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Bisphosphonate Therapy for Osteogenesis Imperfecta

This study has been completed.
Information provided by (Responsible Party):
Linda DiMeglio, MD, Indiana University Identifier:
First received: September 7, 2005
Last updated: February 29, 2016
Last verified: February 2016
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 characterizing the changes effected by oral bisphosphonate therapy and comparing them to a regimen of intravenous bisphosphonate therapy in a group of children with OI and also in children with other disorders that result in low bone mass and fractures.

Condition Intervention Phase
Osteogenesis Imperfecta
Paget Disease of Bone
Drug: Alendronate
Drug: Pamidronate
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Bisphosphonate Therapy for Osteogenesis Imperfecta

Resource links provided by NLM:

Further study details as provided by Indiana University:

Primary Outcome Measures:
  • Bone Mineral Density [ Time Frame: 2 years ]
    By DXA

Secondary Outcome Measures:
  • Pain Assessments [ Time Frame: 6 years ]
  • Bone Turnover Assessments [ Time Frame: 6 years ]

Enrollment: 18
Study Start Date: August 1999
Study Completion Date: August 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Alendronate
1 mg/kg po qd rounded to nearest 10 or 20 mg dose
Drug: Alendronate
Other Name: fosamax
Active Comparator: Pamidronate
3 mg/kg IV q4 months
Drug: Pamidronate

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.


Ages Eligible for Study:   3 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
    • Dentogenesis 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
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Please refer to this study by its identifier: NCT00159419

Sponsors and Collaborators
Indiana University School of Medicine
Principal Investigator: Linda A DiMeglio, MD, MPH Indiana University School of Medicine
  More Information

Responsible Party: Linda DiMeglio, MD, MD, Indiana University Identifier: NCT00159419     History of Changes
Other Study ID Numbers: 9902-30
Study First Received: September 7, 2005
Results First Received: November 12, 2013
Last Updated: February 29, 2016

Keywords provided by Indiana University:
Osteogenesis Imperfecta
Juvenile Pagets

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Bone Diseases
Osteitis Deformans
Bone Diseases, Metabolic
Musculoskeletal Diseases
Bone Diseases, Developmental
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Bone Density Conservation Agents
Physiological Effects of Drugs processed this record on May 25, 2017