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Withdrawn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
About this trial
This is an interventional treatment trial for Limb Girdle Muscular Dystrophy
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
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
NCT ID
NCT02050776
First Posted
January 29, 2014
Last Updated
October 23, 2018
Sponsor
Neurogen Brain and Spine Institute
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Withdrawn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
background
Learn more about this trial
Stem Cell Therapy in Limb Girdle Muscular Dystrophy
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