Nutritional Outcomes After Vitamin A Supplementation in Subjects With SCD

Children's Hospital of Philadelphia (CHOP)

Status: Completed

Conditions: Vitamin A Deficiency in Children; Sickle Cell Anemia in Children

Treatments: Dietary Supplement: retinyl palmitate

Study type: Interventional

Funder types: Other

Identifiers

NCT03632876

13-010081

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 42 patients

Sex: All

Ages: 9+ years old

Volunteers: Accepts Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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42 participants in 3 patient groups

Lower Dose Vitamin A

Active Comparator group

Description:

Subjects with SCD-SS in the lower dose Vitamin A arm receive 3000IU of retinyl palmitate daily for 8 weeks.

Treatment:

Dietary Supplement: retinyl palmitate

Higher Dose Vitamin A

Active Comparator group

Description:

Subjects with SCD-SS in the higher dose Vitamin A arm receive 6000IU of retinyl palmitate daily for 8 weeks.

Treatment:

Dietary Supplement: retinyl palmitate

Healthy Comparison Arm

No Intervention group

Description:

Healthy subjects receive no intervention and undergo comparisons to the two vitamin A supplementation arms at baseline.