A Prospective Natural History Study of Progression of Subjects With Duchenne Muscular Dystrophy.

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Prosensa Therapeutics
ClinicalTrials.gov Identifier:
NCT01753804
First received: December 13, 2012
Last updated: September 24, 2014
Last verified: September 2014
  Purpose

To characterize the natural history and progression of Duchenne Muscular Dystrophy (DMD) to help inform the design of future studies, to capture biomarkers of safety and disease progression and to provide comparative data for the development of rare exons for which formal controlled trials are not feasible.


Condition Intervention
Duchenne Muscular Dystrophy
Other: Observational study

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: A Prospective Natural History Study of Progression of Physical Impairment, Activity Limitation and Quality of Life in Duchenne Muscular Dystrophy.

Resource links provided by NLM:


Further study details as provided by Prosensa Therapeutics:

Primary Outcome Measures:
  • 6 minute walk distance [ Time Frame: Change from visit 1 walking distance ] [ Designated as safety issue: No ]
    Participants are asked to walk at their own preferred speed on a fixed distance for 6 minutes. Subjects are warned of the time and that they may stop earlier if they feel unable to continue. Total distance walked within 6 minutes (or until stopping) is recorded.


Biospecimen Retention:   Samples Without DNA

Blood sampling at 4 time points (first visit then once a year). Urinalysis sampling at 4 time points (first visit then once a year).


Estimated Enrollment: 250
Study Start Date: September 2012
Estimated Study Completion Date: December 2017
Estimated Primary Completion Date: December 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Study participants
All participants will follow the same protocol, including muscle strength and function testing, and blood and urine collection, for a maximum of 7 visits over 3 years.
Other: Observational study
There is no medication or device tested in this study. This is an obversational study on the progression of the disease.

Detailed Description:

This is a prospective study. All DMD patients that fulfil the inclusion/exclusion criteria are eligible although the study is weighted towards ambulant subjects aged 3 years or older. There will be 7 study visits and subjects will be in the study for a maximum of 3 years. Visits will occur every 6 months (+/- 1 month).

Up to 250 DMD subjects planned in the following categories :

  • 75 % ambulant subjects aged between 3 and 18 years at study entry
  • 25% non-ambulant subjects with a maximum age of 18 years at study entry

Subjects will be asked to perform muscle testing assessment with a clinical evaluator, such as walking for 6 minutes, climb stairs, breathe in a tube, see how they can move their arms and legs. They will be asked questions about how they feel overall and perform daily activities. These measurements will be assessed every 6 months.

Urine and blood samples will be collected once a year to measure biomarkers that will allow to have a better overview of DMD.

  Eligibility

Ages Eligible for Study:   3 Years to 18 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population

Subjects diagnosed with DMD resulting from a mutation in the DMD gene which is confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons

Criteria

Inclusion Criteria:

  • Diagnosis of DMD resulting from a mutation in the DMD gene confirmed by a state of the art DNA diagnostic technique covering all DMD gene exons.
  • Age 3 - 18 years
  • Willing and able to comply with protocol requirements
  • Life expectancy of at least 3 years
  • Able to give informed assent and/or consent in writing signed by the subject and/or parent(s)/legal guardian (according to local regulations)

Exclusion Criteria:

  • Current participation in a clinical study with an Investigational Medicinal Product (IMP)
  • Participation within the previous 1 month in a clinical study with an IMP
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01753804

Locations
United States, California
UC Davis Health System
Sacramento, California, United States, 95817
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Argentina
Hospital de Pediatria Prof Dr Juan P Garrahan
Buenos Aires, Argentina
Belgium
Universitair Ziekenhuis
Gent, Belgium
Universitair Ziekenhuis Leuven
Leuven, Belgium
Brazil
Hospital das Clinicas da Faculdade de Medicina da USP
Sao Paulo, Brazil
France
CHU Hopital des enfants
Toulouse, France
Germany
Universitaetsklinikum Essen
Essen, Germany
Universitaetsklinikum Freiburg
Freiburg, Germany
Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, Italy
Policlinico Univsersitario Agostino Gemelli
Rome, Italy
Netherlands
Leids Universitair Medisch Centrum
Leiden, Netherlands
UMC St. Radboud
Nijmegen, Netherlands
Sweden
Drottning Silvias Barn- ochungdomssjukhus
Goteborg, Sweden
Turkey
Hacettepe University Medical Faculty
Ankara, Turkey
Sponsors and Collaborators
Prosensa Therapeutics
Investigators
Principal Investigator: Nathalie Goemans, MD UZ Leuven, Belgium
  More Information

Additional Information:
No publications provided

Responsible Party: Prosensa Therapeutics
ClinicalTrials.gov Identifier: NCT01753804     History of Changes
Other Study ID Numbers: PRO-DMD-01
Study First Received: December 13, 2012
Last Updated: September 24, 2014
Health Authority: Belgium: Ethics Committee

Keywords provided by Prosensa Therapeutics:
DMD
Myopathy
Natural History
Muscular dystrophy
Biomarkers
Muscle testing

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

ClinicalTrials.gov processed this record on October 01, 2014