Status:
COMPLETED
Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot Project.
Lead Sponsor:
Indiana University
Conditions:
Autosomal Dominant Hypophosphatemic Rickets
Eligibility:
All Genders
25+ years
Phase:
NA
Brief Summary
The purpose of the study is to gain a better understanding of the effect of iron on fibroblast growth factor 23 (FGF23) in the inherited disorder, autosomal dominant hypophosphatemic rickets (ADHR). A...
Detailed Description
Iron will be provided in an open label treatment to all enrolled subjects. Iron levels will be monitored in blood and doses adjusted with the target of getting the iron levels to or a little above 100...
Eligibility Criteria
Inclusion
- FGF Mutation in either Arginine 176 or arginine 179
- able and willing to provide consent or have a parent that is able/willing to consent, if a minor
- either serum iron \<50mcg/dl (regardless of phosphate or intact FGF23 concentration); or iron between 500 and 100mcg/dl with serum phosphorus value below 3.0mg/dl for adults or less than or equal to 0.5 mg/dl the lower limit of normal for age in children and intact FGF23 about 30pg/ml
- age \>2 years
- May be receiving treatment with phosphate and calcitriol, but must be willing to undergo dose adjustments by the investigators if iron resolves the phosphate wasting defect.
Exclusion
- malignancy within the last 5 years, except treated squamous or basal cell skin carcinoma
- terminal illness/hospice.
- severe end-organ disease, e.g. cardiovascular, pulmonary, etc, which may limit ability to complete study.
- estimated GFR \<45ml/min/1.73m2, calculated using MDRD formula for adults or modified Schwartz equation for children
- pregnancy or plan on becoming pregnant
Key Trial Info
Start Date :
August 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 12 2019
Estimated Enrollment :
8 Patients enrolled
Trial Details
Trial ID
NCT02233322
Start Date
August 1 2014
End Date
November 12 2019
Last Update
November 14 2019
Active Locations (1)
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1
Indiana University School of Medicine
Indianapolis, Indiana, United States, 46202