PPMO: Phase 1 study of SRP-5051 in people with Duchenne amenable to Exon 51 Skipping

A Phase 1 first-in-human, open-label, multi-center study to evaluate the safety, tolerability and pharmacokinetics of single escalating doses of SRP-5051 in individuals with Duchenne amenable to exon 51 skipping.

Questions and Answers

What stage is this research study?

Study 5051-101 is an open-label Phase 1 study that is currently recruiting participants in the US and Canada. Participants who complete the study may be eligible to enroll into an extension study.

What is the goal or purpose of this research?

This Phase 1 study is being conducted to assess the safety, tolerability, and pharmacokinetics of SRP-5051, in participants 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:

  • Diagnosis of Duchenne muscular dystrophy
  • Deletion mutation in the dystrophin gene amenable to exon 51 skipping
  • Male, age 12 years and older
  • Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks prior to screening
  • Has not had treatment with eteplirsen or drisapersen within 6 months prior to screening
  • No participation in experimental gene therapy for treatment of Duchenne at any time
  • Stable cardiac and pulmonary function
  • Note: There are no requirements for ambulation status

Additional criteria apply. Ultimately, the Principal Investigator (study doctor) determines whether or not a participant meets the requirements and is eligible for participation in the study based on the protocol.

Where will this study take place?

This study is currently being conducted at over 10 clinical sites in the United States and Canada. To view participating study sites, visit www.clinicaltrials.gov (Identifier NCT03375255).

What can be expected by participating in this study?

Participants will receive one dose of SRP-5051 via IV infusion, followed by approximately 12 weeks of safety observation. Participants will stay in the clinic for 2 nights after receiving study drug. During this time, study doctors will collect blood and urine for pharmacokinetic (PK) tests, and perform other safety assessments such as physical exams and vital sign collection. Participants will return to the clinic for 3 additional visits at Week 2, Week 4, and Week 12. These visits will be one-day visits lasting approximately 3-4 hours during the 12 weeks following dosing. Safety, including side effects and laboratory tests, will be monitored on an ongoing basis for all participants.

After completion of the study, participants may be provided the option to participate in an open-label extension study with monthly dosing of SRP-5051.

The Principal Investigator (study doctor) and/or the study site contact will review all study requirements with potential participants during the informed consent process both for the single dose study, and for the open-label extension study.

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
  • Become more familiar with what participation in a clinical study entails
  • 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.

What is SRP-5051?

SRP-5051 is an investigational drug, meaning it has not been approved by the FDA or any other governmental agency. SRP-5051, is a PPMO compound designed to enable skipping of exon 51. PPMO has a cell-penetrating peptide (CPP) attached to a PMO backbone. The mechanism of action is sequence-specific binding to RNA targets. Pre-clinical (animal) studies have demonstrated targeted delivery to skeletal, cardiac, and smooth muscle animal tissues.*

*Passini et al. Presented at the 13th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS) | 24–27 September 2017 | Bordeaux, France

Will I or my child be paid for participating in this study?

Generally, costs associated with participation in the study (e.g., travel, parking, meals) are reimbursed within fair market value. If there isn’t a site geographically close to you, travel will be provided and arranged by a third party vendor. Additional information regarding reimbursement and travel can be provided by the 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 NCT03375255). You may also email medinfo@sarepta.com or call +1-888-727-3782.

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