Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The ANZCTR website will be unavailable from 1pm until 3pm (AEDT) on Wednesday the 30th of October for website maintenance. Please be sure to log out of the system in order to avoid any loss of data.
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
Query!
Scientific title
Safety and Efficacy of Risedronate in the Treatment of Osteogenesis Imperfecta in Children
Query!
Secondary ID [1]
0
0
HMR4003I/3001
Query!
Secondary ID [2]
0
0
2003100
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Osteogenesis Imperfecta
0
0
Query!
Condition category
Condition code
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Injuries and Accidents
0
0
0
0
Query!
Fractures
Query!
Intervention/exposure
Study type
Interventional
Query!
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
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 12, ITT Population
Query!
Assessment method [1]
0
0
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.
Query!
Timepoint [1]
0
0
Baseline and Month 12
Query!
Secondary outcome [1]
0
0
Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 24, ITT Population
Query!
Assessment method [1]
0
0
Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.
Query!
Timepoint [1]
0
0
Baseline and Month 24
Query!
Secondary outcome [2]
0
0
Percent Change From Baseline Lumbar Spine Bone Mineral Density (BMD) at Month 36, ITT Population
Query!
Assessment method [2]
0
0
Lumbar Spine Bone Mineral Density (BMD) measured by dual-energy x-ray absorptiometry (DXA)and read by central reader.
Query!
Timepoint [2]
0
0
Baseline and Month 36
Query!
Secondary outcome [3]
0
0
Percent Change From Baseline in Total Body BMD at Month 12, ITT Population
Query!
Assessment method [3]
0
0
Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
Query!
Timepoint [3]
0
0
Baseline and Month 12
Query!
Secondary outcome [4]
0
0
Percent Change From Baseline in Total Body BMD at Month 24, ITT Population
Query!
Assessment method [4]
0
0
Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
Query!
Timepoint [4]
0
0
Baseline and Month 24
Query!
Secondary outcome [5]
0
0
Percent Change From Baseline in Total Body BMD at Month 36, ITT Population
Query!
Assessment method [5]
0
0
Percent Change from baseline in Total Body Bone Mineral Density (BMD) measured by DXA.
Query!
Timepoint [5]
0
0
Baseline and Month 36
Query!
Secondary outcome [6]
0
0
Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 12, ITT Population
Query!
Assessment method [6]
0
0
Query!
Timepoint [6]
0
0
Baseline and Month 12
Query!
Secondary outcome [7]
0
0
Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 24, ITT Population
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Baseline and Month 24
Query!
Secondary outcome [8]
0
0
Percent Change From Baseline in Lumbar Spine BMC (Bone Mineral Content) at Month 36, ITT Population
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
Baseline and Month 36
Query!
Secondary outcome [9]
0
0
Percent Change From Baseline in Total Body BMC at Month 12, ITT Population
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Baseline and Month 12
Query!
Secondary outcome [10]
0
0
Percent Change From Baseline in Total Body BMC at Month 24, ITT Population
Query!
Assessment method [10]
0
0
Query!
Timepoint [10]
0
0
Baseline and Month 24
Query!
Secondary outcome [11]
0
0
Percent Change From Baseline in Total Body BMC at Month 36, ITT Population
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
Baseline and Month 36
Query!
Secondary outcome [12]
0
0
Lumbar Spine Z-score - Percent Change From Baseline to Month 12, ITT Population
Query!
Assessment method [12]
0
0
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".
Query!
Timepoint [12]
0
0
Baseline and Month 12
Query!
Secondary outcome [13]
0
0
Lumbar Spine Z-score - Percent Change From Baseline to Month 24, ITT Population
Query!
Assessment method [13]
0
0
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".
Query!
Timepoint [13]
0
0
Baseline and Month 24
Query!
Secondary outcome [14]
0
0
Lumbar Spine Z-score - Percent Change From Baseline to Month 36, ITT Population
Query!
Assessment method [14]
0
0
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".
Query!
Timepoint [14]
0
0
Baseline and Month 36
Query!
Secondary outcome [15]
0
0
Total Body Z-score- Percent Change From Baseline to Month 12, ITT Population
Query!
Assessment method [15]
0
0
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".
Query!
Timepoint [15]
0
0
Baseline and Month 12
Query!
Secondary outcome [16]
0
0
Total Body Z-score- Percent Change From Baseline to Month 24, ITT Population
Query!
Assessment method [16]
0
0
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".
Query!
Timepoint [16]
0
0
Baseline and Month 24
Query!
Secondary outcome [17]
0
0
Total Body Z-score- Percent Change From Baseline to Month 36, ITT Population
Query!
Assessment method [17]
0
0
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".
Query!
Timepoint [17]
0
0
Baseline and Month 36
Query!
Secondary outcome [18]
0
0
Percent Change From Baseline in Lumbar Spine Bone Area at Month 12, ITT Population
Query!
Assessment method [18]
0
0
Measured by DXA.
Query!
Timepoint [18]
0
0
Baseline and Month 12
Query!
Secondary outcome [19]
0
0
Percent Change From Baseline in Lumbar Spine Bone Area at Month 24, ITT Population
Query!
Assessment method [19]
0
0
Measured by DXA.
Query!
Timepoint [19]
0
0
Baseline and Month 24
Query!
Secondary outcome [20]
0
0
Percent Change From Baseline in Lumbar Spine Bone Area at Month 36, ITT Population
Query!
Assessment method [20]
0
0
Measured by DXA.
Query!
Timepoint [20]
0
0
Baseline and Month 36
Query!
Secondary outcome [21]
0
0
Percent Change From Baseline in Total Body Bone Area Month 12, ITT Population
Query!
Assessment method [21]
0
0
Query!
Timepoint [21]
0
0
Baseline and Month 12
Query!
Secondary outcome [22]
0
0
Percent Change From Baseline in Total Body Bone Area Month 24, ITT Population
Query!
Assessment method [22]
0
0
Query!
Timepoint [22]
0
0
Baseline and Month 24
Query!
Secondary outcome [23]
0
0
Percent Change From Baseline in Total Body Bone Area Month 36, ITT Population
Query!
Assessment method [23]
0
0
Query!
Timepoint [23]
0
0
Baseline and Month 36
Query!
Secondary outcome [24]
0
0
New Morphometric Vertebral Fracture at Month 12, ITT Population
Query!
Assessment method [24]
0
0
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.
Query!
Timepoint [24]
0
0
Baseline and Month 12
Query!
Secondary outcome [25]
0
0
New Morphometric Vertebral Fracture at Month 36, ITT Population
Query!
Assessment method [25]
0
0
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.
Query!
Timepoint [25]
0
0
Baseline and Month 36
Query!
Secondary outcome [26]
0
0
Categorization by Number of New Morphometric Vertebral Fracture at Month 12, ITT
Query!
Assessment method [26]
0
0
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.
Query!
Timepoint [26]
0
0
Baseline and Month 12
Query!
Secondary outcome [27]
0
0
Categorization by Number of New Morphometric Vertebral Fracture at Month 36, ITT
Query!
Assessment method [27]
0
0
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.
Query!
Timepoint [27]
0
0
Baseline and Month 36
Query!
Secondary outcome [28]
0
0
Incidence New Vertebral Fractures by SQ (Semi-Quantitative) Score, Patients Aged 4-9 Years, Month 12, ITT Population
Query!
Assessment method [28]
0
0
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.
Query!
Timepoint [28]
0
0
Month 12
Query!
Secondary outcome [29]
0
0
Incidence New Vertebral Fractures by SQ Score, Patients Aged 10-15 Years, Month 12, ITT Population
Query!
Assessment method [29]
0
0
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.
Query!
Timepoint [29]
0
0
Month 12
Query!
Secondary outcome [30]
0
0
Probability of Fracture in 12 Months (Kaplan-Meier Cumulative Incidence), ITT Population
Query!
Assessment method [30]
0
0
Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.
Query!
Timepoint [30]
0
0
Time to First Event (days) up to 12 Months
Query!
Secondary outcome [31]
0
0
Number of Clinical Fractures, Month 12, ITT Population
Query!
Assessment method [31]
0
0
Long bones include radius, ulna, humerus, tibia, fibula, femur, upper limb and lower limb fracture.
Query!
Timepoint [31]
0
0
12 Months
Query!
Secondary outcome [32]
0
0
Serum BAP - Percent Change From Baseline to Month 12, ITT Population
Query!
Assessment method [32]
0
0
Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [32]
0
0
Baseline and 12 Months
Query!
Secondary outcome [33]
0
0
Serum BAP - Percent Change From Baseline to Month 24, ITT Population
Query!
Assessment method [33]
0
0
Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [33]
0
0
Baseline and 24 Months
Query!
Secondary outcome [34]
0
0
Serum BAP - Percent Change From Baseline to Month 36, ITT Population
Query!
Assessment method [34]
0
0
Serum Bone Alkaline Phosphatase (BAP - bone formation marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [34]
0
0
Baseline and 36 Months
Query!
Secondary outcome [35]
0
0
Urine NTX/Cr - Percent Change From Baseline at Month 12, ITT Population
Query!
Assessment method [35]
0
0
Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [35]
0
0
Baseline and Endpoint / Month 12
Query!
Secondary outcome [36]
0
0
Urine NTX/Cr - Percent Change From Baseline at Month 24, ITT Population
Query!
Assessment method [36]
0
0
Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [36]
0
0
Baseline and Month 24
Query!
Secondary outcome [37]
0
0
Urine NTX/Cr - Percent Change From Baseline at Month 36, ITT Population
Query!
Assessment method [37]
0
0
Urine type-I collagen N-telopeptide/creatinine (NTX/Cr; bone resorption marker). Negative percent changes indicate response to treatment.
Query!
Timepoint [37]
0
0
Baseline and Month 36
Query!
Secondary outcome [38]
0
0
Wong-Baker FACES Pain Rating Scale - Change From Baseline to Month 12, ITT Population
Query!
Assessment method [38]
0
0
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.
Query!
Timepoint [38]
0
0
Baseline and Month 12
Query!
Secondary outcome [39]
0
0
Bone Age (Years), Change From Baseline to Month 12, ITT Population
Query!
Assessment method [39]
0
0
Bone Age determined by visual assessment of hand / wrist radiographs.
Query!
Timepoint [39]
0
0
Baseline and Month 12
Query!
Secondary outcome [40]
0
0
Bone Age (Years), Change From Baseline to Month 24, ITT Population
Query!
Assessment method [40]
0
0
Bone Age determined by visual assessment of hand / wrist radiographs.
Query!
Timepoint [40]
0
0
Baseline and Month 24
Query!
Secondary outcome [41]
0
0
Bone Age (Years), Change From Baseline to Month 36, ITT Population
Query!
Assessment method [41]
0
0
Bone Age determined by visual assessment of hand / wrist radiographs.
Query!
Timepoint [41]
0
0
Baseline and Month 36
Query!
Secondary outcome [42]
0
0
Annualized Growth Velocity - Change From Baseline to Month 12, ITT Population
Query!
Assessment method [42]
0
0
Annualized Growth Velocity \[= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)\]
Query!
Timepoint [42]
0
0
Baseline and Month 12
Query!
Secondary outcome [43]
0
0
Annualized Growth Velocity - Change From Baseline to Month 36, ITT Population
Query!
Assessment method [43]
0
0
Annualized Growth Velocity \[= bone age change from baseline x (365.25/time in days between baseline and the bone age measurement)\]
Query!
Timepoint [43]
0
0
Baseline and Month 36
Query!
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.
Query!
Minimum age
4
Years
Query!
Query!
Maximum age
15
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
* Any bisphosphonate use within one year of enrollment
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Completed
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
1/11/2004
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
Query!
Actual
1/03/2010
Query!
Sample size
Target
Query!
Accrual to date
Query!
Final
143
Query!
Recruitment in Australia
Recruitment state(s)
NSW
Query!
Recruitment hospital [1]
0
0
The Children's Hospital at Westmead - Westmead
Query!
Recruitment hospital [2]
0
0
Princess Margaret Hospital for Children - Perth
Query!
Recruitment postcode(s) [1]
0
0
- Westmead
Query!
Recruitment postcode(s) [2]
0
0
- Perth
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
Florida
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Nebraska
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
New York
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Ohio
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Oregon
Query!
Country [6]
0
0
Belgium
Query!
State/province [6]
0
0
Bruxelles
Query!
Country [7]
0
0
Chile
Query!
State/province [7]
0
0
Santiago
Query!
Country [8]
0
0
Czech Republic
Query!
State/province [8]
0
0
Plzen
Query!
Country [9]
0
0
Finland
Query!
State/province [9]
0
0
Helsinki
Query!
Country [10]
0
0
Germany
Query!
State/province [10]
0
0
Koln
Query!
Country [11]
0
0
Hungary
Query!
State/province [11]
0
0
Budapest
Query!
Country [12]
0
0
Italy
Query!
State/province [12]
0
0
Valeggio sul Mincio
Query!
Country [13]
0
0
Poland
Query!
State/province [13]
0
0
Warzawa-Miedzylesie
Query!
Country [14]
0
0
South Africa
Query!
State/province [14]
0
0
Gauteng
Query!
Country [15]
0
0
Spain
Query!
State/province [15]
0
0
Barcelona
Query!
Country [16]
0
0
United Kingdom
Query!
State/province [16]
0
0
Bristol
Query!
Country [17]
0
0
United Kingdom
Query!
State/province [17]
0
0
Glasgow
Query!
Country [18]
0
0
United Kingdom
Query!
State/province [18]
0
0
Sheffield
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Query!
Name
Warner Chilcott
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
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.
Query!
Trial website
https://clinicaltrials.gov/study/NCT00106028
Query!
Trial related presentations / publications
Bishop N, Adami S, Ahmed SF, Anton J, Arundel P, Burren CP, Devogelaer JP, Hangartner T, Hosszu E, Lane JM, Lorenc R, Makitie O, Munns CF, Paredes A, Pavlov H, Plotkin H, Raggio CL, Reyes ML, Schoenau E, Semler O, Sillence DO, Steiner RD. Risedronate in children with osteogenesis imperfecta: a randomised, double-blind, placebo-controlled trial. Lancet. 2013 Oct 26;382(9902):1424-32. doi: 10.1016/S0140-6736(13)61091-0. Epub 2013 Aug 6.
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
Dietrich H Wenderoth, MD
Query!
Address
0
0
Procter and Gamble
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT00106028
Download to PDF