Calcimimetics in Hypophosphatemic Rickets

Study Overview
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Brief Summary
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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.

Official Title
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Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets

Conditions
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Familial Hypophosphatemic Rickets

Intervention / Treatment
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* Drug: Cinacalcet


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with XLH Age 5-21 years old From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months Who have not received Cinacalcet before Exclusion Criteria: Treatment with growth hormone Inability to swallow pills. An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start. Pregnancy History of seizure disorder Abnormal liver functions (which may change the drug's AUC)

Ages Eligible for Study
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Minimum Age: 5 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Cinacalcet<br>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. | Drug: Cinacalcet<br>* 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.<br>* Other names: Sensipar;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries |  | 9 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Familial hypophosphatemic rickets, Cinacalcet, hyperparathyroidism