Osteogenesis ImperfectaPhase 4OINCT00131469
Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
A Phase 4 study testing Teriparatide (FORTEO) in people with Osteogenesis Imperfecta, run by Oregon Health and Science University at 3 sites in United States.
Trial summary
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta (OI). Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Teriparatide (FORTEO)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 79 participants
- Where
- 3 sitesUnited States
- Study dates
- Jun 2005to Jan 2011
- 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 18 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Previous established diagnosis of Osteogenesis Imperfecta AND
- > 2 previous adult fractures, AND/OR
- BMD at lumbar spine, femoral neck or total hip T score < -2.0
Who cannot join
- Open epiphyses.
- History of external beam radiation to the skeleton.
- Pagets disease.
- Bone metastases or skeletal malignancies.
- Total lifetime exposure to any antiresorptive medication < 90 days (Primary Inclusion).
- Treatment with any antiresorptive medication 12 months proceeding enrollment - (Secondary Inclusion).
- Women with OI who are pregnant or unwilling to use 1 form of contraception.
- Vitamin D insufficiency (25-hydroxyvitamin D <15ng/ml)
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Baylor College of Medicine, Department of Molecular and Human Gentics
Houston, Texas, 77030
Oregon Health & Science University
Portland, Oregon, 97239-3098
About this study
Official title: A Study to Assess the Effectiveness of Teriparatide (FORTEO) for Increasing Bone Mass and Improving Bone Strength in Adults Affected With Osteogenesis Imperfecta (OI)
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta (OI).
Read the detailed description
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta (OI). Osteogenesis imperfecta is an inherited disorder of type I collagen, a major component of bones, and is characterized by multiple fractures and deformities. OI affects approximately 1-2 of every 10,000 individuals. Virtually all of the studies of potential treatments for OI have evaluated the effects of medications only on children with OI. There is no cure for osteogenesis imperfecta and there is no established medical therapy for adults with the disorder. There are very limited data concerning the usefulness of parathyroid hormone therapy in OI. An effective anabolic therapy for the treatment of adult patients with OI could be a valuable asset to the affected patients. In this study, the working hypothesis is that individuals affected with OI who are treated with Forteo will experience increased spine and hip bone mineral density and an increase in bone strength. Although Forteo is not expected to change the defect in the collagen produced, but is postulated to increase the quantity of bone formed and improve bone strength.
This will be a placebo controlled, double blinded trial; half the patients will receive Forteo 20 ug/day SQ. Adult patients (age at least 18 yrs) with OI will be enrolled for a treatment duration of 18 months. Blood, urine, and bone density/strength tests will be done during the study to assess efficacy and safety.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- Teriparatide (FORTEO), Placebos
- Keywords
- Osteogenesis Imperfecta, Brittle Bone Disease, Fragility Fractures
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Teriparatide (FORTEO)Drug
Also called FORTEO
Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily
PlacebosDrug
| Study group | Role | What they receive |
|---|---|---|
| Teriparatide (FORTEO) | Active comparator | Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Receives: Drug: Teriparatide (FORTEO) |
| Placebo | Placebo comparator | Daily SQ placebo for 18 months Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Spine Bone Mineral Density (BMD)
bone density by dual energy xray absorptiometry
Measured: baseline and 18 months
Secondary outcomes (1)
Total Hip BMD
bone density by dual energy xray absorptiometry
Measured: baseline and 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2005 (estimated)
- Study startJun 2005
- Primary completionJan 2011
- Study completionJan 2011
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- More trials run by Oregon Health and Science UniversityThe sponsor's other registered studies.
- 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
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 79 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oregon Health and Science University
- Sponsor type
- University, hospital or other
- Collaborators
- Eli Lilly and Company, Osteogenesis Imperfecta Foundation, National Institutes of Health (NIH), National Center for Research Resources (NCRR)
- Responsible party
- Principal investigator, Eric Orwoll, MD, Oregon Health and Science University
- Investigators
- Eric S Orwoll, M.D., Principal investigator, Oregon Health and Science UniversityJay Shapiro, M.D., Principal investigator, Hugo W. Moser Research Institute at Kennedy Krieger, Inc.Brendan Lee, M.D., PhD, Principal investigator, Balor College of MedicineSandra Veith, CRA, Principal investigator, Oregon Health and Science University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00131469
- Sponsor's protocol ID
- IBMD-OI
- Other IDs
- UL1RR024140
- Acronym
- OI
- Status last verified
- Apr 2019
- First submitted
- 16 Aug 2005
- Last updated
- 24 Apr 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Orwoll ES, Shapiro J, Veith S, Wang Y, Lapidus J, Vanek C, Reeder JL, Keaveny TM, Lee DC, Mullins MA, Nagamani SC, Lee B. Evaluation of teriparatide treatment in adults with osteogenesis imperfecta. J Clin Invest. 2014 Feb;124(2):491-8. doi: 10.1172/JCI71101. Epub 2014 Jan 27.
PubMed 24463451
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Study to Assess the Effectiveness of Teriparatide (FORTEO) for Increasing Bone Mass and Improving Bone Strength in Adults Affected With Osteogenesis Imperfecta (OI)
- ClinicalTrials.gov ID
- NCT00131469
- Acronym / sponsor ID
- OI / IBMD-OI
- Conditions
- Osteogenesis Imperfecta
- Keywords
- Osteogenesis Imperfecta, Brittle Bone Disease, Fragility Fractures
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 79 participants (actual)
- Lead sponsor
- Oregon Health and Science UniversityUniversity, hospital or other
- Collaborators
- Eli Lilly and Company, Osteogenesis Imperfecta Foundation, National Institutes of Health (NIH), National Center for Research Resources (NCRR)
- Study start
- Jun 2005
- Primary completion
- Jan 2011 (actual)
- Study completion
- Jan 2011 (actual)
- First posted
- 18 Aug 2005 (estimated)
- Results
- Posted 24 Apr 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta (OI).
Detailed description
The purpose of this study is to determine the effectiveness of teriparatide (FORTEO), which is human parathyroid hormone 1-34, for increasing bone mass and improving bone structure in adults affected with Osteogenesis Imperfecta (OI). Osteogenesis imperfecta is an inherited disorder of type I collagen, a major component of bones, and is characterized by multiple fractures and deformities. OI affects approximately 1-2 of every 10,000 individuals. Virtually all of the studies of potential treatments for OI have evaluated the effects of medications only on children with OI. There is no cure for osteogenesis imperfecta and there is no established medical therapy for adults with the disorder. There are very limited data concerning the usefulness of parathyroid hormone therapy in OI. An effective anabolic therapy for the treatment of adult patients with OI could be a valuable asset to the affected patients. In this study, the working hypothesis is that individuals affected with OI who are treated with Forteo will experience increased spine and hip bone mineral density and an increase in bone strength. Although Forteo is not expected to change the defect in the collagen produced, but is postulated to increase the quantity of bone formed and improve bone strength.
This will be a placebo controlled, double blinded trial; half the patients will receive Forteo 20 ug/day SQ. Adult patients (age at least 18 yrs) with OI will be enrolled for a treatment duration of 18 months. Blood, urine, and bone density/strength tests will be done during the study to assess efficacy and safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Teriparatide (FORTEO) | Active comparator | Drug: Teriparatide (FORTEO)Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months |
| Placebo | Placebo comparator | Drug: PlacebosDaily SQ placebo for 18 months |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Previous established diagnosis of Osteogenesis Imperfecta AND
- > 2 previous adult fractures, AND/OR
- BMD at lumbar spine, femoral neck or total hip T score < -2.0
Key exclusion criteria (8 listed)
- Open epiphyses.
- History of external beam radiation to the skeleton.
- Pagets disease.
- Bone metastases or skeletal malignancies.
- Total lifetime exposure to any antiresorptive medication < 90 days (Primary Inclusion).
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 |
|---|---|---|
| Teriparatide (FORTEO)Also: FORTEOTeriparatide (FORTEO) 20mcg, subcutaneous injection, once daily | Drug | Teriparatide (FORTEO) |
| Placebos | Drug | Placebo |
Primary outcome
Spine Bone Mineral Density (BMD)
Time frame: baseline and 18 months
bone density by dual energy xray absorptiometry
1 secondary outcome
Total Hip BMD
Time frame: baseline and 18 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 24463451Orwoll ES, Shapiro J, Veith S, Wang Y, Lapidus J, Vanek C, Reeder JL, Keaveny TM, Lee DC, Mullins MA, Nagamani SC, Lee B. Evaluation of teriparatide treatment in adults with osteogenesis imperfecta. J Clin Invest. 2014 Feb;124(2):491-8. doi: 10.1172/JCI71101. Epub 2014 Jan 27.
- Osteogenesis Imperfecta FoundationLink provided by the sponsor.
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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.
Eric S Orwoll, M.D. principal investigator
Oregon Health and Science University
Jay Shapiro, M.D. principal investigator
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Brendan Lee, M.D., PhD principal investigator
Balor College of Medicine
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Teriparatide (FORTEO) in people with Osteogenesis Imperfecta, run by Oregon Health and Science University at 3 sites in United States.
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 3 inclusion and 8 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 24 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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. Some participants in this study may receive a placebo. 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 24 Apr 2019). 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?
Spine Bone Mineral Density (BMD) (time frame: baseline and 18 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00131469, which also lists 1 linked publication.
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 24 Apr 2019.