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Bisphosphonate Therapy for Osteogenesis Imperfecta

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ClinicalTrials.gov Identifier: NCT00159419
Recruitment Status : Completed
First Posted : September 12, 2005
Results First Posted : March 31, 2016
Last Update Posted : June 14, 2017
Sponsor:
Information provided by (Responsible Party):
Linda DiMeglio, MD, Indiana University

Brief Summary:
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 or disease Intervention/treatment Phase
Osteogenesis Imperfecta Osteoporosis Paget Disease of Bone Drug: Alendronate Drug: Pamidronate Phase 4

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 : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Bisphosphonate Therapy for Osteogenesis Imperfecta
Study Start Date : August 1999
Actual Primary Completion Date : August 2008
Actual Study Completion Date : August 2008


Arm Intervention/treatment
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



Primary Outcome Measures :
  1. 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.



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Ages Eligible for Study:   3 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
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
    • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00159419


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

Responsible Party: Linda DiMeglio, MD, MD, Indiana University
ClinicalTrials.gov Identifier: NCT00159419     History of Changes
Other Study ID Numbers: 9902-30
First Posted: September 12, 2005    Key Record Dates
Results First Posted: March 31, 2016
Last Update Posted: June 14, 2017
Last Verified: May 2017

Keywords provided by Linda DiMeglio, MD, Indiana University:
Osteogenesis Imperfecta
Fractures
Pediatric
Osteoporosis
Juvenile Pagets

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