Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

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Brief Title

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Official Title

Magnetic Resonance Imaging and Biomarkers for Muscular Dystrophy

Brief Summary

      The purpose of this research study is to determine the potential of magnetic resonance
      imaging, spectroscopy, and whole body imaging to monitor disease progression and to serve as
      an objective outcome measure for clinical trials in Muscular Dystrophy (MD).

      The investigators will compare the muscles of ambulatory or non-ambulatory boys/men with DMD
      with muscles of healthy individuals of the same age and monitor disease progression in those
      with DMD over a 5-10 year period. The amount of muscle damage and fat that the investigators
      measure will also be related to performance in daily activities, such as walking and the loss
      of muscle strength. In a small group of subjects the investigators will also assess the
      effect of corticosteroid drugs on the muscle measurements.

      Additionally, the investigators will map the progression of Becker MD following adults with
      this rare disease. The primary objective is to conduct a multi-centered study to validate the
      potential of non-invasive magnetic resonance imaging and magnetic resonance spectroscopy to
      monitor disease progression and to serve as a noninvasive surrogate outcome measure for
      clinical trials in DMD and BMD. The secondary objective is to characterize the progressive
      involvement of the lower extremity, upper extremity, trunk/respiratory muscles in boys/men
      with DMD and BMD guiding clinical trials.
    

Detailed Description

      The overall objective of this proposal is to validate the potential of noninvasive magnetic
      resonance imaging (MRI) and spectroscopy (MRS) to monitor disease progression and to serve as
      an outcome measure for clinical trials in muscular dystrophies. Duchenne muscular dystrophy
      (DMD) is one of the most devastating genetically linked neuromuscular diseases and is
      characterized by the absence of dystrophin, resulting in progressive muscle weakness, loss of
      walking ability and premature death. Despite the poor prognosis therapeutic interventions
      have been lacking, and outcome measures for clinical trials have been limited to measures of
      muscle function, quality of life, serum biomarkers of muscle breakdown and invasive muscle
      biopsies. Closely related to DMD, Becker muscular dystrophy (BMD) has also been largely
      neglected in therapeutic development, due to its heterogeneity, small patient population,
      lack of outcome measures and uncertainty surrounding the patterns of disease progression,
      which may be mutation-dependent. Additional quantitative outcome measures that are
      noninvasive and sensitive to changes in muscle structure and composition are needed to
      facilitate the rapid translation of promising new interventions from preclinical studies to
      clinical trials in both forms of muscular dystrophy. As such, this project targets the
      development and validation of magnetic resonance as a noninvasive biomarker of disease
      progression in muscular dystrophy. Using a multi-site research design this study will
      characterize the intramuscular lipid content, cellular muscle damage and contractile area in
      the lower and/or upper extremity muscles of 200 ambulatory or non-ambulatory boys/men with
      DMD, 105 ambulatory or non-ambulatory men with BMD, and 110 healthy age matched boys/men
      using a combination of sophisticated MRI and MRS technologies. The trunk and respiratory
      muscles will be characterized in a subgroup of subjects (80 DMD, 20 BMD and 10 controls). In
      order to assess the sensitivity of each MR measure individually as well as composite MR
      measures (combination of muscles) to disease progression, all boys/men with DMD or BMD will
      be re-evaluated in yearly or 6 month intervals. The predictive outcome value of MRI/MRS will
      be further evaluated by determining the relationship between changes in MR measures and loss
      in muscle strength and/or functional ability. Using MRI/MRS we will also examine the effect
      of initiating corticosteroid treatment on skeletal muscle characteristics and composition.
      Examination of muscles in BMD patients will allow us to increase our understanding of how
      much dystrophin is needed to protect the muscle. To this end we will specifically examine the
      relationship between the MR phenotype (e.g. fast or slow increases in fat fraction) and
      dystrophin mutations, dystrophin expression and other histological markers. Finally, to
      ensure the rigor of this study we will examine the day-day reproducibility, inter MR system
      reproducibility and inter-validate the MR measures using localized MRS (golden standard). We
      anticipate that the MR techniques developed and validated in this study will be suitable for
      clinical trials in a wide range of muscular dystrophies and other neuromuscular diseases. In
      addition, MR characterization may serve as a powerful tool to further advance our
      understanding of the pathogenesis of muscular dystrophy and help guide the design of future
      trials.
    


Study Type

Observational


Primary Outcome

Change from baseline in intramuscular lipid up to 3-10 years

Secondary Outcome

 Change from baseline in muscle T2 up to 5-10 years

Condition

Duchenne Muscular Dystrophy


Study Arms / Comparison Groups

 Age Matched Controls
Description:  Age matched non-affected (non-DMD) boys * This arm is full
Age matched non-affected men, matched for men with Becker MD *Recruiting

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information



Estimated Enrollment

550

Start Date

September 1, 2020

Completion Date

August 2025

Primary Completion Date

August 2025

Eligibility Criteria

        Inclusion Criteria for boys with DMD:

        1. Ambulatory and non-ambulatory males (ages 5-30 at baseline testing) previously diagnosed
        with DMD based on:

          -  clinical features with onset of symptoms before age five

          -  elevated serum creatine kinase level or

          -  absence of dystrophin expression, as determined by immunostain or western blot (<2%)
             and/or DNA confirmation of a dystrophin mutation *Subjects will not be excluded based
             on corticosteroid treatment or other clinical trials

        Inclusion Criteria for adults with Becker MD:

          1. Ambulatory males (ages 18-62) without disease or injury to the lower extremities

          2. Specific recruitment of a subset of individuals with deletion mutations in the
             dystrophin gene involving either exon 51 or exon 45.

        Inclusion Criteria for age matched controls for Becker MD subjects:

        1. Ambulatory males (ages 18-62) without disease or injury to the lower and/or upper
        extremities will be eligible to participate in this study

        Exclusion Criteria:

          1. Males with a contraindication to an MR examination

          2. Males with unstable medical problems

          3. Males who are not able to cooperate during testing

          4. Males with a secondary condition that may impact muscle metabolism, muscle function or
             functional ability (i.e. cerebral palsy, endocrine disorders, mitochondrial disease)

          5. Daytime ventilation

          6. Implantable Cardioverter Defibrillator- (ICD) or pace maker

          7. Healthy boys/men who participate in competitive sports specific training in excess of
             8 hours per week
      

Gender

Male

Ages

5 Years - 62 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

William Rooney, PhD, 352-273-6100, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01484678

Organization ID

IRB201700056-N

Secondary IDs

R01AR056973

Responsible Party

Sponsor

Study Sponsor

University of Florida

Collaborators

 Oregon Health and Science University

Study Sponsor

William Rooney, PhD, Principal Investigator, Oregan Health and Science University


Verification Date

July 2021