Study of Fordadistrogene Movaparvovec in Early Stage Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne Clinical Trial

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05429372
• Other study ID #: C3391008
• Secondary ID: 2021-003379-33
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: August 8, 2022
• Est. completion date: January 3, 2029

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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The primary analysis will occur when all participants have completed visits through Week 52 (or withdrawn from the study prior to Week 52).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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 10
• Est. completion date: January 3, 2029
• Est. primary completion date: December 27, 2024
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 2 Years to 3 Years

Eligibility

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.

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Genetic:
• PF-06939926
All participants will receive a single dose of PF-06939926 on Day 1.

Locations

Country	Name	City	State
Australia	Perth Children's Hospital	Nedlands	Western Australia
Australia	The Royal Children's Hospital Melbourne	Parkville	Victoria
Australia	The Children's Hospital at Westmead	Westmead	New South Wales
United States	UF Health Shands Hospital	Gainesville	Florida
United States	University of Florida	Gainesville	Florida
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	CTSI Clinical Research Center	Salt Lake City	Utah
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 Hospital	Salt Lake City	Utah
United States	University of Utah Hospital & Clinics Investigational Drug Services	Salt Lake City	Utah
United States	University of Utah Imaging and Neurosciences Center	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events		Through Week 52
Primary	Number of participants with abnormal hematology test results	Blood samples will be collected from subjects for the analysis of hematology	Through Week 52
Primary	Number of participants with abnormal biochemistry test results	Blood samples will be collected from subjects for the analysis of biochemistry	Through Week 52
Primary	Number of participants with abnormal urine analysis	Urine samples will be collected from subjects for the analysis of urine	Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes in neurological examinations		Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes in body weight		Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes in vital signs		Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes on cardiac troponin I		Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)		Through Week 52
Primary	Number of participants with abnormal and clinically relevant changes on echocardiogram		Through Week 52
Secondary	Distribution of mini-dystrophin expression in muscle	Mini-dystrophin distribution from a muscle biopsy will be assessed by immunofluorescence	At Week 9, Week 52 and Year 5 (if available)
Secondary	Level of mini-dystrophin expression in muscle	Mini-dystrophin expression level from a muscle biopsy will be assessed by liquid chromatography mass spectrometry	At Week 9, Week 52 and Year 5 (if available)
Secondary	Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events		Through 5 years
Secondary	Number of participants with abnormal hematology test results	Blood samples will be collected from subjects for the analysis of hematology	Through 5 years
Secondary	Number of participants with abnormal biochemistry test results	Blood samples will be collected from subjects for the analysis of biochemistry	Through 5 years
Secondary	Number of participants with abnormal urine analysis	Urine samples will be collected from subjects for the analysis of urine	Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes in neurological examinations		Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes in body weight		Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes in vital signs		Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes on cardiac troponin I		Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)		Through 5 years
Secondary	Number of participants with abnormal and clinically relevant changes on echocardiogram		Through 5 years

External Links

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