Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00195936
Recruitment Status : Completed
First Posted : September 20, 2005
Last Update Posted : January 19, 2021
Sponsor:
Information provided by (Responsible Party):
Children's Mercy Hospital Kansas City

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.

Condition or disease Intervention/treatment Phase
Hypophosphatemic Rickets, X-Linked Dominant Drug: Cinacalcet Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Effect of Calcimimetic (Cinacalcet) on Phosphate-Induced Hyperparathyroidism in Children With Hypophosphatemic Rickets
Actual Study Start Date : June 2005
Actual Primary Completion Date : February 2008
Actual Study Completion Date : February 2008


Arm Intervention/treatment
Experimental: Overall Study
Calcimimetics as an Adjuvant Treatment for Familial Hypophosphatemic Rickets
Drug: Cinacalcet
Single oral dose of Cinacalcet for children with Hypophosphatemic Rickets




Primary Outcome Measures :
  1. Change of serum Parathyroid Hormone (PTH) with use of cinacalcet [ Time Frame: Baseline, 30 minutes, and 4 hours post dose ]

Secondary Outcome Measures :
  1. Change of mineral homeostasis; ionized calcium, total calcium, and phosphate with use of calcimimetic [ Time Frame: Baseline, 30 minutes, and 4 hours post dose ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00195936


Locations
Layout table for location information
United States, Missouri
Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics
Kansas City, Missouri, United States, 64108
Sponsors and Collaborators
Children's Mercy Hospital Kansas City
Investigators
Layout table for investigator information
Principal Investigator: Rachel Levy-Olomucki, MD Section of Pediatric Nephrology, Children's Mercy Hospitals and Clinics
Layout table for additonal information
Responsible Party: Children's Mercy Hospital Kansas City
ClinicalTrials.gov Identifier: NCT00195936    
Other Study ID Numbers: 05 02-027
First Posted: September 20, 2005    Key Record Dates
Last Update Posted: January 19, 2021
Last Verified: January 2021
Keywords provided by Children's Mercy Hospital Kansas City:
Cinacalcet
PTH
XLH
children and adolescents with hypophosphatemic rickets (XLH)
Additional relevant MeSH terms:
Layout table for MeSH terms
Rickets
Rickets, Hypophosphatemic
Familial Hypophosphatemic Rickets
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Metabolic Diseases
Calcium Metabolism Disorders
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
Malnutrition
Nutrition Disorders
Hypophosphatemia
Phosphorus Metabolism Disorders
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Cinacalcet
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Calcimimetic Agents
Hormone Antagonists
Hormones, Hormone Substitutes, and Hormone Antagonists