Calcimimetics in Hypophosphatemic Rickets (CHR)
Currently, large oral doses of phosphate and 1,25(OH)2D (calcitriol) are the standard treatment of patients with familial hypophosphatemic rickets (XLH). While this therapy is effective in healing the rickets, it is often limited by development of complications due to the high dose of medications required to achieve cure. Among them are the development of calcifications in the kidneys and secondary hyperparathyroidism (HPT) which in some patients may cause complications like high blood calcium level, high blood pressure and damage to the kidney. A drug to treat secondary hyperparathyroidism was just developed. In a short term study we found that it might help the treatment of XLH, by allowing the use of lower doses of the both phosphate and calcitriol. In the present study we will learn if indeed the addition of this new medicine (Cinacalcet) to the long-term treatment will allow the use of lower doses of both phosphate and calcitriol and consequently lower the risk of complications.
|Study Design:||Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Effect of Cinacalcet on the Long-Term Treatment of Familial Hypophosphatemic Rickets|
- doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]
|Study Start Date:||February 2009|
|Estimated Primary Completion Date:||June 2010 (Final data collection date for primary outcome measure)|
Stable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood and urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done.
Stable patients with XLH already treated with Phosphate and calcitriol will add Cinacalcet to their treatment regimen. Sequential monitoring of blood nand urine biochemical variables will follow, based on which adjustments to the doses of the 3 medications will be done.
Other Name: Sensipar
Please refer to this study by its ClinicalTrials.gov identifier: NCT00844740
|United States, Missouri|
|Children's Mercy Hospital|
|Kansas City, Missouri, United States, 64108|