An efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta
- Conditions
- Osteogenesis imperfectaNot Applicable
- Registration Number
- ISRCTN95879580
- Lead Sponsor
- ovartis Pharmaceuticals (Canada)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- All
- Target Recruitment
- 20
1. Children, male or female, 2 weeks to less than 12 months of age, at least at 38 weeks gestational age
2. Any child with phenotypic OI type II, III or IV
3. No previous treatment with bisphosphonates
4. Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed. The assessment will be make 2 weeks after the first assessment and the sample must be a urine collection after a 4-hour fast
1. Blood oxygen saturation of less than 90% in room air
2. Serum creatinine level greater than 56 µmol/L
3. Any clinically significant clinical laboratory abnormalities at screening
4. Treatment with any investigational drug within the past 30 days
5. Patients who are unlikely to be able to complete the study or comply with the visit schedule
6. Any disease or planned therapy which will interfere with the procedures or data collection of this trial
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants with severe osteogenesis imperfecta aged between 2 weeks to 1 year of age at entry, compared to historical controls. The efficacy of zoledronic acid will be demonstrated if it is shown to be a gain in Z-score of at least 1.
- Secondary Outcome Measures
Name Time Method Effect of zoledronic acid on the change in whole body bone mineral content after 12 and 24 months of treatment relative to baseline compared to historical controls in infants 2 weeks to 1 year of age.