Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
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). There is no established medical therapy for adults with the disorder. Virtually all of the studies reviewing potential treatments for OI have evaluated the effects of medications only on children with OI. There is no data concerning the usefulness of parathyroid hormone therapy in OI. The working hypothesis is that adults affected with OI who are treated with FORTEO will experience increased spine and hip bone mineral density and an increase in bone width and thickness. Adult patients with OI will be enrolled in this study for 18 months. Half the patients will receive PTH (FORTEO) and the other half placebo (no active drug). Blood, urine, and bone density tests will be done during the study for safety monitoring.
There are three clinic sites for this study; Oregon Health & Science University (Portland, OR), Kennedy Krieger Institute (Johns Hopkins University, Baltimore, MD), and Baylor College of Medicine (Houston, TX).
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, the major component of bones, characterized by multiple fractures and fragile bones. OI affects approximately 1 to 2 out of every 10,000 individuals of all racial and ethnic origins. There is no cure for osteogenesis imperfecta and there is no established medical therapy for adults with the disorder. Virtually all of the studies reviewing potential treatments for OI have evaluated the effects of medications only on children with OI. Early studies included the use of anabolic steroids, sodium fluoride and testosterone, Vitamins C and D, and calcitonin 1, 4 with minimal or no improvement in bone formation. There is no data concerning the usefulness of parathyroid hormone therapy in OI. Daily low-dose administration of parathyroid hormone (PTH) produces a bone building effect on bone. An effective bone building therapy available for the treatment of adult patients with OI would be an extremely attractive and valuable asset not only to the affected patients but also to the medical community at large.
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 width and thickness. Although FORTEO is not expected to change the defect in the collagen produced, it is expected to increase the quantity of bone formed and improve bone thickness. Therefore, the researchers hypothesize that overall bone strength will be enhanced in OI and fracture incidence will be reduced. Patients with OI will be enrolled in this study for about 18 months. Half the patients will receive PTH (FORTEO) and the other half placebo (no active drug). Blood, urine, and bone density tests will be done during the study for safety monitoring and to see if the PTH is working. Participants will be recruited through the Bone and Mineral Disease clinics, Pediatrics Clinics, Orthopedic clinics, from families of OI patients, the Osteogenesis Imperfecta Foundation, and the general public. The OI Foundation is very enthusiastic about the study and has agreed to alert their membership concerning it. The study will be conducted at each site's General Clinical Research Center.
Study Design
Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Conditions
Osteogenesis Imperfecta
Intervention
Teriparatide (FORTEO)
Location
Kennedy Krieger Institute
Baltimore
Maryland
United States
21205
Status
Recruiting
Source
Oregon Health and Science University
Results (where available)
Links
- Source: http://clinicaltrials.gov/show/NCT00131469
- Information obtained from ClinicalTrials.gov on July 15, 2010
Medical and Biotech [MESH] Definitions
Osteogenesis Imperfecta
Autosomal dominant COLLAGEN DISEASES resulting from defective biosynthesis of COLLAGEN TYPE I and characterized by brittle, osteoporotic, and easily fractured bones. It may also present with blue sclerae, loose joints, and imperfect dentin formation. There are four major types, I-IV.
Teriparatide
A polypeptide that consists of the 1-34 amino-acid fragment of human PARATHYROID HORMONE, the biologically active N-terminal region. The acetate form is given by intravenous infusion in the differential diagnosis of HYPOPARATHYROIDISM and PSEUDOHYPOPARATHYROIDISM. (Reynolds JEF(Ed): Martindale: The Extra Pharmacopoeia (electronic version). Micromedex, Inc, Englewood, CO, 1995)
Chondrogenesis
The formation of cartilage. This process is directed by CHONDROCYTES which continually divide and lay down matrix during development. It is sometimes a precursor to OSTEOGENESIS.
Amelogenesis Imperfecta
A clinically and genetically heterogeneous group of hereditary conditions characterized by malformed DENTAL ENAMEL, usually involving DENTAL ENAMEL HYPOPLASIA and/or TOOTH HYPOMINERALIZATION.
Osteogenesis
The process of bone formation. Histogenesis of bone including ossification.
Clinical Trials
Bisphosphonate Therapy for Osteogenesis Imperfecta
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 ch...
This study is being conducted to collect information from subjects with osteoporosis regarding their experience using the Forteo B Pen to self-administer teriparatide in the community sett...
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures. There are no approved drugs for the treatment of OI in children, even though some intravenous (IV) b...
Zoledronic Acid in Children (1 -17 Years) With Severe Osteogenesis Imperfecta
This is an extension study to examine the long-term safety and efficacy of zoledronic acid in patients who have completed the core CZOL446H2202 study.
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 t...
PubMed Articles
Distal patellar tendon avulsion fracture in a football player with osteogenesis imperfecta.
Proximal patellar tendon ruptures have been described in adults with osteogenesis imperfecta, but distal avulsions from the tibial tubercle have not. This is the first case reporting a distal patellar...
New perspectives on osteogenesis imperfecta.
A new paradigm has emerged for osteogenesis imperfecta as a collagen-related disorder. The more prevalent autosomal dominant forms of osteogenesis imperfecta are caused by primary defects in type I co...
The genetic basis for skeletal disease. Osteogenesis imperfecta and genetic abnormalities.
Patients with osteogenesis imperfecta (OI) represent various degrees of bone fragility and accompany many clinical manifestations such as dentinogenesis imperfecta, blue sclera, growth disturbance, he...
Recent Advances in Osteogenesis Imperfecta.
"Osteogenesis imperfecta" is a term used to describe a group of genetic disorders of variable phenotype usually defined by recurrent fractures, low bone mass, and skeletal fragility. Most cases are as...
The genetic basis for skeletal disease. Osteogenesis imperfecta.
Osteogenesis imperfecta (OI) is characterized by its bone fragility. But the spectrum of the severity of the fragility is widely distributed. Recently, amino-bisphosphonate has been introduced into th...
