Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

Multiple Sclerosis Clinical Trial

Official title:

Autologous Hematopoietic Stem Cell Transplantation for Refractory Multiple Sclerosis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06228781
• Other study ID #: IRB2023-YX-233-01
• Status: Not yet recruiting
• Phase: N/A
• Start date: December 1, 2024
• Est. completion date: January 1, 2029

Study information

• Verified date: April 2024
• Source: Tianjin Medical University General Hospital
• Contact: Qiang Liu, M.D.,Ph.D
• Phone: +86 15022439149
• Email: qliu[@]tmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 20
• Est. completion date: January 1, 2029
• Est. primary completion date: January 1, 2029
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 60 Years

Eligibility

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.

Related Conditions & MeSH terms

• Multiple Sclerosis
• Sclerosis

Intervention

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 transplant.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Tianjin Medical University General Hospital

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Time to MS treatment failure	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.	3 years
Secondary	Transplant related morbidity	Rate of transplant related events.	3 years
Secondary	Transplant related mortality	Rate of transplant related death.	3 years
Secondary	Immune reconstitution following transplant	Rate of immune reconstitution following transplant.	3 years
Secondary	Hematopoietic reconstitution following transplant	Rate of hematopoietic reconstitution following transplant.	3 years
Secondary	Imaging changes associated with the disease activity	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.	3 years