A Study of Etelcalcetide in Pediatric Subjects With Secondary Hyperparathyroidism and Chronic Kidney Disease on Hemodialysis
- Conditions
- Secondary HyperparathyroidismChronic Kidney Disease
- Interventions
- Registration Number
- NCT03633708
- Lead Sponsor
- Amgen
- 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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 56
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Etelcalcetide Etelcalcetide Randomized in a 3:1 ratio to receive etelcalcetide in addition to standard of care Control Etelcalcetide Randomized in a 3:1 ratio to receive etelcalcetide in addition standard of care alone (control arm)
- Primary Outcome Measures
Name Time Method Proportion of participants with ≥ 30% reduction from baseline in intact parathyroid hormone (iPTH) level during the efficacy assessment phase (EAP) 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
Name Time Method Achievement of ≥ 30% reduction in iPTH from baseline on two consecutive visits Up to approximately 30 Weeks To characterize change in laboratory markers of CKD following etelcalcetide treatment.
Incidence of adverse events Day 1 to 30 days after last dose of etelcalcetide (up to approximately 30 weeks) To characterize the safety of etelcalcetide treatment based on adverse events. Nature, frequency, severity, and relationship to treatment of all adverse events, including those of special interest reported during the study.
Percentage change from baseline in corrected total serum calcium Baseline and Weeks 20-27 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 Baseline and Weeks 20-27 Percentage change from baseline in corrected total serum phosphorous during the EAP (defined as weeks 20 through 27).
Frequency of hypocalcemia Up to approximately 30 Weeks Occurrence of hypocalcemia at any point in time, assessed by serum chemistry.
Change in Tanner Stage Week -2 and Week 27 Changes in tanner stage at scheduled visits.
Minimum serum concentration (Cmin) of etelcalcetide 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.
Change from baseline in heart rate Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 To characterize the safety of etelcalcetide treatment based on vital signs.
Number of participants with corrected serum calcium levels at any time during the study Up to approximately 30 Weeks Occurrence of corrected serum Ca levels \<8.0 mg/dL (2.0 mmol/L) for subjects 2 to \< 18 years of age and \<8.6 mg/dL (2.15 mmol/L) for subjects 28 days to \<2 years of age during the study.
Number of participants with serum phosphorous levels below normal for age Up to approximately 30 Weeks Occurrence of serum phosphorous levels below the lower limit of normal for age.
Number of participants with predialysis iPTH levels below normal Up to approximately 30 Weeks Occurrence of predialysis iPTH levels below the lower limit of normal for age.
Change from baseline in diastolic blood pressure Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 To characterize the safety of etelcalcetide treatment based on vital signs.
Change in height Day 1 and Week 27 Changes in height at scheduled visits.
Change in weight Week -2, Day 1, and Week 27 Changes in weight at scheduled visits.
Maximum serum concentration (Cmax) of etelcalcetide 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.
Change from baseline in systolic blood pressure Week -2, Week -1, Day1, and Weeks 4, 8, 12, 16, 20, 24, and 27 To characterize the safety of etelcalcetide treatment based on vital signs.
Mean change from baseline in predialysis iPTH Baseline and Weeks 20-27 Mean change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Percentage change from baseline in predialysis iPTH Baseline and Weeks 20-27 Percentage change from baseline in predialysis iPTH during the EAP (defined as weeks 20 through 27).
Trial Locations
- Locations (43)
Firat Universitesi Hastanesi
🇹🇷Elazig, Turkey
Childrens Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Childrens Hospital Colorado
🇺🇸Aurora, Colorado, United States
Childrens Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Mount Sinai Kidney Center - B1 Renal Treatment
🇺🇸New York, New York, United States
Cincinnati Childrens Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Cleveland Clinic Foundation
🇺🇸Cleveland, Ohio, United States
The Childrens Hospital at Oklahoma University Medical Center
🇺🇸Oklahoma City, Oklahoma, United States
Childrens Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Childrens Medical Center Dallas
🇺🇸Dallas, Texas, United States
Primary Childrens Hospital Outpatient Services
🇺🇸Salt Lake City, Utah, United States
Hospital Italiano
🇦🇷Cuidad Autonoma de Buenos Aires, Buenos Aires, Argentina
Fresenius Escobar
🇦🇷Escobar, Buenos Aires, Argentina
Centro Infantil Del Rinon
🇦🇷San Miguel de Tucuman, Tucuman, Argentina
Fortis Flt Lt Rajan Dhall Hospital
🇮🇳New Delhi, Delhi, India
All India Institute of Medical Sciences
🇮🇳New Delhi, Delhi, India
Sir Ganga Ram Hospital
🇮🇳New Delhi, Delhi, India
Manipal Hospital
🇮🇳Bangalore, Karnataka, India
KLES Dr Prabhakar Kore Hospital and Medical Research Centre
🇮🇳Belagavi, Karnataka, India
NRS Medical College and Hospital
🇮🇳Kolkata, West Bengal, India
Asan Medical Center
🇰🇷Seoul, Korea, Republic of
Seoul National University Hospital
🇰🇷Seoul, Korea, Republic of
Pusan National University Yangsan Hospital
🇰🇷Yangsan-si, Gyeongsangnam-do, Korea, Republic of
Hospital Raja Perempuan Zainab II
🇲🇾Kota Bharu, Kelantan, Malaysia
Hospital TuanKu Jaafar
🇲🇾Seremban, Negri Sembilan, Malaysia
Hospital Wanita Dan Kanak-Kanak Kuala Lumpur
🇲🇾Kuala Lumpur, Wilayah Persekutuan, Malaysia
SBHI Pediatrics city clinical hospital of Saint Vladimir
🇷🇺Moscow, Russian Federation
SBHI Children's City Multidisciplinary Clinical Specialized Center of High Medical Technologies
🇷🇺Saint Petersburg, Russian Federation
State Budgetary Healthcare Institution Samara Regional Clinical Hospital na V D Seredavin
🇷🇺Samara, Russian Federation
National University Hospital
🇸🇬Singapore, Singapore
Kaohsiung Veterans General Hospital
🇨🇳Kaohsiung, Taiwan
National Cheng Kung University Hospital
🇨🇳Tainan, Taiwan
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Linkou Chang Gung Memorial Hospital
🇨🇳Taoyuan, Taiwan
Hacettepe Universitesi Tip Fakultesi Hastanesi
🇹🇷Ankara, Turkey
Baskent Universitesi Ankara Hastanesi
🇹🇷Ankara, Turkey
Gazi Universitesi Saglik Arastirma ve Uygulama Merkezi Gazi Hastanesi
🇹🇷Ankara, Turkey
Ankara Bilkent Sehir Hastanesi
🇹🇷Ankara, Turkey
Istanbul Universitesi Cerrahpasa Tip Fakultesi
🇹🇷Istanbul, Turkey
Marmara Universitesi Tip Fakultesi Hastanesi
🇹🇷Istanbul, Turkey
Ege Universitesi Tip Fakultesi Hastanesi
🇹🇷Izmir, Turkey
Erciyes Universitesi Tip Fakultesi Hastanesi
🇹🇷Kayseri, Turkey
National Childrens Specializated Hospital Okhmadit
🇺🇦Kyiv, Ukraine