Phase 2 open label study of Eteplirsen in young children with Duchenne amenable to Exon 51 Skipping (Study 4658-102)

An Open-label Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Eteplirsen in Young Patients With Duchenne Amenable to Exon 51 Skipping

Questions and Answers

What stage is this research study?

Study 4658-102 is a Phase 2 study that is currently recruiting participants for Cohort 2 (6 – 24 months) in Europe. Cohort 1 (24 – 48 months) is fully enrolled.

What is the purpose of this research?

This study is being conducted to assess the safety, tolerability, pharmacokinetics, and efficacy of eteplirsen, an investigational exon 51 skipping therapy, in young patients with Duchenne who are amenable to exon 51 skipping.

Who is sponsoring this study?

The study is sponsored by Sarepta Therapeutics, Inc.

What are the eligibility requirements of this study?

Eligibility criteria include but are not limited to:

  • Male between 6 months to 24 months of age (inclusive)
  • Diagnosis of Duchenne with a deletion mutation amenable to exon 51 skipping
  • Parent(s) or legal guardian(s) who is willing to provide written informed consent

Key exclusion criteria include:

  • 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 Duchenne
  • Clinically significant laboratory abnormality
  • Any other condition that could interfere with the patient’s participation

The Principal Investigator (study doctor) determines whether or not a patient meets the requirements and is eligible for participation in the study.

Where will this study take place?

This study is currently being conducted at five clinical sites in Europe. To view participating study sites, visit www.clinicaltrials.gov (Identifier NCT03218995).

What can be expected by participating in this study?

All patients enrolled in the study receive weekly treatment with the investigational therapy eteplirsen, for at least 48 weeks and up to 96 weeks. Safety is regularly assessed throughout the study via the collection of side effect information, laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations. Exploratory efficacy assessments including age- appropriate functional measurements are also being collected.

Will I be paid for allowing my child to participate in this study?

Reasonable costs associated with participation in the study (e.g., travel, parking, meals) are reimbursed. Additional information regarding reimbursement can be provided by a participating study site.

Where can I learn more about this clinical study?

To learn more about this study, you or your doctor may contact the study research staff using the contacts posted on www.clinicaltrials.gov (Identifier NCT03218995). You may also email medinfo@sarepta.com or call +1-617-274-4000.

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