An efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta
| ISRCTN | ISRCTN95879580 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN95879580 |
| ClinicalTrials.gov (NCT) | NCT00982124 |
| Protocol serial number | SCH-INFOI |
| Sponsor | Novartis Pharmaceuticals (Canada) |
| Funder | Novartis Pharmaceuticals Canada |
- Submission date
- 23/09/2009
- Registration date
- 04/01/2010
- Last edited
- 10/05/2019
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Other
Prospectively registered
Protocol
Statistical analysis plan
Results
Individual participant data
Record updated in last year
Plain English summary of protocol
Not provided at time of registration
Contact information
Dr Francis H Glorieux
Scientific
Scientific
Shriners Hospitals for Children
1529 Cedar Avenue
Montreal
H3G 1A6
Canada
| ncyr@shriners.mcgill.ca |
Study information
| Primary study design | Interventional |
|---|---|
| Study design | International multicentre open-label efficacy and safety trial |
| Secondary study design | Non randomised study |
| Study type | Participant information sheet |
| Scientific title | An international, multicentre, open-label, efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta |
| Study objectives | This is an international, multicentre, open-label efficacy and safety trial. The primary objective is to evaluate the change in lumbar spine bone mineral density Z-score at month 24 relative to baseline using intravenous zoledronic acid compared to untreated historical controls in infants with severe osteogenensis imperfecta, who are between 2 weeks and 1 year of age, all inclusive. |
| Ethics approval(s) | Faculty of Medicine, McGill University Institutional Review Board, 08/06/2009, ref: A06-M73-06A |
| Health condition(s) or problem(s) studied | Osteogenesis imperfecta |
| Intervention | All patients will receive an initial infusion of zoledronic acid at 0.0125 mg per kg body weight, followed by infusions of zoledronic acid given every three months at a dose of 0.025 mg per kg body weight, administered as a 30 to 45-minute infusion. There will be a total of 10 visits over the 24 month period of time for all patients. The total number of doses is 8. All zoledronic acid patients will be hospitalised for 48 hours at the first administration of zoledronic acid to monitor for drug reactions. Ionised calcium will be measure pre-dose, 12 hours, 24, 36 and 48 hours post-dose during the hospitalisation period. Sites will call all patients at scheduled monthly visits for determination of adverse events and concomitant medications throughout the study, except for those months where there is a scheduled on-site visit. Dual energy x-ray absorptiometry (DXA) measurements of the lumbar spine and total body and radiological skeletal survey will be done at screening or at first administration of zoledronic acid, the 12 month visit (visit 6) and final visit (visit 10). Twenty infants will be enrolled; enrolment will be competitive. |
| Intervention type | Drug |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | Zoledronic acid |
| Primary outcome measure(s) |
Change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants with severe osteogenesis imperfecta aged between 2 weeks to 1 year of age at entry, compared to historical controls. The efficacy of zoledronic acid will be demonstrated if it is shown to be a gain in Z-score of at least 1. |
| Key secondary outcome measure(s) |
Effect of zoledronic acid on the change in whole body bone mineral content after 12 and 24 months of treatment relative to baseline compared to historical controls in infants 2 weeks to 1 year of age. |
| Completion date | 31/12/2012 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 2 Weeks |
| Upper age limit | 12 Months |
| Sex | All |
| Target sample size at registration | 20 |
| Key inclusion criteria | 1. Children, male or female, 2 weeks to less than 12 months of age, at least at 38 weeks gestational age 2. Any child with phenotypic OI type II, III or IV 3. No previous treatment with bisphosphonates 4. Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed. The assessment will be make 2 weeks after the first assessment and the sample must be a urine collection after a 4-hour fast |
| Key exclusion criteria | 1. Blood oxygen saturation of less than 90% in room air 2. Serum creatinine level greater than 56 µmol/L 3. Any clinically significant clinical laboratory abnormalities at screening 4. Treatment with any investigational drug within the past 30 days 5. Patients who are unlikely to be able to complete the study or comply with the visit schedule 6. Any disease or planned therapy which will interfere with the procedures or data collection of this trial |
| Date of first enrolment | 01/10/2009 |
| Date of final enrolment | 31/12/2012 |
Locations
Countries of recruitment
- United Kingdom
- Australia
- Belgium
- Brazil
- Canada
- Finland
- France
- South Africa
- United States of America
Study participating centre
Shriners Hospitals for Children
Montreal
H3G 1A6
Canada
H3G 1A6
Canada
Results and Publications
| Individual participant data (IPD) Intention to share | No |
|---|---|
| IPD sharing plan summary | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Participant information sheet | Participant information sheet | 11/11/2025 | 11/11/2025 | No | Yes |
Editorial Notes
10/05/2019: No publications found. Verifying results with principal investigator.
02/10/2017: No publications found, verifying study status with principal investigator.
