Calcimimetics in Hypophosphatemic Rickets (CHR)

December 15, 2020 updated by: Children's Mercy Hospital Kansas City

Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

Study Type

Interventional

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 years to 21 years (Child, Adult)

Accepts Healthy Volunteers

Yes

Genders Eligible for Study

All

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • Sensipar

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
Time Frame: 9 months
9 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Children's Mercy Hospital Kansas City

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

March 1, 2009

Primary Completion (Anticipated)

March 1, 2012

Study Completion (Anticipated)

March 1, 2012

Study Registration Dates

First Submitted

February 12, 2009

First Submitted That Met QC Criteria

February 13, 2009

First Posted (Estimate)

February 16, 2009

Study Record Updates

Last Update Posted (Actual)

December 17, 2020

Last Update Submitted That Met QC Criteria

December 15, 2020

Last Verified

December 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 08 09-152

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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