This study will be comprised of 2 parts: Part 1 (dose escalation) will be conducted to 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 be conducted for the selection of a high dose (100 mg/kg versus 200 mg/kg) and its comparison with the 30 mg/kg dose of eteplirsen, in approximately 144 participants with genetically confirmed deletion mutations amenable to treatment by skipping exon 51.
|Full study title
|A Randomized, Double-Blind, Dose Finding and Comparison Study of the Safety and Efficacy of a High Dose of Eteplirsen, Preceded by an Open-Label Dose Escalation, in Patients with Duchenne Muscular Dystrophy With Deletion Mutations Amenable to Exon 51 Skipping
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.
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; and Eteplirsen
(previous or current use).
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
Barry J Byrne, MD, PhDPediatric Cardiologist
Participate in a study
Here are some general steps to consider when participating in a research study:
Contact the research team
Call or email the research team listed within the specific clinical trial or study to let them know that you're interested. A member of the research team, such as the researcher or study coordinator, will be available to tell you more about the study and to answer any questions or concerns you may have.
Primary contactBarry Byrne
Get screened to confirm eligibility
You may be asked to take part in prescreening to make sure you are eligible for a study. The prescreening process ensures it is safe for you to participate. During the prescreening process, you will be asked some questions and you may also be asked to schedule tests or procedures to confirm your eligibility.
Provide your consent to participate
If you are eligible and want to join the clinical trial or study, a member of the research team will ask for your consent to participate. To give consent, you will be asked to read and sign a consent form for the study. This consent form explains the study's purpose, procedures, risks, benefits and provides other important information, such as the study team's contact information.
If you decide to participate in a clinical trial or study, the research team will keep you informed of the study requirements and what you will need to do to throughout the study. For some trials or studies, your health care provider may work with the research team to ensure there are no conflicts with other medications or treatments.