deltatrials
Completed NA INTERVENTIONAL 1-arm NCT01594853

Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy

Exercise Study of Function and Pathology for Women With X-ALD

Sponsor: European Leukodystrophy Association

Interventions exercise training
Updated 6 times since 2017 Last updated: Dec 14, 2017 Started: May 31, 2012 Primary completion: Oct 31, 2014 Completion: Oct 31, 2014
This information is for research purposes only and is not medical advice. Consult a healthcare provider before making any medical decision.

Listed as NCT01594853, this NA trial focuses on X-linked Adrenoleukodystrophy and remains completed. Sponsored by European Leukodystrophy Association, it has been updated 6 times since 2012, reflecting limited change activity. This study adds to the evidence base for this therapeutic area through structured, versioned documentation.

Study Description(click to expand)

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...

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.

Status Flow

~Jan 2017 – ~Jun 2018 · 17 months · monthly snapshotCompleted~Jun 2018 – ~Sep 2018 · 3 months · monthly snapshotCompleted~Sep 2018 – ~Jan 2021 · 28 months · monthly snapshotCompleted~Jan 2021 – ~Jul 2024 · 42 months · monthly snapshotCompleted~Jul 2024 – ~Sep 2024 · 2 months · monthly snapshotCompleted~Sep 2024 – present · 19 months · monthly snapshotCompleted

Change History

6 versions recorded
  1. Sep 2024 — Present [monthly]

    Completed NA

  2. Jul 2024 — Sep 2024 [monthly]

    Completed NA

  3. Jan 2021 — Jul 2024 [monthly]

    Completed NA

  4. Sep 2018 — Jan 2021 [monthly]

    Completed NA

  5. Jun 2018 — Sep 2018 [monthly]

    Completed NA

Show 1 earlier version
  1. Jan 2017 — Jun 2018 [monthly]

    Completed NA

    First recorded

May 2012

Trial started

Per CT.gov start date — pre-dates our first snapshot

Eligibility Summary

No eligibility information available.

Contact Information

Sponsor contact:
  • European Leukodystrophy Association
  • Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Data source: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

For direct contact, visit the study record on ClinicalTrials.gov .

Study Locations