Safety & Tolerability of Cinacalcet in Pediatric Patients With Chronic Kidney Disease and Secondary Hyperparathyroidism

Phase 2

Terminated

• Conditions: Hyperparathyroidism, Secondary; Chronic Kidney Disease
• Interventions: Drug: Cinacalcet hydrochloride; Drug: Standard of Care

• Registration Number: NCT01439867
• Lead Sponsor: Amgen

Brief Summary

The primary objective was to characterize corrected serum calcium levels on treatment with cinacalcet in pediatric patients with secondary hyperparathyroidism (HPT).

Detailed Description

This is a multicenter, 26-week, single-arm, open-label, safety study. Participants were to remain on study for 26 weeks or until time of kidney transplantation, whichever came first.

The study and enrollment was placed on partial clinical hold in February 2013 which resulted in changes to the protocol. The study was restarted in April 2014 following these changes.

Participants who completed the 26-week study or were on study when the study was closed in June 2016 were eligible to participate in an open-label extension study (Study 20140159; NCT02341417).

Study Timeline

• Study First Submitted: 2011-09-22
• Study First Submitted QC: 2011-09-22
• Study First Posted: 2011-09-23
• Study Start: 2012-06-22
• Primary Completion: 2016-06-03
• Study Completion: 2016-06-03
• Disposition First Submitted: 2017-04-25
• Disposition First Submitted QC: 2017-04-25
• Disposition First Posted: 2017-04-26
• Results First Submitted: 2017-07-13
• Results First Submitted QC: 2017-07-13
• Results First Posted: 2017-08-11
• Status Verified: 2020-06
• Last Update Submitted: 2020-06-12
• Last Update Posted: 2020-06-17

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 18

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Cinacalcet	Cinacalcet hydrochloride	Prior to the partial clinical hold, the starting dose was 0.25 mg/kg (based on dry weight) and was titrated upwards (maximum allowed daily dose of 4.2 mg/kg) based on plasma intact parathyroid hormone (iPTH), corrected serum calcium levels obtained monthly, and adverse signs and symptoms. After the partial clinical hold the starting dose was 0.20 mg/kg (based on dry weight) and was titrated upwards (maximum allowed daily dose of 2.5 or 60 mg, whichever was lower) based on plasma iPTH, corrected serum calcium levels obtained monthly, weekly monitoring of ionized calcium levels, and adverse signs and symptoms. All participants also received standard of care, which may have included vitamin D sterols.
Cinacalcet	Standard of Care	Prior to the partial clinical hold, the starting dose was 0.25 mg/kg (based on dry weight) and was titrated upwards (maximum allowed daily dose of 4.2 mg/kg) based on plasma intact parathyroid hormone (iPTH), corrected serum calcium levels obtained monthly, and adverse signs and symptoms. After the partial clinical hold the starting dose was 0.20 mg/kg (based on dry weight) and was titrated upwards (maximum allowed daily dose of 2.5 or 60 mg, whichever was lower) based on plasma iPTH, corrected serum calcium levels obtained monthly, weekly monitoring of ionized calcium levels, and adverse signs and symptoms. All participants also received standard of care, which may have included vitamin D sterols.

Primary Outcome Measures

Name	Time	Method
Percentage of Participants With Hypocalcemia	26 weeks	Hypocalcemia was defined as corrected serum calcium levels \< 9.0 mg/dL (2.25 mmol/L) for participants aged 28 days to \< 2 years, and \< 8.4 mg/dL (2.1 mmol/L) for participants aged ≥ 2 years to \< 6 years at any time during the study.

Secondary Outcome Measures

Name	Time	Method
Percentage of Participants Who Achieved > 30% Reduction in iPTH From Baseline at Any Two Consecutive Measurements	26 weeks	A participant was considered to have achieved \> 30% reduction in iPTH from baseline at any 2 consecutive measurements if percent change of any two consecutive post-baseline iPTH values were \< -30% regardless if there was a missing value in between.
Percentage of Participants Who Achieved ≥ 30% Reduction in iPTH From Baseline During the Study	26 weeks	A participant was considered to have achieved ≥ 30% reduction in iPTH if the percent change of any post-baseline iPTH value was ≤ -30% from baseline.
Percentage of Participants Who Achieved iPTH Values Between 200 and 300 pg/mL at Any Two Consecutive Measurements	26 weeks	A participant was considered to have achieved iPTH between 200 and 300 pg/mL (21.2 and 31.8 pmol/L) at any 2 consecutive measurements if any two consecutive post-baseline iPTH values were within the range regardless if there was a missing value in between. The analysis included all enrolled subjects with at least 1 post-baseline assessment.
Percent Change From Baseline in Intact Parathyroid Hormone (iPTH)	Baseline and weeks 3, 7, 11, 15, 19, 22, and 24
Percentage of Participants With Corrected Serum Calcium Levels < 8.8 mg/dL (2.2 mmol/L) During the Study	26 weeks
Percent Change From Baseline in Corrected Serum Calcium	Baseline and weeks 3, 7, 11, 15, 19, 22, and 24
Percent Change From Baseline in Serum Phosphorous	Baseline and weeks 3, 7, 11, 15, 19, 22, and 24
Percent Change From Baseline in Calcium Phosphorus Product (Ca x P)	Baseline and weeks 3, 7, 11, 15, 19, 22, and 24
Dose- and Weight-Normalized Maximum Plasma Concentration (Cmax) of Cinacalcet	Week 12
Percentage of Participants Who Achieved iPTH Values < 300 pg/mL During the Study	26 weeks	A participant was considered to have achieved iPTH \< 300 pg/mL (31.8 pmol/L) during the study if any post-baseline iPTH value was \< 300 pg/mL.
Dose- and Weight-Normalized Area Under the Plasma Concentration-time Curve From Time 0 to the Time of Last Quantifiable Concentration (AUClast) for Cinacalcet	Week 12

Trial Locations

Locations (1)

Research Site; 🇸🇰 Kosice, Slovakia