Osteogenesis ImperfectaPhase 4NCT00159419
Bisphosphonate Therapy for Osteogenesis Imperfecta
A Phase 4 study testing Alendronate and Pamidronate in people with Osteogenesis Imperfecta and Osteoporosis, run by Indiana University.
Trial summary
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." We, the researchers at Indiana University School of Medicine, are characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate… Read more about this study
- Who is it for?
- Ages 3 to 21 years
- Conditions the study is for
- Osteogenesis Imperfecta, Osteoporosis, Paget Disease of Bone
- What is being tested?
- Alendronate and Pamidronate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- No sites listed yet
- Study dates
- Aug 1999to Aug 2008
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 3 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) <2.5 standard deviations (SD) for age plus two of the following:
- Family history of OI
- Frequent fractures
- Blue sclerae
- Multiple wormian bones on skull x-ray
- Hearing disturbance
Show all 10Show fewer
- Dentinogenesis imperfecta
- Age between 3 and 21 years at the start of the study period.
- Children must be able to swallow whole tablets
- Parents of children must be able to understand protocol and give informed consent.
Who cannot join
- Therapy with bisphosphonates during the past 12 months.
- Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.
- Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption
About this study
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." We, the researchers at Indiana University School of Medicine, are characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate therapy in a group of children with OI and also in children with other disorders that result in low bone mass and fractures.
Read the detailed description
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." OI is an inherited disorder of collagen synthesis. Collagen is the major structural protein of the matrix of tendons, skin, and bones. Affected persons have low bone mineral density (and experience multiple fractures and progressive bony deformity). In its most severe form, the disorder is lethal in infancy. We plan to characterize the changes effected by oral bisphosphonate therapy and compare them to a regimen of intravenous bisphosphonate therapy in a group of children with OI.
Additionally, we have begun to treat patients with OI and other conditions of low bone mineralization for age who are not eligible for the standard protocol (too young, history of abdominal pain, etc.) with bisphosphonate. We also plan to screen the parents and siblings of our patients diagnosed with osteogenesis imperfecta, in order to determine if they also have osteoporosis.
- Conditions
- Osteogenesis Imperfecta, Osteoporosis, Paget Disease of Bone
- Being tested
- Alendronate, Pamidronate
- Keywords
- Osteogenesis Imperfecta, Fractures, Pediatric, Osteoporosis, Juvenile Pagets
- Medical subject headings
- Osteogenesis Imperfecta, Osteoporosis, Osteitis Deformans, Fractures, Bone
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlendronateDrug
Also called fosamax
PamidronateDrug
| Study group | Role | What they receive |
|---|---|---|
| Alendronate | Active comparator | 1 mg/kg po qd rounded to nearest 10 or 20 mg dose Receives: Drug: Alendronate |
| Pamidronate | Active comparator | 3 mg/kg IV q4 months Receives: Drug: Pamidronate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bone Mineral Density
By Dual-energy x-ray absorptiometry. Results were reported as z-scores as well as as absolute values. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Sep 2005 (estimated)
- Study startAug 1999
- Primary completionAug 2008
- Study completionAug 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Linda DiMeglio, MD, Indiana University
- Investigators
- Linda A DiMeglio, MD, MPH, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00159419
- Sponsor's protocol ID
- 9902-30
- Status last verified
- May 2017
- First submitted
- 7 Sep 2005
- Last updated
- 14 Jun 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 14 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Bisphosphonate Therapy for Osteogenesis Imperfecta
- ClinicalTrials.gov ID
- NCT00159419
- Acronym / sponsor ID
- 9902-30
- Conditions
- Osteogenesis Imperfecta, Osteoporosis, Paget Disease of Bone
- Keywords
- Osteogenesis Imperfecta, Fractures, Pediatric, Osteoporosis, Juvenile Pagets
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Study start
- Aug 1999
- Primary completion
- Aug 2008 (actual)
- Study completion
- Aug 2008 (actual)
- First posted
- 12 Sep 2005 (estimated)
- Results
- Posted 31 Mar 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." We, the researchers at Indiana University School of Medicine, are characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate therapy in a group of children with OI and also in children with other disorders that result in low bone mass and fractures.
Detailed description
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." OI is an inherited disorder of collagen synthesis. Collagen is the major structural protein of the matrix of tendons, skin, and bones. Affected persons have low bone mineral density (and experience multiple fractures and progressive bony deformity). In its most severe form, the disorder is lethal in infancy. We plan to characterize the changes effected by oral bisphosphonate therapy and compare them to a regimen of intravenous bisphosphonate therapy in a group of children with OI.
Additionally, we have begun to treat patients with OI and other conditions of low bone mineralization for age who are not eligible for the standard protocol (too young, history of abdominal pain, etc.) with bisphosphonate. We also plan to screen the parents and siblings of our patients diagnosed with osteogenesis imperfecta, in order to determine if they also have osteoporosis.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Alendronate | Active comparator | Drug: Alendronate1 mg/kg po qd rounded to nearest 10 or 20 mg dose |
| Pamidronate | Active comparator | Drug: Pamidronate3 mg/kg IV q4 months |
Who may qualify
- Ages
- 3 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (10 listed)
- Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) <2.5 standard deviations (SD) for age plus two of the following:
- Age between 3 and 21 years at the start of the study period.
- Children must be able to swallow whole tablets
- Parents of children must be able to understand protocol and give informed consent.
Key exclusion criteria (3 listed)
- Therapy with bisphosphonates during the past 12 months.
- Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.
- Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption
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 |
|---|---|---|
| AlendronateAlso: fosamax | Drug | Alendronate |
| Pamidronate | Drug | Pamidronate |
Primary outcome
Bone Mineral Density
Time frame: 2 years
By Dual-energy x-ray absorptiometry. Results were reported as z-scores as well as as absolute values. The Z-score indicates the number of standard deviations away from the mean. A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher. Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jun 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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.
Linda A DiMeglio, MD, MPH principal investigator
Indiana University
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Alendronate and Pamidronate in people with Osteogenesis Imperfecta and Osteoporosis, run by Indiana University.
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 10 inclusion and 3 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 14 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 Mar 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Bone Mineral Density (time frame: 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 14 Jun 2017.