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Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Primary Purpose

Muscular Dystrophy, Duchenne

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
PF-06939926
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Early Stage Duchenne Muscular Dystrophy, DMD, gene therapy, fordadistrogene movaparvovec

Eligibility Criteria

2 Years - 3 Years (Child)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • Confirmed diagnosis of DMD by prior genetic testing.

Exclusion Criteria:

  • Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive.
  • Positive test performed by Pfizer for neutralizing antibodies to AAV9.
  • Any prior treatment with gene therapy.
  • Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through).
  • Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD.
  • Abnormality in specified laboratory tests, including blood counts, liver and kidney function.

Sites / Locations

  • UF Health Shands Hospital
  • University of Florida
  • The Children's Hospital of Philadelphia
  • The Children's Hospital of Philadelphia
  • CTSI Clinical Research Center
  • University of Utah Imaging and Neurosciences Center
  • University of Utah Hospital & Clinics Investigational Drug Services
  • Primary Children's Hospital
  • University of Utah Clinical Neurosciences Center
  • University of Utah Hospital
  • The Children's Hospital at Westmead
  • The Royal Children's Hospital Melbourne
  • Perth Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PF-06939926

Arm Description

Outcomes

Primary Outcome Measures

Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Number of participants with abnormal hematology test results
Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal biochemistry test results
Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal urine analysis
Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in neurological examinations
Number of participants with abnormal and clinically relevant changes in body weight
Number of participants with abnormal and clinically relevant changes in vital signs
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Number of participants with abnormal and clinically relevant changes on echocardiogram

Secondary Outcome Measures

Distribution of mini-dystrophin expression in muscle
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
Level of mini-dystrophin expression in muscle
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Number of participants with abnormal hematology test results
Blood samples will be collected from subjects for the analysis of hematology
Number of participants with abnormal biochemistry test results
Blood samples will be collected from subjects for the analysis of biochemistry
Number of participants with abnormal urine analysis
Urine samples will be collected from subjects for the analysis of urine
Number of participants with abnormal and clinically relevant changes in neurological examinations
Number of participants with abnormal and clinically relevant changes in body weight
Number of participants with abnormal and clinically relevant changes in vital signs
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Number of participants with abnormal and clinically relevant changes on echocardiogram

Full Information

First Posted
October 8, 2021
Last Updated
July 6, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT05429372
Brief Title
Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy
Official Title
A PHASE 2, MULTICENTER, SINGLE-ARM STUDY TO EVALUATE THE SAFETY AND DYSTROPHIN EXPRESSION AFTER FORDADISTROGENE MOVAPARVOVEC (PF-06939926) ADMINISTRATION IN MALE PARTICIPANTS WITH EARLY STAGE DUCHENNE MUSCULAR DYSTROPHY
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
August 8, 2022 (Actual)
Primary Completion Date
July 9, 2024 (Anticipated)
Study Completion Date
June 17, 2028 (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
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The study will evaluate the safety and dystrophin expression following gene therapy in boys with Duchenne Muscular Dystrophy (DMD). It is a single-arm, non-randomized, open-label study
Detailed Description
The study will assess the safety and tolerability of fordadistrogene movaparvovec gene therapy. Approximately 10 participants will be enrolled in the study and receive a single IV infusion of PF-06939926; there is no placebo arm. The study includes boys who are at least 2 years old and less than 4 years old (including 3 year olds up until their 4th birthday). All boys will need to be negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening. The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52). All participants will be followed in the study for 5 years after treatment with gene therapy.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Early Stage Duchenne Muscular Dystrophy, DMD, gene therapy, fordadistrogene movaparvovec

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PF-06939926
Arm Type
Experimental
Intervention Type
Genetic
Intervention Name(s)
PF-06939926
Other Intervention Name(s)
Fordadistrogene Movaparvovec
Intervention Description
All participants will receive a single dose of PF-06939926 on Day 1.
Primary Outcome Measure Information:
Title
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Time Frame
Through Week 52
Title
Number of participants with abnormal hematology test results
Description
Blood samples will be collected from subjects for the analysis of hematology
Time Frame
Through Week 52
Title
Number of participants with abnormal biochemistry test results
Description
Blood samples will be collected from subjects for the analysis of biochemistry
Time Frame
Through Week 52
Title
Number of participants with abnormal urine analysis
Description
Urine samples will be collected from subjects for the analysis of urine
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes in neurological examinations
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes in body weight
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes in vital signs
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Time Frame
Through Week 52
Title
Number of participants with abnormal and clinically relevant changes on echocardiogram
Time Frame
Through Week 52
Secondary Outcome Measure Information:
Title
Distribution of mini-dystrophin expression in muscle
Description
Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence
Time Frame
At Week 9, Week 52 and Year 5 (if available)
Title
Level of mini-dystrophin expression in muscle
Description
Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry
Time Frame
At Week 9, Week 52 and Year 5 (if available)
Title
Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events
Time Frame
Through 5 years
Title
Number of participants with abnormal hematology test results
Description
Blood samples will be collected from subjects for the analysis of hematology
Time Frame
Through 5 years
Title
Number of participants with abnormal biochemistry test results
Description
Blood samples will be collected from subjects for the analysis of biochemistry
Time Frame
Through 5 years
Title
Number of participants with abnormal urine analysis
Description
Urine samples will be collected from subjects for the analysis of urine
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes in neurological examinations
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes in body weight
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes in vital signs
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes on cardiac troponin I
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)
Time Frame
Through 5 years
Title
Number of participants with abnormal and clinically relevant changes on echocardiogram
Time Frame
Through 5 years

10. Eligibility

Sex
Male
Gender Based
Yes
Gender Eligibility Description
Male participants age ≥2 to <4 years, at Screening (Visit 1)
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
3 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Confirmed diagnosis of DMD by prior genetic testing. Exclusion Criteria: Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive. Positive test performed by Pfizer for neutralizing antibodies to AAV9. Any prior treatment with gene therapy. Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through). Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD. Abnormality in specified laboratory tests, including blood counts, liver and kidney function.
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
UF Health Shands Hospital
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
University of Florida
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
The Children's Hospital of Philadelphia
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19146
Country
United States
Facility Name
CTSI Clinical Research Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of Utah Imaging and Neurosciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84108
Country
United States
Facility Name
University of Utah Hospital & Clinics Investigational Drug Services
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
Primary Children's Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
University of Utah Clinical Neurosciences Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Utah Hospital
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
The Children's Hospital at Westmead
City
Westmead
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
The Royal Children's Hospital Melbourne
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Perth Children's Hospital
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia

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=C3391008
Description
To obtain contact information for a study center near you, click here.

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Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

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