Cell Therapy in Limb Girdle Muscular Dystrophy
Primary Purpose
Limb Girdle Muscular Dystrophy
Status
Withdrawn
Phase
Phase 1
Locations
India
Study Type
Interventional
Intervention
Stem Cell
Sponsored by
About this trial
This is an interventional treatment trial for Limb Girdle Muscular Dystrophy focused on measuring Limb Girdle Muscular Dystrophy, Autologous Bone Marrow Mononuclear cell therapy, Stem Cell
Eligibility Criteria
Inclusion Criteria:
- age group of 15 years-60 years
- 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 Cell
Arm Description
Outcomes
Primary Outcome Measures
Change in Functional Independence Measure (FIM) scale
Functional Independence Measure scale assesses the patients ability to carry out activities of daily living. At the follow up of 1 year, every patient in this study will be reevaluated on this scale.
Secondary Outcome Measures
Change in Manual Muscle Testing (MMT)
Manual muscle testing is a procedure for the evaluation of the function and strength of individual muscles and muscle groups based on the effective performance of a movement in relation to the forces of gravity and manual resistance. On the follow up after 1 year, every patient will be reassessed on MMT to record changes in their muscle strength after stem cell therapy.
Full Information
NCT ID
NCT02245711
First Posted
September 12, 2014
Last Updated
October 23, 2018
Sponsor
Neurogen Brain and Spine Institute
1. Study Identification
Unique Protocol Identification Number
NCT02245711
Brief Title
Cell Therapy in Limb Girdle Muscular Dystrophy
Official Title
Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy
Study Type
Interventional
2. Study Status
Record Verification Date
October 2018
Overall Recruitment Status
Withdrawn
Study Start Date
December 2010 (undefined)
Primary Completion Date
January 2015 (Actual)
Study Completion Date
June 2015 (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 Limb Girdle Muscular Dystrophy patients.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Limb Girdle Muscular Dystrophy
Keywords
Limb Girdle Muscular Dystrophy, Autologous Bone Marrow Mononuclear cell therapy, Stem Cell
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 Cell
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
Stem Cell
Primary Outcome Measure Information:
Title
Change in Functional Independence Measure (FIM) scale
Description
Functional Independence Measure scale assesses the patients ability to carry out activities of daily living. At the follow up of 1 year, every patient in this study will be reevaluated on this scale.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Change in Manual Muscle Testing (MMT)
Description
Manual muscle testing is a procedure for the evaluation of the function and strength of individual muscles and muscle groups based on the effective performance of a movement in relation to the forces of gravity and manual resistance. On the follow up after 1 year, every patient will be reassessed on MMT to record changes in their muscle strength after stem cell therapy.
Time Frame
1 year
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:
age group of 15 years-60 years
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
Facility Information:
Facility Name
Neurogen brain and spine institute
City
Navi Mumbai
State/Province
Maharashtra
ZIP/Postal Code
400706
Country
India
12. IPD Sharing Statement
Learn more about this trial
Cell Therapy in Limb Girdle Muscular Dystrophy
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