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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02500381




Registration number
NCT02500381
Ethics application status
Date submitted
14/07/2015
Date registered
16/07/2015
Date last updated
8/07/2020

Titles & IDs
Public title
Study of SRP-4045 and SRP-4053 in DMD Patients
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
Secondary ID [1] 0 0
4045-301
Universal Trial Number (UTN)
Trial acronym
ESSENCE
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SRP-4045
Treatment: Drugs - SRP-4053
Treatment: Drugs - Placebo

Experimental: SRP-4045 - Patients amenable to exon 45 skipping will receive SRP-4045 intravenous (IV) infusions, weekly, at 30 mg/kg for up to 96 weeks in double-blinded period. This will be followed by an open label extension period in which all patients will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks in OL period (up to Week 144 in study).

Experimental: SRP-4053 - Patients amenable to exon 53 skipping will receive SRP-4053 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in double-blinded period. This will be followed by an open label extension period in which all patients will receive open-label active treatment of SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks in OL period (up to Week 144 in study).

Placebo Comparator: Placebo followed by SRP-4045 or SRP-4053 - Patients amenable to exon 45 or 53 skipping will receive SRP-4045 or SRP-4053 placebo-matching IV infusions, weekly, at 30 mg/kg for up to 96 weeks in double-blinded period. This will be followed by an open label extension period in which all patients will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV for 48 weeks in OL period (up to Week 144 in study).


Treatment: Drugs: SRP-4045
SRP-4045 solution for IV infusion

Treatment: Drugs: SRP-4053
SRP-4053 solution for IV infusion

Treatment: Drugs: Placebo
SRP-4045 or SRP-4053 placebo-matching solution for IV infusion

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 96
Timepoint [1] 0 0
Baseline and Week 96
Secondary outcome [1] 0 0
Change from Baseline the Total Distance Walked During 6-Minute Walk Test (6MWT) at Week 144 (Week 48 of the OL period)
Timepoint [1] 0 0
Baseline, Week 144
Secondary outcome [2] 0 0
Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
Timepoint [2] 0 0
Baseline and Weeks 48 or 96
Secondary outcome [3] 0 0
Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Timepoint [3] 0 0
Baseline and Weeks 48 or 96
Secondary outcome [4] 0 0
Ability to Rise Independently From the Floor
Timepoint [4] 0 0
Week 96, Week 144
Secondary outcome [5] 0 0
Time to Loss of Ambulation (LOA)
Timepoint [5] 0 0
Baseline, Week 96, Week 144
Secondary outcome [6] 0 0
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96 and Week 144 - The NSAA is a functional scale devised from the Hammersmith Scale of Motor Ability specifically for use in ambulant children with Duchenne muscular dystrophy (DMD). It consists of 17 activities graded 0 (unable to perform), 1 (performs with modifications), 2 (normal movement). The scale assesses activities required to remain functionally ambulant (example, rise from the floor), activities that can be difficult even early in the disease (e.g. standing on heels) and activities that are known to progressively deteriorate over time (stand from a chair, walk). NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Timepoint [6] 0 0
Baseline, Week 96, Week 144
Secondary outcome [7] 0 0
Change From Baseline in Forced Vital Capacity Percent (FVC%) Predicted at Week 96 and Week 144
Timepoint [7] 0 0
Baseline, Week 96, Week 144

Eligibility
Key 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

- Intact right and left biceps or 2 alternative upper muscle groups

- Mean 6MWT greater than or equal 300 meters and less than or equal to 450 meters

- Stable pulmonary function: forced vital capacity (FVC) equal to or greater than 50%
predicted
Minimum age
7 Years
Maximum age
13 Years
Gender
Males
Can healthy volunteers participate?
No
Key exclusion criteria
- Treatment with gene therapy at any time

- Previous treatment with SMT C1100, 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 (other than
deflazacort) 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Queensland Children's Hospital - Brisbane
Recruitment hospital [2] 0 0
Royal Children's Hospital Melbourne - Parkville
Recruitment hospital [3] 0 0
The Royal Children Hospital - Parkville
Recruitment hospital [4] 0 0
Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
4101 - Brisbane
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment postcode(s) [3] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Georgia
Country [6] 0 0
United States of America
State/province [6] 0 0
Illinois
Country [7] 0 0
United States of America
State/province [7] 0 0
Iowa
Country [8] 0 0
United States of America
State/province [8] 0 0
Kansas
Country [9] 0 0
United States of America
State/province [9] 0 0
Massachusetts
Country [10] 0 0
United States of America
State/province [10] 0 0
Missouri
Country [11] 0 0
United States of America
State/province [11] 0 0
Nevada
Country [12] 0 0
United States of America
State/province [12] 0 0
New York
Country [13] 0 0
United States of America
State/province [13] 0 0
Ohio
Country [14] 0 0
United States of America
State/province [14] 0 0
Oregon
Country [15] 0 0
United States of America
State/province [15] 0 0
Pennsylvania
Country [16] 0 0
United States of America
State/province [16] 0 0
Texas
Country [17] 0 0
United States of America
State/province [17] 0 0
Utah
Country [18] 0 0
United States of America
State/province [18] 0 0
Virginia
Country [19] 0 0
United States of America
State/province [19] 0 0
Wisconsin
Country [20] 0 0
Belgium
State/province [20] 0 0
Ghent
Country [21] 0 0
Belgium
State/province [21] 0 0
Leuven
Country [22] 0 0
Belgium
State/province [22] 0 0
Liège
Country [23] 0 0
Bulgaria
State/province [23] 0 0
Sofia-Grad
Country [24] 0 0
Canada
State/province [24] 0 0
Alberta
Country [25] 0 0
Canada
State/province [25] 0 0
British Columbia
Country [26] 0 0
Canada
State/province [26] 0 0
Ontario
Country [27] 0 0
Czechia
State/province [27] 0 0
Brno
Country [28] 0 0
Czechia
State/province [28] 0 0
Praha
Country [29] 0 0
France
State/province [29] 0 0
Haute-Garonne
Country [30] 0 0
France
State/province [30] 0 0
Nantes
Country [31] 0 0
France
State/province [31] 0 0
Paris
Country [32] 0 0
Germany
State/province [32] 0 0
Essen
Country [33] 0 0
Germany
State/province [33] 0 0
Freiburg
Country [34] 0 0
Germany
State/province [34] 0 0
Munich
Country [35] 0 0
Greece
State/province [35] 0 0
Maroussi
Country [36] 0 0
Hungary
State/province [36] 0 0
Budapest
Country [37] 0 0
Israel
State/province [37] 0 0
Petah Tikvah
Country [38] 0 0
Italy
State/province [38] 0 0
Ferrara
Country [39] 0 0
Italy
State/province [39] 0 0
Genoa
Country [40] 0 0
Italy
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Messina
Country [41] 0 0
Italy
State/province [41] 0 0
Rome
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Poland
State/province [42] 0 0
Mazowieckie
Country [43] 0 0
Spain
State/province [43] 0 0
Barcelona
Country [44] 0 0
Spain
State/province [44] 0 0
Valencia
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Sweden
State/province [45] 0 0
Göteborg
Country [46] 0 0
Sweden
State/province [46] 0 0
Stockholm
Country [47] 0 0
United Kingdom
State/province [47] 0 0
Glasgow
Country [48] 0 0
United Kingdom
State/province [48] 0 0
Leeds
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United Kingdom
State/province [49] 0 0
Liverpool
Country [50] 0 0
United Kingdom
State/province [50] 0 0
London
Country [51] 0 0
United Kingdom
State/province [51] 0 0
Newcastle upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Sarepta Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The main objective of this study is to evaluate the efficacy of SRP-4045 and SRP-4053
compared to placebo in Duchenne muscular dystrophy (DMD) patients with out-of-frame deletion
mutations amenable to skipping exon 45 and exon 53, respectively.
Trial website
https://clinicaltrials.gov/show/NCT02500381
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Sarepta Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Medical Information
Address 0 0
Country 0 0
Phone 0 0
+1 800-690-2003
Fax 0 0
Email 0 0
clinicaltrials@sarepta.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02500381