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Not yet recruiting NCT06228781

Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

No phase Interventional Multiple Sclerosis

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Autologous haemopoietic stem cell transplantation.
Who it may be relevant to
Registry conditions: Multiple Sclerosis. Basic parameters: 18 years — 60 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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.

Interventions

  • Procedure 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 transp

Primary outcome measures

  • 3 year MS activity free survival [Time frame: 3 year follow-up post transplant]
Secondary outcome measures (6)
  • Time to MS treatment failure [Time frame: 3 years]
  • Transplant related morbidity [Time frame: 3 years]
  • Transplant related mortality [Time frame: 3 years]
  • Immune reconstitution following transplant [Time frame: 3 years]
  • Hematopoietic reconstitution following transplant [Time frame: 3 years]
  • Imaging changes associated with the disease activity [Time frame: 3 years]

Eligibility criteria

Inclusion criteria

  • 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;

Exclusion criteria

  • 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Basic science

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT06228781 · IRB2023-YX-233-01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗