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 ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Francis H. Glorieux, Shriners Hospitals for Children
First received: September 21, 2009
Last updated: June 4, 2013
Last verified: June 2013
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.
Drug: Zoledronic Acid
||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Supportive Care
||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
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:
| Study Start Date:
| Estimated Study Completion Date:
| Estimated Primary Completion Date:
||May 2015 (Final data collection date for primary outcome measure)
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
|Ages Eligible for Study:
||up to 12 Months
|Genders Eligible for Study:
|Accepts Healthy Volunteers:
- 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.
- 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.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00982124
|Shriners Hospital for Children
|Montreal, Quebec, Canada, H3G 1A6 |
Shriners Hospitals for Children
||Francis H. Glorieux, MD, PhD
No publications provided
||Francis H. Glorieux, Principal Investigator, Shriners Hospitals for Children
History of Changes
|Other Study ID Numbers:
||SHC-INFOI, IRB - A06-M73-06A, Health Canada - 9427-S1926-24C
|Study First Received:
||September 21, 2009
||June 4, 2013
||Canada: Food and Drug Regulations
Canada: Therapeutic Products Directorate's Guideline for Good Clinical Practice
Keywords provided by Shriners Hospitals for Children:
Infants with moderate to severe Osteogenesis Imperfecta
Additional relevant MeSH terms:
ClinicalTrials.gov processed this record on July 22, 2014
Bone Diseases, Developmental
Genetic Diseases, Inborn
Connective Tissue Diseases
Bone Density Conservation Agents
Physiological Effects of Drugs