Clinical trial

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

Name

C3391008

Description

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

Trial arms

Trial start

2022-08-08

Estimated PCD

2024-12-27

Trial end

2029-01-03

Status

Active (not recruiting)

Phase

Early phase I

Treatment

PF-06939926

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

Arms:

PF-06939926

Other names:

Fordadistrogene Movaparvovec

Size

Primary endpoint

Incidence and severity of Treatment-Emergent Adverse Events and Serious Adverse Events

Through Week 52

Number of participants with abnormal hematology test results

Through Week 52

Number of participants with abnormal biochemistry test results

Through Week 52

Number of participants with abnormal urine analysis

Through Week 52

Number of participants with abnormal and clinically relevant changes in neurological examinations

Through Week 52

Number of participants with abnormal and clinically relevant changes in body weight

Through Week 52

Number of participants with abnormal and clinically relevant changes in vital signs

Through Week 52

Number of participants with abnormal and clinically relevant changes on cardiac troponin I

Through Week 52

Number of participants with abnormal and clinically relevant changes on electrocardiogram (ECG)

Through Week 52

Number of participants with abnormal and clinically relevant changes on echocardiogram

Through Week 52

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.

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 10, 'type': 'ACTUAL'}}

Updated at

2024-04-04

1 organization

1 product

2 indications

Organization

Product

Indication

Indication