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| ID | Type | Description | Link |
|---|---|---|---|
| 2023-508510-42-00 | Registry Identifier | CTIS (EU) |
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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.
The study will assess the efficacy of PF-06939926 gene therapy on ambulatory function while also monitoring its safety. Approximately 99 boys with DMD will be enrolled and randomly assigned to one of two groups: approximately two thirds will be in Cohort 1 and receive gene therapy at the start of the study; approximately one third will be in Cohort 2 and receive placebo at the start of the study and receive gene therapy after one year, as long as it remains safe to do so. The treatment (PF-06939926 gene therapy or placebo) will be given as an intravenous infusion lasting up to 2 hours.
The study includes boys who are at least 4 years old and less than 8 years old (including 7 year olds up until their 8th birthday). All boys will need to be on a daily dose of glucocorticoids (prednisone, prednisolone, or deflazacort) for at least 3 months prior to enrolling and to stay on daily glucocorticoids for the first 2 years of the study. All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
The primary outcome of the study will be assessed at 52 weeks. All participants will be followed in the study for 15 years after treatment with gene therapy. Participants who received fordadistrogene movaparvovec in Pfizer studies C3391001 and C3391008 or are currently enrolled in Pfizer study C3391011 will be allowed to roll over into the long-term safety follow-up period of this study and will be considered Cohort 3.
The study medication, all medical tests associated with the study, and the visits to the study sites are free of charge. Participants will also be supported for travel costs associated with study visits.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Other | Approximately two thirds of participants will be randomized to Cohort 1. |
|
| Cohort 2 | Other | Approximately one third of participants will be randomized to Cohort 2. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06939926 | Genetic | PF-06939926 will be administered as a single IV infusion at Year 1 for Cohort 1. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is defined as the last non-missing NSAA total score collected prior to Year 1 drug administration. | Baseline, Week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Normal Dystrophin Expression Level in Muscle Biopsies by Liquid Chromatography Mass Spectrometry (LC-MS) Based on LLQV Peptide at Week 52 | The LC-MS assay measured the LLQVAVEDR (LLQV) peptide that detected full-length endogenous dystrophin as well as the mini-dystrophin transgene protein. | Baseline, Week 52 |
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Key inclusion criteria:
Key exclusion criteria:
Positive test performed by Pfizer for neutralizing antibodies to AAV9
Any treatment designed to increase dystrophin expression within 6 months prior to screening (e.g., Translarna™, EXONDYS 51™, VYONDYS 53™)
Any prior treatment with gene therapy
Any non-healed injury that may impact functional testing (eg NSAA)
Abnormality in specified laboratory tests, including blood counts, liver and kidney function
Any of the following genetic abnormalities in the dystrophin gene:
Male
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72202 | United States | ||
| Arkansas Children's |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41722591 | Derived | Muntoni F, Nascimento A, Shin J, Guglieri M, Stettner GM, Veerapandiyan A, Gallo S, Shi H, Gundapaneni B, Neelakantan S, Lobello K, Shen Q, Levy DI, Mercuri E; CIFFREO Study Group. Safety and efficacy of fordadistrogene movaparvovec in ambulatory participants with Duchenne muscular dystrophy (CIFFREO): a phase 3, double-blind, randomised, placebo-controlled study. Lancet Neurol. 2026 Mar;25(3):245-255. doi: 10.1016/S1474-4422(26)00036-0. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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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.
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Primary Completion Date (PCD) defined as the time point when at least 90 randomized participants received investigational product and completed the one-year follow-up/week 52 visit.
A total of 114 participants were enrolled and received at least one dose of study treatment. Results are reported based on primary completion date of Week 52.
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| ID | Title | Description |
|---|---|---|
| FG000 | Cohort 1 | Participants were randomized to receive a single dose of fordadistrogene Movaparvovec 2*10^14 (2E14) vector genomes per kilogram body weight (vg/kg) on Day 1 (Year 1 Day 1) and a single dose of matching placebo on Day 390 (Year 2 Day 1). |
| FG001 | Cohort 2 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Dec 28, 2023 | May 6, 2025 |
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Parallel up to the measurement of the primary outcome at Week 52. At the beginning of study Year 2 participants who were originally assigned to placebo will have the opportunity to receive PF-06939926. All participants will be followed for 5 years following treatment with PF-06939926.
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The study will be quadruple blind.
| Placebo | Other | Placebo will be administered as a single IV infusion at Year 1 for Cohort 2. |
|
| Placebo | Other | Placebo will be administered as a single IV infusion at Year 2 for Cohort 1. |
|
| PF-06939926 | Genetic | PF-06939926 will be administered as a single IV infusion at Year 2 for Cohort 2 |
|
| Change From Baseline in Percent of Muscle Fibers Expressing Mini-Dystrophin in Muscle Biopsies by Immunofluorescence at Week 52 |
Muscle fibers expressing mini-dystrophin transgene protein were evaluated by immunofluorescent staining using the mini-dystrophin specific antibody which only recognized the mini-dystrophin transgene protein. |
| Baseline, Week 52 |
| Change From Baseline in Serum Creatine Kinase (CK) Concentration at Week 52 | The CK results were analyzed by the central laboratory. | Baseline, Week 52 |
| Least Square Mean of Proportion of Skills Gained Based on the Individual Items of the NSAA at Week 52 | Proportion of skills gained at Week 52 were expressed as a proportion of the number of skills at Baseline that could be gained (numerator was number of items on NSAA gained at Week 52, with response 1 or 2 and denominator was number of items on NSAA with score 0 at baseline). The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. | Baseline, Week 52 |
| Least Square Mean of Proportion of Skills Either Improved or Maintained Based on the Individual Items of the NSAA at Week 52 | Proportion of skills either improved or maintained at Week 52 was expressed as a proportion of the number of items at Baseline that could be improved or maintained (numerator was the number of items on NSAA improved or maintained at Week 52 and denominator is number of items on NSAA which is 17). The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. | Baseline, Week 52 |
| Change From Baseline in 10 Meter Run/Walk Velocity at Week 52 | Velocity was calculated based on the time it took to complete the 10-meter run/walk test. | Baseline, Week 52 |
| Change From Baseline in Rise From Floor Velocity at Week 52 | Velocity was calculated based on the time it took to rise from floor. | Baseline, Week 52 |
| Change From Baseline in Modified Pediatric Outcome Data Collection Instrument (PODCI)- Transfer and Basic Mobility Core Scale at Week 52 | Modified PODCI- transfer and basic mobility core scale (parent of pediatric participant) consisted of 11 items that assessed how caregivers of participants evaluated a participant's ability to walk, stand, and perform activities of daily living. The scale produced an independent, standardized score ranging from 0-100, with lower scores representing lower levels of function. | Baseline, Week 52 |
| Change From Baseline in Modified PODCI- Sports and Physical Functioning Core Scale at Week 52 | Modified PODCI- sports and physical functioning core scale (parent of pediatric participant) consisted of 21 items that assessed how caregivers of participants evaluated a participant's ability to perform recreational activities. The scale produced an independent, standardized score ranging from 0-100, with lower scores representing lower levels of function. | Baseline, Week 52 |
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| UCLA Medical Center | Los Angeles | California | 90095 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | 52242 | United States |
| KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Fairway | Fairway | Kansas | 66205 | United States |
| KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) - Rainbow | Kansas City | Kansas | 66160 | United States |
| University of Kansas Hospital - Investigational Pharmacy | Kansas City | Kansas | 66160 | United States |
| University of Kansas Hospital - Pediatric and Pediatric ICU - Operating Room | Kansas City | Kansas | 66160 | United States |
| University of Kansas Medical Center | Kansas City | Kansas | 66160 | United States |
| Pediatric Cardiology | Prairie Village | Kansas | 66208 | United States |
| Lenox Baker Children's Hospital | Durham | North Carolina | 27705 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| Primary Childrens Hospital | Salt Lake City | Utah | 84113 | United States |
| University of Utah Clinical Neurosciences Center | Salt Lake City | Utah | 84132 | United States |
| University of Utah Hospital | Salt Lake City | Utah | 84132 | United States |
| Seattle Children's | Seattle | Washington | 98105 | United States |
| The Children's Hospital at Westmead | Westmead | New South Wales | 2145 | Australia |
| The Royal Children's Hospital Melbourne | Parkville | Victoria | 3052 | Australia |
| Perth Children's Hospital | Nedlands | Western Australia | 6009 | Australia |
| UZ Gent | Ghent | 9000 | Belgium |
| UZ leuven | Leuven | 3000 | Belgium |
| Alberta Children's Hospital | Calgary | Alberta | T3B 6A8 | Canada |
| Children's Hospital - London Health Sciences Centre | London | Ontario | N6A 4G5 | Canada |
| Childrens Hospital of Eastern Ontario | Ottawa | Ontario | K1H8L1 | Canada |
| The Hospital For Sick Children | Toronto | Ontario | M5G 1X8 | Canada |
| CHU de Nantes- Hotel Dieu | Nantes | 44093 | France |
| Hopital Necker | Paris | 75015 | France |
| Charité - Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Universitatsklinikum Essen | Essen | 45147 | Germany |
| Hadassah University Medical Center, Ein Kerem | Jerusalem | 91120 | Israel |
| Schneider Children's Medical Center of Israel | Petach Tikvah | 4920235 | Israel |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS | Roma | 00168 | Italy |
| IRCCS Ospedale Pediatrico Bambino Gesù | Rome | 00165 | Italy |
| Nagoya City University Hospital | Nagoya | Aichi-ken | 467-8602 | Japan |
| Hyogo College of Medicine College Hospital | Nishinomiya | Hyōgo | 663-8501 | Japan |
| National Center of Neurology and Psychiatry | Tokyo | 187-8551 | Japan |
| Saint Petersburg State Paediatric Medical University | Saint Petersburg | 194100 | Russia |
| State Autonomous Healthcare Institution of Sverdlovsk Region Children's City Clinical Hospital No 9 | Yekaterinburg | 620134 | Russia |
| Pusan National University Yangsan Hospital | Yangsan | Gyeongsangnam-do | 50612 | South Korea |
| Seoul National University Hospital | Seoul | 03080 | South Korea |
| Samsung Medical Center | Seoul | 06351 | South Korea |
| Hospital Sant Joan de Déu | Esplugues de Llobregat | Barcelona | 08950 | Spain |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital Universitari i Politecnic La Fe de Valencia | Valencia | 46026 | Spain |
| Inselspital, University Children's Hospital Berne | Bern | 3010 | Switzerland |
| Universitaets-Kinderspital Zuerich | Zurich | 8008 | Switzerland |
| Kaohsiung Medical University Chung-Ho Memorial Hospital | Kaohsiung City | 807 | Taiwan |
| National Taiwan University Hospital | Taipei | 100 | Taiwan |
| Taipei Veterans General Hospital | Taipei | 11217 | Taiwan |
| The Newcastle upon Tyne Hospitals NHS Foundation Trust, Royal Victoria Infirmary | Newcastle upon Tyne | England | NE1 4LP | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | Merseyside | L12 2AP | United Kingdom |
| Great Ormond Street Institute of Child Health | London | WCIN 1EH | United Kingdom |
Participants were randomized to receive a single dose of matching placebo on Day 1 (Year 1 Day 1) and a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 390 (Year 2 Day 1). |
| Received Treatment Year 1 Day 1 |
|
| Received Treatment Year 2 Day 1 |
|
| COMPLETED | Completed indicates participants completed the Week 52 as of PCD |
|
| NOT COMPLETED |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Cohort 1 | Participants were randomized to receive a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 1 (Year 1 Day 1) and a single dose of matching placebo on Day 390 (Year 2 Day 1). |
| BG001 | Cohort 2 | Participants were randomized to receive a single dose of matching placebo on Day 1 (Year 1 Day 1) and a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 390 (Year 2 Day 1). |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | Years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. Baseline NSAA total score is defined as the last non-missing NSAA total score collected prior to Year 1 drug administration. | Full analysis set (FAS) through Week 52 included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline, Week 52 |
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| Secondary | Change From Baseline in Percent Normal Dystrophin Expression Level in Muscle Biopsies by Liquid Chromatography Mass Spectrometry (LC-MS) Based on LLQV Peptide at Week 52 | The LC-MS assay measured the LLQVAVEDR (LLQV) peptide that detected full-length endogenous dystrophin as well as the mini-dystrophin transgene protein. | FAS through Week 52 included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | % normal dystrophin expression level | Baseline, Week 52 |
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| Secondary | Change From Baseline in Percent of Muscle Fibers Expressing Mini-Dystrophin in Muscle Biopsies by Immunofluorescence at Week 52 | Muscle fibers expressing mini-dystrophin transgene protein were evaluated by immunofluorescent staining using the mini-dystrophin specific antibody which only recognized the mini-dystrophin transgene protein. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Percentage of muscle fibers | Baseline, Week 52 |
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| Secondary | Change From Baseline in Serum Creatine Kinase (CK) Concentration at Week 52 | The CK results were analyzed by the central laboratory. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Units per Liter (U/L) | Baseline, Week 52 |
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| Secondary | Least Square Mean of Proportion of Skills Gained Based on the Individual Items of the NSAA at Week 52 | Proportion of skills gained at Week 52 were expressed as a proportion of the number of skills at Baseline that could be gained (numerator was number of items on NSAA gained at Week 52, with response 1 or 2 and denominator was number of items on NSAA with score 0 at baseline). The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Proportion of skills | Baseline, Week 52 |
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| Secondary | Least Square Mean of Proportion of Skills Either Improved or Maintained Based on the Individual Items of the NSAA at Week 52 | Proportion of skills either improved or maintained at Week 52 was expressed as a proportion of the number of items at Baseline that could be improved or maintained (numerator was the number of items on NSAA improved or maintained at Week 52 and denominator is number of items on NSAA which is 17). The NSAA was a 17-item test that graded performance of various functional skills using the following scale: 0 (unable to achieve independently), 1 (modified method but achieves goal independent of physical assistance from another), and 2 ("normal"- no obvious modification of activity). Total score was calculated as the sum of all 17 individual item responses and ranged from 0 (worst) to 34 (fully independent function) with higher scores indicating better function. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Proportion of skills | Baseline, Week 52 |
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| Secondary | Change From Baseline in 10 Meter Run/Walk Velocity at Week 52 | Velocity was calculated based on the time it took to complete the 10-meter run/walk test. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Meter per second | Baseline, Week 52 |
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| Secondary | Change From Baseline in Rise From Floor Velocity at Week 52 | Velocity was calculated based on the time it took to rise from floor. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Meter per second | Baseline, Week 52 |
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| Secondary | Change From Baseline in Modified Pediatric Outcome Data Collection Instrument (PODCI)- Transfer and Basic Mobility Core Scale at Week 52 | Modified PODCI- transfer and basic mobility core scale (parent of pediatric participant) consisted of 11 items that assessed how caregivers of participants evaluated a participant's ability to walk, stand, and perform activities of daily living. The scale produced an independent, standardized score ranging from 0-100, with lower scores representing lower levels of function. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline, Week 52 |
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| Secondary | Change From Baseline in Modified PODCI- Sports and Physical Functioning Core Scale at Week 52 | Modified PODCI- sports and physical functioning core scale (parent of pediatric participant) consisted of 21 items that assessed how caregivers of participants evaluated a participant's ability to perform recreational activities. The scale produced an independent, standardized score ranging from 0-100, with lower scores representing lower levels of function. | FAS included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Here, "Overall Number of Participants Analyzed" signifies participants evaluable for this outcome measure. | Posted | Least Squares Mean | 95% Confidence Interval | Score on a scale | Baseline, Week 52 |
|
From Year 1 Day 1 up to Week 52
Safety analysis set through Week 52 included all eligible participants who were randomly assigned and received a single dose of study drug on Day 1 (Year 1 Day 1). Same event may appear as both SAE and non-SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Cohort 1 | Participants were randomized to receive a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 1 (Year 1 Day 1) and a single dose of matching placebo on Day 390 (Year 2 Day 1). | 0 | 79 | 25 | 79 | 77 | 79 |
| EG001 | Cohort 2 | Participants were randomized to receive a single dose of matching placebo on Day 1 (Year 1 Day 1) and a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 390 (Year 2 Day 1). | 0 | 35 | 5 | 35 | 20 | 35 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Thrombotic microangiopathy | Blood and lymphatic system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Bradycardia | Cardiac disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Myocarditis | Cardiac disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Enterocolitis | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Gastrointestinal disorder | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hepatitis | Hepatobiliary disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hepatitis acute | Hepatobiliary disorders | MedDRA v27.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Fibula fracture | Injury, poisoning and procedural complications | MedDRA v27.0 | Systematic Assessment |
| |
| Tibia fracture | Injury, poisoning and procedural complications | MedDRA v27.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Troponin I increased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hypophagia | Metabolism and nutrition disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Myositis | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Rhabdomyolysis | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hypertensive encephalopathy | Nervous system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Ear infection | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA v27.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA v27.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA v27.0 | Systematic Assessment |
| |
| Gamma-glutamyltransferase increased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Glutamate dehydrogenase increased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Platelet count decreased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| SARS-CoV-2 test positive | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Troponin I increased | Investigations | MedDRA v27.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Dehydration | Metabolism and nutrition disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA v27.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v27.0 | Systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer Clinical Trials.gov Call Center | Pfizer Inc. | 18007181021 | ClinicalTrials.gov_Inquiries@pfizer.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 8, 2024 | May 6, 2025 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
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| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
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| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
| Participants |
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| Participants |
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Participants were randomized to receive a single dose of matching placebo on Day 1 (Year 1 Day 1) and a single dose of fordadistrogene Movaparvovec 2E14 vg/kg on Day 390 (Year 2 Day 1).
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| Participants |
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