Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

Not Applicable

Not yet recruiting

• Conditions: Multiple Sclerosis
• Interventions: Procedure: Autologous haemopoietic stem cell transplantation

• Registration Number: NCT06228781
• Lead Sponsor: Tianjin Medical University General Hospital

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-01-19
• Study First Submitted QC: 2024-01-19
• Study First Posted: 2024-01-29
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-09
• Last Update Posted: 2024-04-10
• Study Start: 2024-12-01
• Primary Completion: 2029-01-01
• Study Completion: 2029-01-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 20

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;

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hematopoietic Stem Cell Transplantation	Autologous haemopoietic stem cell transplantation	Patients will undergo stem cell transplantation for the treatment of refractory MS

Primary Outcome Measures

Name	Time	Method
3 year MS activity free survival	3 year follow-up post transplant	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.

Secondary Outcome Measures

Name	Time	Method
Time to MS treatment failure	3 years	Disease activity and disability will be assessed with clinical relapse, appearance of a new or Gd-enhancing lesion on MRI, or sustained progression of EDSS score and quality of life.
Transplant related morbidity	3 years	Rate of transplant related events.
Transplant related mortality	3 years	Rate of transplant related death.
Immune reconstitution following transplant	3 years	Rate of immune reconstitution following transplant.
Hematopoietic reconstitution following transplant	3 years	Rate of hematopoietic reconstitution following transplant.
Imaging changes associated with the disease activity	3 years	Imaging changes include: new or enlarging T2-weighted lesion count and new T1-weighted lesion count at all scans after baseline; T2-weighted lesion volume; Gd-enhanced lesion count and volume; and total volume of non-enhancing T1-weighted lesions on all MRI scans.