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Pediatric Chronic Kidney Disease Safety and Efficacy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01277510
Recruitment Status : Terminated (Study was suspended in agreement between sponsor and FDA due to concerns about the study design after a fatality had occurred in the presence of hypocalcemia.)
First Posted : January 17, 2011
Results First Posted : May 15, 2015
Last Update Posted : February 4, 2019
Sponsor:
Information provided by (Responsible Party):
Amgen

Tracking Information
First Submitted Date  ICMJE January 13, 2011
First Posted Date  ICMJE January 17, 2011
Results First Submitted Date  ICMJE April 29, 2015
Results First Posted Date  ICMJE May 15, 2015
Last Update Posted Date February 4, 2019
Study Start Date  ICMJE June 2011
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 29, 2015)
Percentage of Participants Achieving ≥ 30% Reduction in Mean iPTH From Baseline to the Efficacy Assessment Phase [ Time Frame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30 ]
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.
Original Primary Outcome Measures  ICMJE
 (submitted: January 13, 2011)
To demonstrate the efficacy of cinacalcet for reducing the plasma intact parathyroid hormone level (iPTH) by ≥ 30% [ Time Frame: From baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
Change History Complete list of historical versions of study NCT01277510 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 29, 2015)
  • Percentage of Participants Achieving Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During the Efficacy Assessment Phase [ Time Frame: From Baseline to the Efficacy Assessment Phase (EAP), Weeks 25-30 ]
    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 [ Time Frame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30. ]
    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 [ Time Frame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30. ]
    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 [ Time Frame: From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
    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 [ Time Frame: From Baseline to end of Efficacy Assessment at Week 30 ]
    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 [ Time Frame: End of double-blind phase (Week 30) until end of the open-label phase (Week 60) ]
    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 [ Time Frame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30. ]
    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.
Original Secondary Outcome Measures  ICMJE
 (submitted: January 13, 2011)
  • To demonstrate the impact of cinacalcet on corrected total serum calcium level [ Time Frame: From baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
  • To demonstrate the impact of cinacalcet on serum phosphorus level [ Time Frame: From baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
  • To demonstrate the impact of cinacalcet on the calcium x phosphorus product (Ca x P) [ Time Frame: From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
  • Evaluate number of participants with adverse events, and frequency of episodes of hypocalcemia [ Time Frame: Throughout entire treatment period, up to 60 weeks ]
  • To demonstrate the efficacy of cinacalcet for lowering the plasma iPTH level to ≤ 300 pg/mL (31.8 pmol/L) [ Time Frame: From baseline to end of Efficacy Assessment Period, assessed up to 30 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Pediatric Chronic Kidney Disease Safety and Efficacy
Official Title  ICMJE 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
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Chronic Kidney Disease
  • Hyperparathyroidism
  • Hyperparathyroidism, Secondary
  • Kidney Disease
  • Secondary Hyperparathyroidism
Intervention  ICMJE
  • Drug: cinacalcet capsule
    Cinacalcet was prepared for oral administration as both capsules for sprinkling and film coated tablets for swallowing.
    Other Name: Sensipar, Mimpara
  • Drug: placebo
    Placebo tablets and capsules for sprinkling identical to active treatment.
  • Drug: Standard of Care
    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.
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    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.
    Interventions:
    • Drug: placebo
    • Drug: Standard of Care
  • Experimental: Cinacalcet
    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.
    Interventions:
    • Drug: cinacalcet capsule
    • Drug: Standard of Care
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: September 12, 2014)
43
Original Estimated Enrollment  ICMJE
 (submitted: January 13, 2011)
100
Actual Study Completion Date  ICMJE April 2014
Actual Primary Completion Date April 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 17 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   Germany,   Hungary,   Mexico,   Poland,   Russian Federation,   Slovakia,   Spain,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01277510
Other Study ID Numbers  ICMJE 20070208
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Amgen
Study Sponsor  ICMJE Amgen
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: MD Amgen
PRS Account Amgen
Verification Date May 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP