A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.

Duchenne Muscular Dystrophy Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05689164
• Other study ID #: C3391011
• Status: Recruiting
• Phase: Phase 3
• Start date: March 13, 2023
• Est. completion date: May 15, 2039

Study information

• Verified date: February 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: May 15, 2039
• Est. primary completion date: May 15, 2039
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 0 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Biological:
• fordadistrogene movaparvovec
gene therapy administered in a previous study.

Locations

Country	Name	City	State
United States	Duke Children's Health Center	Durham	North Carolina
United States	Duke Lenox Baker Children's	Durham	North Carolina
United States	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	UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)	Los Angeles	California
United States	UCLA Medical Center	Los Angeles	California
United States	Primary Children's Hospital	Salt Lake City	Utah
United States	University of Utah Clinical Neurosciences Center	Salt Lake City	Utah
United States	University of Utah Craig H. Neilsen Rehabilitation Hospital	Salt Lake City	Utah
United States	University of Utah Hospital	Salt Lake City	Utah
United States	University of Utah Imaging and Neurosciences Center	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with serious adverse events		At least annually from 5 through 10 years after dosing in the interventional study.
Primary	Percentage of participants with serious adverse events		At least annually from 5 through 10 years after dosing in the interventional study.
Primary	Number of participants with adverse events considered related to treatment		At least annually from 5 through 10 years after dosing in the interventional study.
Primary	Percentage of particpants with adverse events considered related to treatment		At least annually from 5 through 10 years after dosing in the interventional study
Primary	Number of participants with malignancy adverse event		At least annually from 5 through 10 years after dosing in the interventional study
Primary	Number of participants with clinically significant findings in electrocardiogram (ECG) assessments		Annually from 5 through 10 years after dosing in the interventional study.
Primary	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
Primary	Number of participants with clinically significant findings in echocardiogram parameters		Annually from 5 through 10 years after dosing in the interventional study
Secondary	Change from pre-dose in the ability to walk 10 meters unassisted		Annually from 5 through 10 years after dosing in the interventional study.
Secondary	Change from pre-dose in the ability to climb stairs.		Annually from 5 through 10 years after dosing in the interventional study
Secondary	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
Secondary	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
Secondary	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
Secondary	Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram		Annually from 5 through 10 years after dosing in the interventional study
Secondary	Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument		Annually from 5 through 10 years after dosing in the interventional study
Secondary	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
Secondary	Age when percent predicted forced vital capacity <30%		Annually from 5 through 10 years after dosing in the interventional study
Secondary	Age at loss of ambulation		From 5 through 10 years after dosing in the interventional study
Secondary	Age at death		From 5 through 10 years after dosing in the interventional study
Secondary	Cause of death		From 5 through 10 years after dosing in the interventional study
Secondary	Glucocorticoid use dose and frequency		At least annually from 5 through 10 years after dosing in the interventional study

External Links

•   To obtain contact information for a study center near you, click here.