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

Phase 1

• Conditions: Multiple Sclerosis
• Interventions: Biological: Autologous Hematopoietic Stem Cell; Drug: BEAM Regimen

• Registration Number: NCT03113162
• Lead Sponsor: Makati Medical Center

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2015-05-29
• Study First Submitted: 2017-04-10
• Study First Submitted QC: 2017-04-10
• Study First Posted: 2017-04-13
• Status Verified: 2017-05
• Last Update Submitted: 2017-05-03
• Last Update Posted: 2017-05-05
• Primary Completion: 2020-05-29
• Study Completion: 2022-05-29

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 15

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Autologous Hematopoietic Stem Cell with BEAM Regimen	BEAM Regimen	Autologous HSCT following Reduced-Intensity BEAM Regimen
Autologous Hematopoietic Stem Cell with BEAM Regimen	Autologous Hematopoietic Stem Cell	Autologous HSCT following Reduced-Intensity BEAM Regimen

Primary Outcome Measures

Name	Time	Method
Safety: Adverse Events	12 months	Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities

Secondary Outcome Measures

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

Trial Locations

Locations (1)

Makati Medical Center; 🇵🇭 Makati, Philippines