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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.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00982124
First Posted: September 22, 2009
Last Update Posted: April 19, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborator:
Novartis
Information provided by (Responsible Party):
Francis H. Glorieux, Shriners Hospitals for Children
  Purpose
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: Intervention Model: Single Group Assignment
Masking: None (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 Francis H. Glorieux, 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 ]

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 ]

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

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 12 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00982124


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

Responsible Party: Francis H. Glorieux, Principal Investigator, Shriners Hospitals for Children
ClinicalTrials.gov Identifier: NCT00982124     History of Changes
Other Study ID Numbers: SHC-INFOI
IRB - A06-M73-06A
Health Canada - 9427-S1926-24C
First Submitted: September 21, 2009
First Posted: September 22, 2009
Last Update Posted: April 19, 2016
Last Verified: April 2016

Keywords provided by Francis H. Glorieux, Shriners Hospitals for Children:
Osteogenesis Imperfecta
Infants with moderate to severe Osteogenesis Imperfecta

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Zoledronic acid
Diphosphonates
Bone Density Conservation Agents
Physiological Effects of Drugs