ESSENCE: Phase 3a study of SRP-4045 and SRP-4053 in people with Duchenne amenable to Exons 45 and 53 Skipping

A 96 Week Trial for Individuals with Duchenne Muscular Dystrophy Amenable to either Exon 45 or Exon 53 skipping.

Questions and Answers

What stage is this research study?

This trial is a Phase 3s study.

What is the goal or purpose of this study?

The purpose of this research study is to evaluate the safety and efficacy of SRP-4045 and SRP-4053 in individuals with Duchenne who have a deletion that is amenable to exon 45 or exon 53 skipping.

Who is sponsoring this study?

This study is sponsored by Sarepta Therapeutics.

Who might be eligible to participate in this study?

As of April 2018, ESSENCE has reached targeted enrollment in the US and is no longer enrolling patients in the US. Enrollment is continuing in other countries. The study is enrolling individuals with DMD with deletions amenable to exon 45 or exon 53 skipping. Twice as many patients will receive active treatment as will receive placebo (2:1 randomization).

Eligibility criteria include but are not limited to:

  • Genotypically confirmed diagnosis of Duchenne, with genetic deletion amenable to exon 45 or exon 53 skipping
  • Male, 7 – 13 years of age
  • Stable dose of oral corticosteroids for at least 24 weeks before entry
  • Stable pulmonary and cardiac function
  • Average 6MWT (6-Minute Walk Test) between ≥300 – ≤450 meters

Additional criteria apply and will be reviewed with patients during the screening process. The Principal Investigator (study doctor) determines whether a patient meets the inclusion criteria for the study, and therefore whether or not the patient is eligible to participate.

Where will this study take place?

~80 global sites are planned to participate in ESSENCE. Please see clinicaltrials.gov for updates (Identifier NCT02500381).

Will all individuals enrolled in this trial receive treatment with the investigational therapy?

No, some patients will receive active treatment and some patients will receive placebo (2:1 randomization). However, all patients will receive active treatment in the Open Label Period after completing the 96 Week Double Blind Period.

Why is Sarepta including a placebo group of patients in this study?

Comparing patients receiving active drug against patients receiving placebo helps to understand how a drug affects patients and to determine whether the investigational agent is effective and safe.

Why should I consider participation in this study?

While no benefit can be guaranteed from participation in any clinical study, enrolled participants may:

  • Have access to clinicians with expertise in the treatment of Duchenne
  • Contribute to medical research and what is currently known about the progression of Duchenne
  • Contribute to information that may accelerate the development of Duchenne therapies.

Choosing to participate in a clinical study is an important and personal decision. It is recommended that you speak with your doctor, family members, and/or friends about participating in this study.

Will I be paid for participating in this study?

Generally, travel costs associated with participation in the study will be paid up-front, and incidental costs (parking, meals, etc.) are reimbursed. We do not offer a stipend for participation in the ESSENCE trial. Additional information regarding reimbursement and compensation can be provided by a participating study site.

If enrolled, what can I expect during the study?

The Principal Investigator (study doctor) and/or the study site contact will review study requirements with all patients during the screening process.

All patients will receive weekly infusions over 96 weeks. Patients in the treated group will receive an infusion of investigational therapy, and patients in the untreated group will receive a placebo infusion. Neither the doctor nor the patient will know whether or not the infusion is placebo or investigational therapy during the 96 weeks. After 96 weeks, an open-label extension will continue for up to 48 weeks. During the open-label extension, all patients will receive investigational agent, and no patients will receive placebo.

Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six minute walk test. All patients will undergo a muscle biopsy at baseline and a second muscle biopsy at either Week 48 or Week 96.

Safety will be assessed through the collection of side effect information, laboratory tests, electrocardiograms (ECGs), echocardiograms (ECHOs), vital signs, and physical examinations throughout the study.

Blood samples will be taken periodically throughout the study to assess levels of drug in the bloodstream (pharmacokinetics) and to monitor patient safety.

Where can I learn more about this clinical study?

ESSENCE has reached targeted enrollment in the US and is no longer enrolling patients in the US. Enrollment is continuing in other countries. To learn more information about the ESSENCE study, please visit www.clinicaltrials.gov(Identifier NCT02500381) or essencetrial.com. You may also email medinfo@sarepta.com or call +1-888-727-3782.

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