Back to resultsA Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
Primary Purpose
Secondary Hyperparathyroidism, Chronic Kidney Disease
Status
Recruiting
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Etelcalcetide
Sponsored by
About this trial
This is an interventional treatment trial for Secondary Hyperparathyroidism focused on measuring sHPT, CKD, pediatric
Eligibility 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.
Sites / Locations
- Childrens Hospital of Los AngelesRecruiting
- Childrens Hospital Colorado
- Childrens Mercy HospitalRecruiting
- Mount Sinai Kidney Center - B1 Renal Treatment
- Cincinnati Childrens Hospital Medical CenterRecruiting
- Cleveland ClinicRecruiting
- The Childrens Hospital at Oklahoma University Medical Center
- Childrens Hospital of PhiladelphiaRecruiting
- Childrens Medical Center Dallas
- Primary Childrens Hospital Outpatient ServicesRecruiting
- Hospital Italiano de La PlataRecruiting
- Fresenius EscobarRecruiting
- Centro Infantil Del RinonRecruiting
- Fortis Flt Lt Rajan Dhall HospitalRecruiting
- All India Institute of Medical SciencesRecruiting
- Sir Ganga Ram HospitalRecruiting
- Manipal HospitalRecruiting
- KLES Dr Prabhakar Kore Hospital and Medical Research CentreRecruiting
- NRS Medical College and HospitalRecruiting
- Asan Medical CenterRecruiting
- Seoul National University HospitalRecruiting
- Pusan National University Yangsan HospitalRecruiting
- Hospital Raja Perempuan Zainab IIRecruiting
- Hospital TuanKu JaafarRecruiting
- Hospital Wanita Dan Kanak-Kanak Kuala LumpurRecruiting
- SBHI Pediatrics city clinical hospital of Saint VladimirRecruiting
- Municipal Children Hospital 1Recruiting
- State Budgetary Healthcare Institution Samara Regional Clinical Hospital na V D SeredavinRecruiting
- National University HospitalRecruiting
- Kaohsiung Veterans General HospitalRecruiting
- National Cheng Kung University HospitalRecruiting
- National Taiwan University HospitalRecruiting
- Linkou Chang Gung Memorial HospitalRecruiting
- Hacettepe Universitesi Tip Fakultesi Ihsan Dogramaci Cocuk HastanesiRecruiting
- Baskent Universitesi Ankara HastanesiRecruiting
- Gazi Universitesi Tip FakultesiRecruiting
- Istanbul Universitesi Cerrahpasa Tip FakultesiRecruiting
- Ege Universitesi Tip FakultesiRecruiting
- Erciyes Universitesi Tip Fakultesi Mustafa Eraslan ve Fevzi Mercan Cocuk HastanesiRecruiting
- National Childrens Specializated Hospital OkhmaditRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
Etelcalcetide
Control
Arm Description
Randomized in a 3:1 ratio to receive etelcalcetide in addition to standard of care
Randomized in a 3:1 ratio to receive etelcalcetide in addition standard of care alone (control arm)
Outcomes
Primary Outcome Measures
Proportion of participants with ≥ 30% reduction from baseline in intact parathyroid hormone (iPTH) level during the efficacy assessment phase (EAP)
Achievement of at least a 30% reduction from baseline in mean iPTH during the EAP (defined as weeks 20 through 27).
Secondary Outcome Measures
Maximum serum concentration (Cmax) of etelcalcetide
Cmax will be collected and reported for the etelcalcetide arm only.
Minimum serum concentration (Cmin) of etelcalcetide
Cmin will be collected and reported for the etelcalcetide arm only.
Incidence of adverse events
Frequency of hypocalcemia
Occurrence of hypocalcemia at any point in time, assessed by serum chemistry.
Number of participants with corrected serum calcium levels at any time during the study
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.
Number of participants with serum phosphorous levels below normal for age
Number of participants with predialysis iPTH levels below normal
Change from baseline in systolic blood pressure
Change from baseline in diastolic blood pressure
Change from baseline in heart rate
Change in Tanner Stage
Change in height
Change in weight
Achievement of ≥ 30% reduction in iPTH from baseline on two consecutive visits
Mean change from baseline in predialysis iPTH
Mean change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Percentage change from baseline in predialysis iPTH
Percentage change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Percentage change from baseline in corrected total serum calcium
Percentage change from baseline in corrected total serum calcium during the EAP (defined as weeks 20 through 27).
Percentage change from baseline in corrected total serum phosphorous
Percentage change from baseline in corrected total serum phosphorous during the EAP (defined as weeks 20 through 27).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03633708
Brief Title
A Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
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
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 29, 2019 (Actual)
Primary Completion Date
January 30, 2026 (Anticipated)
Study Completion Date
January 30, 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Amgen
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This is a Phase 3 Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Secondary Hyperparathyroidism, Chronic Kidney Disease
Keywords
sHPT, CKD, pediatric
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
56 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Etelcalcetide
Arm Type
Experimental
Arm Description
Randomized in a 3:1 ratio to receive etelcalcetide in addition to standard of care
Arm Title
Control
Arm Type
Active Comparator
Arm Description
Randomized in a 3:1 ratio to receive etelcalcetide in addition standard of care alone (control arm)
Intervention Type
Drug
Intervention Name(s)
Etelcalcetide
Other Intervention Name(s)
Parsabiv
Intervention Description
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
Primary Outcome Measure Information:
Title
Proportion of participants with ≥ 30% reduction from baseline in intact parathyroid hormone (iPTH) level during the efficacy assessment phase (EAP)
Description
Achievement of at least a 30% reduction from baseline in mean iPTH during the EAP (defined as weeks 20 through 27).
Time Frame
Baseline and Weeks 20-27
Secondary Outcome Measure Information:
Title
Maximum serum concentration (Cmax) of etelcalcetide
Description
Cmax will be collected and reported for the etelcalcetide arm only.
Time Frame
10-30 minutes post dose on Day 1 and 10-30 minutes post dose on Weeks 5, 9, 13, 17, and 21
Title
Minimum serum concentration (Cmin) of etelcalcetide
Description
Cmin will be collected and reported for the etelcalcetide arm only.
Time Frame
10-30 minutes post dose on Day 1 and 10-30 minutes post dose on Weeks 5, 9, 13, 17, and 21
Title
Incidence of adverse events
Time Frame
Day 1 to 30 days after last dose of etelcalcetide (up to approximately 30 weeks)
Title
Frequency of hypocalcemia
Description
Occurrence of hypocalcemia at any point in time, assessed by serum chemistry.
Time Frame
Up to approximately 30 Weeks
Title
Number of participants with corrected serum calcium levels at any time during the study
Description
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.
Time Frame
Up to approximately 30 Weeks
Title
Number of participants with serum phosphorous levels below normal for age
Time Frame
Up to approximately 30 Weeks
Title
Number of participants with predialysis iPTH levels below normal
Time Frame
Up to approximately 30 Weeks
Title
Change from baseline in systolic blood pressure
Time Frame
Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27
Title
Change from baseline in diastolic blood pressure
Time Frame
Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27
Title
Change from baseline in heart rate
Time Frame
Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27
Title
Change in Tanner Stage
Time Frame
Week -2 and Week 27
Title
Change in height
Time Frame
Day 1 and Week 27
Title
Change in weight
Time Frame
Week -2, Day 1, and Week 27
Title
Achievement of ≥ 30% reduction in iPTH from baseline on two consecutive visits
Time Frame
Up to approximately 30 Weeks
Title
Mean change from baseline in predialysis iPTH
Description
Mean change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Time Frame
Baseline and Weeks 20-27
Title
Percentage change from baseline in predialysis iPTH
Description
Percentage change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Time Frame
Baseline and Weeks 20-27
Title
Percentage change from baseline in corrected total serum calcium
Description
Percentage change from baseline in corrected total serum calcium during the EAP (defined as weeks 20 through 27).
Time Frame
Baseline and Weeks 20-27
Title
Percentage change from baseline in corrected total serum phosphorous
Description
Percentage change from baseline in corrected total serum phosphorous during the EAP (defined as weeks 20 through 27).
Time Frame
Baseline and Weeks 20-27
10. Eligibility
Sex
All
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Amgen Call Center
Phone
866-572-6436
Email
medinfo@amgen.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
MD
Organizational Affiliation
Amgen
Official's Role
Study Director
Facility Information:
Facility Name
Childrens Hospital of Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Individual Site Status
Recruiting
Facility Name
Childrens Hospital Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Terminated
Facility Name
Childrens Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Individual Site Status
Recruiting
Facility Name
Mount Sinai Kidney Center - B1 Renal Treatment
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Individual Site Status
Completed
Facility Name
Cincinnati Childrens Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Individual Site Status
Recruiting
Facility Name
Cleveland Clinic
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44195
Country
United States
Individual Site Status
Recruiting
Facility Name
The Childrens Hospital at Oklahoma University Medical Center
City
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
Country
United States
Individual Site Status
Completed
Facility Name
Childrens Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Name
Childrens Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Individual Site Status
Terminated
Facility Name
Primary Childrens Hospital Outpatient Services
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Individual Site Status
Recruiting
Facility Name
Hospital Italiano de La Plata
City
Cuidad Autonoma de Buenos Aires
State/Province
Buenos Aires
ZIP/Postal Code
C1199ABB
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Fresenius Escobar
City
Escobar
State/Province
Buenos Aires
ZIP/Postal Code
B1625DUG
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Centro Infantil Del Rinon
City
San Miguel de Tucuman
State/Province
Tucuman
ZIP/Postal Code
4000
Country
Argentina
Individual Site Status
Recruiting
Facility Name
Fortis Flt Lt Rajan Dhall Hospital
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110 070
Country
India
Individual Site Status
Recruiting
Facility Name
All India Institute of Medical Sciences
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110029
Country
India
Individual Site Status
Recruiting
Facility Name
Sir Ganga Ram Hospital
City
New Delhi
State/Province
Delhi
ZIP/Postal Code
110060
Country
India
Individual Site Status
Recruiting
Facility Name
Manipal Hospital
City
Bangalore
State/Province
Karnataka
ZIP/Postal Code
560 017
Country
India
Individual Site Status
Recruiting
Facility Name
KLES Dr Prabhakar Kore Hospital and Medical Research Centre
City
Belagavi
State/Province
Karnataka
ZIP/Postal Code
590010
Country
India
Individual Site Status
Recruiting
Facility Name
NRS Medical College and Hospital
City
Kolkata
State/Province
West Bengal
ZIP/Postal Code
700014
Country
India
Individual Site Status
Recruiting
Facility Name
Asan Medical Center
City
Seoul
ZIP/Postal Code
05505
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
110-744
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Pusan National University Yangsan Hospital
City
Yangsan-si, Gyeongsangnam-do
ZIP/Postal Code
50612
Country
Korea, Republic of
Individual Site Status
Recruiting
Facility Name
Hospital Raja Perempuan Zainab II
City
Kota Bharu
State/Province
Kelantan
ZIP/Postal Code
15586
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Hospital TuanKu Jaafar
City
Seremban
State/Province
Negri Sembilan
ZIP/Postal Code
70300
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
Hospital Wanita Dan Kanak-Kanak Kuala Lumpur
City
Kuala Lumpur
State/Province
Wilayah Persekutuan
ZIP/Postal Code
50300
Country
Malaysia
Individual Site Status
Recruiting
Facility Name
SBHI Pediatrics city clinical hospital of Saint Vladimir
City
Moscow
ZIP/Postal Code
107014
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
Municipal Children Hospital 1
City
Saint Petersburg
ZIP/Postal Code
198205
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
State Budgetary Healthcare Institution Samara Regional Clinical Hospital na V D Seredavin
City
Samara
ZIP/Postal Code
443095
Country
Russian Federation
Individual Site Status
Recruiting
Facility Name
National University Hospital
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Individual Site Status
Recruiting
Facility Name
Kaohsiung Veterans General Hospital
City
Kaohsiung
ZIP/Postal Code
81362
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
National Cheng Kung University Hospital
City
Tainan
ZIP/Postal Code
70403
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10041
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Linkou Chang Gung Memorial Hospital
City
Taoyuan
ZIP/Postal Code
33305
Country
Taiwan
Individual Site Status
Recruiting
Facility Name
Hacettepe Universitesi Tip Fakultesi Ihsan Dogramaci Cocuk Hastanesi
City
Ankara
ZIP/Postal Code
06230
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Baskent Universitesi Ankara Hastanesi
City
Ankara
ZIP/Postal Code
06490
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Gazi Universitesi Tip Fakultesi
City
Ankara
ZIP/Postal Code
06500
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Istanbul Universitesi Cerrahpasa Tip Fakultesi
City
Istanbul
ZIP/Postal Code
34098
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Ege Universitesi Tip Fakultesi
City
Izmir
ZIP/Postal Code
35040
Country
Turkey
Individual Site Status
Recruiting
Facility Name
Erciyes Universitesi Tip Fakultesi Mustafa Eraslan ve Fevzi Mercan Cocuk Hastanesi
City
Kayseri
ZIP/Postal Code
38039
Country
Turkey
Individual Site Status
Recruiting
Facility Name
National Childrens Specializated Hospital Okhmadit
City
Kyiv
ZIP/Postal Code
01135
Country
Ukraine
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request
IPD Sharing 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.
IPD Sharing 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.
IPD Sharing URL
http://www.amgen.com/datasharing
Links:
URL
http://www.amgentrials.com
Description
AmgenTrials clinical trials website
Learn more about this trial
A Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
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