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An international, multicenter, efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta

Conditions
Children suffering from severe Osteogenesis Imperfecta
MedDRA version: 14.1Level: PTClassification code 10031243Term: Osteogenesis imperfectaSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Registration Number
EUCTR2011-000745-21-BE
Lead Sponsor
Schriners Hospitals for Children
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Authorised-recruitment may be ongoing or finished
Sex
All
Target Recruitment
15
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.
•No previous treatment with bisphosphonates.
•Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed. The assessment will be made 2 weeks after the first assessment and the sample must be a urine collection after a 4-hour fast.

Are the trial subjects under 18? yes
Number of subjects for this age range: 2
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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.

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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