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

This study has been completed.
Information provided by (Responsible Party):
Francis H. Glorieux, Shriners Hospitals for Children Identifier:
First received: September 21, 2009
Last updated: April 18, 2016
Last verified: April 2016
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.

Condition Intervention Phase
Osteogenesis Imperfecta
Drug: Zoledronic Acid
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
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

Resource links provided by NLM:

Further study details as provided by Shriners Hospitals for Children:

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. [ Time Frame: 3 times during 10 visits within 2 years ] [ Designated as safety issue: Yes ]

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. [ Time Frame: 3 times during 10 visits within 2 years ] [ Designated as safety issue: Yes ]

Enrollment: 14
Study Start Date: October 2007
Study Completion Date: April 2016
Primary Completion Date: September 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment Arm (only)
Zoledronic acid infusion
Drug: Zoledronic Acid
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).
Other Name: Aclasta


Ages Eligible for Study:   up to 12 Months
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

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.
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Please refer to this study by its identifier: NCT00982124

Canada, Quebec
Shriners Hospital for Children
Montreal, Quebec, Canada, H4A0A9
Sponsors and Collaborators
Shriners Hospitals for Children
Principal Investigator: Francis H. Glorieux, MD, PhD McGill University
  More Information

Responsible Party: Francis H. Glorieux, Principal Investigator, Shriners Hospitals for Children Identifier: NCT00982124     History of Changes
Other Study ID Numbers: SHC-INFOI  IRB - A06-M73-06A  Health Canada - 9427-S1926-24C 
Study First Received: September 21, 2009
Last Updated: April 18, 2016
Health Authority: Canada: Health Canada
Canada: Therapeutic Products Directorate's Guideline for Good Clinical Practice

Keywords provided by Shriners Hospitals for Children:
Osteogenesis Imperfecta
Infants with moderate to severe Osteogenesis Imperfecta

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Bone Diseases
Bone Diseases, Developmental
Collagen Diseases
Connective Tissue Diseases
Genetic Diseases, Inborn
Musculoskeletal Diseases
Zoledronic acid
Bone Density Conservation Agents
Physiological Effects of Drugs processed this record on May 23, 2016