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Trial record 4 of 20 for:    "Osteogenesis imperfecta"
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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 is currently recruiting participants.
Verified March 2013 by Shriners Hospitals for Children
Sponsor:
Collaborator:
Novartis
Information provided by (Responsible Party):
Francis H. Glorieux, Shriners Hospitals for Children
ClinicalTrials.gov Identifier:
NCT00982124
First received: September 21, 2009
Last updated: March 26, 2013
Last verified: March 2013
History of Changes
  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: 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:

U.S. FDA Resources

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 ]

Estimated Enrollment: 20
Study Start Date: October 2007
Estimated Study Completion Date: December 2015
Estimated Primary Completion Date: May 2015 (Final data collection date for primary outcome measure)
Intervention Details:
    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

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

Inclusion Criteria:

  • Infants, 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.
  • No previous treatment with bisphosphonate.
  • Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed. The assessment will be made 2 weeks after the first assessment and the sample must be a urine collection after a 4 hour fast.

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 to 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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00982124

Contacts
Contact: Michaela Durigova, Clinical Research Coordinator 514-282-7158 mdurigova@shriners.mcgill.ca

Locations
Canada, Quebec
Shriners Hospital for Children Recruiting
Montreal, Quebec, Canada, H3G 1A6
Contact: Michaela Durigova, Clinical Research Coordinator     514-282-7158     mdurigova@shriners.mcgill.ca    
Principal Investigator: Francis H. Glorieux, MD. PhD            
Sponsors and Collaborators
Shriners Hospitals for Children
Novartis
Investigators
Principal Investigator: Francis H. Glorieux, MD, PhD McGill University
  More Information

No publications provided

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
Study First Received: September 21, 2009
Last Updated: March 26, 2013
Health Authority: Canada: Food and Drug Regulations
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
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
Pharmacologic Actions

ClinicalTrials.gov processed this record on May 16, 2013