Osteogenesis ImperfectaPhase 3INFOINCT00982124
An Efficacy and Safety Trial of Intravenous Zoledronic Acid in Infants Less Than One Year of Age, With Severe Osteogenesis Imperfecta
A Phase 3 study testing Zoledronic Acid in people with Osteogenesis Imperfecta, run by Shriners Hospitals for Children at 1 site in Canada.
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. Read more about this study
- Who is it for?
- Ages Up to 12 months
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Zoledronic Acid
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteCanada
- Study dates
- Oct 2007to Apr 2016
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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
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
- Being tested
- Zoledronic Acid
- Keywords
- Osteogenesis Imperfecta, Infants with moderate to severe Osteogenesis Imperfecta
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Zoledronic AcidDrug
Also called Aclasta
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).
| Study group | Role | What they receive |
|---|---|---|
| Treatment Arm (only) | Experimental | Zoledronic acid infusion Receives: Drug: Zoledronic Acid |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 3 times during 10 visits within 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Sep 2009 (estimated)
- Study startOct 2007
- Primary completionSep 2015
- Study completionApr 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shriners Hospitals for Children
- Sponsor type
- University, hospital or other
- Collaborators
- Novartis
- Responsible party
- Principal investigator, Francis H. Glorieux, Shriners Hospitals for Children
- Investigators
- Francis H. Glorieux, MD, PhD, Principal investigator, McGill University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00982124
- Sponsor's protocol ID
- SHC-INFOI
- Other IDs
- IRB - A06-M73-06A, Health Canada - 9427-S1926-24C
- Acronym
- INFOI
- Status last verified
- Apr 2016
- First submitted
- 21 Sep 2009
- Last updated
- 19 Apr 2016
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Apr 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00982124
- Acronym / sponsor ID
- INFOI / SHC-INFOI
- Conditions
- Osteogenesis Imperfecta
- Keywords
- Osteogenesis Imperfecta, Infants with moderate to severe Osteogenesis Imperfecta
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Shriners Hospitals for ChildrenUniversity, hospital or other
- Collaborators
- Novartis
- Study start
- Oct 2007
- Primary completion
- Sep 2015 (actual)
- Study completion
- Apr 2016 (actual)
- First posted
- 22 Sep 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Supportive care
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Arm (only) | Experimental | Drug: Zoledronic AcidZoledronic acid infusion |
Who may qualify
- Ages
- Up to 12 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Zoledronic AcidAlso: AclastaEach 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). | Drug | Treatment Arm (only) |
Primary outcome
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
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Apr 2016.
Related trials for Osteogenesis Imperfecta
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Francis H. Glorieux, MD, PhD principal investigator
McGill University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Zoledronic Acid in people with Osteogenesis Imperfecta, run by Shriners Hospitals for Children at 1 site in Canada.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 19 Apr 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Apr 2016.