Vitamin D for Sickle Cell Anemia

(ViDAS-2 Trial)

Overseen By Margaret T Lee, MD

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Columbia University

Must not be taking: Corticosteroids, Anticonvulsants, Thiazides, Lithium

Disqualifiers: Rickets, Hypercalcemia, Liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

Duration: 24 months

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests whether daily vitamin D can reduce lung problems in children and teens with sickle cell disease. Lung issues pose a significant concern for those with this condition, and vitamin D might lower the risk of these complications. The study compares daily doses of vitamin D (daily oral vitamin D3, 3,333 IU) to larger doses taken less frequently (bolus oral vitamin D3, 100,000 IU) to determine which is more effective. Children and teens aged 3 to 20 with sickle cell disease may be suitable participants, especially if they frequently experience lung-related health challenges. As a Phase 2 trial, this research focuses on evaluating the treatment's effectiveness in an initial, smaller group.

Will I have to stop taking my current medications?

If you are currently using corticosteroids (except inhaled ones), anticonvulsants, thiazide diuretics, or lithium carbonate, you cannot participate in the trial. Also, if you are taking medications for lung issues related to sickle cell disease, your doses must have been stable for at least three months before joining the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that both daily and monthly vitamin D3 supplements are safe for children with sickle cell disease. One study found that children who took daily vitamin D3 for 12 weeks experienced no major side effects, indicating its safety for regular use. Another study discovered that a high dose of vitamin D3 taken once a month was also safe and effectively improved vitamin D levels. Thus, children with sickle cell disease can safely take either daily or monthly vitamin D3 without harm. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about using vitamin D3 for sickle cell anemia because it offers a potential new approach to managing this condition. Unlike traditional treatments that focus on managing pain and preventing complications, vitamin D3 might improve overall bone health and immune function, which are often compromised in individuals with sickle cell anemia. The daily oral dose of 3,333 IU is a straightforward, non-invasive method that could enhance the quality of life for patients. Additionally, the monthly bolus of 100,000 IU provides an alternative dosing strategy that could be more convenient for some patients. These features make vitamin D3 an intriguing candidate for improving outcomes in sickle cell anemia beyond the existing standard of care.

What evidence suggests that daily oral vitamin D3 might be an effective treatment for sickle cell disease?

Research has shown that vitamin D supplements can boost vitamin D levels, which is crucial for children with sickle cell disease. In this trial, participants will receive either a daily dose of vitamin D3 or a monthly bolus dose. One study found that taking vitamin D3 daily significantly increased vitamin D levels in just eight weeks. This increase might help lower the risk of breathing problems, which are common and serious in sickle cell disease. Another study found that administering vitamin D3 once a month reduced the rate of respiratory illnesses by more than 50% in the second year. These findings suggest that both daily and monthly vitamin D3 could be beneficial, but daily doses might work faster. ^{1 2 6 7 8}

Who Is on the Research Team?

Gary M Brittenham, MD

Principal Investigator

Columbia University

Margaret T Lee, MD

Principal Investigator

Columbia University

Are You a Good Fit for This Trial?

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.

Inclusion Criteria

I am between 3 and 20 years old.

I have been diagnosed with sickle cell disease.

Exclusion Criteria

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

I am willing and able to follow the clinical trial's requirements.

I am currently taking corticosteroids, but not through inhalation.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment

Participants receive daily oral vitamin D3 or monthly bolus vitamin D3 for 24 months

24 months

Monthly visits (in-person) for monitoring and tests

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Bolus oral vitamin D3, 100,000 IU
• Daily oral vitamin D3, 3,333 IU
• Placebo oral tablet

Trial Overview 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.

How Is the Trial Designed?

Group I: Daily oral vitamin D3 Experimental Treatment 1 Intervention

Oral vitamin D3, 3,333 IU

Group II: Monthly bolus oral vitamin D3 Active Control 2 Interventions

Bolus oral vitamin D3, 100,000 IU

Who Is Running the Clinical Trial?

Columbia University

Lead Sponsor

Trials

1,529

Recruited

2,832,000+

Gary M Brittenham, MD

Lead Sponsor

Trials

3

Recruited

140+

FDA Office of Orphan Products Development

Collaborator

Trials

73

Recruited

6,400+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11263414/

Safety and efficacy of monthly high-dose vitamin D3 ... Monthly oral high-dose vitamin D supplementation was safe and effective in improving vitamin D levels, HGS, and HRQoL in SCD children and healthy subjects.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03417947

Vitamin D Supplementation in Children With Sickle Cell ... The working hypothesis is that administration of a single oral bolus of 300,000 IU of vitamin D3 to SCD children will result in the attainment of vitamin D ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5941998/

Randomized phase 2 trial of monthly vitamin D to prevent ... Annual rates of respiratory illness in sickle cell disease decreased by >50% during the second year of monthly doses of oral vitamin D3.

4. clinicaltrials.gov clinicaltrials.gov/study/NCT04170348

Daily Vitamin D for Sickle-cell Respiratory Complications 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 ...

5. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT03417947/

Trial | NCT03417947 The working hypothesis is that administration of a single oral bolus of 300,000 IU of vitamin D3 to SCD children will result in the attainment of vitamin D ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6059995/

Response to Long-term Vitamin D Therapy for Bone Disease ... In our small cohort receiving high-dose monthly bolus doses of oral vitamin D3 for 24 months, all patients had 25(OH)D levels that normalized along with ...

7. ashpublications.org ashpublications.org/bloodadvances/article/2/9/969/15839/Randomized-phase-2-trial-of-monthly-vitamin-D-to

Randomized phase 2 trial of monthly vitamin D to prevent ... Annual rates of respiratory illness in sickle cell disease decreased by >50% during the second year of monthly doses of oral vitamin D3.

8. thelancet.com thelancet.com/journals/landia/article/PIIS2213-8587(24)00348-6/fulltext

Vitamin D supplementation to prevent acute respiratory ... Randomized phase 2 trial of monthly vitamin D to prevent respiratory complications in children with sickle cell disease. Blood Adv. 2018; 2 ...