Calcimimetics in Hypophosphatemic Rickets

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

Status

Withdrawn

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00844740

Acronym

CHR

Enrollment

Registered

2009-02-16

Start date

2009-03-01

Completion date

2012-03-01

Last updated

2020-12-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Familial Hypophosphatemic Rickets

Keywords

Familial hypophosphatemic rickets, Cinacalcet, hyperparathyroidism

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.

Interventions

DRUGCinacalcet

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.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

5 Years to 21 Years

Healthy volunteers

Yes

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)

Design outcomes

Primary

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

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026