- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01323608
The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study
Study Overview
Detailed Description
Specific Aim: To determine the effect of various doses of vitamin D supplementation on serum concentration of 25 hydroxy vitamin D (25OHD) and urinary calcium excretion in adults and children with TM.
Hypothesis: Vitamin D supplementation at doses that result in serum 25OHD levels >30 ng/ml result in high rates of hypercalciuria (i.e. increased urinary calcium excretion) in children and adults with TM.
The pilot study will be performed at Weill Cornell Medical College, Payson 695.
- Children and adults with TM, will be divided into 4 groups: "standard" vitamin D dose (equivalent to 400 IU/day), "intermediate" vitamin D dose (equivalent to 1000 IU/day), "high" vitamin D dose (equivalent to 2,000 IU/day) and a "placebo" group that will receive an inactive ingredient. 10 subjects will be assigned to each group. The duration of the study will be 3 months.
- While some studies found no difference between vitamin D2 and D3, others suggest that vitamin D3 may be more advantageous in restoring 25 OHD concentrations. For these reasons, vitamin D3 will be used in this protocol.
- Study schedule with study interventions and frequencies is set out below (see table). All 4 groups will adhere to the same schedule. After obtaining consent, subjects will have a "Baseline Visit", which will include: 1) a complete physical examination; 2) laboratory evaluation that includes measurement of 25OHD, which is the major circulating form of vitamin D and reflects the body's vitamin D stores. Additional blood and urine samples will be obtained for measurement of urinary calcium excretion and serum Calcium and parathyroid hormone (PTH); and 3) Dietary calcium intake questionnaire. 4) Start of study medication.
- Subjects will then return every 2-4 weeks for routine blood transfusions and as required for the management of Thalassemia. To ensure compliance, study medications will be administered by study personnel during these routine visits. Vitamin D supplies will be packaged according to one week worth requirement, i.e. as 1,000 IU/day x 7days=7,000 IU for the "intermediate" vitamin D group. The dose that will be administered during these visits will depend on the interval between transfusions, i.e. 7,000 IU x 2weeks, for a subject of the "intermediate" dose who is transfused every two weeks.
- At the end of 3 month treatment, subjects will have a final visit, that is also scheduled to coincide with a routine transfusion visit. Subjects will have the same evaluation and procedures as in "Baseline Visit" : 1) a complete physical examination; 2) fasting laboratory evaluation that includes measurement of 25OHD, urinary calcium excretion and serum Calcium and PTH; and 3) Dietary calcium intake questionnaire.
- Subjects will be asked to take calcium supplementation at the dose of 500 mg daily for the 6-8 year old and 1,000 mg daily for the 9-18 year old subjects. This intervention will ensure adequate calcium intake in both groups.
All labs will be processed at the CTSC Core Lab. Study meds will be distributed by the central pharmacy. Investigational drug will be administered by study personnel during the transfusion visits to ensure compliance.
Eligible subjects will be assigned to a study group following a block type of enrollment. To ensure an equal, or near equal sex and age distribution, study groups will be matched according to sex and age. Half of the subjects in each group will be composed of pediatric subjects and the other half will be composed of adult subjects.
Study Schedule and Procedures:
- Baseline visit: Physical Exam, 25OHD, Intact PTH & Serum Calcium, 24 hr & spot urine collection for Ca/Crea, Calcium intake questionnaire, Vitamin D Supplementation/Placebo.
- Every 2-4 weeks: Administration of Vitamin D/Placebo
- 3 Month Visit: Physical Exam, 25OHD, Intact PTH & Serum Calcium, 24 hr & spot urine collection for Ca/Crea, Calcium intake questionnaire, and Vitamin D Supplementation/ Placebo.
Primary study endpoint:
Changes in 25OHD concentrations and calcium excretion with the various vitamin D3 doses will be determined at the end of the study and constitute the primary endpoints.
Study Type
Enrollment (Anticipated)
Phase
- Phase 4
Contacts and Locations
Study Contact
- Name: Maria Vogiatzi, MD
- Phone Number: 212-746-3462
- Email: mvogiatz@med.cornell.edu
Study Contact Backup
- Name: Patricia J Giardina, MD
- Phone Number: 212-746-3415
- Email: pjgiardi@med.cornell.edu
Study Locations
-
-
New York
-
New York, New York, United States, 10065
- Weill Cornell Medical College
-
Contact:
- Maria Vogiatzi, MD
- Phone Number: 212-746-3462
- Email: mvogiatz@med.cornell.edu
-
Contact:
- Patricia J Giardina, MD
- Phone Number: 212-746-3415
- Email: pjgiardi@med.cornell.edu
-
Principal Investigator:
- Maria Vogiatzi, MD
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Thalassemia Major (TM)
- 25 OHD: 15-29 ng/ml
- Age 6 to 60 years
- Albumin corrected serum Calcium: Normal (8.5-10.5 mg/dl)
Exclusion Criteria:
- Other thalassemia syndromes
- 25 OHD concentrations < 15 ng/ml or ≥30 ng/ml
- Subjects younger than 6 years
- Hypoparathyroidism
- Abnormal albumin corrected serum Ca (i.e. total calcium <8.5 or > 10.5 mg/dl)
- Medications that may adversely affect vitamin D metabolism (anticonvulsants) or absorption
- End stage renal, heart, or liver disease
- History of Nephrolithiasis or Nephrocalcinosis
- Diseases associated with hypercalciuria (ie. Sarcoidosis, Cushing syndrome, and Wilson disease to name a few)
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Diagnostic
- Allocation: Non-Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Placebo Comparator: Placebo
|
Placebo
|
Experimental: Low Vitamin D Group
Subjects in this group will receive the equivalent of 400 IU/day.
|
Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day.
|
Experimental: Intermediate Vitamin D group
|
Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day.
|
Experimental: High Vitamin D Group
|
Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Vitamin D Dose Response Curve
Time Frame: 3 Months
|
To perform a dose response curve for vitamin D supplementation study and determine the relationship between vitamin D doses and serum 25OHD concentrations and urinary calcium excretion in children and adults with TM.
|
3 Months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Vitamin D Dose Response Curve
Time Frame: 3 Months
|
To determine changes in serum calcium and PTH concentrations with various vitamin D doses
|
3 Months
|
Collaborators and Investigators
Investigators
- Principal Investigator: Maria Vogiatzi, MD, Weill Medical College of Cornell University
Study record dates
Study Major Dates
Study Start
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Hematologic Diseases
- Genetic Diseases, Inborn
- Anemia
- Anemia, Hemolytic, Congenital
- Anemia, Hemolytic
- Hemoglobinopathies
- Thalassemia
- beta-Thalassemia
- Physiological Effects of Drugs
- Micronutrients
- Vitamins
- Bone Density Conservation Agents
- Calcium-Regulating Hormones and Agents
- Vitamin D
- Cholecalciferol
Other Study ID Numbers
- 1102011521
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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