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

• ClinicalTrials.gov Identifier: NCT00982124
• Recruitment Status: Completed
• First Posted: September 22, 2009
• Last Update Posted: April 19, 2016
• Sponsor: Shriners Hospitals for Children
• Collaborator: Novartis
• Information provided by (Responsible Party): Francis H. Glorieux, Shriners Hospitals for Children

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.

Condition or disease	Intervention/treatment	Phase
Osteogenesis Imperfecta	Drug: Zoledronic Acid	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 14 participants
• 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
• Study Start Date: October 2007
• Actual Primary Completion Date: September 2015
• Actual Study Completion Date: April 2016

Arms and Interventions

Arm	Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :

1. 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 :

1. 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 ]

Eligibility Criteria

• 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

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 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; Bone Density Conservation Agents; Physiological Effects of Drugs