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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
OTHER
Masking
NONE
Enrollment
7
gene therapy administered in a previous study.
Reed Neurological Research Center
Los Angeles, California, United States
UCLA Children's Heart Center
Los Angeles, California, United States
UCLA Clinical Lab Services
Los Angeles, California, United States
Number of participants with serious adverse events
Time frame: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of participants with serious adverse events
Time frame: At least annually from 5 through 10 years after dosing in the interventional study.
Number of participants with adverse events considered related to treatment
Time frame: At least annually from 5 through 10 years after dosing in the interventional study.
Percentage of particpants with adverse events considered related to treatment
Time frame: At least annually from 5 through 10 years after dosing in the interventional study
Number of participants with malignancy adverse event
Time frame: 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
Time frame: 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
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Number of participants with clinically significant findings in echocardiogram parameters
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the ability to walk 10 meters unassisted
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UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)
Los Angeles, California, United States
UCLA Medical Center
Los Angeles, California, United States
Duke Lenox Baker Children's
Durham, North Carolina, United States
Duke Children's Health Center
Durham, North Carolina, United States
University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, United States
University of Utah Hospital
Salt Lake City, Utah, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
...and 2 more locations
Time frame: Annually from 5 through 10 years after dosing in the interventional study.
Change from pre-dose in the ability to climb stairs.
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score
Time frame: 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
Time frame: 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)
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument
Time frame: 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
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Age when percent predicted forced vital capacity <30%
Time frame: Annually from 5 through 10 years after dosing in the interventional study
Age at loss of ambulation
Time frame: From 5 through 10 years after dosing in the interventional study
Age at death
Time frame: From 5 through 10 years after dosing in the interventional study
Cause of death
Time frame: From 5 through 10 years after dosing in the interventional study
Glucocorticoid use dose and frequency
Time frame: At least annually from 5 through 10 years after dosing in the interventional study