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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT02500381
Ethics application status
Date submitted
14/07/2015
Date registered
16/07/2015

Titles & IDs
Public title
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
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
2015-002069-52
Secondary ID [2] 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 - Participants amenable to exon 45 skipping will receive SRP-4045 IV infusions, weekly, at 30 mg/kg for up to 96 weeks in the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the study).

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

Placebo comparator: Placebo followed by SRP-4045 or SRP-4053 - Participants 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 the double-blinded period. This will be followed by an open-label extension period in which all participants will receive open-label active treatment of SRP-4045 or SRP-4053 at 30 mg/kg/week IV infusions for 48 weeks (up to Week 144 in the 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 4-Step Ascend Velocity at Week 96
Assessment method [1] 0 0
Timepoint [1] 0 0
Baseline, Week 96
Secondary outcome [1] 0 0
Change from Baseline in the Total Distance Walked During 6MWT at Week 96
Assessment method [1] 0 0
Timepoint [1] 0 0
Baseline, Week 96
Secondary outcome [2] 0 0
Change from Baseline in Rise from Floor Velocity at Week 96
Assessment method [2] 0 0
Timepoint [2] 0 0
Baseline, Week 96
Secondary outcome [3] 0 0
Change From Baseline in the 4-Step Ascend Velocity at Week 144
Assessment method [3] 0 0
Timepoint [3] 0 0
Baseline, Week 144
Secondary outcome [4] 0 0
Change From Baseline in Total Distance Walked During the 10-meter walk/run (10-MWR) Velocity
Assessment method [4] 0 0
Timepoint [4] 0 0
Baseline, Week 96
Secondary outcome [5] 0 0
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96
Assessment method [5] 0 0
The NSAA is a clinician administered scale that rates the participant's performance on various functional activities. During this assessment, participants will be asked to perform 17 different functional activities, including a 10 meter walk/run, rising from a sit to standing, standing on 1 leg, climbing a box step, descending a box step, rising from lying to sitting, rising from the floor, lifting the head, standing on heels, and jumping. Participants will be graded as follows: 2 = achieves goal without any assistance; 1 = modified method but achieves goal independent of physical assistance from another person; and 0 = unable to achieve goal independently. NSAA Total Score ranges from 0 to 34, with a score of 34 implying normal function.
Timepoint [5] 0 0
Baseline, Week 96
Secondary outcome [6] 0 0
Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
Assessment method [6] 0 0
Timepoint [6] 0 0
Baseline, Week 48 or Week 96
Secondary outcome [7] 0 0
Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Assessment method [7] 0 0
Timepoint [7] 0 0
Baseline, Week 48 or Week 96

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 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
Minimum age
6 Years
Maximum age
13 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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.

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
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/09/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
17/10/2025
Actual
Sample size
Target
Accrual to date
Final
228
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Royal Children's Hospital Melbourne - Parkville
Recruitment hospital [2] 0 0
Queensland Children's Hospital - South Brisbane
Recruitment hospital [3] 0 0
Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 0 0
4101 - South Brisbane
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
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Georgia
Country [5] 0 0
United States of America
State/province [5] 0 0
Illinois
Country [6] 0 0
United States of America
State/province [6] 0 0
Iowa
Country [7] 0 0
United States of America
State/province [7] 0 0
Kansas
Country [8] 0 0
United States of America
State/province [8] 0 0
Massachusetts
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
Nevada
Country [11] 0 0
United States of America
State/province [11] 0 0
New York
Country [12] 0 0
United States of America
State/province [12] 0 0
Ohio
Country [13] 0 0
United States of America
State/province [13] 0 0
Oregon
Country [14] 0 0
United States of America
State/province [14] 0 0
Pennsylvania
Country [15] 0 0
United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Utah
Country [17] 0 0
United States of America
State/province [17] 0 0
Virginia
Country [18] 0 0
United States of America
State/province [18] 0 0
Wisconsin
Country [19] 0 0
Argentina
State/province [19] 0 0
Ciudad Autonoma de Buenos Aires
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
Denmark
State/province [29] 0 0
København Ø
Country [30] 0 0
France
State/province [30] 0 0
Haute-Garonne
Country [31] 0 0
France
State/province [31] 0 0
Nantes
Country [32] 0 0
France
State/province [32] 0 0
Paris
Country [33] 0 0
Germany
State/province [33] 0 0
Berlin
Country [34] 0 0
Germany
State/province [34] 0 0
Essen
Country [35] 0 0
Germany
State/province [35] 0 0
Freiburg
Country [36] 0 0
Greece
State/province [36] 0 0
Maroussi
Country [37] 0 0
Greece
State/province [37] 0 0
Thessaloniki
Country [38] 0 0
Hungary
State/province [38] 0 0
Budapest
Country [39] 0 0
India
State/province [39] 0 0
Gujarat
Country [40] 0 0
India
State/province [40] 0 0
Maharashtra
Country [41] 0 0
Ireland
State/province [41] 0 0
Dublin
Country [42] 0 0
Israel
State/province [42] 0 0
Petah Tikvah
Country [43] 0 0
Italy
State/province [43] 0 0
Ferrara
Country [44] 0 0
Italy
State/province [44] 0 0
Genoa
Country [45] 0 0
Italy
State/province [45] 0 0
Messina
Country [46] 0 0
Italy
State/province [46] 0 0
Milano
Country [47] 0 0
Italy
State/province [47] 0 0
Rome
Country [48] 0 0
Korea, Republic of
State/province [48] 0 0
Seoul
Country [49] 0 0
Korea, Republic of
State/province [49] 0 0
Yangsan
Country [50] 0 0
Mexico
State/province [50] 0 0
Culiacán
Country [51] 0 0
Mexico
State/province [51] 0 0
Durango
Country [52] 0 0
Poland
State/province [52] 0 0
Mazowieckie
Country [53] 0 0
Poland
State/province [53] 0 0
Gdansk
Country [54] 0 0
Russian Federation
State/province [54] 0 0
Moscow
Country [55] 0 0
Russian Federation
State/province [55] 0 0
Yekaterinburg
Country [56] 0 0
Serbia
State/province [56] 0 0
Belgrade
Country [57] 0 0
Spain
State/province [57] 0 0
Barcelona
Country [58] 0 0
Spain
State/province [58] 0 0
Valencia
Country [59] 0 0
Sweden
State/province [59] 0 0
Göteborg
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Glasgow
Country [61] 0 0
United Kingdom
State/province [61] 0 0
Leeds
Country [62] 0 0
United Kingdom
State/province [62] 0 0
Liverpool
Country [63] 0 0
United Kingdom
State/province [63] 0 0
London
Country [64] 0 0
United Kingdom
State/province [64] 0 0
Newcastle upon Tyne
Country [65] 0 0
United Kingdom
State/province [65] 0 0
Oxford

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT02500381