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Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05429372
Recruitment Status : Active, not recruiting
First Posted : June 23, 2022
Last Update Posted : April 4, 2024
Sponsor:
Information provided by (Responsible Party):
Pfizer

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

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Genetic: PF-06939926 Phase 2

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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52).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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : August 8, 2022
Estimated Primary Completion Date : December 27, 2024
Estimated Study Completion Date : January 3, 2029


Arm Intervention/treatment
Experimental: PF-06939926 Genetic: PF-06939926
All participants will receive a single dose of PF-06939926 on Day 1.
Other Name: Fordadistrogene Movaparvovec




Primary Outcome Measures :
  1. Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events [ Time Frame: Through Week 52 ]
  2. Number of participants with abnormal hematology test results [ Time Frame: Through Week 52 ]
    Blood samples will be collected from subjects for the analysis of hematology

  3. Number of participants with abnormal biochemistry test results [ Time Frame: Through Week 52 ]
    Blood samples will be collected from subjects for the analysis of biochemistry

  4. Number of participants with abnormal urine analysis [ Time Frame: Through Week 52 ]
    Urine samples will be collected from subjects for the analysis of urine

  5. Number of participants with abnormal and clinically relevant changes in neurological examinations [ Time Frame: Through Week 52 ]
  6. Number of participants with abnormal and clinically relevant changes in body weight [ Time Frame: Through Week 52 ]
  7. Number of participants with abnormal and clinically relevant changes in vital signs [ Time Frame: Through Week 52 ]
  8. Number of participants with abnormal and clinically relevant changes on cardiac troponin I [ Time Frame: Through Week 52 ]
  9. Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG) [ Time Frame: Through Week 52 ]
  10. Number of participants with abnormal and clinically relevant changes on echocardiogram [ Time Frame: Through Week 52 ]

Secondary Outcome Measures :
  1. Distribution of mini-dystrophin expression in muscle [ Time Frame: At Week 9, Week 52 and Year 5 (if available) ]
    Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence

  2. Level of mini-dystrophin expression in muscle [ Time Frame: At Week 9, Week 52 and Year 5 (if available) ]
    Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry

  3. Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events [ Time Frame: Through 5 years ]
  4. Number of participants with abnormal hematology test results [ Time Frame: Through 5 years ]
    Blood samples will be collected from subjects for the analysis of hematology

  5. Number of participants with abnormal biochemistry test results [ Time Frame: Through 5 years ]
    Blood samples will be collected from subjects for the analysis of biochemistry

  6. Number of participants with abnormal urine analysis [ Time Frame: Through 5 years ]
    Urine samples will be collected from subjects for the analysis of urine

  7. Number of participants with abnormal and clinically relevant changes in neurological examinations [ Time Frame: Through 5 years ]
  8. Number of participants with abnormal and clinically relevant changes in body weight [ Time Frame: Through 5 years ]
  9. Number of participants with abnormal and clinically relevant changes in vital signs [ Time Frame: Through 5 years ]
  10. Number of participants with abnormal and clinically relevant changes on cardiac troponin I [ Time Frame: Through 5 years ]
  11. Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG) [ Time Frame: Through 5 years ]
  12. Number of participants with abnormal and clinically relevant changes on echocardiogram [ Time Frame: Through 5 years ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   2 Years to 3 Years   (Child)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Male participants age ≥2 to <4 years, at Screening (Visit 1)
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05429372


Locations
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United States, Florida
UF Health Shands Hospital
Gainesville, Florida, United States, 32610
University of Florida
Gainesville, Florida, United States, 32610
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19146
United States, Utah
CTSI Clinical Research Center
Salt Lake City, Utah, United States, 84108
University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, United States, 84108
University of Utah Hospital & Clinics Investigational Drug Services
Salt Lake City, Utah, United States, 84112
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, United States, 84132
University of Utah Hospital
Salt Lake City, Utah, United States, 84132
Australia, New South Wales
The Children's Hospital at Westmead
Westmead, New South Wales, Australia, 2145
Australia, Victoria
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Australia, Western Australia
Perth Children's Hospital
Nedlands, Western Australia, Australia, 6009
Sponsors and Collaborators
Pfizer
Investigators
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Study Director: Pfizer CT.gov Call Center Pfizer
Additional Information:
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Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT05429372    
Other Study ID Numbers: C3391008
2021-003379-33 ( EudraCT Number )
First Posted: June 23, 2022    Key Record Dates
Last Update Posted: April 4, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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.
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pfizer:
Early Stage Duchenne Muscular Dystrophy
DMD
gene therapy
fordadistrogene movaparvovec
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked