Sickle Cell DiseasePhase 1/2NCT01276587
Study of Vitamin D in Children With Sickle Cell Disease
A Phase 1/2 study testing Vitamin D3 in people with Sickle Cell Disease, run by Gary M Brittenham, MD at 1 site in United States.
Trial summary
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels considered sufficient (30 ng/mL) but well below the threshold for toxicity (150 ng/mL) in… Read more about this study
- Who is it for?
- Ages 3 to 20 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Vitamin D3
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 1 siteUnited States
- Study dates
- Jan 2011to Jun 2011
- Phase
- Phase 1/2
- 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
- patients with sickle cell disease
- 3 to 20 years old
- pregnant females with sickle cell disease are eligible
Who cannot join
- no informed consent or assent
- unable or unwilling to comply with requirements of the clinical trial
- participation in another clinical trial
- history of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, such as primary hyperparathyroidism, malignancy, familial hypocalciuric hypercalcemia, William's syndrome and other rare causes
- therapy with thiazide diuretics or lithium carbonate
- known renal or liver disease
Show all 12Show fewer
- known malabsorption syndrome and inflammatory bowel disease
- chronic use of corticosteroids, excluding inhaled steroids
- current use of anticonvulsants (phenytoin, phenobarbital, carbamazepine)
- current intake of vitamin D and calcium supplements
- initiation of hydroxyurea or iron chelation therapy within the past 3 months
- serum 25hydroxyvitamin D >60 ng/mL
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: Pilot Study of Vitamin D Therapy to Prevent Respiratory Complications in Children With Sickle Cell Disease
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels considered sufficient (30 ng/mL) but well below the threshold for toxicity (150 ng/mL) in children with sickle cell disease. Information from this study will be crucial before we perform a larger clinical trial to determine the effects of vitamin D in reducing respiratory complications in patients with sickle cell disease.
Read the detailed description
Sickle cell disease is a genetic red blood cell disorder that affects an estimated 89,000 Americans, predominantly those of African ancestry. The leading causes of morbidity and of death in sickle cell disease are respiratory complications, particularly a life-threatening lung disease unique to sickle cell disease called the "acute chest syndrome". An infectious trigger and a pro-inflammatory state appear to be critical mechanisms of the respiratory problems in sickling disorders. Emerging evidence that vitamin D has antimicrobial and anti-inflammatory functions in the respiratory tract, and the recognition of widespread vitamin D insufficiency in sickle cell children provide a compelling new rationale for vitamin D supplementation in sickle cell disease.
This will be an open label, single arm study to assess the efficacy and safety of oral vitamin D3 100,000 IU administered monthly for six months in children and adolescents with sickle cell disease. Twelve pediatric patients with sickle cell disease, 3-20 years old, will be recruited. The primary outcome measure (efficacy and safety) will be serum 25-hydroxyvitamin D concentration. Other safety measures will include serum calcium and urinary calcium and creatinine. Serial measurements of serum 25-hydroxyvitamin D, serum chemistries, and urinary calcium and creatinine will be performed at baseline entry, monthly for six months during treatment with vitamin D3, and at study exit. Other measures relevant to our planned Phase 2 clinical trial, including markers of bone turnover, immune function, and inflammation, will also be obtained at baseline, midpoint and exit. Recruitment and enrollment of subjects is expected to be for 3 months; study assessments will be for 7 months (1 month screening and 6 months treatment); and, the remaining 2 months will be devoted to data collation and analysis.
Study Procedures
Screening: After signing written informed consent by a parent or legal guardian (and assent, if applicable) or patient, eligible participants will undergo a screening examination including a standardized history and physical examination.
A venous blood sample will be obtained for baseline screening measures including serum 25-hydroxyvitamin D, serum chemistries, and urine calcium and creatinine. Within one month, eligible participants who do not have any of the exclusion criteria will return for enrollment in the pilot study.
Intervention: Participants will be seen monthly for administration of oral vitamin D3 100,000 IU under the direct observation by the Clinical Research Nurse. History and examination will be performed to capture symptoms and signs of adverse events. Questionnaires to collect data on vitamin D and calcium dietary intake and respiratory events will be administered. Venous blood and urine samples for study assessments (serum 25-hydroxyvitamin D, serum albumin, calcium and phosphate, urine calcium and creatinine) will be obtained at each visit prior to administration of study drug.
- Conditions
- Sickle Cell Disease
- Being tested
- Vitamin D3
- Keywords
- sickle cell disease, children, vitamin D, safety
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin D3Drug
Also called Vitamin D
Oral vitamin D3 100,000 IU administered monthly for six months in children and adolescents with sickle cell disease
| Study group | Role | What they receive |
|---|---|---|
| Single arm | Experimental | Not described Receives: Drug: Vitamin D3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum 25-hydroxyvitamin D Concentration
Serum 25-hydroxyvitamin D level was measured at 6 months.
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2011 (estimated)
- Study startJan 2011
- Primary completionJun 2011
- Study completionJun 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gary M Brittenham, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Gary M Brittenham, MD, Columbia University
- Investigators
- Margaret T Lee, MD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01276587
- Sponsor's protocol ID
- AAAF2598
- Status last verified
- Mar 2018
- First submitted
- 12 Jan 2011
- Last updated
- 12 Apr 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 12 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Vitamin D Therapy to Prevent Respiratory Complications in Children With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT01276587
- Acronym / sponsor ID
- AAAF2598
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, children, vitamin D, safety
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Gary M Brittenham, MDUniversity, hospital or other
- Study start
- Jan 2011
- Primary completion
- Jun 2011 (actual)
- Study completion
- Jun 2011 (actual)
- First posted
- 13 Jan 2011 (estimated)
- Results
- Posted 12 Apr 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This pilot study aims to answer the question whether monthly oral vitamin D3 supplementation, 100,000 IU, will be safe and effective in raising serum 25-hydroxyvitamin D (form of vitamin D measured in the blood) to levels considered sufficient (30 ng/mL) but well below the threshold for toxicity (150 ng/mL) in children with sickle cell disease. Information from this study will be crucial before we perform a larger clinical trial to determine the effects of vitamin D in reducing respiratory complications in patients with sickle cell disease.
Detailed description
Sickle cell disease is a genetic red blood cell disorder that affects an estimated 89,000 Americans, predominantly those of African ancestry. The leading causes of morbidity and of death in sickle cell disease are respiratory complications, particularly a life-threatening lung disease unique to sickle cell disease called the "acute chest syndrome". An infectious trigger and a pro-inflammatory state appear to be critical mechanisms of the respiratory problems in sickling disorders. Emerging evidence that vitamin D has antimicrobial and anti-inflammatory functions in the respiratory tract, and the recognition of widespread vitamin D insufficiency in sickle cell children provide a compelling new rationale for vitamin D supplementation in sickle cell disease.
This will be an open label, single arm study to assess the efficacy and safety of oral vitamin D3 100,000 IU administered monthly for six months in children and adolescents with sickle cell disease. Twelve pediatric patients with sickle cell disease, 3-20 years old, will be recruited. The primary outcome measure (efficacy and safety) will be serum 25-hydroxyvitamin D concentration. Other safety measures will include serum calcium and urinary calcium and creatinine. Serial measurements of serum 25-hydroxyvitamin D, serum chemistries, and urinary calcium and creatinine will be performed at baseline entry, monthly for six months during treatment with vitamin D3, and at study exit. Other measures relevant to our planned Phase 2 clinical trial, including markers of bone turnover, immune function, and inflammation, will also be obtained at baseline, midpoint and exit. Recruitment and enrollment of subjects is expected to be for 3 months; study assessments will be for 7 months (1 month screening and 6 months treatment); and, the remaining 2 months will be devoted to data collation and analysis.
Study Procedures
Screening: After signing written informed consent by a parent or legal guardian (and assent, if applicable) or patient, eligible participants will undergo a screening examination including a standardized history and physical examination.
A venous blood sample will be obtained for baseline screening measures including serum 25-hydroxyvitamin D, serum chemistries, and urine calcium and creatinine. Within one month, eligible participants who do not have any of the exclusion criteria will return for enrollment in the pilot study.
Intervention: Participants will be seen monthly for administration of oral vitamin D3 100,000 IU under the direct observation by the Clinical Research Nurse. History and examination will be performed to capture symptoms and signs of adverse events. Questionnaires to collect data on vitamin D and calcium dietary intake and respiratory events will be administered. Venous blood and urine samples for study assessments (serum 25-hydroxyvitamin D, serum albumin, calcium and phosphate, urine calcium and creatinine) will be obtained at each visit prior to administration of study drug.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm | Experimental | Drug: Vitamin D3 |
Who may qualify
- Ages
- 3 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- patients with sickle cell disease
- 3 to 20 years old
- pregnant females with sickle cell disease are eligible
Key exclusion criteria (12 listed)
- no informed consent or assent
- unable or unwilling to comply with requirements of the clinical trial
- participation in another clinical trial
- history of hypercalcemia or diagnosis of any medical condition associated with hypercalcemia, such as primary hyperparathyroidism, malignancy, familial hypocalciuric hypercalcemia, William's syndrome and other rare causes
- therapy with thiazide diuretics or lithium carbonate
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Vitamin D3Also: Vitamin DOral vitamin D3 100,000 IU administered monthly for six months in children and adolescents with sickle cell disease | Drug | Single arm |
Primary outcome
Serum 25-hydroxyvitamin D Concentration
Time frame: Up to 6 months
Serum 25-hydroxyvitamin D level was measured at 6 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Apr 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Sickle Cell Disease
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Margaret T Lee, MD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Vitamin D3 in people with Sickle Cell Disease, 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 3 inclusion and 12 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 12 Apr 2023. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 12 Apr 2023). 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?
Serum 25-hydroxyvitamin D Concentration (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 12 Apr 2023.