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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/NCT05429372
Registration number
NCT05429372
Ethics application status
Date submitted
8/10/2021
Date registered
23/06/2022
Date last updated
4/04/2024
Titles & IDs
Public title
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
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Scientific title
A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
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Secondary ID [1]
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2021-003379-33
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Secondary ID [2]
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C3391008
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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:
Muscular Dystrophy, Duchenne
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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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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - PF-06939926
Experimental: PF-06939926 -
Other interventions: PF-06939926
All participants will receive a single dose of PF-06939926 on Day 1.
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Intervention code [1]
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Other interventions
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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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Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
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Assessment method [1]
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Timepoint [1]
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Through Week 52
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Primary outcome [2]
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Number of participants with abnormal hematology test results
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Assessment method [2]
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Blood samples will be collected from subjects for the analysis of hematology
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Timepoint [2]
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Through Week 52
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Primary outcome [3]
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Number of participants with abnormal biochemistry test results
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Assessment method [3]
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Blood samples will be collected from subjects for the analysis of biochemistry
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Timepoint [3]
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Through Week 52
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Primary outcome [4]
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Number of participants with abnormal urine analysis
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Assessment method [4]
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Urine samples will be collected from subjects for the analysis of urine
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Timepoint [4]
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Through Week 52
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Primary outcome [5]
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Number of participants with abnormal and clinically relevant changes in neurological examinations
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Assessment method [5]
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Timepoint [5]
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Through Week 52
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Primary outcome [6]
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Number of participants with abnormal and clinically relevant changes in body weight
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Assessment method [6]
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Timepoint [6]
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Through Week 52
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Primary outcome [7]
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Number of participants with abnormal and clinically relevant changes in vital signs
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Assessment method [7]
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Timepoint [7]
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Through Week 52
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Primary outcome [8]
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Number of participants with abnormal and clinically relevant changes on cardiac troponin I
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Assessment method [8]
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Timepoint [8]
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Through Week 52
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Primary outcome [9]
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Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
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Assessment method [9]
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Timepoint [9]
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Through Week 52
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Primary outcome [10]
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Number of participants with abnormal and clinically relevant changes on echocardiogram
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Assessment method [10]
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Timepoint [10]
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Through Week 52
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Secondary outcome [1]
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Distribution of mini-dystrophin expression in muscle
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Assessment method [1]
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Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
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Timepoint [1]
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At Week 9, Week 52 and Year 5 (if available)
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Secondary outcome [2]
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Level of mini-dystrophin expression in muscle
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Assessment method [2]
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Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
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Timepoint [2]
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At Week 9, Week 52 and Year 5 (if available)
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Secondary outcome [3]
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Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
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Assessment method [3]
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Timepoint [3]
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Through 5 years
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Secondary outcome [4]
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Number of participants with abnormal hematology test results
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Assessment method [4]
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Blood samples will be collected from subjects for the analysis of hematology
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Timepoint [4]
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Through 5 years
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Secondary outcome [5]
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Number of participants with abnormal biochemistry test results
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Assessment method [5]
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Blood samples will be collected from subjects for the analysis of biochemistry
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Timepoint [5]
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Through 5 years
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Secondary outcome [6]
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Number of participants with abnormal urine analysis
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Assessment method [6]
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Urine samples will be collected from subjects for the analysis of urine
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Timepoint [6]
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Through 5 years
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Secondary outcome [7]
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Number of participants with abnormal and clinically relevant changes in neurological examinations
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Assessment method [7]
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Timepoint [7]
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Through 5 years
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Secondary outcome [8]
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Number of participants with abnormal and clinically relevant changes in body weight
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Assessment method [8]
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Timepoint [8]
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Through 5 years
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Secondary outcome [9]
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Number of participants with abnormal and clinically relevant changes in vital signs
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Assessment method [9]
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Timepoint [9]
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Through 5 years
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Secondary outcome [10]
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Number of participants with abnormal and clinically relevant changes on cardiac troponin I
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Assessment method [10]
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Timepoint [10]
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Through 5 years
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Secondary outcome [11]
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Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
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Assessment method [11]
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Timepoint [11]
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Through 5 years
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Secondary outcome [12]
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Number of participants with abnormal and clinically relevant changes on echocardiogram
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Assessment method [12]
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Timepoint [12]
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Through 5 years
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Eligibility
Key inclusion criteria
- Confirmed diagnosis of DMD by prior genetic testing.
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Minimum age
2
Years
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Maximum age
3
Years
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Sex
Males
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation
(exon deletion, exon duplication, insertion, or point mutation) affecting any exon
between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and
exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
- Positive test performed by Pfizer for neutralizing antibodies to AAV9.
- Any prior treatment with gene therapy.
- Any treatment designed to increase dystrophin expression within 6 months prior to
screening (including, but not limited to, exon-skipping and nonsense read through).
- Previous or current treatment with oral glucocorticoids or other immunosuppressive
agents for the indication of DMD.
- Abnormality in specified laboratory tests, including blood counts, liver and kidney
function.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
N/A
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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
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Active, not recruiting
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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
8/08/2022
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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
3/01/2029
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Actual
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Sample size
Target
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Accrual to date
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Final
10
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Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
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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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The Royal Children's Hospital Melbourne - Parkville
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Recruitment hospital [3]
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Perth Children's Hospital - Nedlands
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Recruitment postcode(s) [1]
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2145 - Westmead
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment postcode(s) [3]
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6009 - Nedlands
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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Florida
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Country [2]
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United States of America
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State/province [2]
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Pennsylvania
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Country [3]
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United States of America
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State/province [3]
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Utah
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Pfizer
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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
The study will evaluate the safety and dystrophin expression following gene therapy in boys
with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05429372
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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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Pfizer CT.gov Call Center
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Address
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Pfizer
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05429372
Download to PDF