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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01277510




Registration number
NCT01277510
Ethics application status
Date submitted
13/01/2011
Date registered
17/01/2011
Date last updated
29/06/2020

Titles & IDs
Public title
Pediatric Chronic Kidney Disease Safety and Efficacy
Scientific 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
Secondary ID [1] 0 0
20070208
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Chronic Kidney Disease 0 0
Hyperparathyroidism 0 0
Hyperparathyroidism, Secondary 0 0
Kidney Disease 0 0
Secondary Hyperparathyroidism 0 0
Condition category
Condition code
Renal and Urogenital 0 0 0 0
Kidney disease
Renal and Urogenital 0 0 0 0
Other renal and urogenital disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - cinacalcet capsule
Treatment: Drugs - placebo
Treatment: Drugs - Standard of Care

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.

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.


Treatment: Drugs: cinacalcet capsule
Cinacalcet was prepared for oral administration as both capsules for sprinkling and film coated tablets for swallowing.

Treatment: Drugs: placebo
Placebo tablets and capsules for sprinkling identical to active treatment.

Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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.
Timepoint [1] 0 0
From Baseline to the Efficacy Assessment Phase, Weeks 25-30
Secondary outcome [1] 0 0
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.
Timepoint [1] 0 0
From Baseline to the Efficacy Assessment Phase (EAP), Weeks 25-30
Secondary outcome [2] 0 0
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."
Timepoint [2] 0 0
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
Secondary outcome [3] 0 0
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.
Timepoint [3] 0 0
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
Secondary outcome [4] 0 0
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.
Timepoint [4] 0 0
From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks
Secondary outcome [5] 0 0
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.
Timepoint [5] 0 0
From Baseline to end of Efficacy Assessment at Week 30
Secondary outcome [6] 0 0
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.
Timepoint [6] 0 0
End of double-blind phase (Week 30) until end of the open-label phase (Week 60)
Secondary outcome [7] 0 0
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.
Timepoint [7] 0 0
From Baseline to the Efficacy Assessment Phase, Weeks 25-30.

Eligibility
Key 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
Minimum age
6 Years
Maximum age
17 Years
Gender
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Terminated
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC
Recruitment hospital [1] 0 0
Research Site - Randwick
Recruitment hospital [2] 0 0
Research Site - Westmead
Recruitment hospital [3] 0 0
Research Site - Herston
Recruitment hospital [4] 0 0
Research Site - Parkville
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment postcode(s) [3] 0 0
4029 - Herston
Recruitment postcode(s) [4] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Massachusetts
Country [6] 0 0
United States of America
State/province [6] 0 0
Missouri
Country [7] 0 0
United States of America
State/province [7] 0 0
New Jersey
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
Texas
Country [14] 0 0
United States of America
State/province [14] 0 0
Virginia
Country [15] 0 0
Belgium
State/province [15] 0 0
Bruxelles
Country [16] 0 0
Belgium
State/province [16] 0 0
Edegem
Country [17] 0 0
Belgium
State/province [17] 0 0
Gent
Country [18] 0 0
Belgium
State/province [18] 0 0
Leuven
Country [19] 0 0
Germany
State/province [19] 0 0
Heidelberg
Country [20] 0 0
Germany
State/province [20] 0 0
Marburg
Country [21] 0 0
Hungary
State/province [21] 0 0
Budapest
Country [22] 0 0
Hungary
State/province [22] 0 0
Debrecen
Country [23] 0 0
Hungary
State/province [23] 0 0
Pecs
Country [24] 0 0
Hungary
State/province [24] 0 0
Szeged
Country [25] 0 0
Mexico
State/province [25] 0 0
Distrito Federal
Country [26] 0 0
Mexico
State/province [26] 0 0
Aguascalientes
Country [27] 0 0
Poland
State/province [27] 0 0
Gdansk
Country [28] 0 0
Poland
State/province [28] 0 0
Gorzow Wielkopolski
Country [29] 0 0
Poland
State/province [29] 0 0
Lodz
Country [30] 0 0
Poland
State/province [30] 0 0
Warszawa
Country [31] 0 0
Russian Federation
State/province [31] 0 0
Moscow
Country [32] 0 0
Russian Federation
State/province [32] 0 0
Saint Petersburg
Country [33] 0 0
Russian Federation
State/province [33] 0 0
Samara
Country [34] 0 0
Slovakia
State/province [34] 0 0
Banska Bystrica
Country [35] 0 0
Slovakia
State/province [35] 0 0
Bratislava
Country [36] 0 0
Slovakia
State/province [36] 0 0
Kosice
Country [37] 0 0
Spain
State/province [37] 0 0
Cataluña
Country [38] 0 0
Spain
State/province [38] 0 0
Cataluña
Country [39] 0 0
Spain
State/province [39] 0 0
Comunidad Valenciana
Country [40] 0 0
Spain
State/province [40] 0 0
PaÃ-s Vasco
Country [41] 0 0
Spain
State/province [41] 0 0
País Vasco
Country [42] 0 0
Spain
State/province [42] 0 0
Madrid

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Amgen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT01277510
Trial related presentations / publications
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.
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. Review.
Public notes

Contacts
Principal investigator
Name 0 0
MD
Address 0 0
Amgen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT01277510