Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta

• Status: Recruiting
• Trial end date: 2023-05-06
• Target enrollment: 0
• Participant gender: All

Summary

The primary objective of this study is to evaluate the pharmacokinetics (PK) profile following multiple subcutaneous (SC) doses of romosozumab in children and adolescents with Osteogenesis Imperfecta (OI).

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Amgen
• Treatments: Calcium; Vitamin D

Criteria

Inclusion Criteria:

• Ambulatory male or female children 5 to 18 years of age upon entry into screening

• Clinical diagnosis of OI defined as a clinical history consistent with type I-IV OI as determined by presence of expected phenotype and lack of additional features unrelated to type I-IV OI

Exclusion Criteria

• History of an electrophoresis pattern inconsistent with type I to type IV OI

• History of known mutation in a gene other than collagen type I alpha/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other metabolic bone disease

• History of other bone diseases that affect bone metabolism (eg, osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis, osteopetrosis, hypophosphatasia)

• History of Kawasaki disease, rheumatic myocarditis, ischemic cardiomyopathy, inherited cardiomyopathies, valvular heart disease, nephrotic syndrome, familial hypercholesterolemia, stroke, or any thromboembolic disorder

• Unhealed fracture as defined by orthopedic opinion

• Symptoms associated with skull abnormalities such as basilar invagination, basilar impression or Chiari malformation

• Prior treatment with anti-sclerostin antibody, fluoride or strontium, parathyroid hormone (PTH) within 12 months prior to screening, denosumab within 12 months or zoledronic acide with in 6 months prior to first dose

• Less than 2 evaluable vertebrae by DXA evaluation in the region of interest, L1 L4, as confirmed by the central imaging laboratory.