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| ID | Type | Description | Link |
|---|---|---|---|
| NCT05689164 | Registry Identifier | ClinicalTrials.gov |
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All participants who have received fordadistrogene movaparvovec in any Pfizer study will now be assessed for long-term safety in 1 combined study: C3391003
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The purpose of this study is to understand the safety and effects of an experimental gene therapy called fordadistrogene movaparvovec. We are seeking participants from previous Pfizer interventional studies. We will follow participants' experience in this study for 10 years after the end of their previous study. Participants will have 1 annual onsite visit and a few annual remote visits. The exact number of remote visits will be decided by their study doctor.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| All participants | Other | All participants enrolled in the study. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| fordadistrogene movaparvovec | Biological | gene therapy administered in a previous study. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with serious adverse events | At least annually from 5 through 10 years after dosing in the interventional study. | |
| Percentage of participants with serious adverse events | At least annually from 5 through 10 years after dosing in the interventional study. | |
| Number of participants with adverse events considered related to treatment | At least annually from 5 through 10 years after dosing in the interventional study. | |
| Percentage of particpants with adverse events considered related to treatment | At least annually from 5 through 10 years after dosing in the interventional study | |
| Number of participants with malignancy adverse event | At least annually from 5 through 10 years after dosing in the interventional study | |
| Number of participants with clinically significant findings in electrocardiogram (ECG) assessments | Annually from 5 through 10 years after dosing in the interventional study. | |
| Number of participants with clinically significant findings in cardiac troponin I laboratory examinations | Annually from 5 through 10 years after dosing in the interventional study | |
| Number of participants with clinically significant findings in echocardiogram parameters | Annually from 5 through 10 years after dosing in the interventional study |
| Measure | Description | Time Frame |
|---|---|---|
| Change from pre-dose in the ability to walk 10 meters unassisted | Annually from 5 through 10 years after dosing in the interventional study. | |
| Change from pre-dose in the ability to climb stairs. | Annually from 5 through 10 years after dosing in the interventional study |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Reed Neurological Research Center | Los Angeles | California | 90095 | United States | ||
| UCLA Children's Heart Center |
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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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| Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score | Annually from 5 through 10 years after dosing in the interventional study |
| Change from pre-dose in the North Star Ambulatory Assessment total score | Applicable to a sub-set of participants only | Annually from 5 through 10 years after dosing in the interventional study |
| Change from pre-dose in percent of predicted forced vital capacity (%pFVC) and percent predicted peak expiratory flow (%pPEF) | Annually from 5 through 10 years after dosing in the interventional study |
| Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram | Annually from 5 through 10 years after dosing in the interventional study |
| Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument | Annually from 5 through 10 years after dosing in the interventional study |
| Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure | Applicable to non-ambulatory participants only | Annually from 5 through 10 years after dosing in the interventional study |
| Age when percent predicted forced vital capacity <30% | Annually from 5 through 10 years after dosing in the interventional study |
| Age at loss of ambulation | From 5 through 10 years after dosing in the interventional study |
| Age at death | From 5 through 10 years after dosing in the interventional study |
| Cause of death | From 5 through 10 years after dosing in the interventional study |
| Glucocorticoid use dose and frequency | At least annually from 5 through 10 years after dosing in the interventional study |
| Los Angeles |
| California |
| 90095 |
| United States |
| UCLA Clinical Lab Services | Los Angeles | California | 90095 | United States |
| UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center) | Los Angeles | California | 90095 | United States |
| UCLA Medical Center | Los Angeles | California | 90095 | United States |
| Duke Lenox Baker Children's | Durham | North Carolina | 27705 | United States |
| Duke Children's Health Center | Durham | North Carolina | 27710 | United States |
| University of Utah Imaging and Neurosciences Center | Salt Lake City | Utah | 84108 | United States |
| University of Utah Hospital | 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 Craig H. Neilsen Rehabilitation Hospital | Salt Lake City | Utah | 84132 | United States |
| 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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