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
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
Now accepting new patients
Contact Information
Stanford University
School of Medicine
300 Pasteur Drive
Stanford,
CA
94305
Keith Van Haren, MD
Recruiting