Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 1 April 2024
• Main ID: NCT02500381
• Date of registration: 14/07/2015
• Prospective Registration: Yes
• Primary sponsor: Sarepta Therapeutics, Inc.
• Public title: Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD) ESSENCE
• Scientific title: 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: September 28, 2016
• Target sample size: 228
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT02500381
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Argentina, Australia, Belgium, Bulgaria, Canada, Czechia, Denmark, France, Germany, Greece, Hungary, India, Ireland, Israel, Italy, Korea, Republic of, Mexico, Poland, Russian Federation, Serbia, Spain, Sweden, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Sarepta Therapeutics, Inc.

Key inclusion & exclusion criteria

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 or 2 alternative upper 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

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: SRP-4045

Drug: Placebo

Drug: SRP-4053

Primary Outcome(s)

Change From Baseline in the Total Distance Walked During 6MWT at Week 96 [Time Frame: Baseline, Week 96]

Secondary Outcome(s)

Participant's Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore [Time Frame: Week 96, Week 144]

Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96 [Time Frame: Baseline, Week 48 or Week 96]

Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period) [Time Frame: Baseline, Week 144]

Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144 [Time Frame: Baseline, Week 96 and Week 144]

Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96 [Time Frame: Baseline, Week 48 or Week 96]

Change From Baseline in the NSAA Total Score at Week 96 and Week 144 [Time Frame: Baseline, Week 96 and Week 144]

Time to Loss of Ambulation (LOA) [Time Frame: Baseline, Week 96, and Week 144]

Secondary ID(s)

4045-301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors