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Bisphosphonate Therapy for Osteogenesis Imperfecta

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Linda DiMeglio, MD, Indiana University
ClinicalTrials.gov Identifier:
NCT00159419
First received: September 7, 2005
Last updated: September 26, 2011
Last verified: September 2011
  Purpose

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
Osteoporosis
Paget Disease of Bone
Drug: Alendronate
Drug: Pamidronate
Phase 4

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
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 measured 4 times monthly [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]
  • fracture rates [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Audiologic effects (annual) [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]
  • dental effects (annual) [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]
  • pain assessments [ Time Frame: 6 years ] [ Designated as safety issue: No ]
  • bone turnover assessments [ Time Frame: 6 years ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 30
Study Start Date: August 1999
Study Completion Date: August 2008
Primary Completion Date: August 2008 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Alendronate
    10 mg po q day
    Other Name: fosamax
    Drug: Pamidronate
    1mg/kg/day x 3days
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.

  Eligibility

Ages Eligible for Study:   3 Years to 21 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00159419

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

No publications provided

Responsible Party: Linda DiMeglio, MD, MD, Indiana University
ClinicalTrials.gov Identifier: NCT00159419     History of Changes
Other Study ID Numbers: 9902-30
Study First Received: September 7, 2005
Last Updated: September 26, 2011
Health Authority: United States: Institutional Review Board

Keywords provided by Indiana University:
Osteogenesis Imperfecta
Fractures
Pediatric
Osteoporosis
Juvenile Pagets

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Bone Diseases
Osteitis Deformans
Osteoporosis
Bone Diseases, Developmental
Bone Diseases, Metabolic
Collagen Diseases
Connective Tissue Diseases
Genetic Diseases, Inborn
Musculoskeletal Diseases
Osteochondrodysplasias
Diphosphonates
Bone Density Conservation Agents
Pharmacologic Actions
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on November 27, 2014