Back to resultsA Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
Primary Purpose
Duchenne Muscular Dystrophy
Status
Recruiting
Phase
Phase 3
Locations
United States
Study Type
Interventional
Intervention
fordadistrogene movaparvovec
Sponsored by
About this trial
This is an interventional other trial for Duchenne Muscular Dystrophy focused on measuring Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy
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
Sites / Locations
- Duke Lenox Baker Children'sRecruiting
- Duke Children's Health CenterRecruiting
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
All participants
Arm Description
All participants enrolled in the study.
Outcomes
Primary Outcome Measures
Number of participants with serious adverse events
Percentage of participants with serious adverse events
Number of participants with adverse events considered related to treatment
Percentage of particpants with adverse events considered related to treatment
Number of participants with malignancy adverse event
Number of participants with clinically significant findings in electrocardiogram (ECG) assessments
Number of participants with clinically significant findings in cardiac troponin I laboratory examinations
Number of participants with clinically significant findings in echocardiogram parameters
Secondary Outcome Measures
Change from pre-dose in the ability to walk 10 meters unassisted
Change from pre-dose in the ability to climb stairs.
Change from pre-dose in the Performance of Upper Limb (PUL) 2.0 entry score
Change from pre-dose in the North Star Ambulatory Assessment total score
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)
Change from pre-dose in left ventricular ejection fraction (LVEF) on echocardiogram
Change from pre-dose in the Modified Pediatric Outcomes Data Collection Instrument
Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure
Applicable to non-ambulatory participants only
Age when percent predicted forced vital capacity <30%
Age at loss of ambulation
Age at death
Cause of death
Glucocorticoid use dose and frequency
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT05689164
Brief Title
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
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 13, 2023 (Actual)
Primary Completion Date
May 15, 2039 (Anticipated)
Study Completion Date
May 15, 2039 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
Duchenne Muscular Dystrophy, Muscular Dystrophy, Duchenne, Genetic Therapy, Gene Therapy
7. Study Design
Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
250 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
All participants
Arm Type
Other
Arm Description
All participants enrolled in the study.
Intervention Type
Biological
Intervention Name(s)
fordadistrogene movaparvovec
Intervention Description
gene therapy administered in a previous study.
Primary Outcome Measure Information:
Title
Number of participants with serious adverse events
Time Frame
At least annually from 5 through 10 years after dosing in the interventional study.
Title
Percentage of participants with serious adverse events
Time Frame
At least annually from 5 through 10 years after dosing in the interventional study.
Title
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.
Title
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
Title
Number of participants with malignancy adverse event
Time Frame
At least annually from 5 through 10 years after dosing in the interventional study
Title
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.
Title
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
Title
Number of participants with clinically significant findings in echocardiogram parameters
Time Frame
Annually from 5 through 10 years after dosing in the interventional study
Secondary Outcome Measure Information:
Title
Change from pre-dose in the ability to walk 10 meters unassisted
Time Frame
Annually from 5 through 10 years after dosing in the interventional study.
Title
Change from pre-dose in the ability to climb stairs.
Time Frame
Annually from 5 through 10 years after dosing in the interventional study
Title
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
Title
Change from pre-dose in the North Star Ambulatory Assessment total score
Description
Applicable to a sub-set of participants only
Time Frame
Annually from 5 through 10 years after dosing in the interventional study
Title
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
Title
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
Title
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
Title
Change from pre-dose in the Upper Limb Function Patient Reported Outcome Measure
Description
Applicable to non-ambulatory participants only
Time Frame
Annually from 5 through 10 years after dosing in the interventional study
Title
Age when percent predicted forced vital capacity <30%
Time Frame
Annually from 5 through 10 years after dosing in the interventional study
Title
Age at loss of ambulation
Time Frame
From 5 through 10 years after dosing in the interventional study
Title
Age at death
Time Frame
From 5 through 10 years after dosing in the interventional study
Title
Cause of death
Time Frame
From 5 through 10 years after dosing in the interventional study
Title
Glucocorticoid use dose and frequency
Time Frame
At least annually from 5 through 10 years after dosing in the interventional study
10. Eligibility
Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
0 Years
Accepts Healthy Volunteers
No
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Pfizer CT.gov Call Center
Phone
1-800-718-1021
Email
ClinicalTrials.gov_Inquiries@pfizer.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
Duke Lenox Baker Children's
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27705
Country
United States
Individual Site Status
Recruiting
Facility Name
Duke Children's Health Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Individual Site Status
Recruiting
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
IPD Sharing URL
https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=C3391011
Description
To obtain contact information for a study center near you, click here.
Learn more about this trial
A Study to Understand the Long-term Safety and Effects of an Experimental Gene Therapy for Duchenne Muscular Dystrophy.
We'll reach out to this number within 24 hrs