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

NCT ID: NCT00982124

Last Updated: 2016-04-19

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 14 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-10-31
• Study Completion Date: 2016-04-30

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.

Conditions

Osteogenesis Imperfecta

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: NONE

Study Groups

Treatment Arm (only)

Zoledronic acid infusion

Group Type: EXPERIMENTAL

Zoledronic Acid

Intervention Type: DRUG

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).

Interventions

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).

Intervention Type: DRUG

Other Intervention Names

Aclasta

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.

• Maximum Eligible Age: 12 Months
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis

INDUSTRY

Sponsor Role: collaborator

Shriners Hospitals for Children

OTHER

Sponsor Role: lead

Responsible Party

Francis H. Glorieux

Principal Investigator

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Francis H. Glorieux, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

McGill University

Locations

Shriners Hospital for Children

Montreal, Quebec, Canada

Countries

Canada

Other Identifiers

IRB - A06-M73-06A

Identifier Type: -

Identifier Source: secondary_id

Health Canada - 9427-S1926-24C

Identifier Type: -

Identifier Source: secondary_id

SHC-INFOI

Identifier Type: -

Identifier Source: org_study_id