Clinical Trial Details
— Status: Completed
Administrative data
| NCT number |
NCT00195936 |
| Other study ID # |
05 02-027 |
| Secondary ID |
|
| Status |
Completed |
| Phase |
Phase 1
|
| First received |
|
| Last updated |
|
| Start date |
June 2005 |
| Est. completion date |
February 2008 |
Study information
| Verified date |
January 2021 |
| Source |
Children's Mercy Hospital Kansas City |
| Contact |
n/a |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Interventional
|
Clinical Trial Summary
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.
Description:
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.
