Evidence map›Paper›PMID 37090939›Full record

ArticleNeurology. Genetics2023

A Phase 1 Study of Oral Vitamin D

Keith P Van Haren, Kristen Cunanan, Avni Awani, Meng Gu, Dalia Peña, Lindsay C Chromik, Michal Považan, Nicole C Rossi, Jordan Goodman, Vandana Sundaram and 9 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Neurology. Genetics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.3field-weighted citation impact, top 23% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

4 citing papers in PubMed, 8 citations in OpenAlex.

  1. Observational
  2. Characterizing circulating biomarkers for childhood dementia disorders: A scoping review of clinical trials.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Article
  3. Article
  4. Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

19 authors at 2 institutions in 2 countries.

Keith P Van HarenDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.ORCID https://orcid.org/0000-0002-0233-9361
Kristen CunananDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Avni AwaniDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Meng GuDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Dalia PeñaDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Lindsay C ChromikDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Michal PovažanDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Nicole C RossiDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Jordan GoodmanDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Vandana SundaramDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Jennifer WinterbottomDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Gerald V RaymondDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Tina CowanDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Gregory M EnnsDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Emmanuelle WaubantDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.ORCID https://orcid.org/0000-0001-5188-0157
Lawrence SteinmanDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Peter B BarkerDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Daniel SpielmanDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Ali FatemiDepartment of Neurology (K.P.V.H., A.A., D.P., L.C.C., N.C.R., J.W., L.S.), Department of Pediatrics (K.P.V.H., T.C., G.M.E., L.S.), Quantitative Sciences Unit (K.C., V.S.) and Department of Radiology (M.G., D.S.), Stanford University School of Medicine Palo Alto, CA; Russell H. Morgan Department of Radiology and Radiological Science (M.P., P.B.B.), The Johns Hopkins University School of Medicine; The Kennedy Krieger Institute (M.P., P.B.B., A.F.); Department of Genetic Medicine (G.V.R.), The Johns Hopkins University School of Medicine, Baltimore, MD; Department of Pathology (T.C.), Stanford University School of Medicine, Palo Alto, CA; Department of Neurology (E.W.), University of California at San Francisco, ; and Department of Neurology (A.F.), The Johns Hopkins University School of Medicine, Baltimore, MD.
Kennedy Krieger Institute · USJohns Hopkins University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background and Objectives: There are no therapies for preventing cerebral demyelination in X-linked adrenoleukodystrophy (ALD). Higher plasma vitamin D levels have been linked to lower risk of inflammatory brain lesions. We assessed the safety and pharmacokinetics of oral vitamin D dosing regimens in boys and young men with ALD. Methods: In this open-label, multicenter, phase 1 study, we recruited boys and young men with ALD without brain lesions to a 12-month study of daily oral vitamin D Results: Between October 2016 and June 2019, we enrolled 21 participants (n = 12, fixed-dose regimen; n = 9, weight-stratified regimen) with a median age of 6.7 years (range: 1.9-22 years) and median weight of 20 kg (range: 11.7-85.5 kg). The number of participants achieving target vitamin D levels was similar in both groups at 6 months (fixed dose: 92%; weight stratified: 78%) and 12 months (fixed dose: 67%; weight stratified: 67%). Among the 12 participants in the fixed-dose regimen, half had asymptomatic elevations in either urine calcium:creatinine or plasma 25-hydroxyvitamin D; no laboratory deviations occurred with the weight-stratified regimen. Glutathione levels in the brain, but not the blood, increased significantly between baseline and 12 months. Discussion: Our vitamin D dosing regimens were well tolerated and achieved target 25-hydroxyvitamin D levels in most participants. Brain glutathione levels warrant further study as a biomarker for vitamin D and ALD. Classification of Evidence: This study provides Class IV evidence that fixed or weight-stratified vitamin D supplementation achieved target levels of 25-hydroxyvitamin D in boys and young men with X-ALD without brain lesions.

Identifiers

PMID37090939
PMCPMC10117697
OpenAlexW4362471214

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