Study of Teriparatide (FORTEO) to Treat Adults With Osteogenesis Imperfecta
Information source: Oregon Health and Science University
Information obtained from ClinicalTrials.gov on February 07, 2013
Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Osteogenesis Imperfecta
Intervention: Teriparatide (FORTEO) (Drug)
Phase: Phase 4
Status: Recruiting
Sponsored by: Oregon Health and Science University Official(s) and/or principal investigator(s):
Eric S Orwoll, M.D., Principal Investigator, Affiliation: Oregon Health and Science University
Robert Steiner, M.D., Principal Investigator, Affiliation: Oregon Health and Science University
Jay Shapiro, M.D., Principal Investigator, Affiliation: Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Branden Lee, M.D., PhD, Principal Investigator, Affiliation: Balor College of Medicine
Sandra Veith, CRA, Principal Investigator, Affiliation: Oregon Health and Science University
Sven Prevrhal, PhD, Principal Investigator, Affiliation: University of California at San Francisco
Peter Byers, M.D., Principal Investigator, Affiliation: Universtiy of Washington
Overall contact:
Sandra L Veith, CCRA, Phone: 503 494.5630, Email: veithsa@ohsu.edu
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).
Clinical Details
Official title: A Study to Assess the Effectiveness of Teriparatide (FORTEO) for Increasing Bone Mass and Improving Bone Structure in Adults Affected With Osteogenesis Imperfecta (OI)
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator), Primary Purpose: Treatment
Primary outcome: The primary aim of this study is to assess whether an increase in bone mineral density (BMD) will result from the use of rhPTH.
Secondary outcome: A secondary aim is to determine if PTH therapy will decrease fragility fractures in individuals affected with 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, 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.
Eligibility
Minimum age: 18 Years.
Maximum age: 85 Years.
Gender(s): Both.
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)
Locations and Contacts
Sandra L Veith, CCRA, Phone: 503 494.5630, Email: veithsa@ohsu.edu
Kennedy Krieger Institute, Baltimore, Maryland 21205, United States; Recruiting
Jay Shapiro, MD, Phone: 443-923-2703, Email: shapiro@kennedykrieger.org
Pamela Melvin, RN, Phone: 443-923-2707, Email: melvin@kennedykrieger.org
Jay Shapiro, MD, Principal Investigator
Oregon Health & Science University, Portland, Oregon 97239-3098, United States; Recruiting
Sandra L Veith, CCRA, Phone: 503-494-5630, Email: veithsa@ohsu.edu
Eric S Orwoll, M.D., Principal Investigator
Baylor College of Medicine, Department of Molecular and Human Gentics, Houston, Texas 77030, United States; Recruiting
Branden Lee, MD, PhD
Mary A Mullins, RN, Phone: 832-822.4263, Email: mullins@bcm.tmc.edu
Branden Lee, MD, PhD, Principal Investigator
Additional Information
Starting date: June 2005
Last updated: February 3, 2009
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