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

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy - ESSENCE

Status

Recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2022/09/045929

Enrollment

222

Registered

2022-09-27

Start date

Unknown

Completion date

Unknown

Last updated

2023-01-09

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Health Condition 1: G710- Muscular dystrophy

Interventions

Intervention1: SRP-4053 Drug: SRP-4053 SRP-4053 solution for IV infusion Other Names: Golodirsen VYONDYS 53: Participants amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30

Eligibility

Inclusion criteria

Inclusion criteria: 1. Genotypically confirmed DMD, with genetic deletion amenable to exon 45 or exon 53 skipping 2. 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). 3. Intact right and left biceps or 2 alternative upper muscle groups 4. Mean 6MWT >=300 meters and 5. Stable pulmonary function: forced vital capacity (FVC) >=50% predicted

Exclusion criteria

Exclusion criteria: 1.Treatment with gene therapy at any time 2. 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 3. Current or previous treatment with any other experimental treatment within 12 weeks prior to Week 1 4. Major surgery within 3 months prior to Week 1 5. Presence of other clinically significant illness 6. Other inclusion/exclusion criteria may apply.

Design outcomes

Primary

Measure	Time frame
The primary objective of this study is to evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared with placebo on ambulation, endurance, and muscle function as measured by the 6-minute walk test (6MWT).Timepoint: Change from Baseline at Week 96 in 6MWT	—

Secondary

Measure	Time frame
1. Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period) 2. Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96 3. Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96 4. Participants Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore 5. The Time to Loss of Ambulation (LOA) 6. Change From Baseline in the NSAA Total Score at Week 96 and Week 144 7. Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144Timepoint: 1) Change from Baseline at week 144 ( week 48 of the open label extension period) 2) Change from Baseline at week 48 or 96 3) Change from Baseline at week 48 or 96 4) Week 96, Week 144 5) Baseline, Week 96, and Week 144 6) Change from Baseline at week 96 and 144 7) Change from Baseline at week 96 and 144	—

Measure

Time frame

1. Change from Baseline in the Total Distance Walked During 6MWT at Week 144 (Week 48 of the Open-Label Extension Period) 2. Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96 3. Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96 4. Participants Ability to Rise Independently From the Floor, as indicated by a North Star Ambulatory Assessment (NSAA) Subscore 5. The Time to Loss of Ambulation (LOA) 6. Change From Baseline in the NSAA Total Score at Week 96 and Week 144 7. Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144Timepoint: 1) Change from Baseline at week 144 ( week 48 of the open label extension period) 2) Change from Baseline at week 48 or 96 3) Change from Baseline at week 48 or 96 4) Week 96, Week 144 5) Baseline, Week 96, and Week 144 6) Change from Baseline at week 96 and 144 7) Change from Baseline at week 96 and 144

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Countries

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

Contacts

Public ContactRashmi Chitgupi

PPD Pharmaceutical development India Private Limited

rashmi.chitgupi@ppd.com912266022900

Outcome results

None listed

Source: CTRI (via WHO ICTRP) · Data processed: Feb 4, 2026