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 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/Phase 2

Eligibility


Criteria for enrollment to screening:

   1. Molecular confirmation of X-linked ALD (VLCFA elevation & ABCD1 mutation)

   2. Male

   3. Age 3yrs - 12yrs at screening

Criteria for assignment to drug:

   1. Plasma 25-hydroxy vitamin D level ≤ 35ng/ml in past 90 days

   2. MRI brain in past 30 days 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

3 Years - 12 Years

Genders Eligible for Study

Male

Now accepting new patients

Contact Information

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