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Completed

NCT Number: NCT03632876

Nutritional Outcomes After Vitamin A Supplementation in Subjects With SCD

This study establishes the safety and efficacy of vit A supplementation doses (3000 and 6000 IU/d) over 8 weeks in children with SCD-SS, ages 9 and older and test the impact of vit A supplementation on key functional and clinical outcomes. Additionally, vitamin A status is assessed in healthy children ages 9 and older to compare to subjects with SCD-SS.

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Key information

Conditions

Sickle Cell Anemia in Children Vitamin A Deficiency in Children

Age range

9 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States

About this study

Suboptimal vitamin A (vit A) status is prevalent in children with type SS sickle cell disease (SCD-SS) and associated with hospitalizations and poor growth and hematological status. Preliminary data in children with SCD-SS show that vit A supplementation at the dose recommended for healthy children failed to improve vit A status, resulting in no change in hospitalizations, growth or dark adaptation. This indicates an increased vit A requirement most likely due to chronic inflammation, low vit A intake and possible stool or urine loss. The dose of vit A needed to optimize vit A status in subjects with SCD-SS is unknown.

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Sickle cell disease, SS genotype (subjects with sickle cell disease only)
  • Usual state of good health (no hospitalizations, emergency room visits, or unscheduled acute illness clinic visits for two weeks prior to screening)
  • Commitment to a 119-day study (subjects with sickle cell disease only), or a 4-day study (healthy volunteers only)

Exclusion criteria

  • Hydroxyurea initiated within the previous 6 weeks (subjects with sickle cell disease only)
  • History of stroke (subjects with sickle cell disease only)
  • Other chronic conditions that may affect growth, dietary intake or nutritional status
  • Retinoic acid (topical or oral), weight loss medication and/or lipid lowering medications
  • Subjects with a BMI greater than 98th percentile for age and sex
  • Pregnant or lactating females (subjects who become pregnant during the course of the study will not continue participation)
  • Liver function tests >4 x upper limit of reference range
  • Participation in another study with impact on vitamin A status (subjects with sickle cell disease only)
  • Use of multi-vitamin or commercial nutritional supplements containing vitamin A (those who are willing to discontinue these supplements, with the approval of the medical care team, will be eligible for the study after a 1 month washout period. Subjects taking nutritional products without vitamin A will be eligible)
  • Inability to swallow pills (subjects with sickle cell disease only)

Treatment and study plan

retinyl palmitate

Dietary Supplement

The intervention is a daily vitamin A supplement.

Primary outcomes

  1. Serum Vitamin A status

    Time frame: Change from baseline after supplementation for 8 weeks

    Serum vitamin A as measured by retinol

Secondary outcomes

  1. Vitamin A toxicity

    Time frame: Change from baseline after supplementation for 8 weeks

    Retinyl palmitate

  2. Height Z-score

    Time frame: Change from baseline after supplementation for 8 weeks

    Measured on a stadiometer, compared to Center for Disease Control (CDC) reference standard to create a z-score

  3. Weight Z-score

    Time frame: Change from baseline after supplementation for 8 weeks

    Measured on a standing scale, compared to CDC reference standard to create a z-score

  4. BMI Z-score

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated using kg/m^2 and compared to CDC reference standards

  5. Fat-free Mass

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from dual-energy x-ray absorptiometry (DEXA) scan

  6. Fat-free Mass

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from DEXA scan

  7. Fat Mass

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from DEXA scan

  8. Upper arm muscle area

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from mid-upper arm circumference

  9. Upper arm fat area

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from mid-upper arm circumference and triceps skinfold thickness

  10. Muscle strength

    Time frame: Change from baseline after supplementation for 8 weeks

    Directly measured with Biodex Multi-Joint System 3 Pro

  11. Jump strength

    Time frame: Change from baseline after supplementation for 8 weeks

    Directly measured with Force Plate

  12. Upper limb strength

    Time frame: Change from baseline after supplementation for 8 weeks

    Directly measured with hand-grip strength dynamometer

  13. Muscle function

    Time frame: Change from baseline after supplementation for 8 weeks

    Directly measured with Bruininks-Oseretsky Test of Motor Proficiency

  14. Dietary Intake

    Time frame: Change from baseline after supplementation for 8 weeks

    Analysis of a three-day food record

  15. Coefficient of fat absorption

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from 72-hour stool collection and dietary fat intake

  16. Hemoglobin

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through spectral absorption

  17. Hematocrit

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through spectral absorption

  18. Fetal hemoglobin

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative flow cytometry

  19. Mean corpuscular volume

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative flow cytometry

  20. Mean corpuscular hemoglobin

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from hemoglobin mass and erythrocyte count

  21. Mean corpuscular hemoglobin concentration

    Time frame: Change from baseline after supplementation for 8 weeks

    Calculated from hemoglobin divided by hematocrit

  22. Reticulocyte count

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative flow cytometry

  23. Retinol binding protein, serum

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative nephelometry

  24. Retinol binding protein, urine

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative nephelometry

  25. Urine creatinine

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative spectrophotometry

  26. Serum creatinine

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative spectrophotometry

  27. Serum alanine aminotransferase

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative enzymatic assay

  28. Serum aspartate aminotransferase

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative enzymatic assay

  29. Serum gamma glutamyltransferase

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative enzymatic assay

  30. Serum alkaline phosphatase

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative enzymatic assay

  31. Serum bilirubin

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative quantitative spectrophotometry

  32. High-sensitivity c-reactive protein

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative quantitative immunoturbidimetry

  33. Tumor necrosis factor alpha

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative quantitative multiplex bead assay

  34. White blood cell count

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through automated cell count

  35. White blood cell differential

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through automated cell count

  36. Lymphocyte subtypes

    Time frame: Change from baseline after supplementation for 8 weeks

    Direct measurement through quantitative flow cytometry

Other outcomes

  1. Total body vitamin A status via Stable Isotope Dilution

    Time frame: Change from baseline after supplementation for 8 weeks

    compartmental modeling of [13C10]-retinyl acetate, measured by high performance liquid chromatography/mass spectroscopy

Sponsors and collaborators

Lead sponsor

Children's Hospital of Philadelphia

Other

Collaborators

  • Newcastle University
  • Penn State University

Registry information

Official study title

Vitamin A in Sickle Cell Disease: Improving Sub-optimal Status With Supplementation

Important dates

Study start
2015
Primary completion
2016
Study completion
2016
First posted
Aug 16, 2018
Registry last updated
Aug 16, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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