Stem Cell Therapy in Limb Girdle Muscular Dystrophy


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.

Full Title of Study: “The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: March 2013


  • Biological: Autologous bone marrow mononuclear cell transplantation
    • bone marrow derived mononuclear cells are administered intrathecally and intramuscularly in limb girdle muscular dystrophy patients

Arms, Groups and Cohorts

  • Experimental: Stem Cells
    • autologous bone marrow mononuclear cell transplantation

Clinical Trial Outcome Measures

Primary Measures

  • Functional Independence Measure
    • Time Frame: 6 months

Secondary Measures

  • Manual Muscle Testing
    • Time Frame: 6 months

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 15 Years

Maximum Age: 60 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Neurogen Brain and Spine Institute
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Alok K Sharma, M.S., M.Ch, Principal Investigator, Neurogen Brain and Spine Institute


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.

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.