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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
Timepoint [1] 0 0
Through Week 52
Primary outcome [2] 0 0
Number of participants with abnormal hematology test results
Timepoint [2] 0 0
Through Week 52
Primary outcome [3] 0 0
Number of participants with abnormal biochemistry test results
Timepoint [3] 0 0
Through Week 52
Primary outcome [4] 0 0
Number of participants with abnormal urine analysis
Timepoint [4] 0 0
Through Week 52
Primary outcome [5] 0 0
Number of participants with abnormal and clinically relevant changes in neurological examinations
Timepoint [5] 0 0
Through Week 52
Primary outcome [6] 0 0
Number of participants with abnormal and clinically relevant changes in body weight
Timepoint [6] 0 0
Through Week 52
Primary outcome [7] 0 0
Number of participants with abnormal and clinically relevant changes in vital signs
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
Timepoint [8] 0 0
Through Week 52
Primary outcome [9] 0 0
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Timepoint [9] 0 0
Through Week 52
Primary outcome [10] 0 0
Number of participants with abnormal and clinically relevant changes on echocardiogram
Timepoint [10] 0 0
Through Week 52
Secondary outcome [1] 0 0
Distribution of mini-dystrophin expression in muscle
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
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
Timepoint [3] 0 0
Through 5 years
Secondary outcome [4] 0 0
Number of participants with abnormal hematology test results
Timepoint [4] 0 0
Through 5 years
Secondary outcome [5] 0 0
Number of participants with abnormal biochemistry test results
Timepoint [5] 0 0
Through 5 years
Secondary outcome [6] 0 0
Number of participants with abnormal urine analysis
Timepoint [6] 0 0
Through 5 years
Secondary outcome [7] 0 0
Number of participants with abnormal and clinically relevant changes in neurological examinations
Timepoint [7] 0 0
Through 5 years
Secondary outcome [8] 0 0
Number of participants with abnormal and clinically relevant changes in body weight
Timepoint [8] 0 0
Through 5 years
Secondary outcome [9] 0 0
Number of participants with abnormal and clinically relevant changes in vital signs
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
Timepoint [10] 0 0
Through 5 years
Secondary outcome [11] 0 0
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Timepoint [11] 0 0
Through 5 years
Secondary outcome [12] 0 0
Number of participants with abnormal and clinically relevant changes on echocardiogram
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
NA
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
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

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

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests


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