Education and Training
A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
In this pilot study, the investigators will assess the safety of two high-dose regimens of oral vitamin D supplementation and measure the effects of vitamin D supplementation on markers of oxidative stress and inflammation in the blood and brain of study participants before, during, and after taking vitamin D supplements.
The goal of the study is to establish research measures (i.e. biomarkers) and an optimal dose for vitamin D supplementation in boys with the X-linked adrenoleukodystrophy (ALD) genotype.
Stanford is currently not accepting patients for this trial.
Stanford Investigator(s):
Intervention(s):
- dietary supplement: vitamin D3
Eligibility
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
Ages Eligible for Study
18 Months - 25 Years
Genders Eligible for Study
Male
Not currently accepting new patients for this trial
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Keith Van Haren, MD
Not Recruiting