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

Primary Purpose

Multiple Sclerosis

Status

Unknown status

Phase

Phase 1

Locations

Philippines

Study Type

Interventional

Intervention

Autologous Hematopoietic Stem Cell

BEAM Regimen

About this trial

This is an interventional treatment trial for Multiple Sclerosis focused on measuring multiple sclerosis, HSCT

Eligibility Criteria

18 Years - 60 Years (Adult)All SexesDoes not accept 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

Sites / Locations

Makati Medical CenterRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Autologous Hematopoietic Stem Cell with BEAM Regimen

Arm Description

Autologous HSCT following Reduced-Intensity BEAM Regimen

Outcomes

Primary Outcome Measures

Safety: Adverse Events

Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities

Secondary Outcome Measures

Efficacy: EDSS Score

Measurement of disease progression by change in baseline of EDSS score

Efficacy: RAND-36 Score

Measurement of Quality of Life by change in baseline of RAND-36 score

Full Information

NCT ID

NCT03113162

First Posted

April 10, 2017

Last Updated

May 3, 2017

Sponsor

Makati Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT03113162

Brief Title

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

2. Study Status

Record Verification Date

May 2017

Overall Recruitment Status

Unknown status

Study Start Date

May 29, 2015 (Actual)

Primary Completion Date

May 29, 2020 (Anticipated)

Study Completion Date

May 29, 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Makati Medical Center

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Multiple Sclerosis

Keywords

multiple sclerosis, HSCT

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

15 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Autologous Hematopoietic Stem Cell with BEAM Regimen

Arm Type

Experimental

Arm Description

Autologous HSCT following Reduced-Intensity BEAM Regimen

Intervention Type

Biological

Intervention Name(s)

Autologous Hematopoietic Stem Cell

Intervention Type

Drug

Intervention Name(s)

BEAM Regimen

Other Intervention Name(s)

BCNU, Etoposide, Cytarabine, Melphalan

Intervention Description

Reduced-intensity BEAM for Immunoablation

Primary Outcome Measure Information:

Title

Safety: Adverse Events

Description

Type, occurence, severity, timing, seriousness and relatedness of adverse events and laboratory abnormalities

Time Frame

12 months

Secondary Outcome Measure Information:

Title

Efficacy: EDSS Score

Description

Measurement of disease progression by change in baseline of EDSS score

Time Frame

1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion

Title

Efficacy: RAND-36 Score

Description

Measurement of Quality of Life by change in baseline of RAND-36 score

Time Frame

1 month post-infusion, 3 months month post-infusion, 6 months month post-infusion, 12 months month post-infusion

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Marviel T Berboso, RN

Phone

8888999

Ext

3613

Inquiry.CTC@makatimed.net.ph

First Name & Middle Initial & Last Name or Official Title & Degree

Jose Maria C Avila, MD

Phone

8888999

Ext

3614

stemcell@makatimed.net.ph

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Darwin Albert A Dasig, MD

Organizational Affiliation

Makati Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Makati Medical Center

City

Makati

ZIP/Postal Code

1229

Country

Philippines

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Marviel T Berboso, RN

Phone

8888999

Ext

3613

Inquiry.CTC@makatimed.net.ph

12. IPD Sharing Statement

Plan to Share IPD

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

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