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

Makati Medical Center

Status and phase: Unknown; Phase 1

Conditions: Multiple Sclerosis

Treatments: Biological: Autologous Hematopoietic Stem Cell; Drug: BEAM Regimen

Study type: Interventional

Funder types: Other

Identifiers

NCT03113162

MMCIRB 2015-024

Details and patient eligibility

About

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.

Enrollment: 15 estimated patients

Sex: All

Ages: 18 to 60 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Autologous Hematopoietic Stem Cell with BEAM Regimen

Experimental group

Description:

Autologous HSCT following Reduced-Intensity BEAM Regimen

Treatment:

Drug: BEAM Regimen

Biological: Autologous Hematopoietic Stem Cell