Calcimimetics in Hypophosphatemic Rickets
Information source: Children's Mercy Hospital Kansas City
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Familial Hypophosphatemic Rickets
Intervention: Cinacalcet (Drug)
Phase: N/A
Status: Enrolling by invitation
Sponsored by: Children's Mercy Hospital Kansas City
Summary
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.
Clinical Details
Official title: Effect of Cinacalcet on the Long-Term Treatment of Familial Hypophosphatemic Rickets
Study design: Allocation: Non-Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
Eligibility
Minimum age: 5 Years.
Maximum age: 21 Years.
Gender(s): Both.
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)
Locations and Contacts
Children's Mercy Hospital, Kansas City, Missouri 64108, United States
Additional Information
Starting date: February 2009
Last updated: February 13, 2009
|