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Clinical trial

A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications.

ID
Name
SRPT-Dup-US-001
Description
This is an 48-week open-label study to determine the efficacy and safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 for the treatment of boys with duchenne muscular dystrophy who have a single exon duplication of either exon 45, 51 or 53, respectively. There will be weekly infusions and two muscle biopsies at baseline and at month 12.
Trial arms
Trial start
2020-02-18
Estimated PCD
2021-09-01
Trial end
2023-09-01
Status
Completed
Phase
Early phase I
Treatment
Amondys 45
This drug is used to target skipping of exon 45 of the dystrophin gene.
Arms:
AMONDYS 45
Other names:
SRP-4045, Casimersen
Exondys 51
This drug is used to target skipping of exon 51 of the dystrophin gene.
Arms:
EXONDYS 51
Other names:
Eteplirsen, AVI-4658
Vyondys 53
This drug is used to target skipping of exon 53 of the dystrophin gene.
Arms:
VYONDYS 53
Other names:
SRP-4053, Golodirsen
Size
3
Primary endpoint
Change in Dystrophin Expression From Baseline Following Treatment With Either AMONDYS 45 (Previously Casimersen), EXONDYS 51 (Previously Eteplirsen ), or VYONDYS 53 (Previously Golodirsen)
Baseline, 1 year
Monitoring for the Development of Unacceptable Toxicity.
1 year
Eligibility criteria
Inclusion Criteria: * Is a male with DMD and has an out-of-frame duplication of either exon 45, 51, or 53, with a normal copy number of all other DMD exons. * Is above age 6 months of age. * Has sufficient muscle mass in a pair of bilateral muscles that will allow for pre- and post-treatment muscle biopsies per PI discretion. * If the subject is ambulant and 4 years old or greater and has been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to Week 1 the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the study. Exclusion Criteria: * Any additional missing exon for DMD that cannot be treated with study drugs. Other inclusion/exclusion criteria apply
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 3, 'type': 'ACTUAL'}}
Updated at
2023-10-26

1 organization

3 products

1 indication

Organization
Kevin Flanigan
Product
Amondys
Indication
Duchenne muscular dystrophy
Product
Exondys 51
Product
Vyondys 53