Clinical Trial: Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot Project.
Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional
Official Title: Iron Therapy for Autosomal Dominant Hypophosphatemic Rickets: A Pilot
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). 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.
Detailed Summary:
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.
Sponsor: Indiana University
Current Primary Outcome: Does increasing serum iron concentrations above 100 mcg/dl in patients with ADHR result in a decrease in intact FGF23. [ Time Frame: FGF23 will be measured at 1, 2, 3, 6, 9, and 12 months ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
Original Secondary Outcome:
Information By: Indiana University
Dates:
Date Received: August 27, 2014
Date Started: August 2014
Date Completion: September 2018
Last Updated: April 12, 2016
Last Verified: April 2016