Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12605000076673
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 randomised phase III study to evaluate the effectiveness prednisolone therapy for late stage Duchenne muscular dystrophy in maintaining lung function
Scientific title
A randomised phase III study to evaluate the effectiveness prednisolone therapy for late stage Duchenne muscular dystrophy in maintaining lung function
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne muscular dystrophy 151 0
Condition category
Condition code
Musculoskeletal 170 170 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
12-month trial of Prednisolone on respiratory function in non-ambulatory boys with Duchenne muscular dystrophy.
Intervention code [1] 63 0
Treatment: Drugs
Comparator / control treatment
Placebo
Control group
Placebo

Outcomes
Primary outcome [1] 209 0
Lung function
Timepoint [1] 209 0
Lung function and echocardiograms are done prior to starting on prednisolone; repeated at 6 and 12 months after starting on prednisolone and the echo will be repeated 12 months after starting on the prednisolone.
Secondary outcome [1] 475 0
Upper limb strength
Timepoint [1] 475 0
Measured prior to commencement of study medication and then every 3 months after starting medication.
Secondary outcome [2] 476 0
Scoliosis
Timepoint [2] 476 0
Assessed clinically and by lateral X-ray prior to commencing study medication, and at the conclusion of the trial.
Secondary outcome [3] 477 0
Bone densitometry
Timepoint [3] 477 0
Measured prior to and at the conclusion of the trial.
Secondary outcome [4] 478 0
Behaviour and quality of life
Timepoint [4] 478 0
Measured prior to commencement of study meds, and then 3 monthly (behaviour) and 6 monthly (QoL).

Eligibility
Key inclusion criteria
Duchenne muscular dystrophy, non-ambulant.
Minimum age
10 Years
Maximum age
Not stated
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)
Hospital Pharmacy will perform randomisation
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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
Stopped early
Data analysis
Reason for early stopping/withdrawal
Date of first participant enrolment
Anticipated
8/01/2005
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
40
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 225 0
Other
Name [1] 225 0
Institute for Neuromuscular Research
Country [1] 225 0
Australia
Primary sponsor type
Hospital
Name
The Children's Hospital at Westmead
Address
Country
Australia
Secondary sponsor category [1] 168 0
None
Name [1] 168 0
None
Address [1] 168 0
Country [1] 168 0

Ethics approval
Ethics application status
Approved

Summary
Brief summary
The study will assess if treatment with Prednisolone has a positive effect in maintaining lung function in non-ambulant boys with Duchenne muscular dystrophy
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 35561 0
Address 35561 0
Country 35561 0
Phone 35561 0
Fax 35561 0
Email 35561 0
Contact person for public queries
Name 9252 0
Sian Rudge
Address 9252 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 9252 0
Australia
Phone 9252 0
+61 2 98451229
Fax 9252 0
+61 2 98451317
Email 9252 0
[email protected]
Contact person for scientific queries
Name 180 0
Sian Rudge
Address 180 0
Clinical Sciences
The Children's Hospital at Westmead
Locked Bag 4001
Westmead NSW 2145
Country 180 0
Australia
Phone 180 0
+61 2 98451229
Fax 180 0
+61 2 98451317
Email 180 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.