Cell Therapy in Limb Girdle Muscular Dystrophy


The purpose of this study was to study the effect of stem cell therapy on Limb Girdle Muscular Dystrophy patients.

Full Title of Study: “Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy”

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: January 2015


  • Biological: Stem Cell

Arms, Groups and Cohorts

  • Experimental: Stem Cell

Clinical Trial Outcome Measures

Primary Measures

  • Change in Functional Independence Measure (FIM) scale
    • Time Frame: 1 year
    • 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 Measures

  • Change in Manual Muscle Testing (MMT)
    • Time Frame: 1 year
    • 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.

Participating in This Clinical Trial

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

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

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