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Stem Cell Therapy in Limb Girdle Muscular Dystrophy

Primary Purpose

Limb Girdle Muscular Dystrophy

Status
Withdrawn
Phase
Phase 1
Locations
India
Study Type
Interventional
Intervention
Autologous bone marrow mononuclear cell transplantation
Sponsored by
Neurogen Brain and Spine Institute
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Limb Girdle Muscular Dystrophy

Eligibility Criteria

15 Years - 60 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • males and females
  • age group of 15 years and above
  • limb girdle muscular dystrophy diagnosed on the basis of clinical presentation
  • Electromyographic and Nerve Conduction velocity findings

Exclusion Criteria:

  • presence of respiratory distress
  • presence of acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus
  • malignancies
  • acute medical conditions such as respiratory infection, fever, hemoglobin less than 8, bleeding tendency, bone marrow disorder, left ventricular ejection fraction < 30%
  • pregnancy or breastfeeding

Sites / Locations

  • Neurogen brain and spine institute

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Stem Cells

Arm Description

autologous bone marrow mononuclear cell transplantation

Outcomes

Primary Outcome Measures

Functional Independence Measure

Secondary Outcome Measures

Manual Muscle Testing

Full Information

First Posted
January 29, 2014
Last Updated
October 23, 2018
Sponsor
Neurogen Brain and Spine Institute
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1. Study Identification

Unique Protocol Identification Number
NCT02050776
Brief Title
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
Official Title
The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study
Study Type
Interventional

2. Study Status

Record Verification Date
October 2018
Overall Recruitment Status
Withdrawn
Study Start Date
December 2008 (undefined)
Primary Completion Date
March 2013 (Actual)
Study Completion Date
December 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Neurogen Brain and Spine Institute

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study was to study the effect of stem cell therapy on the course of the disease in patients with Limb Girdle Muscular Dystrophy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Limb Girdle Muscular Dystrophy

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Stem Cells
Arm Type
Experimental
Arm Description
autologous bone marrow mononuclear cell transplantation
Intervention Type
Biological
Intervention Name(s)
Autologous bone marrow mononuclear cell transplantation
Intervention Description
bone marrow derived mononuclear cells are administered intrathecally and intramuscularly in limb girdle muscular dystrophy patients
Primary Outcome Measure Information:
Title
Functional Independence Measure
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Manual Muscle Testing
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
15 Years
Maximum Age & Unit of Time
60 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: males and females age group of 15 years and above limb girdle muscular dystrophy diagnosed on the basis of clinical presentation Electromyographic and Nerve Conduction velocity findings Exclusion Criteria: presence of respiratory distress presence of acute infections such as Human Immunodeficient Virus/Hepatitis B Virus/Hepatitis C Virus malignancies acute medical conditions such as respiratory infection, fever, hemoglobin less than 8, bleeding tendency, bone marrow disorder, left ventricular ejection fraction < 30% pregnancy or breastfeeding
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Alok K Sharma, M.S., M.Ch
Organizational Affiliation
Neurogen Brain and Spine Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
Neurogen brain and spine institute
City
Mumbai
State/Province
Maharashtra
ZIP/Postal Code
400071
Country
India

12. IPD Sharing Statement

Citations:
PubMed Identifier
24070109
Citation
Sharma A, Sane H, Badhe P, Gokulchandran N, Kulkarni P, Lohiya M, Biju H, Jacob VC. A clinical study shows safety and efficacy of autologous bone marrow mononuclear cell therapy to improve quality of life in muscular dystrophy patients. Cell Transplant. 2013;22 Suppl 1:S127-38. doi: 10.3727/096368913X672136. Epub 2013 Sep 10.
Results Reference
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Stem Cell Therapy in Limb Girdle Muscular Dystrophy

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