- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01594853
Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
December 14, 2017 updated by: Kathleen Zackowski, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Exercise Study of Function and Pathology for Women With X-ALD
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
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.
Study Type
Interventional
Enrollment (Actual)
31
Phase
- Not Applicable
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Maryland
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Baltimore, Maryland, United States, 21205
- Motion Analysis Lab
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
19 years to 68 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Female
Description
Inclusion Criteria:
- confirmed diagnosis, X-ALD heterozygote carrier
- no medical contraindication to participating in a strength training program
- able to follow complex directions as determined by a score of ≤1 on a subset of questions taken from the NIH Stroke scale (Brott et al. 1989)
- hip flexion strength: 6.6-15.8kg
- hip extension strength: up to 18.3 kg
- normal passive range of motion at hips/knees/ankles
- able to walk ≥50m
Exclusion Criteria:
- Evidence of other neurological deficit that could interfere, such as previous stroke or muscle disease
- congestive heart failure
- cancer
- orthopedic conditions
- severe pain that precludes study participation
- seizures
- pregnancy
- other medical condition that precludes participation in an exercise program, e.g., unstable angina, uncontrolled diabetes, uncontrolled hypertension
Healthy controls have the same age and exclusion criteria as women with AMN except that they will not be carriers for X-ALD. They must have normal neurological function.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: exercise
Female carriers as well as healthy age and gender matched individuals will participate in an exercise paradigm.
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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 Names:
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training
Time Frame: Participants are assessed at baseline (visit 1, enrollment), end of training (visit 2, week 12) and post-training (visit 3, week 18).
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Here, we will assess whether the degree of a person's white matter integrity (i.e. for tracts of interest as measured from MRI) predicts their ability to benefit from exercise.
Our prediction is that those individuals with preserved white matter integrity will show the greatest improvement in hip flexor strength.
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Participants are assessed at baseline (visit 1, enrollment), end of training (visit 2, week 12) and post-training (visit 3, week 18).
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Collaborators
Investigators
- Principal Investigator: Kathleen M Zackowski, Ph.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Helpful Links
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
May 1, 2012
Primary Completion (Actual)
October 1, 2014
Study Completion (Actual)
October 1, 2014
Study Registration Dates
First Submitted
May 7, 2012
First Submitted That Met QC Criteria
May 8, 2012
First Posted (Estimate)
May 9, 2012
Study Record Updates
Last Update Posted (Actual)
August 29, 2018
Last Update Submitted That Met QC Criteria
December 14, 2017
Last Verified
December 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Demyelinating Diseases
- Neurologic Manifestations
- Neurobehavioral Manifestations
- Endocrine System Diseases
- Genetic Diseases, Inborn
- Genetic Diseases, X-Linked
- Metabolism, Inborn Errors
- Mental Retardation, X-Linked
- Intellectual Disability
- Heredodegenerative Disorders, Nervous System
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Leukoencephalopathies
- Adrenal Gland Diseases
- Hereditary Central Nervous System Demyelinating Diseases
- Peroxisomal Disorders
- Adrenal Insufficiency
- Adrenoleukodystrophy
Other Study ID Numbers
- NA_00045673
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on X-linked Adrenoleukodystrophy
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bluebird bioTerminatedCerebral Adrenoleukodystrophy (CALD) | Adrenoleukodystrophy (ALD) | X-Linked Adrenoleukodystrophy (X-ALD)United States, United Kingdom, Argentina, Canada, Germany, Italy, Netherlands
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