Duchenne Muscular Dystrophy
Conditions
Keywords
Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy
Brief summary
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.
Interventions
BIOLOGICALfordadistrogene movaparvovec
gene therapy administered in a previous study.
Sponsors
Pfizer
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
0 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study.
Exclusion criteria
* Investigator site staff directly involved in the study and their family members
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 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 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| Change from pre-dose in the ability to walk 10 meters unassisted | Annually 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 | — |
| Age at death | 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 | — |
| 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 | Annually from 5 through 10 years after dosing in the interventional study | Applicable to a sub-set of participants only |
| 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 | Annually from 5 through 10 years after dosing in the interventional study | Applicable to non-ambulatory participants only |
Countries
United States
Outcome results
None listed