Etelcalcetide in Children with SHPT and CKD receiving Haemodialysis
Research type
Research Study
Full title
Phase 3, Single-arm, Open-label, Multidose, Titration, Pharmacokinetic, Pharmacodynamic, and Safety Study of Etelcalcetide in Children and Adolescents ≥ 2 to < 18 Years of age With Secondary Hyperparathyroidism and Chronic Kidney Disease Receiving Maintenance Hemodialysis.
IRAS ID
265503
Contact name
Ben Reynolds
Contact email
Sponsor organisation
Amgen Ltd
Eudract number
2018-004608-21
Duration of Study in the UK
2 years, 7 months, 27 days
Research summary
Secondary hyperparathyroidism (SHPT) is a common complication of chronic kidney disease (CKD) that occurs in the initial stages of CKD and worsens with declining kidney function. SHPT is associated with serious complications in children on dialysis, including a variety of bone abnormalities, growth retardation and an increased risk for early development of cardiac-related illnesses. Widely used traditional therapies to treat SHPT such as vitamin D sterols have the potential to worsen some complications of the disease, and there is poor compliance with cinacalcet, an oral calcimimetic, thereby creating an unmet medical need for this population. Etelcalcetide has been shown to be efficacious with a positive benefit-risk profile in treating adult CKD patients with SHPT and has recently been approved for use in adult patients with SHPT treated with haemodialysis in both the US and Europe. To this day, no previous studies have been conducted in paediatric patients with etelcalcetide other than one single dose pharmacokinetic (PK) study but currently available data suggest that there is a minimal to moderate risk in relation to the potentially significant benefit for the conduction of this study. In this study with only one treatment arm, approximately 20 children aged > 2 to < 18 years old with SHPT and CKD receiving haemodialysis 3 times weekly will be screened and enrolled (if deemed eligible) to receive etelcalcetide. The study will take place in 12 European countries across 20 hospital sites. Each patient will receive a dose of etelcalcetide at the end of each haemodialysis session, while also blood samples will be taken to assess various parameters regarding disease control and for safety monitoring for a total of 26 weeks. Patient safety will be monitored for an additional month after the last dose. The study is expected to last approximately 2 years
REC name
East Midlands - Leicester Central Research Ethics Committee
REC reference
19/EM/0261
Date of REC Opinion
12 Sep 2019
REC opinion
Further Information Favourable Opinion