A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 20 June 2022
• Main ID: NCT02595489
• Date of registration: 29/10/2015
• Prospective Registration: Yes
• Primary sponsor: Stanford University
• Public title: A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
• Scientific title: A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
• Date of first enrolment: November 21, 2016
• Target sample size: 21
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02595489
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Keith Van Haren, MD
• Affiliation: Stanford University

Key inclusion & exclusion criteria

Criteria for enrollment to screening:

1. Molecular confirmation of X-linked ALD (VLCFA elevation & ABCD1 mutation) known in
patient or immediate family member)

2. Male

3. Age 1.5yrs (i.e. 18mos) - 25yrs at screening

Criteria for assignment to drug:

1. Plasma 25-hydroxy vitamin D level = 60ng/ml in past 30 days

2. MRI brain in past 6 months that is negative for evidence of active cerebral
demyelination

Exclusion Criteria:

- history of liver or kidney disease

- history of nephrolithiasis

- history of hyperthyroidism

- history of ulcerative colitis, Crohn's disease, celiac disease

- taking medication interfering with gastrointestinal absorption

- contraindication or inability to complete MRI every 6 months



Age minimum: 18 Months
Age maximum: 25 Years
Gender: Male

Health Condition(s) or Problem(s) studied

X-linked Adrenoleukodystrophy

Intervention(s)

Dietary Supplement: vitamin D3

Primary Outcome(s)

Percent of patients with a plasma 25-OH vitamin D level in the target range (40-80ng/ml) at 6 months [Time Frame: Plasma 25-OH vitamin D will be measured at 6 months]

Percent of patients with a plasma 25-OH vitamin D level in the target range (40-80ng/ml) at 12 months [Time Frame: Plasma 25-OH vitamin D will be measured at 12 months]

Secondary Outcome(s)

Correlation between plasma 25-OH vitamin D and intracellular glutathione levels in peripheral monocytes [Time Frame: Measurements at baseline and 12 months]

Change in glutathione (GSH) levels in brain [Time Frame: Measurements will be obtained at baseline, 6months, and 12months]

Change in protein carbonyl levels in whole blood at baseline and 12 months. [Time Frame: Measurements at baseline and 12months]

Occurrence of serious adverse events [Time Frame: Measurements will be obtained at baseline, 3months, 6months, 9months, 12months]

Change in glutathione (GSH) levels in blood [Time Frame: Measurements will be obtained at baseline, 6months, and 12months]

Change in plasma macrophage inflammatory protein-1b levels [Time Frame: Measurements at baseline and 12 months]

Correlation between appearance of gadolinium enhancing brain lesion on MRI and most recent plasma 25-OH vitamin D level [Time Frame: Brain MRI at baseline, 6, 12, 18, 24, 30, and 36 months study enrollment. Plasma 25-OH vitamin D levels at baseline, 3, 6, 9, 12, 18, 24, 30, 36 months of enrollment.]

Change in plasma interleukin-8 levels [Time Frame: Measurements a baseline and 12 months]

Change in plasma monocyte chemoattractant protein-1 levels [Time Frame: Measurements at baseline and 12 months]

Secondary ID(s)

23596

K23NS087151

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

ALD Connect, Inc.

National Institute of Neurological Disorders and Stroke (NINDS)

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.