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Sponsored by: |
Children's Mercy Hospital Kansas City |
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Information provided by: | Children's Mercy Hospital Kansas City |
ClinicalTrials.gov Identifier: | NCT00844740 |
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.
Condition | Intervention |
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Familial Hypophosphatemic Rickets |
Drug: Cinacalcet |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study |
Official Title: | Effect of Cinacalcet on the Long-Term Treatment of Familial Hypophosphatemic Rickets |
Estimated Enrollment: | 10 |
Study Start Date: | February 2009 |
Estimated Primary Completion Date: | June 2010 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Cinacalcet: Experimental
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.
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Drug: Cinacalcet
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.
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Ages Eligible for Study: | 5 Years to 21 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria:
Exclusion Criteria:
Responsible Party: | Children's Mercy Hospital ( Uri S. Alon, MD ) |
Study ID Numbers: | 08 09-152 |
Study First Received: | February 12, 2009 |
Last Updated: | February 13, 2009 |
ClinicalTrials.gov Identifier: | NCT00844740 History of Changes |
Health Authority: | United States: Institutional Review Board |
Familial hypophosphatemic rickets Cinacalcet hyperparathyroidism |
Vitamin D Deficiency Metabolic Diseases Avitaminosis Bone Diseases, Metabolic Hypophosphatemic Rickets, X-Linked Dominant Bone Diseases Calcitriol Vitamin D Resistant Rickets Metabolism, Inborn Errors Malnutrition Genetic Diseases, Inborn Urologic Diseases |
Hyperparathyroidism Musculoskeletal Diseases Rickets Hypophosphatemic Rickets Genetic Diseases, X-Linked Hypophosphatemia, Familial Hypophosphatemia Nutrition Disorders Kidney Diseases Metabolic Disorder Deficiency Diseases |
Vitamin D Deficiency Metabolic Diseases Avitaminosis Bone Diseases, Metabolic Renal Tubular Transport, Inborn Errors Hypophosphatemic Rickets, X-Linked Dominant Metal Metabolism, Inborn Errors Bone Diseases Calcium Metabolism Disorders Metabolism, Inborn Errors |
Malnutrition Genetic Diseases, Inborn Urologic Diseases Musculoskeletal Diseases Rickets Genetic Diseases, X-Linked Hypophosphatemia, Familial Nutrition Disorders Kidney Diseases Deficiency Diseases |