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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/NCT04281485




Registration number
NCT04281485
Ethics application status
Date submitted
11/02/2020
Date registered
24/02/2020
Date last updated
4/04/2024

Titles & IDs
Public title
Study to Evaluate the Safety and Efficacy of PF-06939926 for the Treatment of Duchenne Muscular Dystrophy
Scientific title
A PHASE 3, MULTICENTER, RANDOMIZED, DOUBLE-BLIND, PLACEBO CONTROLLED STUDY TO EVALUATE THE SAFETY AND EFFICACY OF PF 06939926 FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
Secondary ID [1] 0 0
2019-002921-31
Secondary ID [2] 0 0
C3391003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 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
Other interventions - PF-06939926
Other interventions - Placebo
Other interventions - Placebo
Other interventions - PF-06939926

Other: Cohort 1 - Approximately two thirds of participants will be randomized to Cohort 1.

Other: Cohort 2 - Approximately one third of participants will be randomized to Cohort 2.


Other interventions: PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1.

Other interventions: Placebo
Placebo will be administered as a single IV infusion at Year 1 for Cohort 2.

Other interventions: Placebo
Placebo will be administered as a single IV infusion at Year 2 for Cohort 1.

Other interventions: PF-06939926
PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline in North Star Ambulatory Assessment (NSAA)
Timepoint [1] 0 0
Week 52
Secondary outcome [1] 0 0
Change from Baseline in mini-dystrophin expression level in muscle
Timepoint [1] 0 0
Week 52
Secondary outcome [2] 0 0
Change from Baseline in distribution of mini-dystrophin expression in the muscle
Timepoint [2] 0 0
Week 52
Secondary outcome [3] 0 0
Change from Baseline in serum creatine kinase (CK)
Timepoint [3] 0 0
Week 52
Secondary outcome [4] 0 0
Number of skills gained based on the individual items of the NSAA.
Timepoint [4] 0 0
Week 52
Secondary outcome [5] 0 0
Number of skills improved or maintained based on the individual items of the NSAA
Timepoint [5] 0 0
Week 52
Secondary outcome [6] 0 0
Change from Baseline in the 10-meter run/walk test velocity
Timepoint [6] 0 0
Week 52
Secondary outcome [7] 0 0
Change from Baseline in the rise from floor velocity
Timepoint [7] 0 0
Week 52
Secondary outcome [8] 0 0
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrument (PODCI): Transfer and Basic Mobility Core Scale
Timepoint [8] 0 0
Week 52
Secondary outcome [9] 0 0
Change from Baseline in the Modified Pediatric Outcomes Data Collection Instrucment (PODCI): Sports and Physical Functioning Core Scale
Timepoint [9] 0 0
Week 52

Eligibility
Key inclusion criteria
Key inclusion criteria:

1. Confirmed diagnosis of Duchenne muscular dystrophy by prior genetic testing

2. Receiving a stable daily dose (at least 0.5 mg/kg/day prednisone or prednisolone, or
at least 0.75 mg/kg/day deflazacort) for at least 3 months prior to Screening

3. Ambulatory, as assessed by protocol-specified criteria

Key exclusion criteria:

1. Positive test performed by Pfizer for neutralizing antibodies to AAV9

2. Any treatment designed to increase dystrophin expression within 6 months prior to
screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™)

3. Any prior treatment with gene therapy

4. Any non-healed injury that may impact functional testing (eg NSAA)

5. Abnormality in specified laboratory tests, including blood counts, liver and kidney
function

6. Any of the following genetic abnormalities in the dystrophin gene:

1. Any mutation (exon deletion, exon duplication, insertion, or point mutation)
affecting any exon between exon 9 and exon 13, inclusive; OR

2. A deletion that affects both exon 29 and exon 30;OR

3. A deletion that affects any exons between 56-71, inclusive.
Minimum age
4 Years
Maximum age
7 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
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
5/11/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
18/04/2029
Actual
Sample size
Target
99
Accrual to date
Final
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
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
Country [4] 0 0
United States of America
State/province [4] 0 0
Kansas
Country [5] 0 0
United States of America
State/province [5] 0 0
Pennsylvania
Country [6] 0 0
United States of America
State/province [6] 0 0
Utah
Country [7] 0 0
United States of America
State/province [7] 0 0
Virginia
Country [8] 0 0
United States of America
State/province [8] 0 0
Washington
Country [9] 0 0
Belgium
State/province [9] 0 0
Gent
Country [10] 0 0
Belgium
State/province [10] 0 0
Leuven
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
Ontario
Country [13] 0 0
France
State/province [13] 0 0
Nantes
Country [14] 0 0
France
State/province [14] 0 0
Paris
Country [15] 0 0
Germany
State/province [15] 0 0
North Rhine-westphalia
Country [16] 0 0
Germany
State/province [16] 0 0
Berlin
Country [17] 0 0
Germany
State/province [17] 0 0
Essen
Country [18] 0 0
Israel
State/province [18] 0 0
Jerusalem
Country [19] 0 0
Israel
State/province [19] 0 0
Petach Tikvah
Country [20] 0 0
Italy
State/province [20] 0 0
Roma
Country [21] 0 0
Italy
State/province [21] 0 0
Rome
Country [22] 0 0
Japan
State/province [22] 0 0
Aichi
Country [23] 0 0
Japan
State/province [23] 0 0
Hyogo
Country [24] 0 0
Japan
State/province [24] 0 0
Tokyo
Country [25] 0 0
Korea, Republic of
State/province [25] 0 0
Gyeongsangnam-do
Country [26] 0 0
Korea, Republic of
State/province [26] 0 0
Seoul
Country [27] 0 0
Russian Federation
State/province [27] 0 0
Saint Petersburg
Country [28] 0 0
Russian Federation
State/province [28] 0 0
Yekaterinburg
Country [29] 0 0
Spain
State/province [29] 0 0
Barcelona
Country [30] 0 0
Spain
State/province [30] 0 0
Valencia
Country [31] 0 0
Switzerland
State/province [31] 0 0
Bern
Country [32] 0 0
Switzerland
State/province [32] 0 0
Zurich
Country [33] 0 0
Taiwan
State/province [33] 0 0
Kaohsiung
Country [34] 0 0
Taiwan
State/province [34] 0 0
Taipei
Country [35] 0 0
United Kingdom
State/province [35] 0 0
England
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Merseyside
Country [37] 0 0
United Kingdom
State/province [37] 0 0
London

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

Ethics approval
Ethics application status

Summary
Brief summary
The study will evaluate the safety and efficacy of gene therapy in boys with DMD. It is a
randomized, double-blind, placebo-controlled study with two thirds of participants assigned
to gene therapy. The one third of participants who are randomized to the placebo arm will
have an opportunity for treatment with gene therapy at the beginning of the second year.
Trial website
https://clinicaltrials.gov/ct2/show/NCT04281485
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