An open-label, multicenter, randomized phase 3 study to evaluate the efficacy and safety of Romosozumab compared to Bisphosphonates in children and adolescents with osteogenesis imperfecta

Zusammenfassung der Studie

This is a phase 3 study, the final phase of the drug development process for humans. Researchers will compare the study drug to a standard treatment for osteogenesis imperfecta in children (ages 5 to under 12 years) and adolescents (ages 12 to under 18 years). Neither the participants nor the study doctor can choose the study drug that participants will receive. Participants agree to be randomly assigned to a treatment group. This is like flipping a coin or drawing numbers from a hat. The participants, the study doctor, and the study staff know which study drug each participant receives. The study may last up to 15 months for each participant, including a screening phase of up to 35 days, a 12-month treatment phase, and a 3-month safety follow-up phase.

(BASEC)

Untersuchte Intervention

Participants assigned to the Romosozumab group will receive 5 mg/kg of Romosozumab as a subcutaneous injection every month for 12 months, with a maximum monthly dose of 210 mg. Participants assigned to the comparator group will receive bisphosphonates according to local standard of care (SOC).

All participants must receive vitamin D and calcium supplements throughout the study duration to minimize the risk of hypocalcemia (low calcium levels in the blood). The recommended daily dose is at least 30 to 50 mg/kg per day up to a maximum of 1000 mg of calcium, including dietary intake, and at least 800 IU of vitamin D.

(BASEC)

Untersuchte Krankheit(en)

Osteogenesis imperfecta (OI) is a genetic disorder that prevents the body from forming strong bones in children. This condition is characterized by bones that break easily. There are no approved medications for the treatment of OI in children, except for Neridronate and Pamidronate, which are approved in Italy and Japan, respectively. Currently, physicians use bisphosphonates in children with moderate to severe OI to increase bone mass. However, the risk of bone fractures cannot be clearly reduced, even for long bone fractures. There is an unmet need for other treatments with further solid scientific evidence. Romosozumab increases bone density (stronger bones) and has the potential to reduce the risk of bone fractures.

(BASEC)

Sponsor

Amgen Switzerland AG Amgen Inc. USA

(BASEC)