Thalassemia MajorPhase 4NCT01323608
The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study
A Phase 4 study testing Vitamin D3 in people with Thalassemia Major, run by Weill Medical College of Cornell University at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 to 60 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- Vitamin D3
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2011to Dec 2014
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 6 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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)
Who cannot join
- 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
Show all 9Show fewer
- 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)
Trial locations and contacts
1 site in 1 country.
United States1 site
Weill Cornell Medical College
New York, New York, 10065
Maria Vogiatzi, MD, 212-746-3462, mvogiatz@med.cornell.edu
Patricia J Giardina, MD, 212-746-3415, pjgiardi@med.cornell.edu
About this study
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.
Read the 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.
- Conditions
- Thalassemia Major
- Being tested
- Vitamin D3, Placebo
- 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
- Medical subject headings
- beta-Thalassemia, Thalassemia, Hypercalciuria
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vitamin D3Drug
Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day.
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| Low Vitamin D Group | Experimental | Subjects in this group will receive the equivalent of 400 IU/day. Receives: Drug: Vitamin D3 |
| Intermediate Vitamin D group | Experimental | Not described Receives: Drug: Vitamin D3 |
| High Vitamin D Group | Experimental | Not described Receives: Drug: Vitamin D3 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 3 Months
Secondary outcomes (1)
Vitamin D Dose Response Curve
To determine changes in serum calcium and PTH concentrations with various vitamin D doses
Measured: 3 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2011 (estimated)
- Study startJun 2011
- Primary completionJun 2014 (estimated)
- Study completionDec 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Investigators
- Maria Vogiatzi, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01323608
- Sponsor's protocol ID
- 1102011521
- Status last verified
- Apr 2011
- First submitted
- 24 Mar 2011
- Last updated
- 19 Apr 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study
- ClinicalTrials.gov ID
- NCT01323608
- Acronym / sponsor ID
- 1102011521
- 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
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Study start
- Jun 2011
- Primary completion
- Jun 2014 (estimated)
- Study completion
- Dec 2014 (estimated)
- First posted
- 25 Mar 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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 design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: Placebo |
| Low Vitamin D Group | Experimental | Drug: Vitamin D3Subjects in this group will receive the equivalent of 400 IU/day. |
| Intermediate Vitamin D group | Experimental | Drug: Vitamin D3 |
| High Vitamin D Group | Experimental | Drug: Vitamin D3 |
Who may qualify
- Ages
- 6 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (9 listed)
- 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)
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 D3Vitamin D3 will be given at the equivalent of the following doses: 400, 1000, and 2000 IU/day. | Drug | High Vitamin D Group, Intermediate Vitamin D group, Low Vitamin D Group |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
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.
1 secondary outcome
Vitamin D Dose Response Curve
Time frame: 3 Months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2011.
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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.
Maria Vogiatzi, MD principal investigator
Weill Medical College of Cornell University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Vitamin D3 in people with Thalassemia Major, run by Weill Medical College of Cornell University 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 4 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 19 Apr 2011. 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. Some participants in this study may receive a placebo. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Maria Vogiatzi, MD (212-746-3462, mvogiatz@med.cornell.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Vitamin D Dose Response Curve (time frame: 3 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 19 Apr 2011.