patients unable to give written informed consent in accordance with research ethics board guidelines.

Sponsors & Collaborators

• Tianjin Medical University General Hospital: lead

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 Officials

• Qiang Liu, M.D.,Ph.D

  Tianjin Medical University General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Qiang Liu, M.D.,Ph.D

CONTACT

+86 15022439149; qliu@tmu.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Department of Neurology

Study Record Dates

• First Submitted: January 19, 2024
• First Posted: January 29, 2024
• Study Start (Estimated): December 1, 2026
• Primary Completion (Estimated): January 1, 2029
• Study Completion (Estimated): January 1, 2029
• Last Updated: May 30, 2025

Record last verified: 2025-05