Clinical trial NCT01277510: 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
Overall status: TERMINATED
Phase: PHASE3
Conditions: Chronic Kidney Disease
Hyperparathyroidism
Hyperparathyroidism, Secondary
Kidney Disease
Secondary Hyperparathyroidism
Keywords: dialysis
sensipar
mimpara
hemodialysis
peritoneal dialysis
renal
parathyroid hormone
pediatric
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2011-01-17
Enrollment: 43
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 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.

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

Primary outcomes:
measure: Percentage of Participants Achieving ≥ 30% Reduction in Mean iPTH From Baseline to the Efficacy Assessment Phase
timeFrame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30
Secondary outcomes:
measure: Percentage of Participants Achieving Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During the Efficacy Assessment Phase
timeFrame: From Baseline to the Efficacy Assessment Phase (EAP), Weeks 25-30
measure: Percent Change From Baseline in Mean Corrected Total Serum Calcium During the Efficacy Assessment Period
timeFrame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
measure: Percent Change From Baseline in Mean Serum Phosphorus During the Efficacy Assessment Phase
timeFrame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
measure: Percent Change From Baseline in Mean Phosphorous Product (Ca x P) During the Efficacy Assessment Phase
timeFrame: From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks
measure: Growth Velocity From Baseline to End of Double-blind Phase
timeFrame: From Baseline to end of Efficacy Assessment at Week 30
measure: Growth Velocity From End of Double-blind Phase to End of Open-label Phase
timeFrame: End of double-blind phase (Week 30) until end of the open-label phase (Week 60)
measure: Percent Change From Baseline in Mean Ionized Calcium During the Efficacy Assessment Phase
timeFrame: From Baseline to the Efficacy Assessment Phase, Weeks 25-30.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Research Site
state: Alabama
city: Los Angeles
country: United States
facility: Research Site
state: California
city: San Francisco
country: United States
facility: Research Site
state: California
city: Gainesville
country: United States
facility: Research Site
state: Florida
city: Baltimore
country: United States
facility: Research Site
state: Maryland
city: Boston
country: United States
facility: Research Site
state: Massachusetts
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: Livingston
country: United States
facility: Research Site
state: New Jersey
city: Bronx
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: Portland
country: United States
facility: Research Site
state: Oregon
city: Philadelphia
country: United States
facility: Research Site
state: Pennsylvania
city: Houston
country: United States
facility: Research Site
state: Texas
city: San Antonio
country: United States
facility: Research Site
state: Texas
city: Charlottesville
country: United States
facility: Research Site
state: Virginia
city: Randwick
country: Australia
facility: Research Site
state: New South Wales
city: Westmead
country: Australia
facility: Research Site
state: New South Wales
city: Herston
country: Australia
facility: Research Site
state: Queensland
city: Parkville
country: Australia
facility: Research Site
state: Victoria
city: Bruxelles
country: Belgium
facility: Research Site
city: Edegem
country: Belgium
facility: Research Site
city: Gent
country: Belgium
facility: Research Site
city: Leuven
country: Belgium
facility: Research Site
city: Heidelberg
country: Germany
facility: Research Site
city: Marburg
country: Germany
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Debrecen
country: Hungary
facility: Research Site
city: Pecs
country: Hungary
facility: Research Site
city: Szeged
country: Hungary
facility: Research Site
city: Mexico
country: Mexico
facility: Research Site
state: Distrito Federal
city: Aguascalientes
country: Mexico
facility: Research Site
city: Gdansk
country: Poland
facility: Research Site
city: Gorzow Wielkopolski
country: Poland
facility: Research Site
city: Lodz
country: Poland
facility: Research Site
city: Warszawa
country: Poland
facility: Research Site
city: Warszawa
country: Poland
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: Banska Bystrica
country: Slovakia
facility: Research Site
city: Bratislava
country: Slovakia
facility: Research Site
city: Kosice
country: Slovakia
facility: Research Site
city: Barcelona
country: Spain
facility: Research Site
state: CataluÃ±a
city: Barcelona
country: Spain
facility: Research Site
state: Cataluña
city: Valencia
country: Spain
facility: Research Site
state: Comunidad Valenciana
city: Baracaldo
country: Spain
facility: Research Site
state: PaÃ-s Vasco
city: Baracaldo
country: Spain
facility: Research Site
state: País Vasco
city: Madrid
country: Spain
facility: Research Site
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References:
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.
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.