Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
MeSH terms: Adrenoleukodystrophy
Study type: Interventional

Summary: 
      The purpose is to see how X-linked adrenoleukodystrophy (X-ALD) is associated with strength
      and sensation using MRI, in women with X-ALD. The investigators will also see whether
      exercise can improve these symptoms for women with X-ALD.
    

Detailed description: 
      X-linked adrenoleukodystrophy (X-ALD), a [sex-linked] progressive neurodegenerative disease,
      is caused by a defect in the ABCD1 gene. The disease is expressed in multiple ways, but the
      most common adult form is adrenomyeloneuropathy (AMN), which results in slowly progressive
      changes in muscle tone and weakness, sensory loss, and dysfunction of the autonomic nervous
      system. In a previous study the investigators linked abnormalities in the [brain/spinal cord]
      to lower extremity weakness in men with AMN; however, there have been no studies evaluating
      these relationships in women carriers (i.e., women with AMN). It is unknown, in women with
      AMN, how the pattern of damage in the brain and spinal cord relates to disability and if
      these patterns predict responsiveness to treatment. The investigators hypothesize that by
      using magnetization transfer (MT) and diffusion tensor imaging (DTI), two magnetic resonance
      imaging (MRI) modalities, to track particular changes in the brain and spinal cord will
      predict disability and additionally, who is likely to respond best to a training regimen. The
      investigators expect that these more advanced imaging techniques will be more sensitive and
      accurate quantitative measures of clinical motor function and women with greater loss in the
      spinal cord compared to the brain will benefit most from training to improve disability. To
      test this hypothesis, women with AMN will receive MRI scans at baseline and complete measures
      of global walking and lower extremity impairments of vibration sensation, spasticity, and
      strength at three time-points: baseline, 12 weeks, and 18 weeks after baseline. The group
      will participate in a resistive training program for 12 weeks. MRI data will be correlated to
      changes over time in measures of impairment to determine their relationships. The linking of
      this information will not only be important for better defining disability in women with AMN
      but it will also help to guide physicians and rehabilitation therapists in predicting who is
      likely to respond to rehabilitative interventions, as well as for optimizing the effects of
      future pharmacological interventions.
    

Intervention name(s): exercise training
Intervention type(s): Behavioral
Intervention description(s): The exercise program uses a local Curves® gym to provide an individualized program of strength and cardiovascular training. The program is expected to be performed for 45 minutes 4-6 days per week.
Other intervention name(s): strengthening; Curves
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: exercise; women; training; rehabilitation; leukodystrophy