Clinical trial NCT06228781: Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis
Official title: Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: NA
Conditions: Multiple Sclerosis
Lead sponsor: Tianjin Medical University General Hospital
Sponsor class: OTHER
First posted: 2024-01-29
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: BASIC_SCIENCE

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.


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.

Primary outcomes:
measure: 3 year MS activity free survival
timeFrame: 3 year follow-up post transplant
Secondary outcomes:
measure: Time to MS treatment failure
timeFrame: 3 years
measure: Transplant related morbidity
timeFrame: 3 years
measure: Transplant related mortality
timeFrame: 3 years
measure: Immune reconstitution following transplant
timeFrame: 3 years
measure: Hematopoietic reconstitution following transplant
timeFrame: 3 years
measure: Imaging changes associated with the disease activity
timeFrame: 3 years
Other outcomes:
Locations:
Study officials:
affiliation: Tianjin Medical University General Hospital
name: Qiang Liu, M.D.,Ph.D
role: PRINCIPAL_INVESTIGATOR
References: