NCT04170348

Daily Vitamin D for Sickle-cell Respiratory Complications

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 58 (actual)
• Sponsor: Columbia University · Academic / Other
• Sex: All
• Age: 3 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

This study aims to answer the question whether daily oral vitamin D supplementation can reduce the risk of respiratory or lung complications in children and adolescents with sickle cell disease. Respiratory problems are the leading causes of sickness and of death in sickle cell disease. The investigators hypothesize that daily oral vitamin D3, compared to monthly oral vitamin D, will rapidly increase circulating vitamin D3, and reduce the rate of respiratory complications by 50% or more within the first year of supplementation in children and adolescents with sickle cell disease. This study is funded by the FDA Office of Orphan Products Development (OOPD).

Detailed description

This is a 2-year controlled, double-blind, randomized Phase 2 clinical trial comparing the efficacy in reducing the rate of respiratory events in sickle-cell disease of daily oral vitamin D3 (3,333 IU/d) with monthly bolus oral vitamin D3, (100,000 IU/mo) as a control. The scientific premise of the clinical trial is that circulating concentrations of vitamin D3, the parent compound, are the principal determinant of the anti-infective and immunomodulatory effects of supplementation. Eligible participants will be initially screened to determine their blood vitamin D levels. Those with 25-hydroxyvitamin D levels between 5 and 60 ng/mL will be assigned by chance to one of the two arms for 24 months. Participants will be checked every month and will have periodic blood and urine tests to monitor for any side effects of the study treatments. Children above 5 y/o who can cooperate and understand the procedure will have lung function test at baseline and at 24 months. Showing that a monthly dose of vitamin D reduces lung infections, asthma and the acute chest syndrome could help establish this simple, low-cost treatment as a way to decrease sickness and deaths in children and adolescents with sickle-cell disease.

Conditions

• Sickle Cell Disease
• Anemia, Sickle Cell
• Anemia, Hemolytic, Congenital
• Respiratory Tract Diseases
• Respiration Disorders
• Acute Chest Syndrome
• Lung Diseases
• Asthma
• Respiratory Tract Infections
• Nutrition Disorders
• Deficiency Diseases Vitamin
• Vitamin D Deficiency

Interventions

Type	Name	Description
DRUG	Daily oral vitamin D3, 3,333 IU	Oral vitamin D3, 3,333 IU, will be administered daily.
DRUG	Monthly oral vitamin D3, 100,000 IU	Oral vitamin D3, 100,000 IU, will be administered monthly.
DRUG	Placebo oral tablet	Participants randomized to receive once monthly oral bolus of vitamin D3, will receive placebo on all other days of the month.

Timeline

• Start date: 2020-09-15
• Primary completion: 2024-06-18
• Completion: 2024-06-18
• First posted: 2019-11-20
• Last updated: 2025-09-24
• Results posted: 2025-09-24

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study