Hypocalcemia, Rickets
Conditions
Keywords
Calciferol, Hypocalcemia, Rickets
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.
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.
Interventions
DRUG1,25-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.
Sponsors
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* INCLUSION CRITERIA: Patients with hereditary resistance to calcitrol.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Normal Serum Calcium Concentrations | 1 year average | Normal calcium concentration 8.2-10.6 mg/dL |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Group 1 Patient has rickets (cild) or osteomalacia (adult) of bone | 6 |
| Total | 6 |
Baseline characteristics
| Characteristic | Group 1 |
|---|---|
| Age, Continuous | 15 years |
| Region of Enrollment United States | 6 participants |
| Sex: Female, Male Female | 3 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 0 / 6 |
| serious Total, serious adverse events | 0 / 6 |
Outcome results
Primary
Number of Participants With Normal Serum Calcium Concentrations
Normal calcium concentration 8.2-10.6 mg/dL
Time frame: 1 year average
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Group 1 | Number of Participants With Normal Serum Calcium Concentrations | 6 participants |