An observational study in Adrenoleukodystrophy, sponsored by Masonic Cancer Center, University of Minnesota. Withdrawn at 1 site in United States. Open to male participants aged 1 Year to 5 Years. Per ClinicalTrials.gov, last updated 2018-07-06.
Sponsored by Masonic Cancer Center, University of Minnesota · Observational
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 11th largest by enrollment of the 11 observational ones, is one of 4 that started in 2018, is one of 11 observational ones, and was the 23rd registered.
68 studies on the registry are indexed under Adrenoleukodystrophy; 19 are open to participants now.
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: 1 Year to 5 Years · male · no healthy volunteers.
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. 4 started in 2018.
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:
Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Masonic Cancer Center, University of Minnesota has 8 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 goal of this single institution study is to evaluate boys with adrenoleukodystrophy (ALD) diagnosed early in life, and to prospectively monitor them to determine parameters that will facilitate earlier detection of the childhood cerebral form of the disease. These at-risk subjects will be assessed yearly through travel to the University of Minnesota, where plasma and cerebral spinal fluid (CSF) biomarker studies, MRI based imaging and neuropsychological assessments will be performed at the University of Minnesota Masonic Children's Hospital and Clinics. The MRI and lumbar puncture to obtain CSF will be obtained under sedation. In addition, at intervening 6 months intervals information will be obtained remotely, including surveys and MRI's in their home location. Also at that time blood samples will be obtained locally and shipped to the University of Minnesota for study. There is no therapeutic intent in this study.
While this study will be registered with clinicaltrials.gov, no specific patient recruitment materials will be utilized. However, various Foundations and Patient Groups will be contacted. In addition, we will work with providers, neurologists, genetic counselors, etc. in the areas where newborn screening is taking place. We will also be available for families and providers at other institutions where a diagnosis may be made.
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:
ALD Early Evaluation
To evaluate boys with adrenoleukodystrophy (ALD) diagnosed early in life, and to prospectively determine factors that have a high correlation with the emergence of cerebral ALD.
Time frame: 5 years
Emergence of cerebral disease through imaging
The ability to discern the emergence of cerebral disease through imaging prior to the onset of classic T2 imaging. This will include diffusion tensor imaging (DTI), T1 and T2 rho, RAFF and spectroscopy, in addition to the standard MRI evaluations including gadolinium enhancement.
Time frame: 5 years
Biomarker Study: Inflammation Markers
Markers of inflammation (chitotriosidase, lipidomics including arachidonic acid metabolites and inflammatory cytokines).
Time frame: 5 years
Biomarker Study: Oxidative Stress
Determinations of oxidative stress (including but not limited to total and reduced/oxidized glutathione and 4-hydroxynonenal, or 4-HNE).
Time frame: 5 years
Biomarker Study: Immunologic Activation
Time frame: 5 years
Neuropsychology testing
Extensive neuropsychology testing, including assessments of verbal comprehension, perceptual reasoning, working memory and processing speed.
Time frame: 5 years
Start is estimated for Nov 2018; primary completion for Jul 2023, 56 months later.
How these figures are worked out, and which studies are left out of them.
This study is withdrawn, as verified in Jul 2018. You cannot join it, but the record below documents what was studied.
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Masonic Cancer Center, University of Minnesota →