Clinical trial NCT05689164: A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Official title: Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
Muscular Dystrophy, Duchenne
Genetic Therapy
Gene Therapy
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2023-01-19
Enrollment: 7
Sex: MALE
Minimum age (years): 0
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: OTHER

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with serious adverse events
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study.
measure: Percentage of participants with serious adverse events
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study.
measure: Number of participants with adverse events considered related to treatment
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study.
measure: Percentage of particpants with adverse events considered related to treatment
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study
measure: Number of participants with malignancy adverse event
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study
measure: Number of participants with clinically significant findings in electrocardiogram (ECG) assessments
timeFrame: Annually from 5 through 10 years after dosing in the interventional study.
measure: Number of participants with clinically significant findings in cardiac troponin I laboratory examinations
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Number of participants with clinically significant findings in echocardiogram parameters
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
Secondary outcomes:
measure: Change from pre-dose in the ability to walk 10 meters unassisted
timeFrame: Annually from 5 through 10 years after dosing in the interventional study.
measure: Change from pre-dose in the ability to climb stairs.
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in the North Star Ambulatory Assessment total score
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in percent of predicted forced vital capacity (%pFVC) and percent predicted peak expiratory flow (%pPEF)
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Age when percent predicted forced vital capacity <30%
timeFrame: Annually from 5 through 10 years after dosing in the interventional study
measure: Age at loss of ambulation
timeFrame: From 5 through 10 years after dosing in the interventional study
measure: Age at death
timeFrame: From 5 through 10 years after dosing in the interventional study
measure: Cause of death
timeFrame: From 5 through 10 years after dosing in the interventional study
measure: Glucocorticoid use dose and frequency
timeFrame: At least annually from 5 through 10 years after dosing in the interventional study
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Reed Neurological Research Center
state: California
city: Los Angeles
country: United States
facility: UCLA Children's Heart Center
state: California
city: Los Angeles
country: United States
facility: UCLA Clinical Lab Services
state: California
city: Los Angeles
country: United States
facility: UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)
state: California
city: Los Angeles
country: United States
facility: UCLA Medical Center
state: California
city: Durham
country: United States
facility: Duke Lenox Baker Children's
state: North Carolina
city: Durham
country: United States
facility: Duke Children's Health Center
state: North Carolina
city: Salt Lake City
country: United States
facility: University of Utah Imaging and Neurosciences Center
state: Utah
city: Salt Lake City
country: United States
facility: University of Utah Hospital
state: Utah
city: Salt Lake City
country: United States
facility: Primary Children's Hospital
state: Utah
city: Salt Lake City
country: United States
facility: University of Utah Clinical Neurosciences Center
state: Utah
city: Salt Lake City
country: United States
facility: University of Utah Craig H. Neilsen Rehabilitation Hospital
state: Utah
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: