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.

Lead Sponsor:

Stanford University

Collaborator: ALD Connect, Inc.

Stanford Investigator(s):

Intervention(s):

  • Dietary Supplement: vitamin D3

Phase:

Phase 1

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
Recruiting