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

NCT00982124 | Completed Phase 3

• 3 other identifiers: SHC-INFOIIRB - A06-M73-06AHealth Canada - 9427-S1926-24C
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

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

Keywords

Osteogenesis Imperfecta; Infants with moderate to severe Osteogenesis Imperfecta

Outcome Measures

Primary Outcomes (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.

  3 times during 10 visits within 2 years

Secondary Outcomes (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.

  3 times during 10 visits within 2 years

Study Arms (1)

Treatment Arm (only)

EXPERIMENTAL

Zoledronic acid infusion

Drug: Zoledronic Acid

Interventions

Zoledronic Acid 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).

Also known as: Aclasta

Treatment Arm (only)

Eligibility Criteria

• Age: Up to 12 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shriners Hospitals for Children: lead
• Novartis: collaborator

Study Sites (1)

Shriners Hospital for Children

Montreal, Quebec, H4A0A9, Canada

Location

MeSH Terms

Conditions

Osteogenesis Imperfecta

Interventions

Zoledronic Acid

Condition Hierarchy (Ancestors)

Osteochondrodysplasias; Bone Diseases, Developmental; Bone Diseases; Musculoskeletal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Diphosphonates; Organophosphonates; Organophosphorus Compounds; Organic Chemicals; Imidazoles; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Francis H. Glorieux, MD, PhD

  McGill University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: SUPPORTIVE CARE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: September 21, 2009
• First Posted: September 22, 2009
• Study Start: October 1, 2007
• Primary Completion: September 1, 2015
• Study Completion: April 1, 2016
• Last Updated: April 19, 2016

Record last verified: 2016-04

Locations

CA (1)