Back to resultsAn Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta (INFOI)
Primary Purpose
Osteogenesis Imperfecta
Status
Completed
Phase
Phase 3
Locations
Canada
Study Type
Interventional
Intervention
Zoledronic Acid
Sponsored by
About this trial
This is an interventional supportive care trial for Osteogenesis Imperfecta focused on measuring Osteogenesis Imperfecta, Infants with moderate to severe Osteogenesis Imperfecta
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.
Sites / Locations
- Shriners Hospital for Children
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Treatment Arm (only)
Arm Description
Zoledronic acid infusion
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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.
Full Information
NCT ID
NCT00982124
First Posted
September 21, 2009
Last Updated
April 18, 2016
Sponsor
Shriners Hospitals for Children
Collaborators
Novartis
1. Study Identification
Unique Protocol Identification Number
NCT00982124
Brief Title
An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
Acronym
INFOI
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
2. Study Status
Record Verification Date
April 2016
Overall Recruitment Status
Completed
Study Start Date
October 2007 (undefined)
Primary Completion Date
September 2015 (Actual)
Study Completion Date
April 2016 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Shriners Hospitals for Children
Collaborators
Novartis
4. Oversight
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteogenesis Imperfecta
Keywords
Osteogenesis Imperfecta, Infants with moderate to severe Osteogenesis Imperfecta
7. Study Design
Primary Purpose
Supportive Care
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Treatment Arm (only)
Arm Type
Experimental
Arm Description
Zoledronic acid infusion
Intervention Type
Drug
Intervention Name(s)
Zoledronic Acid
Other Intervention Name(s)
Aclasta
Intervention Description
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).
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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
10. Eligibility
Sex
All
Maximum Age & Unit of Time
12 Months
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Francis H. Glorieux, MD, PhD
Organizational Affiliation
McGill University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Shriners Hospital for Children
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4A0A9
Country
Canada
12. IPD Sharing Statement
Learn more about this trial
An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
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