Clinical trial NCT00982124: An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
Official 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Osteogenesis Imperfecta
Keywords: Osteogenesis Imperfecta
Infants with moderate to severe Osteogenesis Imperfecta
Lead sponsor: Shriners Hospitals for Children
Sponsor class: OTHER
Collaborators: name: Novartis
First posted: 2009-09-22
Enrollment: 14
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

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.


Eligibility 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.

Primary outcomes:
measure: 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.
timeFrame: 3 times during 10 visits within 2 years
Secondary outcomes:
measure: 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.
timeFrame: 3 times during 10 visits within 2 years
Other outcomes:
Locations:
city: Montreal
country: Canada
facility: Shriners Hospital for Children
state: Quebec
Study officials:
affiliation: McGill University
name: Francis H. Glorieux, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: