Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) for Multiple Sclerosis

Study Overview
=================
Brief Summary
-----------------
This is a patient-sponsored study that evaluates the safety and efficacy of reduced-intensity immunoablation followed by a single dose autologous hematopoetic stem cell transplantation in patients diagnosed with multiple sclerosis. Patients are followed-up after 1 month, 3 months, 6 months and 12 months post-transplantation.

Official Title
-----------------
Evaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis

Conditions
-----------------
Multiple Sclerosis

Intervention / Treatment
-----------------
* Biological: Autologous Hematopoietic Stem Cell
* Drug: BEAM Regimen


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Diagnosed with progressive multiple sclerosis with or without relapses EDSS score between 1.5 and 7.0, including documented rapid progression over the previous year unresponsive to conventional therapies or no available treatment options Aged between 18 and 60 with a history of at least one enhancing lesion on brain MRI With absolute neutrophil count ≥ 1,000/mm^3, platelet count ≥ 100,000/mm^3 and hemoglobin ≥ 9.0 g/dL Exclusion Criteria: Patients with cardiac, renal, pulmonary, hepatic, or other organ impairment that would limit their ability to receive dose-intensive immunosuppressive therapy, high-dose chemotherapy, and/or Autologous HSCT Patients with any active or chronic infection e.g. uncontrolled viral, fungal, or bacterial infection Uncontrolled diabetes Patients who are seropositive for HIV1, HIV2, Hepatitis B Surface Antigen, and Hepatitis C Patients whose life expectancy is severely limited by another illness Patients with evidence of myelodysplasia or other non-autoimmune cytopenia Patients having received a cytotoxic agent within one month prior to this study Patients who are pregnant or at risk of pregnancy, including those unwilling to practice Patients with psychiatric illness, mental deficiency, or cognitive dysfunction Patients unable to give written informed consent in accordance with research ethics board guidelines

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 60 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Autologous Hematopoietic Stem Cell with BEAM Regimen<br>Autologous HSCT following Reduced-Intensity BEAM Regimen | Biological: Autologous Hematopoietic Stem Cell<br> <br> Drug: BEAM Regimen<br>* Reduced-intensity BEAM for Immunoablation<br>* Other names: BCNU, Etoposide, Cytarabine, Melphalan;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety: Adverse Events | Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy: EDSS Score | Measurement of disease progression by change in baseline of EDSS score | 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion | 
| Efficacy: RAND-36 Score | Measurement of Quality of Life by change in baseline of RAND-36 score | 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
multiple sclerosis, HSCT