Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon… (NCT03218995) | Clinical Trial Compass
CompletedPhase 2
Study of Eteplirsen in Young Participants With Duchenne Muscular Dystrophy (DMD) Amenable to Exon 51 Skipping
Belgium, France, Italy15 participantsStarted 2017-08-16
Plain-language summary
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
Who can participate
Age range
6 Months – 48 Months
Sex
MALE
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Male between 6 months to 48 months of age (inclusive)
* Diagnosis of DMD with a deletion mutation amenable to exon 51 skipping
* Parent(s) or legal guardian(s) who is willing to provide written informed consent
Exclusion Criteria:
* Received treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing
* Received previous or current treatment with any experimental treatment
* Clinically significant illness other than DMD
* Clinically significant laboratory abnormality
* Any other condition that could interfere with the participation in the study.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and TEAEs Leading to Discontinuation From Study Drug
Timeframe: Baseline up to Week 100
2
Number of Participants With at Least 1 Potentially Clinically Significant Clinical Safety Laboratory Abnormality
Timeframe: Baseline up to Week 100
3
Number of Participants With at Least 1 Markedly Abnormal Vital Sign
Timeframe: Baseline up to Week 100
4
Abnormal Changes From Baseline or Worsening of Physical Examination Findings
Timeframe: Baseline up to Week 100
5
Number of Participants With at Least 1 Markedly Abnormal Electrocardiogram (ECG) and Echocardiogram (ECHO)