Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot Project.
Completed · Not applicable
Conditions studied: Autosomal Dominant Hypophosphatemic Rickets
In brief
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). ADHR is an inherited disorder in which the body makes too much FGF 23 and causes low blood phosphorus levels and bone problems such as rickets (bowed legs in children) or bone pain and weakness in adults. This study is to test whether or not giving iron helps correct the high FGF23 and there by correcting the phosphate problem.
Key facts
- Study ID
- NCT02233322
- Run by
- Indiana University
- People needed
- 8
- Starts
- 2014-08-01
- Expected to finish
- 2019-11-12
- Last updated by the study team
- 2019-11-14
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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.
You may not qualify if…
- 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
Where it is running
- Indiana University School of Medicine — Indianapolis, Indiana, United States
Full record on ClinicalTrials.gov
Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.