Clinical trial NCT03113162: Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) for Multiple Sclerosis
Official title: Evaluation of the Safety and Efficacy of Reduced-intensity Immunoablation and Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Multiple Sclerosis
Keywords: multiple sclerosis
HSCT
Lead sponsor: Makati Medical Center
Sponsor class: OTHER
First posted: 2017-04-13
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 60
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Safety: Adverse Events
timeFrame: 12 months
Secondary outcomes:
measure: Efficacy: EDSS Score
timeFrame: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
measure: Efficacy: RAND-36 Score
timeFrame: 1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion
Other outcomes:
Locations:
city: Makati
country: Philippines
facility: Makati Medical Center
Study officials:
affiliation: Makati Medical Center
name: Darwin Albert A Dasig, MD
role: PRINCIPAL_INVESTIGATOR
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