SRP-4045/SRP-4053 (ESSENCE/4045-301) - A 96 -Week, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients with Duchenne


Frequently Asked Questions (FAQs) about this study:


What stage is this research?

  • This trial is a Phase 3 study that will begin recruiting patients in 2016.


What was the goal or purpose of this study?

  • The main goal of this study is to evaluate the efficacy of SRP-4045 and SRP-4053 compared to placebo in Duchenne muscular dystrophy patients with deletions amenable to skipping exon 45 and exon 53 respectively. Additional objectives include evaluation of safety, pharmacokinetics and biomarkers.


Who is sponsoring this study?

  • This study is sponsored by Sarepta Therapeutics.


Who might be eligible to participate in this study?

  • Researchers are recruiting approximately 99 Duchenne patients with deletions amenable to exon 45 or exon 53 skipping. Of these, 66 patients will be assigned to active treatment and 33 patients will be assigned to placebo. Twice as many patients will receive active treatment as will receive placebo (2:1 randomization).
  • Key inclusion criteria include but are not limited to:
    • Diagnosis of Duchenne, genotypically confirmed, with genetic deletions amenable to exon 45 or exon 53 skipping
    • Male 7 – 16 years of age
    • Stable dose of corticosteroids for at least six months
    • Stable pulmonary and cardiac function
  • 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 and exclusion criteria for the study, and therefore whether or not the patient is eligible to participate.


Where will this study take place?

  • Once study sites have been selected, they will be posted on (Identifier NCT02500381).


Will all boys 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).


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 determine whether it is effective and safe.


Why should I consider participating in this study?

  • While no benefit can be guaranteed from participation in any clinical study, we believe you may:
    • Have access to highly experienced clinicians with strong expertise in treating Duchenne, who might not normally be accessible to you and your family
    • Gain better understanding of Duchenne
    • Have the opportunity to help others by contributing to medical research that may accelerate the development of Duchenne therapies
  • Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.


Will I get paid for participating in this study?

  • Generally, costs associated with participation in the study (e.g., travel, parking, meals) are reimbursed and a stipend may be available. 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.
  • Clinical efficacy will be assessed at periodically 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 over the course of the study.
  • 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).


Where can I learn more about this clinical study?

  • You and your doctor may contact clinical study staff to inquire about the clinical study. Contact information will be posted for the clinical study investigator and coordinator once study sites have been selected and have received approval to conduct the study. Continue to check (Identifier NCT02500381) for information. You may also email This email address is being protected from spambots. You need JavaScript enabled to view it..


*Protocol amendment pending to update study duration to 96 weeks.

 (Updated 5/13/2016)