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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-003379-33 | EudraCT Number |
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All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in one combined study: C3391003
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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
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). 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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| PF-06939926 | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| PF-06939926 | Genetic | All participants will receive a single dose of PF-06939926 on Day 1. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events | Through Week 52 | |
| Number of participants with abnormal hematology test results | Blood samples will be collected from subjects for the analysis of hematology | Through Week 52 |
| Number of participants with abnormal biochemistry test results | Blood samples will be collected from subjects for the analysis of biochemistry | Through Week 52 |
| Number of participants with abnormal urine analysis | Urine samples will be collected from subjects for the analysis of urine | Through Week 52 |
| Number of participants with abnormal and clinically relevant changes in neurological examinations | Through Week 52 | |
| Number of participants with abnormal and clinically relevant changes in body weight | Through Week 52 | |
| Number of participants with abnormal and clinically relevant changes in vital signs | Through Week 52 | |
| Number of participants with abnormal and clinically relevant changes on cardiac troponin I | Through Week 52 | |
| Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG) |
| Measure | Description | Time Frame |
|---|---|---|
| Distribution of mini-dystrophin expression in muscle | Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence | At Week 9, Week 52 and Year 5 (if available) |
| Level of mini-dystrophin expression in muscle |
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Inclusion Criteria:
Exclusion Criteria:
Male participants age ≥2 to <4 years, at Screening (Visit 1)
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UF Health Shands Hospital | Gainesville | Florida | 32610 | United States | ||
| University of Florida |
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| 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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| Through Week 52 |
| Number of participants with abnormal and clinically relevant changes on echocardiogram | Through Week 52 |
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
| At Week 9, Week 52 and Year 5 (if available) |
| Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events | Through 5 years |
| Number of participants with abnormal hematology test results | Blood samples will be collected from subjects for the analysis of hematology | Through 5 years |
| Number of participants with abnormal biochemistry test results | Blood samples will be collected from subjects for the analysis of biochemistry | Through 5 years |
| Number of participants with abnormal urine analysis | Urine samples will be collected from subjects for the analysis of urine | Through 5 years |
| Number of participants with abnormal and clinically relevant changes in neurological examinations | Through 5 years |
| Number of participants with abnormal and clinically relevant changes in body weight | Through 5 years |
| Number of participants with abnormal and clinically relevant changes in vital signs | Through 5 years |
| Number of participants with abnormal and clinically relevant changes on cardiac troponin I | Through 5 years |
| Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG) | Through 5 years |
| Number of participants with abnormal and clinically relevant changes on echocardiogram | Through 5 years |
| Gainesville |
| Florida |
| 32610 |
| United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19146 | United States |
| CTSI Clinical Research Center | Salt Lake City | Utah | 84108 | United States |
| University of Utah Imaging and Neurosciences Center | Salt Lake City | Utah | 84108 | United States |
| University of Utah Hospital & Clinics Investigational Drug Services | Salt Lake City | Utah | 84112 | United States |
| Primary Children's 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 |
| 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 |
| 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 |
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