Trial Search Results
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.
Collaborator: National Institute of Neurological Disorders and Stroke (NINDS)
- Dietary Supplement: vitamin D3
Criteria for enrollment to screening:
1. Molecular confirmation of X-linked ALD (VLCFA elevation & ABCD1 mutation) known in
patient or immediate family member)
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
- 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