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Trial record 1 of 1 for:    NCT03633708
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A Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis

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ClinicalTrials.gov Identifier: NCT03633708
Recruitment Status : Recruiting
First Posted : August 16, 2018
Last Update Posted : July 15, 2022
Sponsor:
Information provided by (Responsible Party):
Amgen

Brief Summary:
This is a Phase 3 Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis

Condition or disease Intervention/treatment Phase
Secondary Hyperparathyroidism Chronic Kidney Disease Drug: Etelcalcetide Phase 3

Detailed Description:

SHPT is a common and serious co-morbidity that develops relatively early in the course of CKD, worsens with declining kidney function, and is associated with serious complications in children on dialysis. Children on dialysis experience a wide spectrum of bone abnormalities and growth retardation, in addition to increased risk for cardiovascular morbidity and mortality that manifests early in their adulthood. Traditional therapies for SHPT (eg, vitamin D sterols) are widely used in the pediatric dialysis population, and have the potential to aggravate complications of the disease by increasing serum calcium (Ca), serum phosphorus, and serum Ca times serum phosphorus product.

Etelcalcetide has been shown to be safe and efficacious in treating adult CKD patients with SHPT by simultaneously controlling intact parathyroid hormone (iPTH), Ca, and phosphorus and has recently been approved for use in adult patients with SHPT treated with hemodialysis in both the United States and Europe. Although no previous studies have been conducted in pediatric patients with etelcalcetide (one single dose pharmacokinetic [PK] study is currently ongoing), Amgen anticipates minimal to moderate risk with a possibility of direct benefit to the pediatric subjects (age 28 days to 18 years) in this study. The burden of complications of SHPT in the pediatric dialysis population and the limitations of current standard therapy, underscore the need for studies of etelcalcetide in these patients to address this unmet medical need and inform the pediatric nephrology community of the potential use of etelcalcetide in children on hemodialysis with critical safety and efficacy data.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 56 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 3, Randomized, Open-label, Controlled, Multiple Dose, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Etelcalcetide in Pediatric Subjects 28 Days to < 18 Years of Age With Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving Maintenance Hemodialysis
Actual Study Start Date : April 29, 2019
Estimated Primary Completion Date : January 30, 2026
Estimated Study Completion Date : June 29, 2026

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Etelcalcetide
Randomized in a 3:1 ratio to receive etelcalcetide in addition to standard of care
Drug: Etelcalcetide
Etelcalcetide has been shown to be safe and efficacious in treating adult CKD patients with SHPT by simultaneously controlling intact parathyroid hormone (iPTH), Ca, and phosphorus and has recently been approved for use in adult patients with SHPT treated with hemodialysis in both the United States and Europe
Other Name: Parsabiv

Active Comparator: Control
Randomized in a 3:1 ratio to receive etelcalcetide in addition standard of care alone (control arm)
Drug: Etelcalcetide
Etelcalcetide has been shown to be safe and efficacious in treating adult CKD patients with SHPT by simultaneously controlling intact parathyroid hormone (iPTH), Ca, and phosphorus and has recently been approved for use in adult patients with SHPT treated with hemodialysis in both the United States and Europe
Other Name: Parsabiv




Primary Outcome Measures :
  1. Proportion of participants with ≥ 30% reduction from baseline in intact parathyroid hormone (iPTH) level during the efficacy assessment phase (EAP) [ Time Frame: Baseline and Weeks 20-27 ]
    Achievement of at least a 30% reduction from baseline in mean iPTH during the EAP (defined as weeks 20 through 27).


Secondary Outcome Measures :
  1. Maximum serum concentration (Cmax) of etelcalcetide [ Time Frame: 10-30 minutes post dose on Day 1 and 10-30 minutes post dose on Weeks 5, 9, 13, 17, and 21 ]
    Cmax will be collected and reported for the etelcalcetide arm only.

  2. Minimum serum concentration (Cmin) of etelcalcetide [ Time Frame: 10-30 minutes post dose on Day 1 and 10-30 minutes post dose on Weeks 5, 9, 13, 17, and 21 ]
    Cmin will be collected and reported for the etelcalcetide arm only.

  3. Incidence of adverse events [ Time Frame: Day 1 to 30 days after last dose of etelcalcetide (up to approximately 30 weeks) ]
  4. Frequency of hypocalcemia [ Time Frame: Up to approximately 30 Weeks ]
    Occurrence of hypocalcemia at any point in time, assessed by serum chemistry.

  5. Number of participants with corrected serum calcium levels at any time during the study [ Time Frame: Up to approximately 30 Weeks ]
    Number of participants achieving serum calcium levels <8.0 mg/dL (2.0 mmol/L) for participants 28 days to < 2 years and <8.6 mg/dL (2.15 mmol/L) for participants <2 years at any time during the study.

  6. Number of participants with serum phosphorous levels below normal for age [ Time Frame: Up to approximately 30 Weeks ]
  7. Number of participants with predialysis iPTH levels below normal [ Time Frame: Up to approximately 30 Weeks ]
  8. Change from baseline in systolic blood pressure [ Time Frame: Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 ]
  9. Change from baseline in diastolic blood pressure [ Time Frame: Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 ]
  10. Change from baseline in heart rate [ Time Frame: Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 ]
  11. Change in Tanner Stage [ Time Frame: Week -2 and Week 27 ]
  12. Change in height [ Time Frame: Day 1 and Week 27 ]
  13. Change in weight [ Time Frame: Week -2, Day 1, and Week 27 ]
  14. Achievement of ≥ 30% reduction in iPTH from baseline on two consecutive visits [ Time Frame: Up to approximately 30 Weeks ]
  15. Mean change from baseline in predialysis iPTH [ Time Frame: Baseline and Weeks 20-27 ]
    Mean change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).

  16. Percentage change from baseline in predialysis iPTH [ Time Frame: Baseline and Weeks 20-27 ]
    Percentage change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).

  17. Percentage change from baseline in corrected total serum calcium [ Time Frame: Baseline and Weeks 20-27 ]
    Percentage change from baseline in corrected total serum calcium during the EAP (defined as weeks 20 through 27).

  18. Percentage change from baseline in corrected total serum phosphorous [ Time Frame: Baseline and Weeks 20-27 ]
    Percentage change from baseline in corrected total serum phosphorous during the EAP (defined as weeks 20 through 27).



Information from the National Library of Medicine

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Ages Eligible for Study:   0 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion criteria

  • Dry weight ≥ 7 kg during screening.
  • Diagnosed with CKD and SHPT undergoing hemodialysis at the time of screening.
  • Diagnosis of SHPT with the mean of the 2 consecutive central laboratory iPTH values ≥ 400 pg/mL (42 pmol/L) during screening, on separate days and within 2 weeks of enrolment.
  • Serum cCa value ≥ 9.0 mg/dL (2.25 mmol/L) for subjects ≥ 2 years of age and older and serum cCa value ≥ 9.6 mg/dL (2.4 mmol/L) for subjects 28 days to < 2 years of age obtained from the central laboratory during screening.
  • Dialysate Ca level ≥ 2.5 mEq/L during screening.
  • SHPT not due to vitamin D deficiency, per investigator assessment.

Exclusion

  • Anticipated or scheduled parathyroidectomy or kidney transplant during the study period.
  • Subject has received a parathyroidectomy within 6 months prior to randomization.
  • Receipt of cinacalcet therapy within 30 days prior to screening assessments and through randomization.
  • Receipt of etelcalcetide within 6 months prior to screening assessments and through randomization.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03633708


Contacts
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Contact: Amgen Call Center 866-572-6436 medinfo@amgen.com

Locations
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Sponsors and Collaborators
Amgen
Investigators
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Study Director: MD Amgen
Additional Information:
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Responsible Party: Amgen
ClinicalTrials.gov Identifier: NCT03633708    
Other Study ID Numbers: 20140315
2017-002411-34 ( EudraCT Number )
First Posted: August 16, 2018    Key Record Dates
Last Update Posted: July 15, 2022
Last Verified: July 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the link below.
URL: http://www.amgen.com/datasharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Amgen:
sHPT
CKD
pediatric
Additional relevant MeSH terms:
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Neoplasm Metastasis
Kidney Diseases
Renal Insufficiency, Chronic
Hyperparathyroidism
Hyperparathyroidism, Secondary
Urologic Diseases
Neoplastic Processes
Neoplasms
Pathologic Processes
Renal Insufficiency
Parathyroid Diseases
Endocrine System Diseases