Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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6 January 2025 |
Main ID: |
NCT02500381 |
Date of registration:
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14/07/2015 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
ESSENCE |
Scientific title:
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A Double-Blind, Placebo-Controlled, Multi-Center Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy |
Date of first enrolment:
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September 28, 2016 |
Target sample size:
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228 |
Recruitment status: |
Active, not recruiting |
URL:
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https://clinicaltrials.gov/ct2/show/NCT02500381 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
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Phase:
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Phase 3
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Countries of recruitment
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Argentina
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Australia
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Belgium
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Bulgaria
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Canada
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Czechia
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Denmark
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France
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Germany
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Greece
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Hungary
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India
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Ireland
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Israel
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Italy
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Korea, Republic of
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Mexico
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Poland
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Russian Federation
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Serbia
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Spain
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Sweden
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United Kingdom
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United States
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Contacts
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Name:
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Medical Director |
Address:
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Telephone:
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Email:
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Affiliation:
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Sarepta Therapeutics, Inc. |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53
skipping
- Stable dose of oral corticosteroids for at least 24 weeks prior to Week 1, and the
dose is expected to remain constant throughout the study (except for modifications
to accommodate changes in weight).
- Intact right and left biceps brachii muscles or 2 alternative upper arm muscle
groups
- Mean 6MWT =300 meters and =450 meters
- Stable pulmonary function: forced vital capacity (FVC) =50% predicted
Exclusion Criteria:
- Treatment with gene therapy at any time
- Previous treatment with SMT C1100 within 1 week prior to Week 1 and previous
treatment with PRO045 (BMN 045), PRO053 (BMN 053), or PRO051 (BMN 051) within 24
weeks prior to Week 1
- Current or previous treatment with any other experimental treatment within 12 weeks
prior to Week 1
- Major surgery within 3 months prior to Week 1
- Presence of other clinically significant illness
- Other inclusion/exclusion criteria may apply.
Age minimum:
6 Years
Age maximum:
13 Years
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Duchenne Muscular Dystrophy
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Intervention(s)
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Drug: SRP-4045
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Drug: SRP-4053
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Drug: Placebo
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Primary Outcome(s)
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Change From Baseline in the 4-Step Ascend Velocity at Week 96
[Time Frame: Baseline, Week 96]
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Secondary Outcome(s)
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Change from Baseline in Rise from Floor Velocity at Week 96
[Time Frame: Baseline, Week 96]
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Change From Baseline in Total Distance Walked During the 10-meter walk/run (10-MWR) Velocity
[Time Frame: Baseline, Week 96]
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Change From Baseline in the 4-Step Ascend Velocity at Week 144
[Time Frame: Baseline, Week 144]
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Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
[Time Frame: Baseline, Week 48 or Week 96]
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Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
[Time Frame: Baseline, Week 48 or Week 96]
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Change from Baseline in the Total Distance Walked During 6MWT at Week 96
[Time Frame: Baseline, Week 96]
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Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96
[Time Frame: Baseline, Week 96]
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Secondary ID(s)
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2024-514698-23-00
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4045-301
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2015-002069-52
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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