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

Purpose

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.

Condition

  • Osteogenesis Imperfecta

Eligibility

Eligible Ages
Between 5 Years and 17 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 3
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Romosozumab
Participants will receive romosozumab once a month (QM) for 12 months.
  • Drug: Romosozumab
    Subcutaneous (SC) injection
    Other names:
    • EVENITY®
Active Comparator
Standard of Care Bisphosphonate
Participants will receive bisphosphonates per local standard of care treatment regimens, as determined by the investigator for 12 months.
  • Drug: Bisphosphonate
    Administration determined by investigator according to the local standard of care

Recruiting Locations

Phoenix Childrens Hospital
Phoenix, Arizona 85016

University of California, Los Angeles Interventional Clinical Trials
Los Angeles, California 90095

Nemours Hospital for Children
Wilmington, Delaware 19803

Nemours Childrens Hospital
Orlando, Florida 32827

University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
Tampa, Florida 33606

Emory University
Atlanta, Georgia 30322

Indiana University
Indianapolis, Indiana 46202

Kennedy Krieger Institute
Baltimore, Maryland 21287

Boston Childrens Hospital
Boston, Massachusetts 02115

Gillette Childrens Hospital and Clinic Saint Paul
Saint Paul, Minnesota 55101

Vanderbilt University Medical Center
Nashville, Tennessee 37212-3157

Marshfield Clinic
Marshfield, Wisconsin 54449

More Details

Status
Recruiting
Sponsor
Amgen

Study Contact

Amgen Call Center
866-572-6436
medinfo@amgen.com