Clinical trial NCT00131469: Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Osteogenesis Imperfecta
Keywords: Osteogenesis Imperfecta
Brittle Bone Disease
Fragility Fractures
Lead sponsor: Oregon Health and Science University
Sponsor class: OTHER
Collaborators: name: Eli Lilly and Company
name: Osteogenesis Imperfecta Foundation
name: National Institutes of Health (NIH)
name: National Center for Research Resources (NCRR)
First posted: 2005-08-18
Enrollment: 79
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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).

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.

Eligibility criteria: 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)

Primary outcomes:
measure: Spine Bone Mineral Density (BMD)
timeFrame: baseline and 18 months
Secondary outcomes:
measure: Total Hip BMD
timeFrame: baseline and 18 months
Other outcomes:
Locations:
city: Baltimore
country: United States
facility: Kennedy Krieger Institute
state: Maryland
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Houston
country: United States
facility: Baylor College of Medicine, Department of Molecular and Human Gentics
state: Texas
Study officials:
affiliation: Oregon Health and Science University
name: Eric S Orwoll, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
name: Jay Shapiro, M.D.
role: PRINCIPAL_INVESTIGATOR
affiliation: Balor College of Medicine
name: Brendan Lee, M.D., PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Oregon Health and Science University
name: Sandra Veith, CRA
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.