Track this trial free. DataLookout checks ClinicalTrials.gov daily and emails you which fields changed on NCT03992430 (status, enrollment, completion dates, endpoints, sites) when this record is updated. Free accounts track up to 5 trials.

Track this trial

A Study to Compare Safety and Efficacy of High Doses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)

Study acronym: MIS51ON
StatusActive, Not Recruiting
PhasePhase 3
Started2020-07-13
View on ClinicalTrials.gov ↗

Amendment history

2026-02-25
Study Status v27
Re-verified, no change to tracked fields
2024-12-18
Study Identification, Study Status, Study Description, Arms and Interventions, Outcome Measures, Eligibility v26
Primary completion date2024-11-30→2026-10-31
Completion date2024-11-30→2026-10-31
Eligibility criteria+51 characters
[...] dose; Vamorolone in the last 12 weeks prior to first dose; and Eteplirsen (previous or current use); and Tamoxifen in the last 4 weeks prior to first dose. Major surgery within 3 months prior to randomization. Pres [...]
Secondary endpoints7 entries, revised
Part 2: Change from Baseline at Week 144 in Forced Vital Capacity Percent Predicted (FVC%p) at Week 144
Primary endpoints2 to 3 entries
Part 2: Change From Baseline at Week 144 in the NSAA Total Score (for Final Analysis) Part 2: Change from Baseline at Week 14472 or Week 96 in NSAA Total Score (for Conditional Efficacy Interim Analysis)
Study title-1 characters
A Study to Compare Safety and Efficacy of a High DoseDoses of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) (MIS51ON)
2024-04-11
Study Status, Contacts/Locations v25
Study sites58→59
2023-11-28
Recruiting→Active, Not Recruiting Study Status, Study Design, Contacts/Locations v24
Trial statusRecruiting→Active, Not Recruiting
Enrollment targetestimated 154→confirmed 160
Study sites60→58
2023-10-04
Study Status, Contacts/Locations v23
Study sites54→60
Show 22 earlier versions
2023-08-16
Study Status, Contacts/Locations v22
Study sites51→54
2023-07-03
Study Status, Contacts/Locations v21
Study sites50→51
2023-05-24
Study Status, Contacts/Locations v20
Study sites52→50
2023-05-09
Study Status, Contacts/Locations v19
Study sites51→52
2023-04-21
Study Status, Contacts/Locations v18
Study sites48→51
2023-04-06
Study Status, Contacts/Locations v17
Study sites45→48
2023-02-16
Study Status, Contacts/Locations v16
Study sites30→45
2023-01-17
Study Status, Contacts/Locations v15
Study sites28→30
2022-12-13
Study Status, Contacts/Locations v14
Study sites23→28
2022-11-04
Study Status, Contacts/Locations v13
Study sites21→23
2022-09-30
Study Status, Contacts/Locations v12
Study sites18→21
2022-08-24
Study Status, Contacts/Locations v11
Study sites15→18
2022-06-18
Study Status, Contacts/Locations v10
Study sites13→15
2022-05-18
Study Status, Study Description, Arms and Interventions, Contacts/Locations v9
Primary completion date2026-02-28→2024-11-30
Completion date2026-02-28→2024-11-30
Study sites10→13
2022-04-08
Study Status, Contacts/Locations v8
Study sites8→10
2022-01-19
Active, Not Recruiting→Recruiting Study Identification, Study Status, Study Description, Study Design, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v7
Trial statusActive, Not Recruiting→Recruiting
Primary completion date2026-02→2026-02-28
Completion date2026-02→2026-02-28
Enrollment target152→154
Eligibility criteria+592 characters
[...] tion mutation of the DMD gene amenable to exon 51 skipping. HaveAmbulatory achievedparticipant, aable meanto 6-minuteperform TTRISE in 10 seconds or less at the time of screening visit. Able to walk testindependently (6MWT)without distanceassistive of greater than equal to (>=) 300 and less than equal to (<=) 450 metersdevices. Have intact right and left biceps muscles or an alternativ [...] 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 pub [...] [...]
Secondary endpoints6 to 7 entries
Part 2: (DoseChange Comparison)From Baseline in Time to Rise From the Floor, Time to Complete 10-Meter Walk/Run, and the Timed Stair Ascend Test Part 2: Change From Baseline in the Total Distance Walked During 6-Minute Walk Test (6MWT) Part 2 (Dose Comparison): Change Fromfrom Baseline in Time to Complete Walk/run, Stairs and Time to Rise Part 2 (Dose Comparison): Annual Rate in Decline of Forced Vital Capacity Percent Predicted (FVC%p) at Week 144 Part 2 (Dose Comparison): Time to Loss of Ambulation (LOA) Part 2 (Dose Comparison): Change From Baseline in Skeletal Muscle Dystrophin Expression [...] [...]
Primary endpoints5 to 2 entries
Part 1 and Part 2 (Dose Finding): IncidencesIncidence of Adverse Events (AEs) Part 2 (Dose Comparison): Change From Baseline in the North Star Ambulatory Assessment (NSAA) Total Score Part 2at (DoseWeek Finding): Dystrophin Expression in Biopsied Muscle Tissue Part 2 (Dose Finding): Pharmacokinetic (PK) Plasma Concentration of Eteplirsen Part 2 (Dose Finding): Tissue Concentration of Eteplirsen From Biopsied Muscle Tissue144
Study sites8 entries, revised
Children's Hospital - London Health Sciences Centre
Study title+9 characters
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Participants With Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)
2021-01-15
Recruiting→Active, Not Recruiting Study Status, Contacts/Locations v6
Trial statusRecruiting→Active, Not Recruiting
Primary completion date2024-10→2026-02
Completion date2024-10→2026-02
Study sites7→8
2020-08-12
Study Status, Contacts/Locations v5
Study sitescontacts updated at 1 of 7 sites
2020-07-30
Study Status, Arms and Interventions, Outcome Measures, Eligibility, Contacts/Locations v4
Start dateestimated 2020-01→confirmed 2020-07-13
Study sites2→7
Eligibility criteria+64 characters
[...] ose or dose equivalent of oral corticosteroids for at least 2412 weeks prior to randomization. Have stable pulmonary functio [...] ent for DMD; some exceptions apply. Previous treatment with drisapersen, ezutromid, or domagrozumabEzutromid in the last 241 week prior to first dose or Drisapersen in the last 36 weeks prior to studyfirst enrollmentdose. Major surgery within 3 months prior to randomization. Pres [...] neuromuscular or genetic disease other than DMD. Presence of any known impairment of renal function and/or other clinically significant illness. Has evidence of cardi [...]
Secondary endpointsdetails revised at 1 of 6 entries
2020-01-16
Study Status, Contacts/Locations v3
Start date2019-12→2020-01
Study sites1→2
2019-12-12
Study Status v2
Start date2019-12-05→2019-12
2019-12-05
Not Yet Recruiting→Recruiting Study Identification, Study Status, Conditions, Contacts/Locations v1
Trial statusNot Yet Recruiting→Recruiting
Start date2019-08→2019-12-05
Study sites0→1
Study title+10 characters
A Study to Compare Safety and Efficacy of a High Dose of Eteplirsen in Duchenne Muscular Dystrophy (DMD) Patients (MIS51ON)
2019-06-18
Original filing
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.
Trial Details
NCT Number NCT03992430
Lead Sponsor Sarepta Therapeutics, Inc.
Conditions Muscular Dystrophy, Duchenne
Enrollment 160 participants
Start Date 2020-07-13
Primary Completion 2026-10-31 (estimated)
Study Completion 2026-10-31 (estimated)
Updated on ClinicalTrials.gov 2026-02-27