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Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2017 by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Sponsor:
Information provided by (Responsible Party):
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier:
NCT01671865
First received: August 21, 2012
Last updated: March 1, 2017
Last verified: March 2017
  Purpose
The purpose of this research study is to identify and study changes in muscle in people with facioscapulohumeral muscular dystrophy using magnetic resonance imaging and spectroscopy.

Condition
Facioscapulohumeral Muscular Dystrophy

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Magnetic Resonance Imaging and Spectroscopy Biomarkers for Facioscapulohumeral Muscular Dystrophy

Resource links provided by NLM:


Further study details as provided by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:

Primary Outcome Measures:
  • Intramuscular fat infiltration on MRI [ Time Frame: Baseline, 3 months, 9 months, 15 months, and 21 months ]

Secondary Outcome Measures:
  • Intramuscular edema on MRI [ Time Frame: Baseline, 3 months, 9 months, 15 months, and 21 months ]
  • Muscle strength testing [ Time Frame: Baseline, 3 months, 9 months, 15 months, and 21 months ]
  • Timed function testing [ Time Frame: Baseline, 3 months, 9 months, 15 months, and 21 months ]

Estimated Enrollment: 75
Actual Study Start Date: March 2012
Estimated Study Completion Date: June 2020
Estimated Primary Completion Date: June 2020 (Final data collection date for primary outcome measure)
Detailed Description:
This research study is being done to study changes in muscle imaging over time in people with facioscapulohumeral muscular dystrophy (FSHD). Whole-body magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS) will be used to evaluate skeletal muscle in study participants. This research is being done to assess how changes in muscle imaging correspond to muscle strength and function. Qualified participants will be asked to complete 5 study visits over 21 months. Each visit will include muscle strength and function testing in addition to the MRI/MRS scan. The investigators plan to use MRI and MRS in developing outcome measures that can be used in future clinical trials for FSHD.
  Eligibility

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
This study will include people with facioscapulohumeral muscular dystrophy confirmed through genetic testing.
Criteria

Inclusion Criteria:

  • Ability to give written informed consent for study participation (a parent or guardian will be asked to provide informed consent for participants younger than 18 years old)
  • Confirmed diagnosis of FSHD through genetic testing (participants will be asked to provide copies of genetic testing results)

Exclusion Criteria:

  • Any contraindication to MRI scanning
  • Inability to complete a physical examination, including strength measurements
  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: NCT01671865

Contacts
Contact: Doris G Leung, MD, PhD 443-923-9521 leungd@kennedykrieger.org

Locations
United States, Maryland
Doris Leung Recruiting
Baltimore, Maryland, United States, 21205
Contact: Doris G Leung, MD, PhD    443-923-9521    leungd@kennedykrieger.org   
Principal Investigator: Doris G Leung, MD, PhD         
Sponsors and Collaborators
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Investigators
Principal Investigator: Doris G Leung, MD, PhD Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
  More Information

Additional Information:
Publications:
Responsible Party: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
ClinicalTrials.gov Identifier: NCT01671865     History of Changes
Other Study ID Numbers: NA_00065256
Study First Received: August 21, 2012
Last Updated: March 1, 2017
Individual Participant Data  
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.:
FSHD
magnetic resonance imaging
magnetic resonance spectroscopy

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Facioscapulohumeral
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on May 22, 2017