X-linked Adrenoleukodystrophy Clinical Trial
Official title:
A Pilot Study of Vitamin D in Boys With X-linked Adrenoleukodystrophy
Verified date | June 2022 |
Source | Stanford University |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 21 |
Est. completion date | June 30, 2020 |
Est. primary completion date | June 30, 2020 |
Accepts healthy volunteers | No |
Gender | Male |
Age group | 18 Months to 25 Years |
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 |
Country | Name | City | State |
---|---|---|---|
United States | Stanford University | Palo Alto | California |
Lead Sponsor | Collaborator |
---|---|
Stanford University | ALD Connect, Inc., Hugo W. Moser Research Institute at Kennedy Krieger, Inc., National Institute of Neurological Disorders and Stroke (NINDS) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percent of patients with a plasma 25-OH vitamin D level in the target range (40-80ng/ml) at 12 months | The investigators expect 100% of patients will be in the target range at 12 months (i.e. oral dose of 4000 IU daily) | Plasma 25-OH vitamin D will be measured at 12 months | |
Primary | Percent of patients with a plasma 25-OH vitamin D level in the target range (40-80ng/ml) at 6 months | The investigators expect that 80% of patients will be in the target range 6 months (i.e. oral dose of 2000 IU daily) | Plasma 25-OH vitamin D will be measured at 6 months | |
Secondary | Correlation between appearance of gadolinium enhancing brain lesion on MRI and most recent plasma 25-OH vitamin D level | For participants developing gadolinium enhancing lesions on MRI, the investigators will compare the most recent preceding 25-OH vitamin D level with the average 25-OH vitamin D level of participants in the study who did not develop gadolinium enhancing lesions. The investigators expect the development of gadolinium enhancing lesion on MRI will correlate with lower vitamin D levels. However, our current study is not sufficiently powered to measure this effect. | Brain MRI at baseline, 6, 12, 18, 24, 30, and 36 months study enrollment. Plasma 25-OH vitamin D levels at baseline, 3, 6, 9, 12, 18, 24, 30, 36 months of enrollment. | |
Secondary | Change in protein carbonyl levels in whole blood at baseline and 12 months. | The investigators expect a decrease in whole blood protein carbonyl levels between baseline and 12 months. | Measurements at baseline and 12months | |
Secondary | Correlation between plasma 25-OH vitamin D and intracellular glutathione levels in peripheral monocytes | The investigators will use flow cytometry to measure intracellular GSH in CD14+ monocytes from participants peripheral blood at baseline and 12 months. The investigators will measure plasma 25-OH vitamin at the same time points. The investigators expect a positive correlation between plasma vitamin D levels and monocyte GSH levels. | Measurements at baseline and 12 months | |
Secondary | Change in glutathione (GSH) levels in blood | The investigators expect a positive correlation between plasma 25-OH vitamin levels and GSH levels in whole blood (measured by tandem mass spectroscopy). | Measurements will be obtained at baseline, 6months, and 12months | |
Secondary | Change in glutathione (GSH) levels in brain | The investigators will examine the correlation between 25-OH vitamin D levels in plasma and total GSH levels in occipital white matter (measured by single-voxel MR spectroscopy). | Measurements will be obtained at baseline, 6months, and 12months | |
Secondary | Occurrence of serious adverse events | The investigators do not expect any participants to develop hypercalcemia (serum calcium >10.7mg/dl) or related serious adverse events (e.g. kidney stones) while taking 2000 IU or 4000 IU daily. | Measurements will be obtained at baseline, 3months, 6months, 9months, 12months | |
Secondary | Change in plasma interleukin-8 levels | The investigators expect a decrease in plasma IL-8 levels between baseline and 12 months | Measurements a baseline and 12 months | |
Secondary | Change in plasma macrophage inflammatory protein-1b levels | The investigators expect a decrease in plasma MIP-1b levels between baseline and 12 months | Measurements at baseline and 12 months | |
Secondary | Change in plasma monocyte chemoattractant protein-1 levels | The investigators expect a decrease in plasma MCP-1 levels between baseline and 12 months | Measurements at baseline and 12 months |
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