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

Osteogenesis Imperfecta Clinical Trial

— INFOI  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00982124
• Other study ID #: SHC-INFOI
• Secondary ID: IRB - A06-M73-06
• Status: Completed
• Phase: Phase 3
• First received: September 21, 2009
• Last updated: April 18, 2016
• Start date: October 2007
• Est. completion date: April 2016

Study information

• Verified date: April 2016
• Source: Shriners Hospitals for Children
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaCanada: Therapeutic Products Directorate's Guideline for Good Clinical Practice
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: April 2016
• Est. primary completion date: September 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A to 12 Months

Eligibility

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.

Study Design

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

Related Conditions & MeSH terms

• Osteogenesis Imperfecta

Intervention

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).

Locations

Country	Name	City	State
Canada	Shriners Hospital for Children	Montreal	Quebec

Sponsors (2)

Lead Sponsor	Collaborator
Shriners Hospitals for Children	Novartis

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.		3 times during 10 visits within 2 years	Yes
Secondary	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.		3 times during 10 visits within 2 years	Yes