The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study

NCT01323608 | Unknown Phase 4

• 1 other identifier: 1102011521
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this pilot study is to determine the effect of various doses of vitamin D supplementation on vitamin D stores and calcium excretion in the urine in subjects with Thalassemia Major (TM). Subjects with TM are routinely placed on vitamin D supplements because they frequently have osteoporosis (a condition in which bone tissue thins and loses density and strength) and low vitamin D stores. The amount of vitamin D supplementation that is required to raise vitamin D stores in optimal levels is not known in TM, and will be determined in this study. Finally, a recent study in TM has linked blood vitamin D levels to urine calcium excretion, which is a risk factor for kidney stones. Therefore, we want to determine changes in calcium excretion with various vitamin D doses and with increasing vitamin D stores. We plan to test 3 doses of vitamin D for 3 months in children and adults with TM. Changes in vitamin D blood levels and urinary calcium will be determined. The results of this pilot study will be used in future studies that will examine the effect of various doses of vitamin D supplementation in the treatment of osteoporosis in TM.

Conditions

Thalassemia Major

Keywords

Thalassemia; hypercalciuria; vitamin D; To conduct a pilot study to determine the effect of various doses of; vitamin D supplementation on vitamin D stores and their association with calcium excretion

Outcome Measures

Primary Outcomes (1)

• Vitamin D Dose Response Curve

  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 Outcomes (1)

• Vitamin D Dose Response Curve

  3 Months

Study Arms (4)

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Low Vitamin D Group

EXPERIMENTAL

Subjects in this group will receive the equivalent of 400 IU/day.

Drug: Vitamin D3

Intermediate Vitamin D group

EXPERIMENTAL

Drug: Vitamin D3

High Vitamin D Group

EXPERIMENTAL

Drug: Vitamin D3

Interventions

Vitamin D3 DRUG

Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day.

High Vitamin D Group; Intermediate Vitamin D group; Low Vitamin D Group

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 6 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Thalassemia Major (TM)
• OHD: 15-29 ng/ml
• Age 6 to 60 years
• Albumin corrected serum Calcium: Normal (8.5-10.5 mg/dl)

You may not qualify if:

• Other thalassemia syndromes
• 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)

Sponsors & Collaborators

• Weill Medical College of Cornell University: lead

Study Sites (1)

Weill Cornell Medical College

New York, New York, 10065, United States

Location

MeSH Terms

Conditions

beta-Thalassemia; Thalassemia; Hypercalciuria

Interventions

Cholecalciferol

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urological Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Cholestenes; Cholestanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Sterols; Vitamin D; Secosteroids; Membrane Lipids; Lipids

Study Officials

• Maria Vogiatzi, MD

  Weill Medical College of Cornell University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Maria Vogiatzi, MD

CONTACT

212-746-3462; mvogiatz@med.cornell.edu

Patricia J Giardina, MD

CONTACT

212-746-3415; pjgiardi@med.cornell.edu

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: March 24, 2011
• First Posted: March 25, 2011
• Study Start: June 1, 2011
• Primary Completion: June 1, 2014
• Study Completion: December 1, 2014
• Last Updated: April 19, 2011

Record last verified: 2011-04

Locations

US (1)