Calcimimetics in Hypophosphatemic Rickets
CHR
Effect of Cinacalcet on the Long-term Treatment of Familial Hypophosphatemic Rickets
1 other identifier
interventional
N/A
1 country
1
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
Started Mar 2009
Typical duration for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
February 12, 2009
CompletedFirst Posted
Study publicly available on registry
February 16, 2009
CompletedStudy Start
First participant enrolled
March 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2012
CompletedStudy Completion
Last participant's last visit for all outcomes
March 1, 2012
CompletedDecember 17, 2020
December 1, 2020
3 years
February 12, 2009
December 15, 2020
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
doses of phosphate and calcitriol required to sustain goals of blood and urine chemistries
9 months
Study Arms (1)
Cinacalcet
EXPERIMENTALStable 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.
Interventions
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.
Eligibility Criteria
You may qualify if:
- Patients with XLH
- Age 5-21 years old
- From the Renal and Endocrinology Clinics who have been in a stable clinical, biochemical and radiological condition for the preceding 9 months
- Who have not received Cinacalcet before
You may not qualify if:
- Treatment with growth hormone
- Inability to swallow pills.
- An orthopedic surgery or braces in the past 9 months, or scheduled within 9 months of start.
- Pregnancy
- History of seizure disorder
- Abnormal liver functions (which may change the drug's AUC)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
February 12, 2009
First Posted
February 16, 2009
Study Start
March 1, 2009
Primary Completion
March 1, 2012
Study Completion
March 1, 2012
Last Updated
December 17, 2020
Record last verified: 2020-12