Amgen Inc. is conducting a pivotal Phase 3 clinical study to evaluate etelcalcetide in pediatric patients with secondary hyperparathyroidism and chronic kidney disease receiving maintenance hemodialysis. The study, which began on December 20, 2019, represents a significant effort to address treatment needs in a vulnerable population that has been less studied in this therapeutic context.
Study Design and Patient Population
The Phase 3 trial follows a single-arm, open-label, multidose design evaluating children and adolescents aged 2 to 18 years with secondary hyperparathyroidism and chronic kidney disease on hemodialysis. The interventional study uses a single-group assignment model with no masking, meaning all participants receive the treatment and both researchers and participants know what treatment is being administered.
The study's primary purpose is treatment-focused, aiming to evaluate the therapeutic effects of etelcalcetide in the specified patient group over a 26-week period. Participants receive etelcalcetide intravenously three times per week, with doses adjusted based on specific health markers to maintain optimal levels of parathyroid hormone, calcium, and phosphorus.
Drug Mechanism and Current Approval Status
Etelcalcetide is already approved for adult patients with secondary hyperparathyroidism on hemodialysis and has demonstrated effectiveness in this population. The drug works by controlling levels of intact parathyroid hormone, calcium, and phosphorus, with the goal of improving patient outcomes through better management of these critical biomarkers.
The intravenous administration three times weekly aligns with the typical hemodialysis schedule, allowing for convenient dosing that can be titrated based on patient response to achieve therapeutic targets.
Clinical Significance and Market Impact
This research addresses a critical need for effective treatments in pediatric patients with secondary hyperparathyroidism and chronic kidney disease, a population that has historically been understudied. The study's focus on children and adolescents represents an important expansion of the evidence base for etelcalcetide beyond its current adult indication.
The ongoing study has the potential to significantly impact Amgen's market position if results demonstrate meaningful clinical benefits. Successful outcomes could expand etelcalcetide's use to a new patient demographic, potentially strengthening Amgen's competitive position in the nephrology market against other companies developing treatments for chronic kidney disease-related conditions.
Study Timeline and Current Status
The study is currently in the recruitment phase, with the latest update submitted on July 15, 2025. The estimated completion date is set for 2025, providing a clear timeline for potential results and subsequent regulatory decisions. These milestone dates are crucial for tracking study progress and anticipated market impacts.
