Trial Review

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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
Trial registered on ANZCTR


Registration number
ACTRN12605000077662
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 non-randomised Phase III study to evaluate the effectiveness of Deflazacort in boys with Duchenne muscular dystrophy in improving muscle strength and function and minimising side effects.
Scientific title
A non-randomised Phase III study to evaluate the effectiveness of Deflazacort in 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 152 0
Condition category
Condition code
Musculoskeletal 171 171 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
12-month trial of Deflazacort vs prednisone given as part of routine clinical care
Intervention code [1] 64 0
Treatment: Drugs
Comparator / control treatment
Control group
Active

Outcomes
Primary outcome [1] 210 0
Improvement in muscle strength
Timepoint [1] 210 0
Measured prior to commencement on deflazacort, then 1, 3, 6, 9 and 12 months after starting the treatment.
Secondary outcome [1] 479 0
Minimum side effects (weight gain, behavioural changes).
Timepoint [1] 479 0

Eligibility
Key inclusion criteria
Duchenne muscular dystrophy, walking independently, failed Prednisone.
Minimum age
4 Years
Maximum age
15 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
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not 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
1/11/2004
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 226 0
Other
Name [1] 226 0
Institute for Neuromuscular Research
Country [1] 226 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Country
Australia
Secondary sponsor category [1] 169 0
None
Name [1] 169 0
None
Address [1] 169 0
Country [1] 169 0

Ethics approval
Ethics application status
Approved

Summary
Brief summary
Trial of Deflazacort, a steroid which is not currently commercially available in Australia, for boys with DMD who have failed Prednisone trials
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 36152 0
Address 36152 0
Country 36152 0
Phone 36152 0
Fax 36152 0
Email 36152 0
Contact person for public queries
Name 9253 0
Sian Rudge
Address 9253 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 9253 0
Australia
Phone 9253 0
+61 2 98451229
Fax 9253 0
+61 2 98451317
Email 9253 0
sianr@chw.edu.au
Contact person for scientific queries
Name 181 0
Sian Rudge
Address 181 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 181 0
Australia
Phone 181 0
+61 2 98451229
Fax 181 0
+61 2 98451317
Email 181 0
sianr@chw.edu.au

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.