Cinacalcet for Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia (Hypophosphatemic Rickets)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01748812
• Date of registration: 11/12/2012
• Prospective Registration: No
• Primary sponsor: National Institute of Dental and Craniofacial Research (NIDCR)
• Public title: Cinacalcet for Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia (Hypophosphatemic Rickets)
• Scientific title: Open-label Dose-titration Study of the Tolerability and Efficacy of Cinacalcet to Treat Fibroblast Growth Factor 23 (FGF23)-Mediated Hypophosphatemia
• Date of first enrolment: November 16, 2012
• Target sample size: 1
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01748812
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Contacts

• Name: Rachel I Gafni, M.D.
• Affiliation: National Institute of Dental and Craniofacial Research (NIDCR)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

1. Chronological age: 18-70 years

2. Diagnosis of a genetic form of FGF23-mediated hypophosphatemia:

1. X-linked hypophosphatemic rickets (XLH)

2. Autosomal dominant hypophosphatemic rickets (ADHR)

3. Autosomal recessive hypophosphatemic rickets (ARHR)

Or, diagnosis of a non-genetic form of FGF23-mediated hypophosphatemia, i.e.
tumor-induced osteomalacia (TIO)

3. Ability to understand and provide informed consent

4. Ability to complete the protocol scheduled assessments and medication
regimen

5. Women of child-bearing potential (not surgically sterile via tubal ligation,
bilateral oophorectomy or hysterectomy, or who are not postmenopausal for greater
than or equal to 1 year) must agree to practice adequate contraception that may
include, but is not limited to, abstinence, monogamous relationship with
vasectomized partner, barrier methods such as condoms, diaphragms, spermicides,
intrauterine devices, and licensed hormonal methods for the duration of the
treatment portion of the study.

EXCLUSION CRITERIA:

1. Chronic or recurrent hypocalcemia defined by a serum calcium < 8.4 mg/dL (2.1
mmol/L)

2. Tertiary hyperparathyroidism as evidenced by concurrent PTH and calcium levels
above the upper limit of normal

3. History of parathyroid surgery and/or hypoparathyroidism

4. Hypercalciuria as defined as > 4 mg/kg/day (0.1 mmol/kg/day) on optimized
conventional therapy (as determined during SOC optimization phase)

5. Moderate to severe hepatic insufficiency as defined by total bilirubin > 2 mg/dL
and serum albumin < 3 g/dL and International Normalized Ratio (INR) >2 OR presence of
ascites or hepatic encephalopathy.

6. A calculated eGFR < 50 mL/min/1.73 m(2), using the CKD-EPI equation

7. History of a non-febrile seizure disorder

8. History of a clinically significant cardiac arrhythmia

9. History of chronic gastrointestinal disease

10. Current therapy (at the time of informed consent) bisphosphonates, calcitonin,
diuretics or medications that may have a significant drug interaction with cinacalcet

11. Known hypersensitivity to cinacalcet or any of its constituents

12. Positive pregnancy test or lactation

13. Use of another investigational agent (i.e., in the context of a clinical trial,
use of an investigational product that may have impact on the study) within the last 3
months



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Osteomalacia

Intervention(s)

Drug: Osteomalacia

Primary Outcome(s)

Evaluate the tolerability of cinacalcet in individuals with FGF23-mediated hypophosphatemia [Time Frame: 4 years]

Secondary ID(s)

13-D-0025

130025

Source(s) of Monetary Support

Please refer to primary and secondary sponsors