Study to Evaluate Efficacy and Safety of Romosozumab Compared With Bisphosphonates in Children and Adolescents With Osteogenesis Imperfecta

Amgen

Status and phase: Enrolling; Phase 3

Conditions: Osteogenesis Imperfecta

Treatments: Drug: Romosozumab; Drug: Bisphosphonate

Study type: Interventional

Funder types: Industry

Identifiers

NCT05972551

20200105

2023-503294-37 (EudraCT Number)

Details and patient eligibility

About

The primary objective of this study is to evaluate the effect of romosozumab treatment for 12-months compared with bisphosphonate(s) on the number of clinical fractures at 12-months; the number of any fractures at 12-months and change in lumbar spine bone mineral density (BMD) Z-score at 6-months.

Enrollment: 106 estimated patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Participant has provided informed consent/assent prior to initiation of any study specific activities/procedures.

OR

• Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines prior to any study-specific activities/procedures being initiated.

• Ambulatory male and female children and adolescents, age 5 to <18 years, including ambulatory with assistance as defined in the pediatric osteogenesis imperfecta (OI) population.

• Clinical diagnosis of OI, defined as clinical history consistent with type I, III, or IV OI as determined by presence of expected phenotype (examples include: facial shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic features, fracture pattern) and lack of additional features unrelated to type I, III, or IV OI (eg, blindness, mental retardation, neuropathy, and craniosynostosis).

  o If familial, also must be autosomal dominant.

• Meets at least one of the following:

  • 3 or more fractures within the previous 2 years, or
  • 1 or more nonvertebral fracture(s) within the previous 2 years and at least 1 prevalent vertebral fracture, or
  • 2 or more prevalent vertebral fractures.

Exclusion criteria

Disease Related

• History of an electrophoresis pattern inconsistent with type I, III or IV OI.
• History of known mutation in a gene other than collagen type I alpha 1/collagen type I alpha 2 (COL1A1/COL1A2) causing OI or other metabolic bone disease.
• History of congenital dislocation of the radial head, interosseous membrane calcification, or exuberant callus formation.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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106 participants in 2 patient groups

Romosozumab

Experimental group

Description:

Participants will receive romosozumab once a month (QM) for 12 months.

Treatment:

Drug: Romosozumab

Standard of Care Bisphosphonate

Active Comparator group

Description:

Participants will receive bisphosphonates per local standard of care treatment regimens, as determined by the investigator for 12 months.

Treatment:

Drug: Bisphosphonate