An interventional study of retinyl palmitate in Sickle Cell Anemia in Children and Vitamin A Deficiency in Children, sponsored by Children's Hospital of Philadelphia. Completed at 1 site in United States. Open to participants aged 9 Years and older, including healthy volunteers. Per ClinicalTrials.gov, last updated 2018-08-16.
Sponsored by Children's Hospital of Philadelphia · Not applicable, Interventional, and Treatment
Among 733 studies in Anemia, Sickle Cell with a site in United States — 134 open to participants now, 185 under way, 16 of them phase 3. Anemia, Sickle Cell · United States.
Among the 733 studies in Anemia, Sickle Cell with a site in United States, this one is the 231st largest by enrollment of the 559 interventional ones that give a count, is one of 38 that started in 2015, is one of 165 with no phase, and was the 421st registered.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
Browse Anemia, Sickle Cell studies →134 of the 733 studies in Anemia, Sickle Cell with a site in United States are open now. Recruiting first, the newest first:
University of Maryland, Baltimore · Sites in United States
Ages 18–88
Mohsen Saidinejad · Sites in United States
Ages 5–20
St. Jude Children's Research Hospital · Sites in United States
Ages up to 22
Emory University · Sites in United States
Ages 8–18
University of Alabama at Birmingham · Sites in United States
Ages 18–50 · Women only
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 177 Anemia, Sickle Cell studies recruiting now, 64 (36%) set no upper age limit and 97 (55%) stop at 75 or younger; 127 take children under 18; 29 take healthy volunteers.
This study: 9 Years and older · all sexes · healthy volunteers too.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
733 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 158 · Phase 2 228 · Phase 3 57 · Phase 4 20
First in 1984; this study’s year is marked. 38 started in 2015.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 42 is close to the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Median enrollment 31 across the 559 interventional studies, 100 across the 168 observational. Of the 506 that completed or were terminated, 207 (41%) have results posted.
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168 of 733 have an industry lead sponsor. The 10 most frequent:
Children's Hospital of Philadelphia is the lead sponsor of 489 studies on the registry; 90 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
Children's Hospital of Philadelphia has 6 studies filed under Anemia, Sickle Cell — fewer than ten, so there is no comparison to draw.
57 of the studies in Anemia, Sickle Cell with a site in United States that are under way reach primary completion within a year, 7 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT07247188Phase 3SanofiStudy of a Single Dose of a 21-valent Pneumococcal Conjugate Vaccine in Children and Adolescents With Sickle Cell Disease | 2027-01-07 |
| NCT04293185Phase 3bluebird bioA Study Evaluating Gene Therapy With BB305 Lentiviral Vector in Sickle Cell Disease | 2027-05 |
| NCT06989567Phase 3Asklepion Pharmaceuticals, LLCA Study of Intravenous L-Citrulline in Patients Aged 6-21 Years Old With Sickle Cell Disease Presenting to Emergency Departments in Acute Vaso-Occlusive Crisis | 2027-06 |
| NCT05329649Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Severe Sickle Cell Disease (SCD) | 2027-06-06 |
| NCT05477563Phase 3Vertex Pharmaceuticals IncorporatedEvaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease | 2027-06-09 |
Add all 57 to your calendar (.ics) → With an account.
Among the 733 studies in Anemia, Sickle Cell with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Subjects with SCD-SS in the lower dose Vitamin A arm receive 3000IU of retinyl palmitate daily for 8 weeks.
Dietary Supplement: retinyl palmitate
Subjects with SCD-SS in the higher dose Vitamin A arm receive 6000IU of retinyl palmitate daily for 8 weeks.
Dietary Supplement: retinyl palmitate
Healthy subjects receive no intervention and undergo comparisons to the two vitamin A supplementation arms at baseline.
The intervention is a daily vitamin A supplement.
Serum Vitamin A status
Serum vitamin A as measured by retinol
Time frame: Change from baseline after supplementation for 8 weeks
Vitamin A toxicity
Retinyl palmitate
Time frame: Change from baseline after supplementation for 8 weeks
Height Z-score
Measured on a stadiometer, compared to Center for Disease Control (CDC) reference standard to create a z-score
Time frame: Change from baseline after supplementation for 8 weeks
Weight Z-score
Measured on a standing scale, compared to CDC reference standard to create a z-score
Time frame: Change from baseline after supplementation for 8 weeks
BMI Z-score
Calculated using kg/m\^2 and compared to CDC reference standards
Time frame: Change from baseline after supplementation for 8 weeks
Fat-free Mass
Calculated from dual-energy x-ray absorptiometry (DEXA) scan
Time frame: Change from baseline after supplementation for 8 weeks
Fat-free Mass
Calculated from DEXA scan
Time frame: Change from baseline after supplementation for 8 weeks
Fat Mass
Calculated from DEXA scan
Time frame: Change from baseline after supplementation for 8 weeks
Upper arm muscle area
Calculated from mid-upper arm circumference
Time frame: Change from baseline after supplementation for 8 weeks
Upper arm fat area
Calculated from mid-upper arm circumference and triceps skinfold thickness
Time frame: Change from baseline after supplementation for 8 weeks
Muscle strength
Directly measured with Biodex Multi-Joint System 3 Pro
Time frame: Change from baseline after supplementation for 8 weeks
Jump strength
Directly measured with Force Plate
Time frame: Change from baseline after supplementation for 8 weeks
Upper limb strength
Directly measured with hand-grip strength dynamometer
Time frame: Change from baseline after supplementation for 8 weeks
Muscle function
Directly measured with Bruininks-Oseretsky Test of Motor Proficiency
Time frame: Change from baseline after supplementation for 8 weeks
Dietary Intake
Analysis of a three-day food record
Time frame: Change from baseline after supplementation for 8 weeks
Coefficient of fat absorption
Calculated from 72-hour stool collection and dietary fat intake
Time frame: Change from baseline after supplementation for 8 weeks
Hemoglobin
Direct measurement through spectral absorption
Time frame: Change from baseline after supplementation for 8 weeks
Hematocrit
Direct measurement through spectral absorption
Time frame: Change from baseline after supplementation for 8 weeks
Fetal hemoglobin
Direct measurement through quantitative flow cytometry
Time frame: Change from baseline after supplementation for 8 weeks
Mean corpuscular volume
Direct measurement through quantitative flow cytometry
Time frame: Change from baseline after supplementation for 8 weeks
Mean corpuscular hemoglobin
Calculated from hemoglobin mass and erythrocyte count
Time frame: Change from baseline after supplementation for 8 weeks
Mean corpuscular hemoglobin concentration
Calculated from hemoglobin divided by hematocrit
Time frame: Change from baseline after supplementation for 8 weeks
Reticulocyte count
Direct measurement through quantitative flow cytometry
Time frame: Change from baseline after supplementation for 8 weeks
Retinol binding protein, serum
Direct measurement through quantitative nephelometry
Time frame: Change from baseline after supplementation for 8 weeks
Retinol binding protein, urine
Direct measurement through quantitative nephelometry
Time frame: Change from baseline after supplementation for 8 weeks
Urine creatinine
Direct measurement through quantitative spectrophotometry
Time frame: Change from baseline after supplementation for 8 weeks
Serum creatinine
Direct measurement through quantitative spectrophotometry
Time frame: Change from baseline after supplementation for 8 weeks
Serum alanine aminotransferase
Direct measurement through quantitative enzymatic assay
Time frame: Change from baseline after supplementation for 8 weeks
Serum aspartate aminotransferase
Direct measurement through quantitative enzymatic assay
Time frame: Change from baseline after supplementation for 8 weeks
Serum gamma glutamyltransferase
Direct measurement through quantitative enzymatic assay
Time frame: Change from baseline after supplementation for 8 weeks
Serum alkaline phosphatase
Direct measurement through quantitative enzymatic assay
Time frame: Change from baseline after supplementation for 8 weeks
Serum bilirubin
Direct measurement through quantitative quantitative spectrophotometry
Time frame: Change from baseline after supplementation for 8 weeks
High-sensitivity c-reactive protein
Direct measurement through quantitative quantitative immunoturbidimetry
Time frame: Change from baseline after supplementation for 8 weeks
Tumor necrosis factor alpha
Direct measurement through quantitative quantitative multiplex bead assay
Time frame: Change from baseline after supplementation for 8 weeks
White blood cell count
Direct measurement through automated cell count
Time frame: Change from baseline after supplementation for 8 weeks
White blood cell differential
Direct measurement through automated cell count
Time frame: Change from baseline after supplementation for 8 weeks
Lymphocyte subtypes
Direct measurement through quantitative flow cytometry
Time frame: Change from baseline after supplementation for 8 weeks
Total body vitamin A status via Stable Isotope Dilution
compartmental modeling of \[13C10\]-retinyl acetate, measured by high performance liquid chromatography/mass spectroscopy
Time frame: Change from baseline after supplementation for 8 weeks
Plan to share: Yes — Individual participant data that underlie the results reported will be shared upon request, after deidentification.
Supporting information: Study protocol, Informed consent form
Time frame: The data will be available immediately upon publication.
Access criteria: Contact brownellj@email.chop.edu. Requestors will need to sign a data access agreement.
Ran from Oct 2015 to primary completion in Sep 2016, 11 months.
Of 165 non-industry interventional studies with no phase under Anemia, Sickle Cell, half reached primary completion within 28 months of starting (the median), counting those still running; a quarter within 17 and three quarters within 54. This study's 11 months is below that.
Counting every sponsor, half of 173 studies reached it within 28 months; a quarter within 15 and three quarters within 49.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Aug 2018. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Retinol palmitate
Children's Hospital of Philadelphia →