Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12605000075684
Ethics application status
Approved
Date submitted
25/07/2005
Date registered
5/08/2005
Date last updated
22/08/2007
Type of registration
Retrospectively registered

Titles & IDs
Public title
A randomized phase III study to evaluate the effectiveness of two different dosing regimens (high dose vs daily) of Prednisone for boys with Duchenne muscular dystrophy in improving muscle strength and function and minimising side effects.
Scientific title
A randomized phase III study to evaluate the effectiveness of two different dosing regimens (high dose vs daily) of Prednisone for boys with Duchenne muscular dystrophy in improving muscle strength and function and minimising side effects.
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne muscular dystrophy 150 0
Condition category
Condition code
Musculoskeletal 169 169 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparison of daily low dose (0.75mg/kg/day) Prednisone to high dose Prednisone over 2 days (10mg/kg/week).
Intervention code [1] 62 0
Treatment: Drugs
Comparator / control treatment
Control group
Dose comparison

Outcomes
Primary outcome [1] 208 0
Muscle strength
Timepoint [1] 208 0
Measured at the start of the trial, and then 1,3,6,9 and 12 months after starting the prednisone
Secondary outcome [1] 474 0
Minimum side effects
Timepoint [1] 474 0

Eligibility
Key inclusion criteria
Diagnosis of Duchenne muscular dystrophy, ambulant, steroid naïve.
Minimum age
4 Years
Maximum age
10 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
No exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Coordinating centre pharmacist will consult the site's randomization table
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Pre-generating a database of randomly permuted balanced blocks. Stratified to age (4 -7 years, 7-10 years)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
1/07/2005
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
140
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 224 0
Other
Name [1] 224 0
Institute for Neuromuscular Research
Country [1] 224 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Country
Australia
Secondary sponsor category [1] 167 0
Other Collaborative groups
Name [1] 167 0
Cooperative International Neuromuscular Research Group
Address [1] 167 0
Country [1] 167 0
United States of America

Ethics approval
Ethics application status
Approved

Summary
Brief summary
To see which regimen of Prednisone - either a low daily dose or a high dose given on the two days of the weekend - gives the best positive effects (improvement in muscle strength) while minimising adverse effects (such as weight gain and behavioural changes)
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35722 0
Address 35722 0
Country 35722 0
Phone 35722 0
Fax 35722 0
Email 35722 0
Contact person for public queries
Name 9251 0
Sian Rudge
Address 9251 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 9251 0
Australia
Phone 9251 0
+61 2 98451229
Fax 9251 0
+61 2 98451317
Email 9251 0
[email protected]
Contact person for scientific queries
Name 179 0
Sian Rudge
Address 179 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 179 0
Australia
Phone 179 0
+61 2 98451229
Fax 179 0
+61 2 98451317
Email 179 0
[email protected]

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

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.