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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT00106028
Registration number
NCT00106028
Ethics application status
Date submitted
18/03/2005
Date registered
21/03/2005
Date last updated
22/04/2013
Titles & IDs
Public title
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
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Scientific title
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
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Secondary ID [1]
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HMR4003I/3001
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Secondary ID [2]
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2003100
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Osteogenesis Imperfecta
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Injuries and Accidents
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0
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Fractures
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - risedronate sodium (Actonel)
Treatment: Drugs - Placebo
Placebo Comparator: Placebo Daily - placebo tablet, once a day for one year then for two years open label risedronate
Experimental: Risedronate Daily - risedronate tablet, once a day for one year then for two years open label risedronate once a day
Treatment: Drugs: risedronate sodium (Actonel)
risedronate tablet once a day for one year followed by risedronate once a day for two years
Treatment: Drugs: Placebo
placebo tablet once a day for one year followed by risedronate once a day for two years
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population
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Assessment method [1]
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Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader. Duplicate scans obtained at screening and Month 12.
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Timepoint [1]
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Baseline and Month 12
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Secondary outcome [1]
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Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 24, ITT Population
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Assessment method [1]
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Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.
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Timepoint [1]
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Baseline and Month 24
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Secondary outcome [2]
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Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 36, ITT Population
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Assessment method [2]
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Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.
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Timepoint [2]
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Baseline and Month 36
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Secondary outcome [3]
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Percent Change From Baseline in Total Body BMD at Month 12, ITT Population
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Assessment method [3]
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Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
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Timepoint [3]
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Baseline and Month 12
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Secondary outcome [4]
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Percent Change From Baseline in Total Body BMD at Month 24, ITT Population
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Assessment method [4]
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Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
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Timepoint [4]
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Baseline and Month 24
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Secondary outcome [5]
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Percent Change From Baseline in Total Body BMD at Month 36, ITT Population
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Assessment method [5]
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Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
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Timepoint [5]
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Baseline and Month 36
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Secondary outcome [6]
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Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 12, ITT Population
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Assessment method [6]
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Timepoint [6]
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Baseline and Month 12
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Secondary outcome [7]
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Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 24, ITT Population
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Assessment method [7]
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Timepoint [7]
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Baseline and Month 24
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Secondary outcome [8]
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Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 36, ITT Population
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Assessment method [8]
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Timepoint [8]
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Baseline and Month 36
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Secondary outcome [9]
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Percent Change From Baseline in Total Body BMC at Month 12, ITT Population
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Assessment method [9]
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Timepoint [9]
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Baseline and Month 12
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Secondary outcome [10]
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Percent Change From Baseline in Total Body BMC at Month 24, ITT Population
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Assessment method [10]
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Timepoint [10]
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Baseline and Month 24
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Secondary outcome [11]
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Percent Change From Baseline in Total Body BMC at Month 36, ITT Population
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Assessment method [11]
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Timepoint [11]
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Baseline and Month 36
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Secondary outcome [12]
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Lumbar Spine Z-score - Percent Change From Baseline to Month 12, ITT Population
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Assessment method [12]
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Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [12]
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0
Baseline and Month 12
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Secondary outcome [13]
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Lumbar Spine Z-score - Percent Change From Baseline to Month 24, ITT Population
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Assessment method [13]
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Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [13]
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Baseline and Month 24
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Secondary outcome [14]
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Lumbar Spine Z-score - Percent Change From Baseline to Month 36, ITT Population
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Assessment method [14]
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Lumbar Spine Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [14]
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Baseline and Month 36
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Secondary outcome [15]
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Total Body Z-score- Percent Change From Baseline to Month 12, ITT Population
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Assessment method [15]
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Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [15]
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Baseline and Month 12
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Secondary outcome [16]
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Total Body Z-score- Percent Change From Baseline to Month 24, ITT Population
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Assessment method [16]
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Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [16]
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Baseline and Month 24
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Secondary outcome [17]
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Total Body Z-score- Percent Change From Baseline to Month 36, ITT Population
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Assessment method [17]
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Total Body Z-score - number of standard deviations a patient's BMD differs from the average BMD of their age, sex, and ethnicity. Positive scores indicate BMD above the mean; Positive values are "best values" and negative values are "worst values".
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Timepoint [17]
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Baseline and Month 36
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Secondary outcome [18]
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Percent Change From Baseline in Lumbar Spine Bone Area at Month 12, ITT Population
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Assessment method [18]
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Measured by DXA.
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Timepoint [18]
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Baseline and Month 12
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Secondary outcome [19]
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Percent Change From Baseline in Lumbar Spine Bone Area at Month 24, ITT Population
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Assessment method [19]
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Measured by DXA.
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Timepoint [19]
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Baseline and Month 24
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Secondary outcome [20]
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Percent Change From Baseline in Lumbar Spine Bone Area at Month 36, ITT Population
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Assessment method [20]
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Measured by DXA.
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Timepoint [20]
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Baseline and Month 36
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Secondary outcome [21]
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Percent Change From Baseline in Total Body Bone Area Month 12, ITT Population
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Assessment method [21]
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Timepoint [21]
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Baseline and Month 12
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Secondary outcome [22]
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Percent Change From Baseline in Total Body Bone Area Month 24, ITT Population
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Assessment method [22]
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Timepoint [22]
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Baseline and Month 24
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Secondary outcome [23]
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Percent Change From Baseline in Total Body Bone Area Month 36, ITT Population
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Assessment method [23]
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0
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Timepoint [23]
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Baseline and Month 36
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Secondary outcome [24]
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New Morphometric Vertebral Fracture at Month 12, ITT Population
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Assessment method [24]
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Morphometric Vertebral Fracture measured by semi-quantitative (SQ) analysis of x-rays using the Genant scoring system at endpoint. (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). New fracture = SQ score is 0 at baseline and >0 at the specified end visit.
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Timepoint [24]
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Baseline and Month 12
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Secondary outcome [25]
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New Morphometric Vertebral Fracture at Month 36, ITT Population
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Assessment method [25]
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Morphometric Vertebral Fracture measured by SQ analysis of x-rays using the Genant scoring system. (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). New fracture = SQ score is 0 at baseline and >0 at the specified end visit.
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Timepoint [25]
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Baseline and Month 36
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Secondary outcome [26]
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Categorization by Number of New Morphometric Vertebral Fracture at Month 12, ITT
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Assessment method [26]
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Patients with 1 or more New Morphometric Vertebral Fracture as measured by SQ analysis of x-rays using the Genant scoring system (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). Incidence = SQ score is 0 at baseline and >0 at post-baseline.
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Timepoint [26]
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0
Baseline and Month 12
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Secondary outcome [27]
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Categorization by Number of New Morphometric Vertebral Fracture at Month 36, ITT
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Assessment method [27]
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Patients with 1 or more New Morphometric Vertebral Fracture as measured by SQ analysis of x-rays using the Genant scoring system (Ref: Genant 1993). SQ-Scores range from 0 (no fracture) to 3 (severe fracture). Incidence = SQ score is 0 at baseline and >0 at post-baseline.
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Timepoint [27]
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Baseline and Month 36
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Secondary outcome [28]
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Incidence New Vertebral Fractures by SQ (Semi-Quantitative) Score, Patients Aged 4-9 Years, Month 12, ITT Population
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Assessment method [28]
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Patients aged 4-9 years with new morphometric vertebral fractures as measured by SQ analysis of x-rays using the Genant scoring system at Month 12 +/- 14 days. (Ref: Genant 1993). SQ Score mild - 0/no fracture to Grade 1, Moderate to Severe - change from 0/no fracture to Grade 2-3.
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Timepoint [28]
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Month 12
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Secondary outcome [29]
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Incidence New Vertebral Fractures by SQ Score, Patients Aged 10-15 Years, Month 12, ITT Population
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Assessment method [29]
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Patients aged 10-15 years with new morphometric vertebral fractures as measured by SQ analysis of x-rays using the Genant scoring system at Month 12 +/- 14 days. (Ref: Genant 1993). SQ Score mild - 0/no fracture to Grade 1, Moderate to Severe - change from 0/no fracture to Grade 2-3.
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Timepoint [29]
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Month 12
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Secondary outcome [30]
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Probability of Fracture in 12 Months (Kaplan-Meier Cumulative Incidence), ITT Population
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Assessment method [30]
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Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.
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Timepoint [30]
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Time to First Event (days) up to 12 Months
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Secondary outcome [31]
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Number of Clinical Fractures, Month 12, ITT Population
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Assessment method [31]
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Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.
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Timepoint [31]
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12 Months
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Secondary outcome [32]
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Serum BAP - Percent Change From Baseline to Month 12, ITT Population
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Assessment method [32]
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Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
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Timepoint [32]
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Baseline and 12 Months
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Secondary outcome [33]
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Serum BAP - Percent Change From Baseline to Month 24, ITT Population
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Assessment method [33]
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Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
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Timepoint [33]
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Baseline and 24 Months
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Secondary outcome [34]
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Serum BAP - Percent Change From Baseline to Month 36, ITT Population
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Assessment method [34]
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Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
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Timepoint [34]
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Baseline and 36 Months
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Secondary outcome [35]
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Urine NTX/Cr - Percent Change From Baseline at Month 12, ITT Population
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Assessment method [35]
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Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
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Timepoint [35]
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Baseline and Endpoint / Month 12
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Secondary outcome [36]
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Urine NTX/Cr - Percent Change From Baseline at Month 24, ITT Population
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Assessment method [36]
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Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
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Timepoint [36]
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Baseline and Month 24
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Secondary outcome [37]
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Urine NTX/Cr - Percent Change From Baseline at Month 36, ITT Population
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Assessment method [37]
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Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
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Timepoint [37]
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Baseline and Month 36
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Secondary outcome [38]
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Wong-Baker FACES Pain Rating Scale - Change From Baseline to Month 12, ITT Population
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Assessment method [38]
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Wong-Baker FACES Pain Rating Scale (pain assessment scale using facial expressions, translated into a range from 0= no pain [smiling face] to 10= worst pain possible [distorted face with tears]; negative values indicate decrease in pain). Reference: Wong DL et al.
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Timepoint [38]
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Baseline and Month 12
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Secondary outcome [39]
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Bone Age (Years), Change From Baseline to Month 12, ITT Population
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Assessment method [39]
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Bone Age determined by visual assessment of hand / wrist radiographs.
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Timepoint [39]
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Baseline and Month 12
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Secondary outcome [40]
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Bone Age (Years), Change From Baseline to Month 24, ITT Population
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Assessment method [40]
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Bone Age determined by visual assessment of hand / wrist radiographs.
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Timepoint [40]
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Baseline and Month 24
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Secondary outcome [41]
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Bone Age (Years), Change From Baseline to Month 36, ITT Population
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Assessment method [41]
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Bone Age determined by visual assessment of hand / wrist radiographs.
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Timepoint [41]
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Baseline and Month 36
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Secondary outcome [42]
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Annualized Growth Velocity - Change From Baseline to Month 12, ITT Population
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Assessment method [42]
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Annualized Growth Velocity [= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)]
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Timepoint [42]
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Baseline and Month 12
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Secondary outcome [43]
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Annualized Growth Velocity - Change From Baseline to Month 36, ITT Population
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Assessment method [43]
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Annualized Growth Velocity [= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)]
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Timepoint [43]
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Baseline and Month 36
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Eligibility
Key inclusion criteria
- OI diagnosis
- increased risk of fracture: either has a history of at least 1 radiographically
confirmed, non-traumatic or low impact fracture plus low bone mineral density (BMD) or
has very low BMD with or without a history of fractures.
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Minimum age
4
Years
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Maximum age
15
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Any bisphosphonate use within one year of enrollment
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/11/2004
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
1/03/2010
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Sample size
Target
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Accrual to date
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Final
143
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Recruitment in Australia
Recruitment state(s)
NSW
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Recruitment hospital [1]
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The Children's Hospital at Westmead - Westmead
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Recruitment hospital [2]
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Princess Margaret Hospital for Children - Perth
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Recruitment postcode(s) [1]
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- Westmead
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Recruitment postcode(s) [2]
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- Perth
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
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0
Florida
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Country [2]
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0
United States of America
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State/province [2]
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0
Nebraska
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Country [3]
0
0
United States of America
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State/province [3]
0
0
New York
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Country [4]
0
0
United States of America
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State/province [4]
0
0
Ohio
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Country [5]
0
0
United States of America
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State/province [5]
0
0
Oregon
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Country [6]
0
0
Belgium
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State/province [6]
0
0
Bruxelles
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Country [7]
0
0
Chile
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State/province [7]
0
0
Santiago
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Country [8]
0
0
Czech Republic
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State/province [8]
0
0
Plzen
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Country [9]
0
0
Finland
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State/province [9]
0
0
Helsinki
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Country [10]
0
0
Germany
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State/province [10]
0
0
Koln
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Country [11]
0
0
Hungary
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State/province [11]
0
0
Budapest
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Country [12]
0
0
Italy
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State/province [12]
0
0
Valeggio sul Mincio
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Country [13]
0
0
Poland
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State/province [13]
0
0
Warzawa-Miedzylesie
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Country [14]
0
0
South Africa
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State/province [14]
0
0
Gauteng
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Country [15]
0
0
Spain
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State/province [15]
0
0
Barcelona
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Country [16]
0
0
United Kingdom
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State/province [16]
0
0
Bristol
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Country [17]
0
0
United Kingdom
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State/province [17]
0
0
Glasgow
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Country [18]
0
0
United Kingdom
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State/province [18]
0
0
Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Warner Chilcott
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Children with Osteogenesis Imperfecta (OI) have bone pain, low bone mass and fractures. There
are no approved drugs for the treatment of OI in children, even though some intravenous (IV)
bisphosphonates are used off-label in some countries. In a single dose, pharmacokinetic
study, data showed that risedronate was well tolerated in 28 children with OI. This three
year study will test the safety and efficacy of risedronate in the treatment of children with
OI. For the first year, patients will be randomized to the risedronate and placebo groups in
a 2:1 ratio. For the second and third years of the study, all patients will receive
risedronate.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT00106028
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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0
Dietrich H Wenderoth, MD
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Address
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0
Procter and Gamble
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for public queries
Name
0
0
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Address
0
0
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Country
0
0
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Phone
0
0
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Fax
0
0
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Email
0
0
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT00106028
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