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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: | NCT00195936 |
This study will measure the effect of cinacalcet (Sensipar) on parathyroid hormone (PTH) secretion in children and adolescents with hypophosphatemic rickets (XLH). The investigators are seeking evidence that patients with XLH may benefit from treatment with cinacalcet by achieving better control of PTH secretion.
Condition | Intervention | Phase |
---|---|---|
Hypophosphatemic Rickets, X-Linked Dominant |
Drug: Cinacalcet |
Phase I |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | Effect of Calcimimetic (Cinacalcet) on Phosphate-Induced Hyperparathyroidism in Children With Hypophosphatemic Rickets |
Study Start Date: | June 2005 |
X-linked hypophosphatemic rickets (XLH) is an X-linked dominant genetic disorder. Common findings are low serum phosphate and inadequate 1,25(OH)2 vitamin D production. It is generally believed that the primary defect in XLH is impaired renal tubular transport of phosphate coupled with abnormal regulation of the enzyme responsible for the 1-alfa hydroxylation of 25(OH) vitamin D. The current treatment of children with XLH is large oral doses of phosphate and 1,25-dihydroxyvitamin D. There are two common side effects to this treatment; nephrocalcinosis and secondary hyperparathyroidism (HPT). The latter at times may cause hypertension, hypercalcemia, and permanent renal damage. The complication of secondary hyperparathyroidism is seen in 20% of the patients. The release of PTH from the glands into the circulation is tightly regulated by serum calcium concentration. The glands "read" serum calcium concentration via Ca sensing receptors (CaR) which are located at the surface of the glands. Calcimimetics are compounds that allosterically modulate the CaR, thereby enhancing its sensitivity to circulating serum calcium concentrations and consequently decreasing PTH secretion. When used in primary HPT, they rapidly reduce PTH level and normalize serum calcium concentration.
Cinacalcet is a calcimimetic agent recently approved by the FDA for treating hypercalcemia in patients with parathyroid carcinoma and secondary HPT in patients with chronic renal disease. Cinacalcet was found to be effective in decreasing both PTH level and the calcium X phosphorous ion product in dialysis patients.
The goal of our proposed acute study is to see whether concomitant administration of Cinacalcet and phosphate, to patients with XLH, will block completely or partially secretion of PTH (day 2), expected to be seen following administration of phosphate alone (day 1). We will also monitor serum phosphate, total calcium, and ionized calcium concentration to learn to what extent, if any, blockage of PTH secretion affects mineral homeostasis under this condition.
If found to be effective in blocking PTH secretion, Cinacalcet will become a candidate for a long-term study in children with XLH to protect them from developing secondary hyperparathyroidism.
Ages Eligible for Study: | 5 Years and older |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contact: Rachel Levy-Olomucki, MD | 816-234-3010 | rlevy@cmh.edu |
United States, Missouri | |
Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics | Recruiting |
Kansas City, Missouri, United States, 64108 | |
Principal Investigator: Rachel Levy-Olomucki, MD |
Principal Investigator: | Rachel Levy-Olomucki, MD | Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics |
Study ID Numbers: | 05 02-027 |
Study First Received: | September 13, 2005 |
Last Updated: | October 22, 2007 |
ClinicalTrials.gov Identifier: | NCT00195936 History of Changes |
Health Authority: | United States: Institutional Review Board |
Cinacalcet PTH XLH children and adolescents with hypophosphatemic rickets (XLH) |
Vitamin D Deficiency Metabolic Diseases Avitaminosis Bone Diseases, Metabolic Hypophosphatemic Rickets, X-Linked Dominant Hormones Bone Diseases 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 |