Text Size

Calcimimetics in Hypophosphatemic Rickets (CHR)

This study is enrolling participants by invitation only.
Sponsor:
Information provided by:
Children's Mercy Hospital Kansas City
ClinicalTrials.gov Identifier:
NCT00844740
First received: February 12, 2009
Last updated: February 13, 2009
Last verified: February 2009
History of Changes
  Purpose

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
Familial Hypophosphatemic Rickets
 Drug: Cinacalcet

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Effect of Cinacalcet on the Long-Term Treatment of Familial Hypophosphatemic Rickets

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Children's Mercy Hospital Kansas City:

Primary Outcome Measures:
  • doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries [ Time Frame: 9 months ] [ Designated as safety issue: Yes ]

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
Experimental: Cinacalcet
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.
 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.
Other Name: Sensipar

  Eligibility

Ages Eligible for Study:   5 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  1. Patients with XLH
  2. Age 5-21 years old
  3. From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months
  4. Who have not received Cinacalcet before

Exclusion Criteria:

  1. Treatment with growth hormone
  2. Inability to swallow pills.
  3. An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start.
  4. Pregnancy
  5. History of seizure disorder
  6. Abnormal liver functions (which may change the drug's AUC)
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00844740

Locations
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Sponsors and Collaborators
Children's Mercy Hospital Kansas City
  More Information

No publications provided

Responsible Party: Uri S. Alon, MD, Children's Mercy Hospital
ClinicalTrials.gov Identifier: NCT00844740     History of Changes
Other Study ID Numbers: 08 09-152
Study First Received: February 12, 2009
Last Updated: February 13, 2009
Health Authority: United States: Institutional Review Board

Keywords provided by Children's Mercy Hospital Kansas City:
Familial hypophosphatemic rickets
Cinacalcet
hyperparathyroidism

Additional relevant MeSH terms:
Rickets
Hypophosphatemic Rickets, X-Linked Dominant
Bone Diseases, Metabolic
Bone Diseases
Musculoskeletal Diseases
Calcium Metabolism Disorders
Metabolic Diseases
Vitamin D Deficiency
Avitaminosis
Deficiency Diseases
 Malnutrition
Nutrition Disorders
Hypophosphatemia, Familial
Renal Tubular Transport, Inborn Errors
Kidney Diseases
Urologic Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metal Metabolism, Inborn Errors
Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on June 18, 2013