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

NCT ID: NCT00131469

Last Updated: 2019-04-24

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 79 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-06-30
• Study Completion Date: 2011-01-31

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.

Conditions

Osteogenesis Imperfecta

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Teriparatide (FORTEO)

Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months

Group Type: ACTIVE_COMPARATOR

Teriparatide (FORTEO)

Intervention Type: DRUG

Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily

Placebo

Daily SQ placebo for 18 months

Group Type: PLACEBO_COMPARATOR

Placebos

Intervention Type: DRUG

Interventions

Teriparatide (FORTEO)

Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily

Intervention Type: DRUG

Placebos

Intervention Type: DRUG

Other Intervention Names

FORTEO

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)

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Eli Lilly and Company

INDUSTRY

Sponsor Role: collaborator

Osteogenesis Imperfecta Foundation

OTHER

Sponsor Role: collaborator

National Institutes of Health (NIH)

NIH

Sponsor Role: collaborator

National Center for Research Resources (NCRR)

NIH

Sponsor Role: collaborator

Oregon Health and Science University

OTHER

Sponsor Role: lead

Responsible Party

Eric Orwoll, MD

Professor Of Medicine

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Eric S Orwoll, M.D.

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Jay Shapiro, M.D.

Role: PRINCIPAL_INVESTIGATOR

Hugo W. Moser Research Institute at Kennedy Krieger, Inc.

Brendan Lee, M.D., PhD

Role: PRINCIPAL_INVESTIGATOR

Balor College of Medicine

Sandra Veith, CRA

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Locations

Kennedy Krieger Institute

Baltimore, Maryland, United States

Oregon Health & Science University

Portland, Oregon, United States

Baylor College of Medicine, Department of Molecular and Human Gentics

Houston, Texas, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 24463451 (View on PubMed)

Related Links

http://oif.org

Osteogenesis Imperfecta Foundation

Other Identifiers

UL1RR024140

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IBMD-OI

Identifier Type: -

Identifier Source: org_study_id