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
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental Romosozumab |
Participants will receive romosozumab once a month (QM) for 12 months. |
|
|
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. |
|
Recruiting Locations
Phoenix Childrens Hospital
Phoenix, Arizona 85016
Phoenix, Arizona 85016
University of California, Los Angeles Interventional Clinical Trials
Los Angeles, California 90095
Los Angeles, California 90095
Nemours Hospital for Children
Wilmington, Delaware 19803
Wilmington, Delaware 19803
Nemours Childrens Hospital
Orlando, Florida 32827
Orlando, Florida 32827
University of South Florida - Carol and Frank Morsani Center for Advanced Health Care
Tampa, Florida 33606
Tampa, Florida 33606
Emory University
Atlanta, Georgia 30322
Atlanta, Georgia 30322
Indiana University
Indianapolis, Indiana 46202
Indianapolis, Indiana 46202
Kennedy Krieger Institute
Baltimore, Maryland 21287
Baltimore, Maryland 21287
Boston Childrens Hospital
Boston, Massachusetts 02115
Boston, Massachusetts 02115
Gillette Childrens Hospital and Clinic Saint Paul
Saint Paul, Minnesota 55101
Saint Paul, Minnesota 55101
Vanderbilt University Medical Center
Nashville, Tennessee 37212-3157
Nashville, Tennessee 37212-3157
Marshfield Clinic
Marshfield, Wisconsin 54449
Marshfield, Wisconsin 54449
More Details
- Status
- Recruiting
- Sponsor
- Amgen