An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta

• Register: ClinicalTrials.gov
• Last refreshed on: 2 May 2016
• Main ID: NCT00982124
• Date of registration: 21/09/2009
• Prospective Registration: No
• Primary sponsor: Shriners Hospitals for Children
• Public title: An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta INFOI
• Scientific title: An International, Multicenter, Open-label, Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
• Date of first enrolment: October 2007
• Target sample size: 14
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00982124
• Study type: Interventional
• Study design: Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Supportive Care
• Phase: Phase 3

Countries of recruitment

Canada

Contacts

• Name: Francis H. Glorieux, MD, PhD
• Affiliation: McGill University

Key inclusion & exclusion criteria

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.



Age minimum: N/A
Age maximum: 12 Months
Gender: Both

Health Condition(s) or Problem(s) studied

Osteogenesis Imperfecta

Intervention(s)

Drug: Zoledronic Acid

Primary Outcome(s)

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. [Time Frame: 3 times during 10 visits within 2 years]

Secondary Outcome(s)

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. [Time Frame: 3 times during 10 visits within 2 years]

Secondary ID(s)

Health Canada - 9427-S1926-24C

IRB - A06-M73-06A

SHC-INFOI

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis