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NCT Number: NCT06228781

Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

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.

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Key information

Conditions

Multiple Sclerosis Autoimmune Diseases Autoimmune Diseases of the Nervous System Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Immune System Diseases Nervous System Diseases

Age range

18 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

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.

Primary outcomes

  1. 3 year MS activity free survival

    Time frame: 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 outcomes

  1. Time to MS treatment failure

    Time frame: 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.

  2. Transplant related morbidity

    Time frame: 3 years

    Rate of transplant related events.

  3. Transplant related mortality

    Time frame: 3 years

    Rate of transplant related death.

  4. Immune reconstitution following transplant

    Time frame: 3 years

    Rate of immune reconstitution following transplant.

  5. Hematopoietic reconstitution following transplant

    Time frame: 3 years

    Rate of hematopoietic reconstitution following transplant.

  6. Imaging changes associated with the disease activity

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Qiang Liu, M.D.,Ph.D

CONTACT

[email protected]

+86 15022439149

Sponsors and collaborators

Lead sponsor

Tianjin Medical University General Hospital

Other

Registry information

Important dates

Study start
2026
Primary completion
2029
Study completion
2029
First posted
Jan 29, 2024
Registry last updated
May 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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