Duchenne Muscular DystrophyPhase 3ESSENCENCT02500381
Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 3 study testing SRP-4045 and SRP-4053 in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 76 sites in 24 countries.
Trial summary
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively. Read more about this study
- Who is it for?
- Ages 6 to 13 years, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- SRP-4045 and SRP-4053
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 228 participants
- Where
- 76 sitesin 24 countries
- Study dates
- 28 Sep 2016to 16 Oct 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 13 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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.
Trial locations and contacts
76 sites in 24 countries.
76 sites in 24 countries
United States23 sites
Center for Integrative Rare Disease Research (CIRDR)
Atlanta, Georgia, 30318
Boston Children's Hospital
Boston, Massachusetts, 02115
Ann and Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center (CCHMC)
Cincinnati, Ohio, 45229
Nationwide Children's Hospital
Columbus, Ohio, 43205
Children's Medical Center Dallas
Dallas, Texas, 75235
University of Florida
Gainesville, Florida, 32610
NW Florida Clinical Research Group, LLC
Gulf Breeze, Florida, 32561
University of Iowa Children's Hospital
Iowa City, Iowa, 52242
University of Kansas, Medical Center
Kansas City, Kansas, 66160
Las Vegas Clinic
Las Vegas, Nevada, 89145
Children's Hospital Los Angeles
Los Angeles, California, 90027
David Geffen School of Medicine, UCLA
Los Angeles, California, 90095
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospital of the King's Daughters
Norfolk, Virginia, 23507
Neuromuscular Research Center
Phoenix, Arizona, 85028
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Shriners Hospital for Children
Portland, Oregon, 97239
University of Rochester Clinical Research Center
Rochester, New York, 14642
University of Utah
Salt Lake City, Utah, 84132
Rady Children's Hospital San Diego/ UCSD
San Diego, California, 92123
St. Louis Children's Hospital
St Louis, Missouri, 63110
Stanford University School of Medicine/Medical Center
Stanford, California, 94305
United Kingdom6 sites
Royal Hospital for Children (Glasgow)
Glasgow, G51 4TF
Leeds Teaching Hospitals NHS Trust
Leeds, LS1 3EX
Alder Hey Childrens Hospital
Liverpool, L12 2AP
Great Ormond Street Hospital (GOSH)
London, WC1N 1EH
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
John Radcliffe Hospital
Oxford, OX3 9DU
Italy5 sites
Azienda Ospedaliero-Universitaria di Ferrara - Arcispedale Sant' Anna
Ferrara, 44124
Istituto Giannina Gaslini
Genoa, 16147
Az Ospedaliera Universitaria Policlinico G Martino
Messina, 98125
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milan, 20133
Policlinico Universitario A Gemelli
Rome, 00168
Canada4 sites
Alberta Childrens Hospital
Calgary, Alberta, T3B 6A8
London Health Sciences Centre
London, Ontario, N6A 5W9
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Children's and Women's Health Centre of British Columbia
Vancouver, British Columbia, V6H 3V4
Germany4 sites
Charité - Universitätsmedizin Berlin
Berlin, 13353
Universitätsklinikum Essen
Essen, 45122
University Hospital Freiburg
Freiburg im Breisgau, 79106
LMU Klinikum der Universität
München, Bavaria, 80337
Australia3 sites
Royal Children's Hospital Melbourne
Parkville, Victoria, 3052
Queensland Children's Hospital
South Brisbane, 4101
Children's Hospital at Westmead
Westmead, 2145
Belgium3 sites
Universitair Ziekenhuis Gent
Ghent, 9000
Universitair Ziekenhuis Leuven
Leuven, 3000
CHR de la Citadelle
Liège, 4000
France3 sites
Reference Centre for Neuromuscular Diseases
Nantes, 44093
Hôpital Armand Trousseau
Paris, 75012
Hôpital Des Enfants
Toulouse, Haute-Garonne, 31059
Spain3 sites
Hospital de La Santa Creu i Sant Pau
Barcelona, 08041
Hospital Sant Joan de Deu
Barcelona, 08950
Hospital Universitari i Politecnic La Fe de Valencia
Valencia
Czechia2 sites
University Hospital Brno
Brno, 61300
Fakultni nemocnice v Motole
Prague, 15008
Greece2 sites
IASO Children's Hospital
Marousi, 15123
Ippokratio General Hospital of Thessaloniki
Thessaloniki, 54642
India2 sites
Royal Instituite of Child Neurosciences
Ahmedabad, Gujarat, 380054
Deenanth Mangeshkar Hospital
Pune, Maharashtra, 411004
Mexico2 sites
Neurociencias Estudios Clínicos S.C
Culiacán, 80020
Instituto de Investigaciones Clínicas para la Salud A.C
Durango, 34000
Poland2 sites
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
Samodzielny Publiczny Centralny Szpital Kliniczny
Warsaw, Masovian Voivodeship, 02-097
Russia2 sites
Federal state budget educational institution of higher education "Russian national research medical university n.a. N.I. Pirogov" of Ministry of healthcare of Russian Federation
Moscow, 125412
State Autonomous Healthcare Institution of Sverdlovsk Region Children's Clinical Hospital No. 9 City of Ekaterinburg
Yekaterinburg
South Korea2 sites
Seoul National University Hospital
Seoul, 03080
Pusan National University Yangsan Hospital
Yangsan, 50612
Bulgaria1 site
University Multiprofile Hospital for Active Treatment Aleksandrovska EAD
Sofia, Sofia-Grad, 1431
Hungary1 site
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
Budapest, 1083
Serbia1 site
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade, 11000
About this study
Official 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
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Read the detailed description
This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible participants with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 milligrams/kilograms (mg/kg) SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group) or placebo for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open-label extension period in which all participants will receive open-label active treatment for 48 weeks (up to Week 144 of study).
The study will enroll approximately 222 participants. Twice as many participants will be randomized to receive active treatment as will receive placebo (2:1 randomization).
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests, such as the 6-minute walk test (6MWT). All participants will undergo a muscle biopsy at baseline and a second muscle biopsy either at Week 48 or Week 96.
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of both drugs.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- SRP-4045, SRP-4053, Placebo
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Ambulatory, Pediatric, Duchenne
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SRP-4045Drug
Also called Casimersen, AMONDYS 45
SRP-4045 solution for IV infusion
SRP-4053Drug
Also called Golodirsen, VYONDYS 53
SRP-4053 solution for IV infusion
PlaceboDrug
SRP-4045 or SRP-4053 placebo-matching solution for IV infusion
| Study group | Role | What they receive |
|---|---|---|
| SRP-4045 | Experimental | 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). Receives: Drug: SRP-4045 |
| SRP-4053 | Experimental | 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). Receives: Drug: SRP-4053 |
| Placebo followed by SRP-4045 or SRP-4053 | Placebo comparator | 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). Receives: Drug: SRP-4045; Drug: SRP-4053; Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change From Baseline in the 4-Step Ascend Velocity at Week 96
Measured: Baseline, Week 96
Secondary outcomes (7)
Change from Baseline in the Total Distance Walked During 6MWT at Week 96
Measured: Baseline, Week 96
Change from Baseline in Rise from Floor Velocity at Week 96
Measured: Baseline, Week 96
Change From Baseline in the 4-Step Ascend Velocity at Week 144
Measured: Baseline, Week 144
Change From Baseline in Total Distance Walked During the 10-meter walk/run (10-MWR) Velocity
Measured: Baseline, Week 96
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96
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.
Measured: Baseline, Week 96
Change from Baseline in Dystrophin Protein Levels Determined by Western Blot at Weeks 48 or 96
Measured: Baseline, Week 48 or Week 96
Change from Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Measured: Baseline, Week 48 or Week 96
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Jul 2015 (estimated)
- Study start28 Sep 2016
- Primary completion12 Nov 2024
- Study completion16 Oct 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Sarepta Therapeutics, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 228 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sarepta Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Sarepta Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02500381
- Sponsor's protocol ID
- 4045-301
- Other IDs
- 2015-002069-52, 2024-514698-23-00, 2015
- Acronym
- ESSENCE
- Status last verified
- Nov 2025
- First submitted
- 14 Jul 2015
- Last updated
- 18 Nov 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Vandekerckhove I, Hanssen B, Peeters N, Dewit T, De Beukelaer N, Van den Hauwe M, De Waele L, Van Campenhout A, De Groote F, Desloovere K. Anthropometric-related percentile curves for muscle size and strength of lower limb muscles of typically developing children. J Anat. 2025 Aug;247(2):348-362. doi: 10.1111/joa.14241. Epub 2025 Mar 17.
PubMed 40098309Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.
PubMed 34105177
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT02500381
- Acronym / sponsor ID
- ESSENCE / 4045-301
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, DMD, Exon 53, Exon 45, Ambulatory, Pediatric, Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 228 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Sep 2016 (actual)
- Primary completion
- 12 Nov 2024 (actual)
- Study completion
- 16 Oct 2025 (actual)
- First posted
- 16 Jul 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.
Detailed description
This is a double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of SRP-4045 and SRP-4053. Eligible participants with out-of-frame deletion mutations amenable to exon 45 or 53 skipping will be randomized to receive once weekly intravenous (IV) infusions of 30 milligrams/kilograms (mg/kg) SRP-4045 or 30 mg/kg SRP-4053 respectively (combined-active group) or placebo for up to 96 weeks (the placebo-controlled period of the trial). This will be followed by an open-label extension period in which all participants will receive open-label active treatment for 48 weeks (up to Week 144 of study).
The study will enroll approximately 222 participants. Twice as many participants will be randomized to receive active treatment as will receive placebo (2:1 randomization).
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests, such as the 6-minute walk test (6MWT). All participants will undergo a muscle biopsy at baseline and a second muscle biopsy either at Week 48 or Week 96.
Safety will be assessed through the collection of adverse events (AEs), laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.
Blood samples will be taken periodically throughout the study to assess the pharmacokinetics of both drugs.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| SRP-4045 | Experimental | Drug: SRP-4045Participants 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). |
| SRP-4053 | Experimental | Drug: SRP-4053Participants 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 followed by SRP-4045 or SRP-4053 | Placebo comparator | Drug: SRP-4045, Drug: SRP-4053, Drug: PlaceboParticipants 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). |
Who may qualify
- Ages
- 6 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (6 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SRP-4045Also: Casimersen, AMONDYS 45SRP-4045 solution for IV infusion | Drug | Placebo followed by SRP-4045 or SRP-4053, SRP-4045 |
| SRP-4053Also: Golodirsen, VYONDYS 53SRP-4053 solution for IV infusion | Drug | Placebo followed by SRP-4045 or SRP-4053, SRP-4053 |
| PlaceboSRP-4045 or SRP-4053 placebo-matching solution for IV infusion | Drug | Placebo followed by SRP-4045 or SRP-4053 |
Primary outcome
Change From Baseline in the 4-Step Ascend Velocity at Week 96
Time frame: Baseline, Week 96
7 secondary outcomes
Change from Baseline in the Total Distance Walked During 6MWT at Week 96
Time frame: Baseline, Week 96
Change from Baseline in Rise from Floor Velocity at Week 96
Time frame: Baseline, Week 96
Change From Baseline in the 4-Step Ascend Velocity at Week 144
Time frame: Baseline, Week 144
Change From Baseline in Total Distance Walked During the 10-meter walk/run (10-MWR) Velocity
Time frame: Baseline, Week 96
Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score at Week 96
Time frame: Baseline, Week 96
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 Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Weeks 48 or 96
Time frame: Baseline, Week 48 or Week 96
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2025.
- Derived from this study: PubMed 40098309Vandekerckhove I, Hanssen B, Peeters N, Dewit T, De Beukelaer N, Van den Hauwe M, De Waele L, Van Campenhout A, De Groote F, Desloovere K. Anthropometric-related percentile curves for muscle size and strength of lower limb muscles of typically developing children. J Anat. 2025 Aug;247(2):348-362. doi: 10.1111/joa.14241. Epub 2025 Mar 17.
- Derived from this study: PubMed 34105177Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Sarepta Therapeutics, Inc.
76 sites in 24 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing SRP-4045 and SRP-4053 in people with Duchenne Muscular Dystrophy, run by Sarepta Therapeutics, Inc. at 76 sites in 24 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 76 sites in United States, United Kingdom, Italy, Canada, Germany and 19 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in the 4-Step Ascend Velocity at Week 96 (time frame: Baseline, Week 96). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02500381, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2025.