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Sickle Cell DiseasePhase 2NCT01443728

Vitamin D for Sickle-cell Respiratory Complications

A Phase 2 study testing Experimental: Vitamin D3 100,000 IU and Active Comparator: Vitamin D3 12,000 IU in people with Sickle Cell Disease and Vitamin D Deficiency, run by Gary M Brittenham, MD at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Read more about this study

Who is it for?
Ages 3 to 20 years
What is being tested?
Experimental: Vitamin D3 100,000 IU and Active Comparator: Vitamin D3 12,000 IU
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
70 participants
Where
1 siteUnited States
Study dates
13 Dec 2011to 15 Feb 2015
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 20 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Diagnosis of sickle cell disease (HbSS, HbSC, HbS Beta-thalassemia)
  • Age 3 to 20 years old

Who cannot join

  • Patient (or parent or guardian) unwilling or unable to provide written informed consent (and assent, if applicable)
  • Patient unable or unwilling to comply with requirements of the clinical trial
  • Participation in other therapeutic clinical trial
  • Current diagnosis of rickets
  • History of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, including primary hyperparathyroidism, malignancy, sarcoidosis, tuberculosis, granulomatous disease, familial hypocalciuric hypercalcemia
  • Current use of corticosteroids, excluding inhaled steroids
Show all 13Show fewer
  • Current use of anticonvulsants (phenytoin, phenobarbital, carbamazepine)
  • Therapy with thiazide diuretics or lithium carbonate
  • Known liver or renal disease
  • Patients taking medications for pulmonary complications of sickle cell disease not on a stable dose of medications, as defined by a change in medications or doses within the three months prior to study entry
  • Patients on chronic red blood cell transfusion therapy
  • Absence of baseline record of respiratory events (respiratory infections, asthma exacerbations, episodes of acute chest syndrome) for the preceding year
  • Pregnancy

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Columbia University Irving Medical Center

    New York, New York, 10032

Other trials in United States

About this study

This study aims to answer the question whether oral vitamin D supplementation can decrease lung complications in children and adolescents with sickle cell disease. Lung complications are the leading causes of morbidity and of death in sickle cell disease. Infections and increased inflammation play important roles in the development of the lung problems in sickle cell disease. Emerging evidence shows that vitamin D helps the immune system to fight infection and to control inflammation and could potentially help prevent respiratory complications in patients with sickle cell disease. The investigators hypothesize that oral vitamin D3, 100,000 IU (2.5 mg), given once a month to a group of children and adolescents with sickle cell disease, will reduce the rate of respiratory events (infection, asthma exacerbation and acute chest syndrome) compared to the rate in a group given standard dose oral vitamin D3, 12,000 IU (0.3 mg) given once a month.

Funding Source - U.S. Food \& Drug Administration, Office of Orphan Products Development

Read the detailed description

This study will be a Phase 2 double-blind randomized clinical trial in 80 patients with sickle cell disease, ages 3 to 20 years-old, comparing a 2-year monthly oral dose of vitamin D3, 100,000 IU (equivalent to 3,300 IU/day) to a standard monthly dose, 12,000 IU (400 IU/day) in reducing the rate of respiratory events (defined as respiratory infections, acute asthma exacerbation, and the acute chest syndrome) in children and adolescents with sickle cell disease in comparison with the rates of respiratory events over a baseline period of one year.

Eligible participants (130 patients) will initially be screened to determine their blood vitamin D levels (serum 25-hydroxyvitamin D). Those with 25-hydroxyvitamin D levels between 5 and 60 ng/mL will be eligible for randomization. At study entry, blood and urine samples will be collected for routine and special blood tests including tests on immune function, inflammation, and bone function. Children above 5 years old will also have lung function and muscle strength tests. Participants will be followed once a month to administer the study medication (oral vitamin D3) and to monitor any side effects from the study medication by history, examination and blood and urine tests. After 12 and 24 months of therapy, the same study procedures at study entry will be repeated.

This study could help establish oral vitamin D3 as a simple, low cost treatment to reduce respiratory complications in children and adolescents with sickle cell disease.

Being tested
Experimental: Vitamin D3 100,000 IU, Active Comparator: Vitamin D3 12,000 IU
Keywords
sickle cell disease, acute chest syndrome, respiratory complications, vitamin D
Medical subject headings
Anemia, Sickle Cell, Vitamin D Deficiency, Acute Chest Syndrome, Asthma, Respiratory Tract Infections

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Experimental: Vitamin D3 100,000 IUDrug

    Also called Cholecalciferol

    Oral vitamin D3, 100,000 IU [2.5 mg] given once a month

  • Active Comparator: Vitamin D3 12,000 IUDrug

    Also called Cholecalciferol

    Standard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month

Study groups in NCT01443728
Study groupRoleWhat they receive
Vitamin D3 100,000 IUExperimentalOral vitamin D3, 100,000 IU [2.5 mg] given once a month
Receives: Drug: Experimental: Vitamin D3 100,000 IU
Vitamin D3 12,000 IUActive comparatorStandard dose oral vitamin D3 12,000 IU [0.3 mg] given once a month
Receives: Drug: Active Comparator: Vitamin D3 12,000 IU

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mean Annual Rate of Respiratory Events

    Defined as respiratory infection, acute asthma exacerbation, and acute chest syndrome.

    Measured: Up to 2 years

Secondary outcomes (13)

  1. Mean 25-Hydroxyvitamin D (25-OHD)

    The overall mean serum 25-OHD concentration will be measured for both groups.

    Measured: 2 years

  2. Forced Vital Capacity (FVC)

    Percent predicted forced vital capacity will be calculated for both groups.

    Measured: Up to 2 years

  3. Forced Expiratory Volume (FEV) in 1 Second (FEV1)

    Percent predicted forced expiratory volume in 1 second will be calculated for both groups.

    Measured: Up to 2 years

  4. FEV1/FVC Ratio

    The ratio of FEV1 to FVC will be calculated (in percentage).

    Measured: Up to 2 years

  5. FEF 25-75

    Percent predicted forced expiratory flow (FEF) during expiration of 25 to 75% of the FVC will be calculated.

    Measured: Up to 2 years

  6. RV/TLC Ratio

    The ratio of residual volume (RV) to total lung capacity (TLC) will be calculated (in percentage).

    Measured: Up to 2 years

  7. DLCO

    Percent predicted of the diffusing capacity for carbon monoxide in the lungs (DLCO) will be measured.

    Measured: Up to 2 years

  8. FeNO

    Fractional exhaled nitric oxide (FeNO) will be measured in parts per billion (ppb).

    Measured: Up to 2 years

  9. MIP

    Maximum inspiratory pressure (MIP) will be measured.

    Measured: Up to 2 years

  10. MEP

    Maximum expiratory pressure (MEP) will be measured.

    Measured: Up to 2 years

  11. Hand-grip, Right

    Muscle strength was measured by looking at hand-grip strength in the right hand.

    Measured: Up to 2 years

  12. Hand-grip, Left

    Muscle strength was measured by looking at hand-grip strength in the left hand.

    Measured: Up to 2 years

  13. Hand-grip, Dominant

    Muscle strength was measured by looking at hand-grip strength in the dominant hand.

    Measured: Up to 2 years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted30 Sep 2011 (estimated)
  2. Study start13 Dec 2011
  3. Primary completion20 Jun 2013
  4. Study completion15 Feb 2015

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
70 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01443728
Sponsor's protocol ID
AAAE3244
Other IDs
R01FD003894
Status last verified
Jul 2024
First submitted
27 Sep 2011
Last updated
9 Aug 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Lee MT, Kattan M, Fennoy I, Arpadi SM, Miller RL, Cremers S, McMahon DJ, Nieves JW, Brittenham GM. Randomized phase 2 trial of monthly vitamin D to prevent respiratory complications in children with sickle cell disease. Blood Adv. 2018 May 8;2(9):969-978. doi: 10.1182/bloodadvances.2017013979.

    PubMed 29712666

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Experimental: Vitamin D3 100,000 IU and Active Comparator: Vitamin D3 12,000 IU in people with Sickle Cell Disease and Vitamin D Deficiency, run by Gary M Brittenham, MD at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 9 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean Annual Rate of Respiratory Events (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01443728, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2024.

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