Vanderbilt Clinical Trials

Study to Evaluate Romosozumab in Children and Adolescents With Osteogenesis Imperfecta

Purpose

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).

Condition

Osteogenesis Imperfecta

Eligibility

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

Inclusion Criteria

Ambulatory male or female children 5 to less than 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 1/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, 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 acid within 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. - Clinically significant valvular heart disease based on local echocardiogram (ECHO) results.

Study Design

Phase: Phase 1
Study Type: Interventional
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Primary Purpose: Treatment
Masking: None (Open Label)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Romosozumab: 12 - < 18 Years of Age	Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.	Drug: Romosozumab Participants will receive multiple doses of romosozumab via a SC injection. Dietary Supplement: Calcium All participants will receive daily supplements of elemental calcium. Dietary Supplement: Vitamin D All participants will receive daily supplementation with vitamin D.
Experimental Romosozumab: 5 - < 12 Years of Age	Participants will receive 1 of 3 dose levels of romosozumab. All participants also received calcium and vitamin D.	Drug: Romosozumab Participants will receive multiple doses of romosozumab via a SC injection. Dietary Supplement: Calcium All participants will receive daily supplements of elemental calcium. Dietary Supplement: Vitamin D All participants will receive daily supplementation with vitamin D.

More Details

Status: Completed
Sponsor: Amgen

Study Contact

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.