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Studies With 1,25-Dihydroxycholecalciferol

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ClinicalTrials.gov Identifier: NCT00001151
Recruitment Status : Terminated (Terminated because of lack of drug supply)
First Posted : November 4, 1999
Results First Posted : November 25, 2013
Last Update Posted : November 25, 2013
Information provided by (Responsible Party):
Stephen Marx, M.D., National Institutes of Health Clinical Center (CC)

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Hypocalcemia Rickets Drug: 1,25-Dihydroxycholecalciferol Phase 2

Detailed Description:

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.

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Studies With 1,25-Dihydroxycholecalciferol
Study Start Date : March 1976
Primary Completion Date : October 2009
Study Completion Date : October 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Rickets
U.S. FDA Resources

Arm Intervention/treatment
Experimental: Drug treatment
1,25-Dihydroxycholecalciferol 5 ug orally per day for 2 years
Drug: 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.

Primary Outcome Measures :
  1. Number of Participants With Normal Serum Calcium Concentrations [ Time Frame: 1 year average ]
    Normal calcium concentration 8.2-10.6 mg/dL

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Patients with hereditary resistance to calcitrol.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00001151

United States, Maryland
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Principal Investigator: Stephen J Marx, MD NIDDK/NIH

Responsible Party: Stephen Marx, M.D., Chief of the Metabolic Diseases Branch of NIDDK/NIH, National Institutes of Health Clinical Center (CC)
ClinicalTrials.gov Identifier: NCT00001151     History of Changes
Other Study ID Numbers: 760081
76-DK-0081 ( Other Identifier: 76-DK-0081 )
First Posted: November 4, 1999    Key Record Dates
Results First Posted: November 25, 2013
Last Update Posted: November 25, 2013
Last Verified: June 2013

Keywords provided by Stephen Marx, M.D., National Institutes of Health Clinical Center (CC):

Additional relevant MeSH terms:
Calcium Metabolism Disorders
Metabolic Diseases
Water-Electrolyte Imbalance
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Vitamin D Deficiency
Deficiency Diseases
Nutrition Disorders
Calcium Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Vasoconstrictor Agents
Growth Substances
Physiological Effects of Drugs
Bone Density Conservation Agents