An interventional study of exercise training in X-linked Adrenoleukodystrophy, sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc.. Completed at 1 site in United States. Open to female participants aged 21 Years to 70 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2018-08-29.
Sponsored by Hugo W. Moser Research Institute at Kennedy Krieger, Inc. · Not applicable, Interventional, and Treatment
Among 39 studies in Adrenoleukodystrophy with a site in United States — 8 open to participants now, 11 under way, 1 of them phase 3. Adrenoleukodystrophy · United States.
Among the 39 studies in Adrenoleukodystrophy with a site in United States, this one is the 15th largest by enrollment of the 26 interventional ones, is one of 2 that started in 2012, is one of 3 with no phase, and was the 11th registered.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
This study's enrollment of 31 is close to the median of 31 across 40 interventional studies indexed under Adrenoleukodystrophy.
Browse Adrenoleukodystrophy studies →8 of the 39 studies in Adrenoleukodystrophy with a site in United States are open now, in 1 country. Each count lists the studies.
Of the 16 Adrenoleukodystrophy studies recruiting now, 7 (44%) set no upper age limit and 9 (56%) stop at 75 or younger; 11 take children under 18; 4 take healthy volunteers.
This study: 21 Years to 70 Years · female · healthy volunteers too.
The full table on the hub →39 studies; the count open now beside each. This study's row is marked.
First in 1992; this study’s year is marked. 2 started in 2012.
Median enrollment 34 across the 26 interventional studies, 65 across the 11 observational. Of the 22 that completed or were terminated, 12 (55%) have results posted.
15 of 39 have an industry lead sponsor. The 10 most frequent:
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. is the lead sponsor of 76 studies on the registry; 19 are open to participants now.
Hugo W. Moser Research Institute at Kennedy Krieger, Inc. has 3 studies filed under Adrenoleukodystrophy — fewer than ten, so there is no comparison to draw.
3 of the studies in Adrenoleukodystrophy with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05819866Phase 3Minoryx Therapeutics, S.L.A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy | 2027-05 |
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
| NCT02254863Joanne Kurtzberg, MDUCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | 2027-10 |
Among the 39 studies in Adrenoleukodystrophy with a site in United States, from their registry updates:
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.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
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.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Female carriers as well as healthy age and gender matched individuals will participate in an exercise paradigm.
Behavioral: exercise training
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.
Also known as: strengthening, Curves
Change in Maximal Voluntary Contraction of the Hip Flexors From Baseline to End of Training and to Post-training
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.
Time frame: Participants are assessed at baseline (visit 1, enrollment), end of training (visit 2, week 12) and post-training (visit 3, week 18).
| Milestone | Female Carriers | Healthy Controls |
|---|---|---|
| Started | 16 | 15 |
| Completed | 14 | 12 |
| Not completed | 2 | 3 |
| Withdrew: Withdrawal by subject | 1 | 1 |
| Withdrew: Lost to follow-up | 1 | 1 |
| Withdrew: Noncompliance with exercise | 0 | 1 |
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.
| kg | Female Carriers | Healthy Controls |
|---|---|---|
| Change post-training | 0.94 ± 1.14 | 0.98 ± 2.01 |
| Change at follow up | 1.32 ± 1.28 | 1.37 ± 1.68 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Female Carriers | 0/14 (0%) | 0/14 (0%) | 0/14 (0%) |
| Healthy Controls | 0/12 (0%) | 0/12 (0%) | 0/12 (0%) |
| Age, Continuous(years) | Female Carriers | Healthy Controls | Total |
|---|---|---|---|
| Mean | 52.1 ± 8.4 | 51.3 ± 12.5 | 51.7 ± 10.5 |
| Sex: Female, Male(Participants) | Female Carriers | Healthy Controls | Total |
|---|---|---|---|
| Female | 14 | 12 | 26 |
| Male | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | Female Carriers | Healthy Controls | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 3 | 4 |
| White | 13 | 9 | 22 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Region of Enrollment(Participants) | Female Carriers | Healthy Controls | Total |
|---|---|---|---|
| United States | 14 | 12 | 26 |
PDFs hosted by the registry, ClinicalTrials.gov.
The record gives a start of May 2012, a primary completion of Oct 2014 and a completion of Oct 2014. It does not say whether these dates are actual or estimated.
Results were first posted in Aug 2018, 3 years 10 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2017. You cannot join it, but the record below documents what was studied.
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Hugo W. Moser Research Institute at Kennedy Krieger, Inc.