Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta

Study Overview
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Brief Summary
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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
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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.

Official Title
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A Study to Assess the Effectiveness of Teriparatide (FORTEO) for Increasing Bone Mass and Improving Bone Strength in Adults Affected With Osteogenesis Imperfecta (OI)

Conditions
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Osteogenesis Imperfecta

Intervention / Treatment
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* Drug: Teriparatide (FORTEO)
* Drug: Placebos


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 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 Exclusion Criteria: 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)

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 85 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Teriparatide (FORTEO)<br>Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months | Drug: Teriparatide (FORTEO)<br>* Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily<br>* Other names: FORTEO;|
| Placebo Comparator: Placebo<br>Daily SQ placebo for 18 months | Drug: Placebos<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Spine Bone Mineral Density (BMD) | bone density by dual energy xray absorptiometry | baseline and 18 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Total Hip BMD | bone density by dual energy xray absorptiometry | baseline and 18 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Osteogenesis Imperfecta, Brittle Bone Disease, Fragility Fractures