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Study of Vitamin D in Children With Sickle Cell Disease

Primary Purpose

Sickle Cell Disease

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Vitamin D3
Sponsored by
Gary M Brittenham, MD
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Sickle Cell Disease focused on measuring sickle cell disease, children, vitamin D, safety

Eligibility Criteria

3 Years - 20 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • patients with sickle cell disease
  • 3 to 20 years old
  • pregnant females with sickle cell disease are eligible

Exclusion Criteria:

  • 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
  • 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

Sites / Locations

  • Columbia University Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single arm

Arm Description

Outcomes

Primary Outcome Measures

Serum 25-hydroxyvitamin D Concentration
Serum 25-hydroxyvitamin D level was measured at 6 months.

Secondary Outcome Measures

Full Information

First Posted
January 12, 2011
Last Updated
March 21, 2023
Sponsor
Gary M Brittenham, MD
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1. Study Identification

Unique Protocol Identification Number
NCT01276587
Brief Title
Study of Vitamin D in Children With Sickle Cell Disease
Official Title
Pilot Study of Vitamin D Therapy to Prevent Respiratory Complications in Children With Sickle Cell Disease
Study Type
Interventional

2. Study Status

Record Verification Date
March 2018
Overall Recruitment Status
Completed
Study Start Date
January 2011 (undefined)
Primary Completion Date
June 2011 (Actual)
Study Completion Date
June 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Gary M Brittenham, MD

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Sickle Cell Disease
Keywords
sickle cell disease, children, vitamin D, safety

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Single arm
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
Vitamin D3
Other Intervention Name(s)
Vitamin D
Intervention Description
Oral vitamin D3 100,000 IU administered monthly for six months in children and adolescents with sickle cell disease
Primary Outcome Measure Information:
Title
Serum 25-hydroxyvitamin D Concentration
Description
Serum 25-hydroxyvitamin D level was measured at 6 months.
Time Frame
Up to 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: patients with sickle cell disease 3 to 20 years old pregnant females with sickle cell disease are eligible Exclusion Criteria: 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 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Margaret T Lee, MD
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States

12. IPD Sharing Statement

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Study of Vitamin D in Children With Sickle Cell Disease

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