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Trial record 7 of 443 for:    muscular dystrophy

Efficacy of Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2

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ClinicalTrials.gov Identifier: NCT02484560
Recruitment Status : Unknown
Verified June 2015 by Alper Dai, MD, University of Gaziantep.
Recruitment status was:  Active, not recruiting
First Posted : June 29, 2015
Last Update Posted : July 1, 2015
Sponsor:
Collaborator:
Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital
Information provided by (Responsible Party):
Alper Dai, MD, University of Gaziantep

Brief Summary:
Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell Phase 1

Detailed Description:

Duchenne Muscular Dystrophy (DMD) is a X-linked genetic disorder primarily affecting males, resulting in an absence of dystrophin which ultimately leads to progressive muscle degeneration. Patients with DMD progressively lose functional abilities of movement, breath, and eventually the ability to circulate blood. Currently, there is no cure for DMD, although several strategies are being tested for treatment, none have yet proven to be sufficient. Children with DMD are generally divided into two groups based on severity or progression of the disease, non-ambulatory and ambulatory. Ambulatory patients are capable of walking independently while non-ambulatory patients cannot walk independently.

The purpose of this study is to investigate the effects of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children with Duchenne Muscular Dystrophy and determine its suitability as a form of treatment.


Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Allogenic Mesenchymal Stem Cell Therapy in Ambulatory and Non-ambulatory Children With Duchenne Muscular Dystrophy - Phase 1-2
Study Start Date : June 2015
Estimated Primary Completion Date : December 2015
Estimated Study Completion Date : December 2015


Arm Intervention/treatment
Experimental: Ambulatory Patients
Ambulatory patients receiving stem cell therapy
Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell
Experimental: Non-Ambulatory Patients
non-ambulatory patients receiving stem cell therapy
Drug: Biological: Umbilical Cord Based Allogenic Mesenchymal Stem Cell



Primary Outcome Measures :
  1. Degree of improvement in patients with Duchenne Muscular Dystrophy after stem cell therapy treatment administered using Northstar Ambulatory Assessment, Magnetic Resonance Imaging & Spectroscopy, muscle strength assessment equipment, and a questionnaire. [ Time Frame: 12 Months ]

    Tests Used in Assement:

    Northstar ambulatory assessment CHAQ (Child Health Assessment Questionnaire) MRI/MRS Muscle Strength Assessment - Myogrip, Myopinch, and Moviplate




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Ages Eligible for Study:   8 Years to 14 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ambulatory and Non-ambulatory patients diagnosed with DMD that is proven both clinically and genetically and are between 5-20 years old who need partial respiratory support daily. Patients with less than or equal to stage 1 NIH, cardiac, liver, and renal function. Patients must also not present any indication of cancer, allergic disease, nor bleeding diathesis.

Exclusion Criteria:

  • Patients who require full respiratory support. Patients have stage II NIH or greater, cardiac, liver, and renal function. Patients present with signs of symptoms of cancer, allergic disease, or bleeding diathesis.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02484560


Locations
Turkey
Gaziantep University Hospital
Gaziantep, Turkey, 27310
Sponsors and Collaborators
University of Gaziantep
Istınye University, Cukurova University, Yıldırım Beyazıt University, Gaziantep Deva Hospital, Gaziantep Public Hospital

Responsible Party: Alper Dai, MD, Lead Investigator, University of Gaziantep
ClinicalTrials.gov Identifier: NCT02484560     History of Changes
Other Study ID Numbers: 56733164/203
First Posted: June 29, 2015    Key Record Dates
Last Update Posted: July 1, 2015
Last Verified: June 2015

Keywords provided by Alper Dai, MD, University of Gaziantep:
Duchenne Muscular Dystrophy
Neuromuscular Diseases
Muscular Dystrophy
Genetic Diseases, X-linked
Musculoskeletal Diseases
Nervous System Diseases

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked