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Pediatric Chronic Kidney Disease Safety and Efficacy

Primary Purpose

Chronic Kidney Disease, Hyperparathyroidism, Hyperparathyroidism, Secondary

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
cinacalcet capsule
placebo
Standard of Care
Sponsored by
Amgen
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Kidney Disease focused on measuring dialysis, sensipar, mimpara, hemodialysis, peritoneal dialysis, renal, parathyroid hormone, pediatric

Eligibility Criteria

6 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • Age 6 to less than 18 years at screening
  • Diagnosed with CKD and SHPT receiving hemodialysis or peritoneal dialysis for ≥ 2 months before randomization
  • Dry weight ≥ 12.5 kg at screening
  • iPTH obtained from the central laboratory must be > 300 pg/mL (31.8 pmol/L)
  • Serum calcium (corrected) obtained from the central laboratory must be ≥ 8.8 mg/dL (2.2 mmol/L)
  • Serum phosphorus obtained from the central laboratory ≥ 4.0 mg/dL (1.3 mmol/L) for children 6 to less than 12 years old, or ≥ 3.5 mg/dL (1.1 mmol/L) for children 12 to less than 18 years old
  • Subjects already receiving vitamin D sterols (either calcitriol or a synthetic analog), a stable dose within the last 2 months prior to randomization
  • Subjects taking growth hormone, a stable dose defined as no change > than 20% in the last 2 months prior to randomization
  • Subjects on anti-convulsant medication must be on a stable dose for 3 months, and have a therapeutic blood level of the anti-convulsant at the time of randomization
  • Subjects must be on a dialysate calcium concentration of ≥ 2.5 mEq/L (1.25 mmol/L) for at least 2 months prior to randomization

Exclusion Criteria:

  • Underwent parathyroidectomy in the last 6 months
  • Anticipated parathyroidectomy within 6 months after randomization
  • Received therapy with cinacalcet (sensipar/mimpara) within the last month
  • A new onset of seizure or worsening of a pre-existing seizure disorder within the last 3 months
  • Scheduled date for kidney transplant from a known living donor that makes completion of the study unlikely

Sites / Locations

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Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

Cinacalcet

Arm Description

Participants received standard of care and placebo once daily for 30 weeks during the double-blind phase. During the open-label phase, participants received cinacalcet with standard of care for an additional 30 weeks. The starting dose of cinacalcet was ≤ 0.20 mg/kg based on dry weight, and could be titrated up according to plasma iPTH and serum calcium levels every 4 weeks up to Week 54 to a maximum dose of 4.2 mg/kg.

Participants received standard of care and cinacalcet once daily for 30 weeks during the double-blind phase. The starting dose of cinacalcet was ≤ 0.20 mg/kg based on dry weight, and could be titrated up according to plasma iPTH and serum calcium levels every 4 weeks until Week 24 to a maximum dose of 4.2 mg/kg. During the open-label phase participants continued to receive cinacalcet with standard of care for an additional 30 weeks.

Outcomes

Primary Outcome Measures

Percentage of Participants Achieving ≥ 30% Reduction in Mean iPTH From Baseline to the Efficacy Assessment Phase
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase (EAP; Weeks 25 - 30). When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available post-baseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.

Secondary Outcome Measures

Percentage of Participants Achieving Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During the Efficacy Assessment Phase
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Percent Change From Baseline in Mean Corrected Total Serum Calcium During the Efficacy Assessment Period
Serum calcium was reported as a corrected value by the central laboratory based on calcium and albumin concentrations: Corrected total calcium (mg/dL) = measured total serum calcium (mg/dL) + 0.8 (4.0 - Serum albumin (g/dL)). The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used."
Percent Change From Baseline in Mean Serum Phosphorus During the Efficacy Assessment Phase
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Percent Change From Baseline in Mean Phosphorous Product (Ca x P) During the Efficacy Assessment Phase
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Growth Velocity From Baseline to End of Double-blind Phase
Linear growth velocity (cm/year) = 52 x change in height (cm) / number of weeks between the two assessments. End of double-blind phase visit was at Week 30 by design but the last assessment in the double-blind phase was used due to the early termination of the study.
Growth Velocity From End of Double-blind Phase to End of Open-label Phase
Linear growth velocity (cm/year) = 52 x change in height (cm) / number of weeks between the two assessments. End of open-label phase visit was at Week 60 by design but the last assessment in the open-label phase was used due to the early termination of the study.
Percent Change From Baseline in Mean Ionized Calcium During the Efficacy Assessment Phase
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.

Full Information

First Posted
January 13, 2011
Last Updated
June 12, 2020
Sponsor
Amgen
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1. Study Identification

Unique Protocol Identification Number
NCT01277510
Brief Title
Pediatric Chronic Kidney Disease Safety and Efficacy
Official Title
A Randomized, Double-blind, Placebo Controlled Study to Assess the Efficacy and Safety of Cinacalcet HCl in Pediatric Subjects With Chronic Kidney Disease and Secondary Hyperparathyroidism Receiving Dialysis
Study Type
Interventional

2. Study Status

Record Verification Date
June 2020
Overall Recruitment Status
Terminated
Why Stopped
Study was put on clinical hold on 30 Jan 2013 following a subject fatality. Study was never restarted and was closed.
Study Start Date
June 28, 2011 (Actual)
Primary Completion Date
April 30, 2014 (Actual)
Study Completion Date
undefined (undefined)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amgen

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this study is to assess the safety and efficacy of adding cinacalcet to the current treatment of secondary hyperparathyroidism in children currently receiving dialysis compared to a treatment regimen that does not include cinacalcet.
Detailed Description
Secondary hyperparathyroidism (SHPT) is a condition that can develop early in patients with chronic kidney disease (CKD), usually gets worse over time, and is known to cause problems for patients on dialysis. Children on dialysis can have a wide range of bone and growth issues, and common treatments have a chance of making these things worse by increasing serum calcium and serum phosphorus. Cinacalcet has been shown to be effective in controlling parathyroid hormone (PTH), calcium and phosphorus in adults. The purpose of this study is to show that including cinacalcet in the treatment of SHPT will lower the levels of intact parathyroid hormone (iPTH) in a larger number of pediatric patients with CKD who are receiving dialysis, compared to a treatment regimen that does not include cinacalcet.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Kidney Disease, Hyperparathyroidism, Hyperparathyroidism, Secondary, Kidney Disease, Secondary Hyperparathyroidism
Keywords
dialysis, sensipar, mimpara, hemodialysis, peritoneal dialysis, renal, parathyroid hormone, pediatric

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
43 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received standard of care and placebo once daily for 30 weeks during the double-blind phase. During the open-label phase, participants received cinacalcet with standard of care for an additional 30 weeks. The starting dose of cinacalcet was ≤ 0.20 mg/kg based on dry weight, and could be titrated up according to plasma iPTH and serum calcium levels every 4 weeks up to Week 54 to a maximum dose of 4.2 mg/kg.
Arm Title
Cinacalcet
Arm Type
Experimental
Arm Description
Participants received standard of care and cinacalcet once daily for 30 weeks during the double-blind phase. The starting dose of cinacalcet was ≤ 0.20 mg/kg based on dry weight, and could be titrated up according to plasma iPTH and serum calcium levels every 4 weeks until Week 24 to a maximum dose of 4.2 mg/kg. During the open-label phase participants continued to receive cinacalcet with standard of care for an additional 30 weeks.
Intervention Type
Drug
Intervention Name(s)
cinacalcet capsule
Other Intervention Name(s)
Sensipar, Mimpara
Intervention Description
Cinacalcet was prepared for oral administration as both capsules for sprinkling and film coated tablets for swallowing.
Intervention Type
Drug
Intervention Name(s)
placebo
Intervention Description
Placebo tablets and capsules for sprinkling identical to active treatment.
Intervention Type
Drug
Intervention Name(s)
Standard of Care
Intervention Description
All participants, regardless of treatment assignment, will receive standard of care with vitamin D sterols (calcitriol and its analogs), as prescribed by the treating physician.
Primary Outcome Measure Information:
Title
Percentage of Participants Achieving ≥ 30% Reduction in Mean iPTH From Baseline to the Efficacy Assessment Phase
Description
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase (EAP; Weeks 25 - 30). When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available post-baseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Time Frame
From Baseline to the Efficacy Assessment Phase, Weeks 25-30
Secondary Outcome Measure Information:
Title
Percentage of Participants Achieving Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During the Efficacy Assessment Phase
Description
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Time Frame
From Baseline to the Efficacy Assessment Phase (EAP), Weeks 25-30
Title
Percent Change From Baseline in Mean Corrected Total Serum Calcium During the Efficacy Assessment Period
Description
Serum calcium was reported as a corrected value by the central laboratory based on calcium and albumin concentrations: Corrected total calcium (mg/dL) = measured total serum calcium (mg/dL) + 0.8 (4.0 - Serum albumin (g/dL)). The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used."
Time Frame
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
Title
Percent Change From Baseline in Mean Serum Phosphorus During the Efficacy Assessment Phase
Description
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Time Frame
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
Title
Percent Change From Baseline in Mean Phosphorous Product (Ca x P) During the Efficacy Assessment Phase
Description
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Time Frame
From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks
Title
Growth Velocity From Baseline to End of Double-blind Phase
Description
Linear growth velocity (cm/year) = 52 x change in height (cm) / number of weeks between the two assessments. End of double-blind phase visit was at Week 30 by design but the last assessment in the double-blind phase was used due to the early termination of the study.
Time Frame
From Baseline to end of Efficacy Assessment at Week 30
Title
Growth Velocity From End of Double-blind Phase to End of Open-label Phase
Description
Linear growth velocity (cm/year) = 52 x change in height (cm) / number of weeks between the two assessments. End of open-label phase visit was at Week 60 by design but the last assessment in the open-label phase was used due to the early termination of the study.
Time Frame
End of double-blind phase (Week 30) until end of the open-label phase (Week 60)
Title
Percent Change From Baseline in Mean Ionized Calcium During the Efficacy Assessment Phase
Description
The efficacy assessment value is based on the scheduled assessment(s) taken during the efficacy assessment phase. When multiple assessments were available, the average of those was used. If an efficacy measurement during the EAP was missing, the mean of the last 2 available postbaseline values in the dose-titration phase was used. If only one post-baseline value was available, this single value was used.
Time Frame
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Age 6 to less than 18 years at screening Diagnosed with CKD and SHPT receiving hemodialysis or peritoneal dialysis for ≥ 2 months before randomization Dry weight ≥ 12.5 kg at screening iPTH obtained from the central laboratory must be > 300 pg/mL (31.8 pmol/L) Serum calcium (corrected) obtained from the central laboratory must be ≥ 8.8 mg/dL (2.2 mmol/L) Serum phosphorus obtained from the central laboratory ≥ 4.0 mg/dL (1.3 mmol/L) for children 6 to less than 12 years old, or ≥ 3.5 mg/dL (1.1 mmol/L) for children 12 to less than 18 years old Subjects already receiving vitamin D sterols (either calcitriol or a synthetic analog), a stable dose within the last 2 months prior to randomization Subjects taking growth hormone, a stable dose defined as no change > than 20% in the last 2 months prior to randomization Subjects on anti-convulsant medication must be on a stable dose for 3 months, and have a therapeutic blood level of the anti-convulsant at the time of randomization Subjects must be on a dialysate calcium concentration of ≥ 2.5 mEq/L (1.25 mmol/L) for at least 2 months prior to randomization Exclusion Criteria: Underwent parathyroidectomy in the last 6 months Anticipated parathyroidectomy within 6 months after randomization Received therapy with cinacalcet (sensipar/mimpara) within the last month A new onset of seizure or worsening of a pre-existing seizure disorder within the last 3 months Scheduled date for kidney transplant from a known living donor that makes completion of the study unlikely
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MD
Organizational Affiliation
Amgen
Official's Role
Study Director
Facility Information:
Facility Name
Research Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Research Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Research Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Research Site
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Research Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Research Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Research Site
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Research Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Research Site
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Research Site
City
Livingston
State/Province
New Jersey
ZIP/Postal Code
07039
Country
United States
Facility Name
Research Site
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Research Site
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27834
Country
United States
Facility Name
Research Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Research Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97227
Country
United States
Facility Name
Research Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Research Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Research Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
Research Site
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22908
Country
United States
Facility Name
Research Site
City
Randwick
State/Province
New South Wales
ZIP/Postal Code
2031
Country
Australia
Facility Name
Research Site
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Research Site
City
Herston
State/Province
Queensland
ZIP/Postal Code
4029
Country
Australia
Facility Name
Research Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Research Site
City
Bruxelles
ZIP/Postal Code
1020
Country
Belgium
Facility Name
Research Site
City
Edegem
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Research Site
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
Research Site
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Research Site
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Research Site
City
Marburg
ZIP/Postal Code
35043
Country
Germany
Facility Name
Research Site
City
Budapest
ZIP/Postal Code
1083
Country
Hungary
Facility Name
Research Site
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
Research Site
City
Pecs
ZIP/Postal Code
7623
Country
Hungary
Facility Name
Research Site
City
Szeged
ZIP/Postal Code
6720
Country
Hungary
Facility Name
Research Site
City
Mexico
State/Province
Distrito Federal
ZIP/Postal Code
04530
Country
Mexico
Facility Name
Research Site
City
Aguascalientes
ZIP/Postal Code
20219
Country
Mexico
Facility Name
Research Site
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
Research Site
City
Gorzow Wielkopolski
ZIP/Postal Code
66-400
Country
Poland
Facility Name
Research Site
City
Lodz
ZIP/Postal Code
93-338
Country
Poland
Facility Name
Research Site
City
Warszawa
ZIP/Postal Code
00-576
Country
Poland
Facility Name
Research Site
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Research Site
City
Moscow
ZIP/Postal Code
107014
Country
Russian Federation
Facility Name
Research Site
City
Saint Petersburg
ZIP/Postal Code
198205
Country
Russian Federation
Facility Name
Research Site
City
Samara
ZIP/Postal Code
443095
Country
Russian Federation
Facility Name
Research Site
City
Banska Bystrica
ZIP/Postal Code
974 09
Country
Slovakia
Facility Name
Research Site
City
Bratislava
ZIP/Postal Code
833 40
Country
Slovakia
Facility Name
Research Site
City
Kosice
ZIP/Postal Code
040 11
Country
Slovakia
Facility Name
Research Site
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08035
Country
Spain
Facility Name
Research Site
City
Barcelona
State/Province
Cataluña
ZIP/Postal Code
08035
Country
Spain
Facility Name
Research Site
City
Valencia
State/Province
Comunidad Valenciana
ZIP/Postal Code
46026
Country
Spain
Facility Name
Research Site
City
Baracaldo
State/Province
PaÃ-s Vasco
ZIP/Postal Code
48903
Country
Spain
Facility Name
Research Site
City
Baracaldo
State/Province
País Vasco
ZIP/Postal Code
48903
Country
Spain
Facility Name
Research Site
City
Madrid
ZIP/Postal Code
28046
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
30506144
Citation
Warady BA, Iles JN, Ariceta G, Dehmel B, Hidalgo G, Jiang X, Laskin B, Shahinfar S, Vande Walle J, Schaefer F. A randomized, double-blind, placebo-controlled study to assess the efficacy and safety of cinacalcet in pediatric patients with chronic kidney disease and secondary hyperparathyroidism receiving dialysis. Pediatr Nephrol. 2019 Mar;34(3):475-486. doi: 10.1007/s00467-018-4116-y. Epub 2018 Nov 30.
Results Reference
background
PubMed Identifier
32367309
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.
Results Reference
background
Links:
URL
http://www.amgentrials.com
Description
AmgenTrials clinical trials website

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Pediatric Chronic Kidney Disease Safety and Efficacy

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