Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets
Primary Purpose
Hypophosphatemic Rickets, X-Linked Dominant
Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Cinacalcet
Sponsored by
About this trial
This is an interventional treatment trial for Hypophosphatemic Rickets, X-Linked Dominant focused on measuring Cinacalcet, PTH, XLH, children and adolescents with hypophosphatemic rickets (XLH)
Eligibility Criteria
Inclusion Criteria: Established patients with XLH Age 5 years old and above Normal serum calcium and creatinine concentrations Exclusion Criteria: Patients with hypersensitivity to any component(s) of cinacalcet Hypocalcaemia Elevated serum creatinine
Sites / Locations
- Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Overall Study
Arm Description
Calcimimetics as an Adjuvant Treatment for Familial Hypophosphatemic Rickets
Outcomes
Primary Outcome Measures
Change of serum Parathyroid Hormone (PTH) with use of cinacalcet
Secondary Outcome Measures
Change of mineral homeostasis; ionized calcium, total calcium, and phosphate with use of calcimimetic
Full Information
NCT ID
NCT00195936
First Posted
September 13, 2005
Last Updated
January 15, 2021
Sponsor
Children's Mercy Hospital Kansas City
1. Study Identification
Unique Protocol Identification Number
NCT00195936
Brief Title
Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets
Official Title
Effect of Calcimimetic (Cinacalcet) on Phosphate-Induced Hyperparathyroidism in Children With Hypophosphatemic Rickets
Study Type
Interventional
2. Study Status
Record Verification Date
January 2021
Overall Recruitment Status
Completed
Study Start Date
June 2005 (Actual)
Primary Completion Date
February 2008 (Actual)
Study Completion Date
February 2008 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Mercy Hospital Kansas City
4. Oversight
5. Study Description
Brief 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.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hypophosphatemic Rickets, X-Linked Dominant
Keywords
Cinacalcet, PTH, XLH, children and adolescents with hypophosphatemic rickets (XLH)
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Overall Study
Arm Type
Experimental
Arm Description
Calcimimetics as an Adjuvant Treatment for Familial Hypophosphatemic Rickets
Intervention Type
Drug
Intervention Name(s)
Cinacalcet
Intervention Description
Single oral dose of Cinacalcet for children with Hypophosphatemic Rickets
Primary Outcome Measure Information:
Title
Change of serum Parathyroid Hormone (PTH) with use of cinacalcet
Time Frame
Baseline, 30 minutes, and 4 hours post dose
Secondary Outcome Measure Information:
Title
Change of mineral homeostasis; ionized calcium, total calcium, and phosphate with use of calcimimetic
Time Frame
Baseline, 30 minutes, and 4 hours post dose
10. Eligibility
Sex
All
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Established patients with XLH
Age 5 years old and above
Normal serum calcium and creatinine concentrations
Exclusion Criteria:
Patients with hypersensitivity to any component(s) of cinacalcet
Hypocalcaemia
Elevated serum creatinine
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Rachel Levy-Olomucki, MD
Organizational Affiliation
Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics
Official's Role
Principal Investigator
Facility Information:
Facility Name
Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
12. IPD Sharing Statement
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Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets
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