Autosomal Dominant Hypophosphatemic Rickets Clinical Trial
Official title:
Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot
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.
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 mcg/dl.
The study will look to see if there is a decrease of FGF23 level. It will also look at how
long does it take to decrease the level of FGF 23 and how long it takes for the serum and
urine phosphate to normalize.
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