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

• Sponsor: Oregon Health and Science University (Other)
• Overall Status: Completed
• CT.gov ID: NCT00131469
• Collaborator: Eli Lilly and Company (Industry), Osteogenesis Imperfecta Foundation (Other), National Institutes of Health (NIH) (NIH), National Center for Research Resources (NCRR) (NIH)
• Enrollment: 79
• Locations: 3
• Arms: 2
• Duration (Months): 67
• Patients Per Site: 26.3
• Patients Per Site Per Month: 0.4

Study Details

Study Description

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).

Condition or Disease	Intervention/Treatment	Phase
Osteogenesis Imperfecta	Drug: Teriparatide (FORTEO); Drug: Placebos	Phase 4

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 79 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Triple (Participant, Care Provider, Investigator)
• Primary Purpose: Treatment
• 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 Start Date: Jun 1, 2005
• Actual Primary Completion Date: Jan 1, 2011
• Actual Study Completion Date: Jan 1, 2011

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Teriparatide (FORTEO) Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months	Drug: Teriparatide (FORTEO); Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily; Other Names: FORTEO
Placebo Comparator: Placebo Daily SQ placebo for 18 months	Drug: Placebos

Outcome Measures

Primary Outcome Measures

1. Spine Bone Mineral Density (BMD) [baseline and 18 months]

   bone density by dual energy xray absorptiometry

Secondary Outcome Measures

1. Total Hip BMD [baseline and 18 months]

   bone density by dual energy xray absorptiometry

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years to 85 Years
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Kennedy Krieger Institute	Baltimore	Maryland	United States	21205
2	Oregon Health & Science University	Portland	Oregon	United States	97239-3098
3	Baylor College of Medicine, Department of Molecular and Human Gentics	Houston	Texas	United States	77030

Sponsors and Collaborators

• Oregon Health and Science University
• Eli Lilly and Company
• Osteogenesis Imperfecta Foundation
• National Institutes of Health (NIH)
• National Center for Research Resources (NCRR)

Investigators

• Principal Investigator: Eric S Orwoll, M.D., Oregon Health and Science University
• Principal Investigator: Jay Shapiro, M.D., Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
• Principal Investigator: Brendan Lee, M.D., PhD, Balor College of Medicine
• Principal Investigator: Sandra Veith, CRA, Oregon Health and Science University

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

• Osteogenesis Imperfecta Foundation

Publications

None provided.

• Responsible Party: Eric Orwoll, MD, Professor Of Medicine, Oregon Health and Science University
• ClinicalTrials.gov Identifier: NCT00131469
• Other Study ID Numbers: IBMD-OI; UL1RR024140
• First Posted: Aug 18, 2005
• Last Update Posted: Apr 24, 2019
• Last Verified: Apr 1, 2019

• Keywords provided by Eric Orwoll, MD, Professor Of Medicine, Oregon Health and Science University: Osteogenesis Imperfecta; Brittle Bone Disease; Fragility Fractures
• Additional relevant MeSH terms: Osteogenesis Imperfecta; Teriparatide

Study Results

Participant Flow

Pre-assignment Detail	One participant was formally enrolled but then dropped out before the first study treatment was administered.

Arm/Group Title	Teriparatide (FORTEO)	Placebo
Arm/Group Description	Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Teriparatide (FORTEO): Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily	Daily SQ placebo for 18 months Placebos
Period Title: Overall Study
STARTED	38	40
COMPLETED	29	27
NOT COMPLETED	9	13

Baseline Characteristics

Arm/Group Title	Teriparatide (FORTEO)	Placebo	Total
Arm/Group Description	Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Teriparatide (FORTEO): Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily	Daily SQ placebo for 18 months Placebos	Total of all reporting groups
Overall Participants	38	40	78
Age (years) [Mean (Standard Deviation) ]
Mean (Standard Deviation) [years]	40.8 (12.9)	41.2 (10.1)	41 (11.5)
Sex: Female, Male (Count of Participants)
Female	22 57.9%	24 60%	46 59%
Male	16 42.1%	16 40%	32 41%
Race and Ethnicity Not Collected (Count of Participants)
Count of Participants [Participants]	0 0%

Outcome Measures

1. Primary Outcome

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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Teriparatide (FORTEO)	Placebo
Arm/Group Description	Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Teriparatide (FORTEO): Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily	Daily SQ placebo for 18 months Placebos
Measure Participants	29	27
Mean (Standard Error) [percentage of change in g/cm2]	6.1 (1.5)	2.8 (1.3)

2. Secondary Outcome

Title	Total Hip BMD
Description	bone density by dual energy xray absorptiometry
Time Frame	baseline and 18 months

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Teriparatide (FORTEO)	Placebo
Arm/Group Description	Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Teriparatide (FORTEO): Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily	Daily SQ placebo for 18 months Placebos
Measure Participants	22	26
Mean (Standard Error) [percentage of change in g/cm2]	2.6 (2)	-2.4 (1.8)

Adverse Events

Time Frame	18 months
Arm/Group Title	Teriparatide (FORTEO)		Placebo
Arm/Group Description	Once daily SQ administration of Teriparatide (FORTEO) 20 ug for 18 months Teriparatide (FORTEO): Teriparatide (FORTEO) 20mcg, subcutaneous injection, once daily		Daily SQ placebo for 18 months Placebos
All Cause Mortality
	Teriparatide (FORTEO)		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0%)		1/40 (2.5%)
Serious Adverse Events
	Teriparatide (FORTEO)		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0%)		0/40 (0%)
Other (Not Including Serious) Adverse Events
	Teriparatide (FORTEO)		Placebo
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	0/38 (0%)		0/40 (0%)

Limitations/Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.