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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05429372




Registration number
NCT05429372
Ethics application status
Date submitted
8/10/2021
Date registered
23/06/2022

Titles & IDs
Public title
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
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
Secondary ID [1] 0 0
2021-003379-33
Secondary ID [2] 0 0
C3391008
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Muscular Dystrophy, Duchenne 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - PF-06939926

Experimental: PF-06939926 -


Treatment: Other: PF-06939926
All participants will receive a single dose of PF-06939926 on Day 1.
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Assessment method [1] 0 0
Timepoint [1] 0 0
Through Week 52
Primary outcome [2] 0 0
Number of participants with abnormal hematology test results
Assessment method [2] 0 0
Blood samples will be collected from subjects for the analysis of hematology
Timepoint [2] 0 0
Through Week 52
Primary outcome [3] 0 0
Number of participants with abnormal biochemistry test results
Assessment method [3] 0 0
Blood samples will be collected from subjects for the analysis of biochemistry
Timepoint [3] 0 0
Through Week 52
Primary outcome [4] 0 0
Number of participants with abnormal urine analysis
Assessment method [4] 0 0
Urine samples will be collected from subjects for the analysis of urine
Timepoint [4] 0 0
Through Week 52
Primary outcome [5] 0 0
Number of participants with abnormal and clinically relevant changes in neurological examinations
Assessment method [5] 0 0
Timepoint [5] 0 0
Through Week 52
Primary outcome [6] 0 0
Number of participants with abnormal and clinically relevant changes in body weight
Assessment method [6] 0 0
Timepoint [6] 0 0
Through Week 52
Primary outcome [7] 0 0
Number of participants with abnormal and clinically relevant changes in vital signs
Assessment method [7] 0 0
Timepoint [7] 0 0
Through Week 52
Primary outcome [8] 0 0
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Assessment method [8] 0 0
Timepoint [8] 0 0
Through Week 52
Primary outcome [9] 0 0
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Assessment method [9] 0 0
Timepoint [9] 0 0
Through Week 52
Primary outcome [10] 0 0
Number of participants with abnormal and clinically relevant changes on echocardiogram
Assessment method [10] 0 0
Timepoint [10] 0 0
Through Week 52
Secondary outcome [1] 0 0
Distribution of mini-dystrophin expression in muscle
Assessment method [1] 0 0
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
Timepoint [1] 0 0
At Week 9, Week 52 and Year 5 (if available)
Secondary outcome [2] 0 0
Level of mini-dystrophin expression in muscle
Assessment method [2] 0 0
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
Timepoint [2] 0 0
At Week 9, Week 52 and Year 5 (if available)
Secondary outcome [3] 0 0
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Assessment method [3] 0 0
Timepoint [3] 0 0
Through 5 years
Secondary outcome [4] 0 0
Number of participants with abnormal hematology test results
Assessment method [4] 0 0
Blood samples will be collected from subjects for the analysis of hematology
Timepoint [4] 0 0
Through 5 years
Secondary outcome [5] 0 0
Number of participants with abnormal biochemistry test results
Assessment method [5] 0 0
Blood samples will be collected from subjects for the analysis of biochemistry
Timepoint [5] 0 0
Through 5 years
Secondary outcome [6] 0 0
Number of participants with abnormal urine analysis
Assessment method [6] 0 0
Urine samples will be collected from subjects for the analysis of urine
Timepoint [6] 0 0
Through 5 years
Secondary outcome [7] 0 0
Number of participants with abnormal and clinically relevant changes in neurological examinations
Assessment method [7] 0 0
Timepoint [7] 0 0
Through 5 years
Secondary outcome [8] 0 0
Number of participants with abnormal and clinically relevant changes in body weight
Assessment method [8] 0 0
Timepoint [8] 0 0
Through 5 years
Secondary outcome [9] 0 0
Number of participants with abnormal and clinically relevant changes in vital signs
Assessment method [9] 0 0
Timepoint [9] 0 0
Through 5 years
Secondary outcome [10] 0 0
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Assessment method [10] 0 0
Timepoint [10] 0 0
Through 5 years
Secondary outcome [11] 0 0
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Assessment method [11] 0 0
Timepoint [11] 0 0
Through 5 years
Secondary outcome [12] 0 0
Number of participants with abnormal and clinically relevant changes on echocardiogram
Assessment method [12] 0 0
Timepoint [12] 0 0
Through 5 years

Eligibility
Key inclusion criteria
* Confirmed diagnosis of DMD by prior genetic testing.
Minimum age
2 Years
Maximum age
3 Years
Sex
Males
Can healthy volunteers participate?
No
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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
8/08/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
3/01/2029
Actual
Sample size
Target
Accrual to date
Final
10
Recruitment in Australia
Recruitment state(s)
NSW,VIC,WA
Recruitment hospital [1] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment hospital [2] 0 0
The Royal Children's Hospital Melbourne - Parkville
Recruitment hospital [3] 0 0
Perth Children's Hospital - Nedlands
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
6009 - Nedlands
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Florida
Country [2] 0 0
United States of America
State/province [2] 0 0
Pennsylvania
Country [3] 0 0
United States of America
State/province [3] 0 0
Utah

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pfizer
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


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 not provided in https://clinicaltrials.gov/study/NCT05429372