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

Not Recruiting

Trial ID: NCT02595489


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.

Official Title

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

Stanford Investigator(s)


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

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


dietary supplement: vitamin D3

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Keith Van Haren, MD