Osteogenesis Imperfecta
Conditions
Keywords
Osteogenesis Imperfecta, Infants with moderate to severe Osteogenesis Imperfecta
Brief summary
The investigators have currently finished conducting an international multi-center trial that compares the efficacy and safety of pamidronate and zoledronate in the treatment of moderate to severe forms of Osteogenesis Imperfecta (OI). This trial has included only children above one year of age. The aim of the current study is to extend the observations of that currently finished study to infants below 1 year of age. Moreover, it is possible to administer zoledronate in a single short infusion instead of the three-day cycles with Pamidronate, therefore decreasing patient and family burdens with shorter stays in the hospital.
Interventions
DRUGZoledronic Acid
Each patient is to receive an initial dose of zoledronic acid of 0.0125 mg per kg body weight, then subsequent doses of zoledronic acid at a dose of 0.025 mg per kg body weight once every 12 to 13 weeks for 104 weeks (total of 8 doses).
Sponsors
Novartis
Shriners Hospitals for Children
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 12 Months
Healthy volunteers
No
Inclusion criteria
* Children, male or female 2 weeks to \< 12 months of age, at least at 38 weeks gestational age. * Any child with phenotypic OI type II, III or IV. * Any child classified as OI type I, V-VIII that has had at least two or more previous fractures of long bone, or vertebral compression fractures, and a low bone mineral density. * No previous treatment with bisphosphonates. * Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed.
Exclusion criteria
* Blood oxygen saturation of less than 90% in room air. * Serum creatinine level greater than 56 µmol/L. * Any clinically significant clinical laboratory abnormalities at screening. * Treatment with any investigational drug within the past 30 days. * Patients who are unlikely to be able to complete the study or comply with the visit schedule. * Any disease or planned therapy which will interfere with the procedures or data collection of this trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The primary objective of this trial is to assess the change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants compared to historical controls. | 3 times during 10 visits within 2 years |
Secondary
| Measure | Time frame |
|---|---|
| To assess the effect of zoledronic acid on the number of clinical fractures &/or vertebral compressions that occur over a two year period compared to untreated historical controls in infants. | 3 times during 10 visits within 2 years |
Countries
Canada
Outcome results
None listed