What is the mechanism of action of this treatment?

Hydroxyurea is a cornerstone treatment for Sickle Cell Disease (SCD) that works by increasing fetal hemoglobin (HbF) production, which reduces the sickling of red blood cells and decreases vaso-occlusive episodes. Red blood cell transfusions are used to lower the proportion of sickle cells in the blood, thereby reducing complications like stroke and severe anemia. Vitamin D supplementation, as studied in trials, aims to improve overall health and reduce respiratory complications, which are common in SCD patients. These treatments are crucial as they address the underlying pathophysiology of SCD, improve quality of life, and reduce the frequency and severity of complications.

What side effects are associated with this type of intervention?

Common treatments for Sickle Cell Disease (SCD) include hydroxyurea and vitamin D3 supplementation. Hydroxyurea is generally well-tolerated but can cause side effects such as bone marrow suppression, leading to decreased white blood cells and platelets, gastrointestinal disturbances, and potential long-term risks like infertility in men. Vitamin D3 supplementation is usually safe but can cause hypercalcemia if taken in excessive amounts, leading to symptoms like nausea, vomiting, weakness, and serious complications such as kidney stones. Regular monitoring and appropriate dosing are essential to minimize these risks.

What prior FDA approvals exist?

The FDA has approved several treatments for Sickle Cell Disease, including hydroxyurea, which reduces the frequency of pain episodes and the need for blood transfusions, and L-glutamine oral powder, which helps reduce acute complications. Voxelotor and crizanlizumab are newer agents that target the underlying pathophysiology of SCD. While these treatments primarily focus on reducing pain and vaso-occlusive crises, the trial studying daily oral Vitamin D3 supplementation aims to reduce respiratory complications, a different but critical aspect of SCD management. This approach is unique as it targets the improvement of overall respiratory health in children and adolescents with SCD.

What other types of research exist?

Promising alternatives to daily oral vitamin D3 supplementation for SCD patients may include high-dose intermittent vitamin D3 supplementation, which could provide similar benefits with less frequent dosing. Additionally, exploring genetic therapies that target respiratory complications or enhance overall immune function in SCD patients could be beneficial. Consulting with a healthcare provider for personalized treatment options is recommended.

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Vitamin Supplement

Vitamin D for Sickle Cell Anemia (ViDAS-2 Trial)

Phase 2

Waitlist Available

Led By Margaret T Lee, MD

Research Sponsored by Gary M Brittenham, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Age 3-20 years old

Diagnosis of sickle cell disease (Hb SS, Hb SC, Hb S-Beta-thalassemia)

Must not have

Patients on chronic red blood cell transfusion therapy

Current use of corticosteroids, excluding inhaled steroids

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline and month 24

Summary

This trial tests if vitamin D3 can lower lung issues in kids with sickle cell disease. It compares different dosing schedules to see which works better. The goal is to find a simple, low-cost way to help these children stay healthier. Vitamin D has been studied for its potential to reduce respiratory complications in children with sickle cell disease due to its ability to fight infections and support the immune system.

Who is the study for?

This trial is for children and adolescents aged 3-20 with sickle cell disease. It's not for those on chronic blood transfusions, using certain seizure meds, unable to consent or follow the study plan, with rickets or high calcium conditions, liver/kidney disease, unstable pulmonary meds within 3 months prior to the study, current corticosteroid use (except inhaled), or in another clinical trial.

What is being tested?

The study tests if daily vitamin D supplements can lower respiratory issues in young people with sickle cell disease. Participants will either take a daily dose of vitamin D3 (3333 IU) or a monthly bolus dose (100000 IU), compared to a placebo group. The goal is to cut respiratory complications by half within one year.

What are the potential side effects?

Potential side effects may include elevated calcium levels which could lead to kidney stones and constipation; too much vitamin D might also cause weakness, fatigue, sleepiness, headache, loss of appetite, dry mouth and metallic taste.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 3 and 20 years old.

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I have been diagnosed with sickle cell disease.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am on a long-term treatment that involves receiving blood transfusions.

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I am currently taking corticosteroids, but not through inhalation.

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My medication for sickle cell lung issues hasn't changed in the last 3 months.

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I am currently taking thiazide diuretics or lithium carbonate.

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I have a history of high calcium levels or conditions causing it.

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I have been diagnosed with rickets.

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I am currently taking medication for seizures.

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I have a known liver or kidney disease.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline and month 24

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline and month 24

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline and month 24 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Rate of Respiratory Events

Secondary study objectives

Change in C-terminal telopeptides of Type I collagen (CTX)

Change in Diffusing Capacity of the Lungs for Carbon Monoxide (DLCO)

Change in Forced Expiratory Flow at 25%-75% vital capacity (FEF25-75)

+21 more

Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: Daily oral vitamin D3Experimental Treatment1 Intervention

Oral vitamin D3, 3,333 IU

Group II: Monthly bolus oral vitamin D3Active Control2 Interventions

Bolus oral vitamin D3, 100,000 IU

0 / 10Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Hydroxyurea is a cornerstone treatment for Sickle Cell Disease (SCD) that works by increasing fetal hemoglobin (HbF) production, which reduces the sickling of red blood cells and decreases vaso-occlusive episodes. Red blood cell transfusions are used to lower the proportion of sickle cells in the blood, thereby reducing complications like stroke and severe anemia. Vitamin D supplementation, as studied in trials, aims to improve overall health and reduce respiratory complications, which are common in SCD patients. These treatments are crucial as they address the underlying pathophysiology of SCD, improve quality of life, and reduce the frequency and severity of complications.

Apparent vitamin B6 deficiency in sickle cell anemia.Hypothesis: Low Vitamin A and D Levels Worsen Clinical Outcomes When Children with Sickle Cell Disease Encounter Parvovirus B19.Rheumatic Manifestations of Haemoglobinopathies.