CompletedNCT03992430ClinicalTrials.gov record

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

Muscular Dystrophy, Duchenne

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THE QUICK READ

What this study involves

The registry does not currently label this study as recruiting. Ask about its status before planning a screening visit.

Study type: Interventional
Phase: Phase 3
Age range: 4 years - 13 years
Sex eligible: Male
Healthy volunteers: Not accepted
Participants: 160 Actual

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

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.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

Inclusion Criteria:

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.

Exclusion Criteria:

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.

Understand screening

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment: Random assignment
Masking: Quadruple masked

Part 1: Eteplirsen

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.

Drug: Eteplirsen

Part 2: Eteplirsen 30 mg/kg

Randomized participants will receive eteplirsen 30 mg/kg once weekly for up to 144 weeks.

Drug: Eteplirsen

Part 2: Eteplirsen 100 mg/kg

Randomized participants will receive eteplirsen 100 mg/kg once weekly for up to 144 weeks.

Drug: Eteplirsen

Part 2: Eteplirsen 200 mg/kg

Randomized participants will receive eteplirsen 200 mg/kg once weekly for up to 144 weeks.

Drug: Eteplirsen

Eteplirsen DRUG

Solution for intravenous (IV) infusion.

Prepare your questions

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.