Clinical trial NCT02138838: Efficacy and Safety of Cinacalcet in Pediatric Patients With Secondary Hyperparathyroidism (SHPT) and Chronic Kidney Disease (CKD) on Dialysis
Official title: A Randomized, Open-label, Controlled Study to Assess the Efficacy and Safety of Cinacalcet HCl in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving Dialysis
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Chronic Kidney Disease, Secondary Hyperparathyroidism
Keywords: Chronic Kidney Disease, Secondary Hyperparathyroidism, Dialysis, Pediatric
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2014-05-15
Enrollment: 55
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The primary objective was to evaluate the efficacy of cinacalcet for reducing the plasma intact parathyroid hormone (iPTH) level by ≥ 30%.

Detailed description: This was a 24-week, randomized, multicenter, open-label, controlled study. Participants were randomized into one of two treatment arms; oral administration of cinacalcet daily in addition to standard of care treatment, or standard of care alone. Randomization was stratified by age group (6 to \< 12 and 12 to \< 18 years of age). All participants received standard of care which could include therapy with Vitamin D sterols, calcium supplementation, and phosphate binders.

Participants in both treatment groups who completed the 20-week treatment period and those who ended the study due to study closure were eligible to enroll in an open-label extension study (20140159; NCT02341417) for further safety follow-up.

Eligibility criteria: Inclusion Criteria:

* Age 6 - \< 18 years
* Diagnosis of SHPT with the mean of the two consecutive central laboratory iPTH values ≥ 300 pg/mL during screening
* Corrected calcium value of ≥ 8.8 mg/dL during screening
* Diagnosis of CKD, receiving either hemodialysis or peritoneal dialysis, for ≥ 30 days prior to screening
* Parent or legally acceptable representative has provided written informed consent and subject has provided written assent when required by institutional guidelines

Exclusion Criteria:

* History of congenital long QT syndrome, second or third degree heart block, ventricular tachyarrhythmias or other conditions associated with prolonged QT interval
* Corrected QT interval (QTc) \> 500 ms, using Bazett's formula
* QTc ≥ 450 to ≤ 500 ms, using Bazett's formula, unless written permission to enroll is provided by the investigator after consultation with a pediatric cardiologist
* Use of grapefruit juice, herbal medications, or potent cytochrome P450 3A4 (CYP3A4) inhibitors (eg, erythromycin, clarithromycin, ketoconazole, itraconazole)
* Use of concomitant medications that may prolong the QTc interval (eg, ondansetron, albuterol)

Primary outcomes:
measure: Percentage of Participants Who Achieved a ≥ 30% Reduction From Baseline In Mean Plasma iPTH During Weeks 11 to 15
timeFrame: Baseline and weeks 11 to 15
measure: Percentage of Participants Who Achieved a ≥ 30% Reduction From Baseline in Mean Plasma Intact Parathyroid Hormone During the Efficacy Assessment Period
timeFrame: Baseline and the efficacy assessment period (EAP), weeks 17 to 20
Secondary outcomes:
measure: Percentage of Participants Who Achieved a Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During Weeks 17 to 20
timeFrame: Efficacy assessment period, weeks 17 to 20
measure: Percent Change in iPTH From Baseline to the Mean Value During Weeks 17 to 20
timeFrame: Baseline and weeks 17 to 20
measure: Change in Corrected Serum Calcium From Baseline to the Mean Value During Weeks 17 to 20
timeFrame: Baseline and weeks 17 to 20
measure: Change in Serum Phosphorus From Baseline to the Mean Value During Weeks 17 to 20
timeFrame: Baseline and weeks 17 to 20
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Research Site
state: California
city: Wilmington
country: United States
facility: Research Site
state: Delaware
city: Atlanta
country: United States
facility: Research Site
state: Georgia
city: Chicago
country: United States
facility: Research Site
state: Illinois
city: Iowa City
country: United States
facility: Research Site
state: Iowa
city: Louisville
country: United States
facility: Research Site
state: Kentucky
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Boston
country: United States
facility: Research Site
state: Massachusetts
city: Detroit
country: United States
facility: Research Site
state: Michigan
city: Minneapolis
country: United States
facility: Research Site
state: Minnesota
city: Jackson
country: United States
facility: Research Site
state: Mississippi
city: Kansas City
country: United States
facility: Research Site
state: Missouri
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: Saint Louis
country: United States
facility: Research Site
state: Missouri
city: West Orange
country: United States
facility: Research Site
state: New Jersey
city: Bronx
country: United States
facility: Research Site
state: New York
city: New York
country: United States
facility: Research Site
state: New York
city: Greenville
country: United States
facility: Research Site
state: North Carolina
city: Cincinnati
country: United States
facility: Research Site
state: Ohio
city: Cleveland
country: United States
facility: Research Site
state: Ohio
city: Columbus
country: United States
facility: Research Site
state: Ohio
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Dallas
country: United States
facility: Research Site
state: Texas
city: Houston
country: United States
facility: Research Site
state: Texas
city: San Antonio
country: United States
facility: Research Site
state: Texas
city: Salt Lake City
country: United States
facility: Research Site
state: Utah
city: Bruxelles
country: Belgium
facility: Research Site
city: Gent
country: Belgium
facility: Research Site
city: Leuven
country: Belgium
facility: Research Site
city: Praha 5
country: Czechia
facility: Research Site
city: Bron cedex
country: France
facility: Research Site
city: Lille
country: France
facility: Research Site
city: Marseille cedex 05
country: France
facility: Research Site
city: Nice cedex 3
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Paris
country: France
facility: Research Site
city: Hannover
country: Germany
facility: Research Site
city: Heidelberg
country: Germany
facility: Research Site
city: Marburg
country: Germany
facility: Research Site
city: Athens
country: Greece
facility: Research Site
city: Thessaloniki
country: Greece
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Szeged
country: Hungary
facility: Research Site
city: Genova
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Torino
country: Italy
facility: Research Site
city: Vilinus
country: Lithuania
facility: Research Site
city: Grafton, Auckland
country: New Zealand
facility: Research Site
city: Krakow
country: Poland
facility: Research Site
city: Lodz
country: Poland
facility: Research Site
city: Warszawa
country: Poland
facility: Research Site
city: Porto
country: Portugal
facility: Research Site
city: Moscow
country: Russian Federation
facility: Research Site
city: Saint Petersburg
country: Russian Federation
facility: Research Site
city: Samara
country: Russian Federation
facility: Research Site
city: Kosice
country: Slovakia
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
state: Cataluña
city: Espluques De LLobregat
country: Spain
facility: Research Site
state: Cataluña
city: Kyiv
country: Ukraine
facility: Research Site
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
citation: Chen P, Sohn W, Narayanan A, Gisleskog PO, Melhem M. Bridging adults and paediatrics with secondary hyperparathyroidism receiving haemodialysis: a pharmacokinetic-pharmacodynamic analysis of cinacalcet. Br J Clin Pharmacol. 2019 Jun;85(6):1312-1325. doi: 10.1111/bcp.13900. Epub 2019 Apr 25.
citation: Warady BA, Ng E, Bloss L, Mo M, Schaefer F, Bacchetta J. Cinacalcet studies in pediatric subjects with secondary hyperparathyroidism receiving dialysis. Pediatr Nephrol. 2020 Sep;35(9):1679-1697. doi: 10.1007/s00467-020-04516-4. Epub 2020 May 4.