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Bisphosphonate Therapy for Osteogenesis Imperfecta

Primary Purpose

Osteogenesis Imperfecta, Osteoporosis, Paget Disease of Bone

Status
Completed
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Alendronate
Pamidronate
Sponsored by
Indiana University School of Medicine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteogenesis Imperfecta focused on measuring Osteogenesis Imperfecta, Fractures, Pediatric, Osteoporosis, Juvenile Pagets

Eligibility Criteria

3 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

    Arms of the Study

    Arm 1

    Arm 2

    Arm Type

    Active Comparator

    Active Comparator

    Arm Label

    Alendronate

    Pamidronate

    Arm Description

    1 mg/kg po qd rounded to nearest 10 or 20 mg dose

    3 mg/kg IV q4 months

    Outcomes

    Primary Outcome Measures

    Bone Mineral Density
    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.

    Secondary Outcome Measures

    Full Information

    First Posted
    September 7, 2005
    Last Updated
    May 17, 2017
    Sponsor
    Indiana University School of Medicine
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00159419
    Brief Title
    Bisphosphonate Therapy for Osteogenesis Imperfecta
    Official Title
    Bisphosphonate Therapy for Osteogenesis Imperfecta
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    May 2017
    Overall Recruitment Status
    Completed
    Study Start Date
    August 1999 (undefined)
    Primary Completion Date
    August 2008 (Actual)
    Study Completion Date
    August 2008 (Actual)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Principal Investigator
    Name of the Sponsor
    Indiana University School of Medicine

    4. Oversight

    Data Monitoring Committee
    Yes

    5. Study Description

    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.
    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.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Osteogenesis Imperfecta, Osteoporosis, Paget Disease of Bone
    Keywords
    Osteogenesis Imperfecta, Fractures, Pediatric, Osteoporosis, Juvenile Pagets

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Phase 4
    Interventional Study Model
    Parallel Assignment
    Masking
    None (Open Label)
    Allocation
    Randomized
    Enrollment
    18 (Actual)

    8. Arms, Groups, and Interventions

    Arm Title
    Alendronate
    Arm Type
    Active Comparator
    Arm Description
    1 mg/kg po qd rounded to nearest 10 or 20 mg dose
    Arm Title
    Pamidronate
    Arm Type
    Active Comparator
    Arm Description
    3 mg/kg IV q4 months
    Intervention Type
    Drug
    Intervention Name(s)
    Alendronate
    Other Intervention Name(s)
    fosamax
    Intervention Type
    Drug
    Intervention Name(s)
    Pamidronate
    Primary Outcome Measure Information:
    Title
    Bone Mineral Density
    Description
    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.
    Time Frame
    2 years

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    3 Years
    Maximum Age & Unit of Time
    21 Years
    Accepts Healthy Volunteers
    No
    Eligibility 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
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Linda A DiMeglio, MD, MPH
    Organizational Affiliation
    Indiana University School of Medicine
    Official's Role
    Principal Investigator

    12. IPD Sharing Statement

    Learn more about this trial

    Bisphosphonate Therapy for Osteogenesis Imperfecta

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