Studies With 1,25-Dihydroxycholecalciferol

NCT ID: NCT00001151

Last Updated: 2013-11-25

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

1976-03-31

Study Completion Date

2009-10-31

Brief Summary

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Vitamin D in the diet undergoes changes in the liver and kidneys to several forms. Patients suffering from disorders with Vitamin D resistance are unable to absorb calcium from food. Patients diagnosed with these disorders will be evaluated and treated with high doses of another form of Vitamin D (1,25-dihydroxyvitamin D3). Patients will be monitored and observed throughout the study to avoid experiencing side effects from the medication.

Detailed Description

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Patients with extreme resistance to 1,25-dihydroxyvitamin D will be evaluated and treated with high doses of 1,25-dihydroxyvitamin D3.

Plan: In previously untreated patients the study will be divided into a control and one or more treatment periods. During the control period, parathyroid status will be assessed by parameters nos. 1\& 2 (below). In previously treated patients maintenance vitamin D will be gradually replaced with 1,25(OH)2D3. This will be accomplished by withdrawal of vitamin D and institution of 1,25(OH)2D3 when the serum calcium shows a downward trend.

1,25(OH)2D3 as 0.25 or 0.5 ug capsules (though IND 20,889) or as a solution of I microgram per ml will be administered orally. In most cases, because of consideration of time and expense, the cooperation of the patient's local physician will be enlisted. The following will be monitored:

1. Serum calcium, phosphorus,alkaline phosphatase,creatinine at twice weekly intervals. After a maintenance dose has been established, this will be decreased to a monthly, and subsequently 3-6 monthly interval.
2. Urine calcium, phosphorus,creatinine and cAMP before therapy and, when appropriate, during therapy.

The dose of 1,25(OH)2D3 will be 0.125 to 50.0 ug/day. Serum calcium will not be allowed to rise above the normal range (2.0 -2.4 mM at NIH). Should hypercalcemia occur, appropriate treatment will be initiated and the drug dosage will be decreased.

Conditions

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Hypocalcemia Rickets

Keywords

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Calciferol Hypocalcemia Rickets

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Drug treatment

1,25-Dihydroxycholecalciferol 5 ug orally per day for 2 years

Group Type EXPERIMENTAL

1,25-Dihydroxycholecalciferol

Intervention Type DRUG

Usual doses of 1,25-dihydroycholecalciferol are 0.25-1 ug per day. All patients in this study received very high doses or 5-20 ug per day.

Interventions

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1,25-Dihydroxycholecalciferol

Usual doses of 1,25-dihydroycholecalciferol are 0.25-1 ug per day. All patients in this study received very high doses or 5-20 ug per day.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

Patients with hereditary resistance to calcitrol.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

NIH

Sponsor Role lead

Responsible Party

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Stephen Marx, M.D.

Chief of the Metabolic Diseases Branch of NIDDK/NIH

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Stephen J Marx, MD

Role: PRINCIPAL_INVESTIGATOR

NIDDK/NIH

Locations

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National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, United States

Site Status

Countries

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United States

References

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Marx SJ, Swart EG Jr, Hamstra AJ, DeLuca HF. Normal intrauterine development of the fetus of a woman receiving extraordinarily high doses of 1,25-dihydroxyvitamin D3. J Clin Endocrinol Metab. 1980 Nov;51(5):1138-42. doi: 10.1210/jcem-51-5-1138.

Reference Type BACKGROUND
PMID: 6893458 (View on PubMed)

Fraser D, Kooh SW, Kind HP, Holick MF, Tanaka Y, DeLuca HF. Pathogenesis of hereditary vitamin-D-dependent rickets. An inborn error of vitamin D metabolism involving defective conversion of 25-hydroxyvitamin D to 1 alpha,25-dihydroxyvitamin D. N Engl J Med. 1973 Oct 18;289(16):817-22. doi: 10.1056/NEJM197310182891601. No abstract available.

Reference Type BACKGROUND
PMID: 4357855 (View on PubMed)

Marx SJ, Spiegel AM, Brown EM, Gardner DG, Downs RW Jr, Attie M, Hamstra AJ, DeLuca HF. A familial syndrome of decrease in sensitivity to 1,25-dihydroxyvitamin D. J Clin Endocrinol Metab. 1978 Dec;47(6):1303-10. doi: 10.1210/jcem-47-6-1303.

Reference Type BACKGROUND
PMID: 233695 (View on PubMed)

Other Identifiers

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76-DK-0081

Identifier Type: OTHER

Identifier Source: secondary_id

760081

Identifier Type: -

Identifier Source: org_study_id