NCT00001151

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 1976

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 1976

Completed
23.7 years until next milestone

First Submitted

Initial submission to the registry

November 3, 1999

Completed
1 day until next milestone

First Posted

Study publicly available on registry

November 4, 1999

Completed
9.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2009

Completed
4.2 years until next milestone

Results Posted

Study results publicly available

November 25, 2013

Completed
Last Updated

November 25, 2013

Status Verified

June 1, 2013

Enrollment Period

33.6 years

First QC Date

November 3, 1999

Results QC Date

November 12, 2010

Last Update Submit

November 21, 2013

Conditions

HypocalcemiaRickets

Keywords

CalciferolHypocalcemiaRickets

Outcome Measures

Primary Outcomes (1)

  • Number of Participants With Normal Serum Calcium Concentrations

    Normal calcium concentration 8.2-10.6 mg/dL

    1 year average

Study Arms (1)

Drug treatment

EXPERIMENTAL

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

Drug: 1,25-Dihydroxycholecalciferol

Interventions

1,25-DihydroxycholecalciferolDRUG

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.

Drug treatment

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with hereditary resistance to calcitrol.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

  • 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.

    PMID: 6893458BACKGROUND

  • 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.

    PMID: 4357855BACKGROUND

  • 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.

    PMID: 233695BACKGROUND

MeSH Terms

Conditions

HypocalcemiaRickets

Interventions

Calcitriol

Condition Hierarchy (Ancestors)

Calcium Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesWater-Electrolyte ImbalanceBone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesVitamin D DeficiencyAvitaminosisDeficiency DiseasesMalnutritionNutrition Disorders

Intervention Hierarchy (Ancestors)

DihydroxycholecalciferolsHydroxycholecalciferolsCholecalciferolCholestenesCholestanesSteroidsFused-Ring CompoundsPolycyclic CompoundsSterolsVitamin DSecosteroidsMembrane LipidsLipids

Results Point of Contact

Title
Stephen J. Marx, M.D./National Institute of Diabetes and Digestive and Kidney Diseases
Organization
NIH
marxs@mail.nih.gov
3014965051

Study Officials

  • Stephen J Marx, MD

    NIDDK/NIH

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
NIH
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief of the Metabolic Diseases Branch of NIDDK/NIH

Study Record Dates

First Submitted

November 3, 1999

First Posted

November 4, 1999

Study Start

March 1, 1976

Primary Completion

October 1, 2009

Study Completion

October 1, 2009

Last Updated

November 25, 2013

Results First Posted

November 25, 2013

Record last verified: 2013-06

Locations

US(1)