ClinicalTrials.gov
ClinicalTrials.gov Menu

Allogeneic Human Umbilical Cord Mesenchymal Stem Cells for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02235844
Recruitment Status : Active, not recruiting
First Posted : September 10, 2014
Last Update Posted : January 24, 2017
Sponsor:
Collaborators:
Aidan Foundation
Neil H. Riordan PhD
Information provided by (Responsible Party):
Allergy and Asthma Consultants, Wichita, Kansas

Brief Summary:
This research study is designed to evaluate the effects of human umbilical cord mesenchymal stem cells (UC-MSCs), on Duchenne's muscular dystrophy (DMD). The potential muscle regenerative and anti-inflammatory properties of UC MSCs position them as a possible treatment option for DMD. Both of these properties could lead to potential benefits for a DMD patient.

Condition or disease Intervention/treatment Phase
Duchenne's Muscular Dystrophy Biological: Umbilical Cord Mesenchymal Stem Cells Phase 1

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Allogeneic Transplantation of Human Umbilical Cord Mesenchymal Stem Cells (UC-MSC) for a Single Male Patient With Duchenne Muscular Dystrophy (DMD)
Study Start Date : September 2014
Estimated Primary Completion Date : September 2017
Estimated Study Completion Date : September 2018


Arm Intervention/treatment
Experimental: Mesenchymal Stem Cells
Umbilical Cord Mesenchymal Stem Cells
Biological: Umbilical Cord Mesenchymal Stem Cells



Primary Outcome Measures :
  1. Adverse Events [ Time Frame: 3 months after final treatment ]
    No occurrence of adverse events


Secondary Outcome Measures :
  1. Change from baseline of weight [ Time Frame: 3 months after final treatment ]
  2. Change of muscle diameter (circumferential measurements) from baseline [ Time Frame: 3 months after final treatment ]
  3. Change from baseline of Pulmonary Maximum Expiratory Pressure [ Time Frame: 3 months after final treatment ]
  4. Change from baseline of Pulmonary Forced Vital Capacity [ Time Frame: 3 months after final treatment ]
  5. Maximum Change from baseline of Predicted Inspiratory Pressure % [ Time Frame: 3 months after final treatment ]
  6. Change from baseline of Predicted Maximum Expiratory Pressure % [ Time Frame: 3 months after final treatment ]
  7. Change from baseline of Predicted Forced Vital Capacity % [ Time Frame: 3 months after final treatment ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   28 Years to 31 Years   (Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Duchenne's Muscular Dystrophy

Exclusion Criteria:


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): NCT02235844


Locations
United States, Kansas
Asthma and Allergy Consultants
Wichita, Kansas, United States, 67205
Sponsors and Collaborators
Allergy and Asthma Consultants, Wichita, Kansas
Aidan Foundation
Neil H. Riordan PhD
Investigators
Principal Investigator: Maurice HV Strickland, MD Allergy and Asthma Consultants of Wichita, KS

Responsible Party: Allergy and Asthma Consultants, Wichita, Kansas
ClinicalTrials.gov Identifier: NCT02235844     History of Changes
Other Study ID Numbers: IND 16026 DMD Single Patient
First Posted: September 10, 2014    Key Record Dates
Last Update Posted: January 24, 2017
Last Verified: January 2017

Keywords provided by Allergy and Asthma Consultants, Wichita, Kansas:
Mesenchymal
Stem Cells
Umbilical Cord
Duchenne's
Muscular Dystrophy

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