Muscular Dystrophy, DuchennePhase 3MIS51ONNCT03992430
A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
A Phase 3 study testing Eteplirsen in people with Muscular Dystrophy, Duchenne, run by Sarepta Therapeutics, Inc. at 59 sites in 26 countries.
Trial summary
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of… Read more about this study
- Who is it for?
- Ages 4 to 13 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Eteplirsen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 59 sitesin 26 countries
- Study dates
- 13 Jul 2020to 31 Oct 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 4 to 13 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
- Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
- Able to walk independently without assistive devices.
- Have intact right and left biceps muscles or an alternative upper arm muscle group.
- Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.
- For ages 7 years and older, has stable pulmonary function (forced vital capacity ≥50 percent (%) of predicted and no requirement for nocturnal ventilation). For ages 4 to 6 years, does not require support from ventilator or non-invasive ventilation at time of screening.
Who cannot join
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
- Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; Eteplirsen (previous or current use); and Tamoxifen in the last 4 weeks prior to first dose.
- Major surgery within 3 months prior to randomization.
- Presence of any other significant neuromuscular or genetic disease other than DMD.
- Presence of any known impairment of renal function and/or other clinically significant illness.
- Has evidence of cardiomyopathy, as defined by left ventricular ejection fraction less than <50% on the screening echocardiogram or Fridericia's correction formula (QTcF) ≥450 millisecond based on the screening electrocardiograms (ECGs).
- Other inclusion/exclusion criteria apply.
Trial locations and contacts
59 sites in 26 countries.
59 sites in 26 countries
India8 sites
Royal Institute of Child Neurosciences
Ahmedabad, 380054
Aster RV Hospital
Bengaluru, 560078
Nizam's Institute of Medical Sciences
Hyderabad, 500082
Jaicare Hospital (A Unit of Sarvee Integra Pvt Ltd.)
Madurai, 626022
All India Institute of Medical Sciences
New Delhi, 110029
Sir Ganga Ram Hospital
New Delhi, 110060
Deenanath Mangeshkar Hospital & Research Centre
Pune, 411004
Christian Medical College
Vellore, 632004
Jordan4 sites
The Specialty Hospital (TSH)/Advanced Clinical Center
Amman, 11194
Istiklal Hosptial (IST)
Amman, 11196
Irbid Specialty Hospital
Irbid, 22110
Pharmaceutical Research Center/Jordan University of Science and Technology
Irbid, 22110
South Korea4 sites
Kyungpook National University Chilgok Hospital
Daegu, 41404
Seoul National University Hospital
Seoul, 03080
Samsung Medical Center
Seoul, 6351
Pusan National University Yangsan Hospital
Yangsan, 50612
United States3 sites
Rare Disease Research, LLC
Atlanta, Georgia, 30318
University of Alabama at Birmingham
Birmingham, Alabama, 35233
University of Florida
Gainesville, Florida, 32610-3010
Colombia3 sites
Hospital Universitario San Ignacio
Bogotá, 110231
Instituto Neurologico de Colombia (INDEC)
Medellín, 50012
Hospital Pablo Tobón Uribe
Medellín, 50034
France3 sites
Hopital Femme Mere Enfant
Bron, 69677
Hopital Armand Trousseau
Paris, 75571
CHRU de Strasbourg
Strasbourg, 67098
Germany3 sites
Charité Universitätsmedizin Berlin CVK
Berlin, 13353
Universitätsklinikum Essen
Essen, 45147
Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106
Serbia3 sites
Clinic for Neurology and Psychiatry for Children and Youth
Belgrade, 11000
University Children's Hospital
Belgrade, 11000
Mother and Child Health Care Institute of Serbia "Dr Vukan Cupic"
Belgrade, 190133
United Kingdom3 sites
Birmingham Heartlands Hospital
Birmingham, B9 5SS
Leeds Teaching Hospitals NHS Trust
Leeds, West Yorkshire, LS1 3EX
UCL Institute of Child Health Great Ormond Street
London, WC1N 1EH
Czechia2 sites
Brno Klinika detske neurologie
Brno, 613 00
Fakultni nemocnice v Motole
Prague, 150 06
Italy2 sites
IRCCS Instituto Gianna Gaslini
Genova, 16147
Fondazione Policlinico Universitario A. Gemelli- IRCCS
Rome, 00168
Mexico2 sites
Neurociencias Estudios Clínicos S.C.
Culiacán, Sinaloa, 80020
Instituto de Investigaciones Clinicas para la Salud A.C
Durango, 34000
Netherlands2 sites
Leids Universitair Medisch Centrum
Leiden, 2333 ZC
Radboud University Nijmegen Medical Centre
Nijmegen, 6525GA
Norway2 sites
Oslo Universitetssykehus HF Rikshospitalet
Oslo, 0450
Children's Department and Department for Children's Habilitation at Stavanger University Hospital
Stavanger, 4011
Spain2 sites
Hospital Sant Joan de Deu
Barcelona, 8950
Hospital Universitari i Politecnic La Fe de Valencia
Valencia, 46026
Taiwan2 sites
Kaohsiung Medical University
Kaohsiung City, 80756
National Taiwan University Hospital
Taipei, 10071
Turkey (Türkiye)2 sites
Akdeniz Universitesi Tip Fakultesi
Antalya, 07059
Mersin University Medical Faculty
Mersin, 33110
Hungary1 site
Semmelweis Egyetem Genomikai Medicina és Ritka Betegsegek Intezete
Budapest, 1082
Ireland1 site
Children's Health Ireland (CHI) at Temple Street
Dublin, D01 XD99
Poland1 site
Klinika Neurologii Rozwojowej
Gdansk, Pomeranian Voivodeship, 80-211
Romania1 site
National Clinical Hospital for Children Neurorehabilitation "Dr. Nicolae Robănescu"
Bucharest, 41408
About this study
Official title: A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Eteplirsen
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, Exon 51, North Star Ambulatory Assessment, Ambulatory, DMD, Pediatric, Duchenne, EXONDYS, MIS51ON
- 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.
EteplirsenDrug
Also called AVI-4658, EXONDYS 51, EXONDYS
Solution for intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Part 1: Eteplirsen | Experimental | Participants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks. Receives: Drug: Eteplirsen |
| Part 2: Eteplirsen 30 mg/kg | Active comparator | Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks. Receives: Drug: Eteplirsen |
| Part 2: Eteplirsen 100 mg/kg | Experimental | Randomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks. Receives: Drug: Eteplirsen |
| Part 2: Eteplirsen 200 mg/kg | Experimental | Randomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks. Receives: Drug: Eteplirsen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Incidence of Adverse Events (AEs)
Measured: Up to Week 148
Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis)
Measured: Baseline, Week 144
Part 2: Change from Baseline at Week 72 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis)
Measured: Baseline, Week 72 or Week 96
Secondary outcomes (7)
Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Measured: Baseline, Week 144
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Measured: Baseline, Week 144
Part 2: Change from Baseline at Week 144 in Forced Vital Capacity Percent Predicted (FVC%p)
Measured: Baseline, Week 144
Part 2: Time to Loss of Ambulation (LOA)
Measured: Baseline up to Week 144
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Measured: Baseline, Postdose (at Week 24, Week 48, or Week 144)
Part 2: Incidence of Adverse Events (AEs)
Measured: Baseline up to Week 148
Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen
Measured: 0 (predose) to 2 hours postdose up to Week 144
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jun 2019
- Study start13 Jul 2020
- Primary completion31 Oct 2026 (estimated)
- Study completion31 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, DuchenneStudies 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
- 160 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
- NCT03992430
- Sponsor's protocol ID
- 4658-402
- Other IDs
- 2018-001762-42, 2024-511492-15-00, 2018
- Acronym
- MIS51ON
- Status last verified
- Feb 2026
- First submitted
- 18 Jun 2019
- Last updated
- 27 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of High Doses of Eteplirsen, Preceded by an Open-label Dose Escalation, in Patients With Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
- ClinicalTrials.gov ID
- NCT03992430
- Acronym / sponsor ID
- MIS51ON / 4658-402
- Conditions
- Muscular Dystrophy, Duchenne
- Keywords
- Duchenne muscular dystrophy, Exon Skipping, Exon 51, North Star Ambulatory Assessment, Ambulatory, DMD, Pediatric, Duchenne
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 160 participants (actual)
- Lead sponsor
- Sarepta Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Jul 2020 (actual)
- Primary completion
- 31 Oct 2026 (estimated)
- Study completion
- 31 Oct 2026 (estimated)
- First posted
- 20 Jun 2019 (actual)
- 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.
Part 1 (dose escalation) will evaluate the safety and tolerability of 2 doses (100 milligrams/kilogram [mg/kg] and 200 mg/kg) of eteplirsen in approximately 10 participants with DMD; Part 2 (dose finding and dose comparison) will evaluate the efficacy and safety of the high doses (100 mg/kg and 200 mg/kg) of eteplirsen compared with that of the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part 1: Eteplirsen | Experimental | Drug: EteplirsenParticipants will receive eteplirsen 100 mg/kg once weekly for at least 4 weeks, followed by eteplirsen 200 mg/kg once weekly for at least 4 weeks. |
| Part 2: Eteplirsen 30 mg/kg | Active comparator | Drug: EteplirsenRandomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks. |
| Part 2: Eteplirsen 100 mg/kg | Experimental | Drug: EteplirsenRandomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks. |
| Part 2: Eteplirsen 200 mg/kg | Experimental | Drug: EteplirsenRandomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks. |
Who may qualify
- Ages
- 4 to 13 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- Be a male with an established clinical diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping.
- Ambulatory participant, able to perform TTRISE in 10 seconds or less at the time of screening visit.
- Able to walk independently without assistive devices.
- Have intact right and left biceps muscles or an alternative upper arm muscle group.
- Have been on a stable dose or dose equivalent of oral corticosteroids for at least 12 weeks prior to randomization and the dose is expected to remain constant (except for modifications to accommodate changes in weight and stress-related needs as per the recently published guidelines throughout the study.
Key exclusion criteria (7 listed)
- Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks prior to randomization.
- Current or previous treatment with any other experimental pharmacologic treatment for DMD or any prior exposure to antisense oligonucleotide, gene therapy or gene editing; except the following: Ezutromid in the last 12 weeks prior to first dose; Drisapersen in the last 36 weeks prior to first dose; Suvodirsen in the last 12 weeks prior to first dose; Vamorolone in the last 12 weeks prior to first dose; Eteplirsen (previous or current use); and Tamoxifen in the last 4 weeks prior to first dose.
- Major surgery within 3 months prior to randomization.
- Presence of any other significant neuromuscular or genetic disease other than DMD.
- Presence of any known impairment of renal function and/or 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 |
|---|---|---|
| EteplirsenAlso: AVI-4658, EXONDYS 51, EXONDYSSolution for intravenous (IV) infusion. | Drug | Part 1: Eteplirsen, Part 2: Eteplirsen 100 mg/kg, Part 2: Eteplirsen 200 mg/kg, Part 2: Eteplirsen 30 mg/kg |
Primary outcome
Part 1: Incidence of Adverse Events (AEs)
Time frame: Up to Week 148
Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis)
Time frame: Baseline, Week 144
Part 2: Change from Baseline at Week 72 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis)
Time frame: Baseline, Week 72 or Week 96
7 secondary outcomes
Part 2: Change From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test
Time frame: Baseline, Week 144
Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT)
Time frame: Baseline, Week 144
Part 2: Change from Baseline at Week 144 in Forced Vital Capacity Percent Predicted (FVC%p)
Time frame: Baseline, Week 144
Part 2: Time to Loss of Ambulation (LOA)
Time frame: Baseline up to Week 144
Part 2: Change From Baseline in Skeletal Muscle Dystrophin Expression
Time frame: Baseline, Postdose (at Week 24, Week 48, or Week 144)
Part 2: Incidence of Adverse Events (AEs)
Time frame: Baseline up to Week 148
Part 2: Pharmacokinetic (PK) Plasma Concentration of Eteplirsen
Time frame: 0 (predose) to 2 hours postdose up to Week 144
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2026.
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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.
59 sites in 26 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Eteplirsen in people with Muscular Dystrophy, Duchenne, run by Sarepta Therapeutics, Inc. at 59 sites in 26 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 6 inclusion and 7 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: under way, not looking for participants, as of the sponsor's last update on 27 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 59 sites in India, Jordan, South Korea, United States, Colombia and 21 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Part 1: Incidence of Adverse Events (AEs) (time frame: Up to Week 148). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Feb 2026.