Clinical trial

Long-term Follow-up Safety and Efficacy Study in Participants With Duchenne Muscular Dystrophy Who Have Received Fordadistrogene Movaparvovec in a Preceding Clinical Study

Name

C3391011

Description

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.

Trial arms

Trial start

2023-03-13

Estimated PCD

2039-05-15

Trial end

2039-05-15

Status

Recruiting

Phase

Early phase I

Treatment

fordadistrogene movaparvovec

gene therapy administered in a previous study.

Arms:

All participants

Size

250

Primary endpoint

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

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE3'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'OTHER', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 250, 'type': 'ESTIMATED'}}

Updated at

2024-02-22

1 organization

1 product

1 indication

Product

Fordadistrogene Movaparvovec

Indication

Duchenne muscular dystrophy

Organization

Pfizer