Pediatric Chronic Kidney Disease Safety and Efficacy

NCT01277510 | Terminated Phase 3 Results DMC

• 1 other identifier: 20070208
• Study Type: interventional
• Target: 43
• Locations: 10 countries
• Sites: 51

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.

Conditions

Chronic Kidney Disease; Hyperparathyroidism; Hyperparathyroidism, Secondary; Kidney Disease; Secondary Hyperparathyroidism

Keywords

dialysis; sensipar; mimpara; hemodialysis; peritoneal dialysis; renal; parathyroid hormone; pediatric

Outcome Measures

Primary Outcomes (1)

• 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.

  From Baseline to the Efficacy Assessment Phase, Weeks 25-30

Secondary Outcomes (7)

• Percentage of Participants Achieving Mean iPTH ≤ 300 pg/mL (31.8 Pmol/L) During the Efficacy Assessment Phase

  From Baseline to the Efficacy Assessment Phase (EAP), Weeks 25-30

• Percent Change From Baseline in Mean Corrected Total Serum Calcium During the Efficacy Assessment Period

  From Baseline to the Efficacy Assessment Phase, Weeks 25-30.

• Percent Change From Baseline in Mean Serum Phosphorus During the Efficacy Assessment Phase

  From Baseline to the Efficacy Assessment Phase, Weeks 25-30.

• Percent Change From Baseline in Mean Phosphorous Product (Ca x P) During the Efficacy Assessment Phase

  From Baseline to end of Efficacy Assessment Period, assessed up to 30 weeks

• Growth Velocity From Baseline to End of Double-blind Phase

  From Baseline to end of Efficacy Assessment at Week 30

Study Arms (2)

Placebo

PLACEBO COMPARATOR

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.

Drug: placebo; Drug: Standard of Care

Cinacalcet

EXPERIMENTAL

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.

Drug: cinacalcet capsule; Drug: Standard of Care

Interventions

cinacalcet capsule DRUG

Cinacalcet was prepared for oral administration as both capsules for sprinkling and film coated tablets for swallowing.

Also known as: Sensipar, Mimpara

Cinacalcet

placebo DRUG

Placebo tablets and capsules for sprinkling identical to active treatment.

Placebo

Standard of Care DRUG

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.

Cinacalcet; Placebo

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Amgen: lead

Study Sites (51)

Research Site

Birmingham, Alabama, 35233, United States

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Los Angeles, California, 90095, United States

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San Francisco, California, 94143, United States

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Gainesville, Florida, 32610, United States

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Baltimore, Maryland, 21287, United States

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Boston, Massachusetts, 02115, United States

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Kansas City, Missouri, 64108, United States

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St Louis, Missouri, 63104, United States

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St Louis, Missouri, 63110, United States

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Livingston, New Jersey, 07039, United States

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The Bronx, New York, 10467, United States

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Greenville, North Carolina, 27834, United States

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Cincinnati, Ohio, 45229, United States

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Portland, Oregon, 97227, United States

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Philadelphia, Pennsylvania, 19104, United States

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Houston, Texas, 77030, United States

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San Antonio, Texas, 78229, United States

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Charlottesville, Virginia, 22908, United States

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Randwick, New South Wales, 2031, Australia

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Westmead, New South Wales, 2145, Australia

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Herston, Queensland, 4029, Australia

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Parkville, Victoria, 3052, Australia

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Brussels, 1020, Belgium

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Edegem, 2650, Belgium

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Ghent, 9000, Belgium

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Leuven, 3000, Belgium

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Heidelberg, 69120, Germany

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Marburg, 35043, Germany

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Budapest, 1083, Hungary

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Debrecen, 4032, Hungary

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Pécs, 7623, Hungary

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Szeged, 6720, Hungary

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Mexico City, Mexico City, 04530, Mexico

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Aguascalientes, 20219, Mexico

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Gdansk, 80-952, Poland

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Gorzów Wielkopolski, 66-400, Poland

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Lodz, 93-338, Poland

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Warsaw, 00-576, Poland

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Warsaw, 04-730, Poland

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Moscow, 107014, Russia

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Saint Petersburg, 198205, Russia

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Samara, 443095, Russia

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Banská Bystrica, 974 09, Slovakia

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Bratislava, 833 40, Slovakia

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Košice, 040 11, Slovakia

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Barakaldo, Basque Country, 48903, Spain

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Barcelona, Catalonia, 08035, Spain

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Barcelona, Cataluña, 08035, Spain

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Barakaldo, PaÃ-s Vasco, 48903, Spain

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Valencia, Valencia, 46026, Spain

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Madrid, 28046, Spain

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Related Publications (2)

• 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.

  PMID: 30506144 BACKGROUND

• 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.

  PMID: 32367309 BACKGROUND

Related Links

• AmgenTrials clinical trials website

MeSH Terms

Conditions

Renal Insufficiency, Chronic; Hyperparathyroidism; Hyperparathyroidism, Secondary; Kidney Diseases

Interventions

Cinacalcet; Standard of Care

Condition Hierarchy (Ancestors)

Renal Insufficiency; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Parathyroid Diseases; Endocrine System Diseases

Intervention Hierarchy (Ancestors)

Naphthalenes; Polycyclic Aromatic Hydrocarbons; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals; Polycyclic Compounds; Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Limitations and Caveats

The study was terminated early with a smaller sample size. However, the study was still sufficiently powered for the double-blind phase. The data collected in the open-label phase is very sparse.

Results Point of Contact

• Title: Study Director
• Organization: Amgen Inc.

866-572-6436

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 13, 2011
• First Posted: January 17, 2011
• Study Start: June 28, 2011
• Primary Completion: April 30, 2014
• Last Updated: June 29, 2020
• Results First Posted: May 15, 2015

Record last verified: 2020-06

Locations

PL (5); BE (4); SL (3); HU (4); DE (2); US (18); AU (4); ES (6); RU (3); ME (2)