- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00159419
Bisphosphonate Therapy for Osteogenesis Imperfecta
Study Overview
Status
Intervention / Treatment
Detailed Description
The study is designed to evaluate the efficacy and safety of "Bisphosphonate Therapy for Osteogenesis Imperfecta (OI)." OI is an inherited disorder of collagen synthesis. Collagen is the major structural protein of the matrix of tendons, skin, and bones. Affected persons have low bone mineral density (and experience multiple fractures and progressive bony deformity). In its most severe form, the disorder is lethal in infancy. We plan to characterize the changes effected by oral bisphosphonate therapy and compare them to a regimen of intravenous bisphosphonate therapy in a group of children with OI.
Additionally, we have begun to treat patients with OI and other conditions of low bone mineralization for age who are not eligible for the standard protocol (too young, history of abdominal pain, etc.) with bisphosphonate. We also plan to screen the parents and siblings of our patients diagnosed with osteogenesis imperfecta, in order to determine if they also have osteoporosis.
Study Type
Enrollment (Actual)
Phase
- Phase 4
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
Diagnosis of OI, as defined by genetic analysis revealing a defect of type I collagen, OR by bone mineral density (BMD) <2.5 standard deviations (SD) for age plus two of the following:
- Family history of OI
- Frequent fractures
- Blue sclerae
- Multiple wormian bones on skull x-ray
- Hearing disturbance
- Dentinogenesis imperfecta
- Age between 3 and 21 years at the start of the study period.
- Children must be able to swallow whole tablets
- Parents of children must be able to understand protocol and give informed consent.
Exclusion Criteria:
- Therapy with bisphosphonates during the past 12 months.
- Other "non-traditional" therapy for OI in the last 6 months, such as growth hormone or anabolic steroids.
- Other chronic diseases besides OI that interfere with bone morphology or gastrointestinal absorption
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Active Comparator: Alendronate
1 mg/kg po qd rounded to nearest 10 or 20 mg dose
|
Other Names:
|
Active Comparator: Pamidronate
3 mg/kg IV q4 months
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Bone Mineral Density
Time Frame: 2 years
|
By Dual-energy x-ray absorptiometry.
Results were reported as z-scores as well as as absolute values.
The Z-score indicates the number of standard deviations away from the mean.
A Z-score of 0 is equal to the mean with negative numbers indicating values lower than the mean and positive values higher.
Higher Z scores indicate a better outcome", or similar, as accurate and appropriate.
|
2 years
|
Collaborators and Investigators
Investigators
- Principal Investigator: Linda A DiMeglio, MD, MPH, Indiana University School of Medicine
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Genetic Diseases, Inborn
- Musculoskeletal Diseases
- Connective Tissue Diseases
- Bone Diseases, Metabolic
- Bone Diseases, Developmental
- Osteochondrodysplasias
- Collagen Diseases
- Osteoporosis
- Bone Diseases
- Osteogenesis Imperfecta
- Osteitis Deformans
- Physiological Effects of Drugs
- Bone Density Conservation Agents
- Alendronate
- Pamidronate
Other Study ID Numbers
- 9902-30
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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