A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03375255
Recruitment Status : Recruiting
First Posted : December 18, 2017
Last Update Posted : August 7, 2018
Information provided by (Responsible Party):
Sarepta Therapeutics

Brief Summary:
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of 5 escalating doses of SRP-5051 administered as a single dose to patients with DMD amenable to exon 51 skipping treatment.

Condition or disease Intervention/treatment Phase
Muscular Dystrophy, Duchenne Drug: SRP-5051 Phase 1

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1 Trial to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Treatment
Actual Study Start Date : December 11, 2017
Estimated Primary Completion Date : January 2019
Estimated Study Completion Date : January 2019

Arm Intervention/treatment
Experimental: SRP-5051

Patients will be sequentially assigned to receive one of the 5 escalating dose levels of SRP-5051 on Day 1.

Patients who complete the study and continue to meet safety eligibility criteria will have the opportunity to enroll in an open-label extension study to continue to receive SRP-5051.

Drug: SRP-5051
Single dose of SRP-5051 administered as an intravenous (IV) infusion.

Primary Outcome Measures :
  1. Number of Patients with Adverse Events (AEs) [ Time Frame: Up to 14 Weeks ]
    An AE is any untoward medical occurrence in a clinical trial patient, which does not necessarily have a causal relationship with the investigational drug. An AE can, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurs during or after administration of the study drug whether or not considered related to the study drug.

Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51 skipping treatment
  • Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration with continued dosing of oral corticosteroids while participating in the study*, or has not received corticosteroids for at least 12 weeks prior to study drug administration and will not initiate dosing of oral corticosteroids while participating in the study

Exclusion Criteria:

  • Has a left ventricular ejection fraction (LVEF) less than (<) 40 percent (%) based on an echocardiogram (ECHO) performed within 3 months prior to Screening or at the Screening visit
  • Has a QTcF >= 450 millisecond (msec) on the Screening electrocardiogram (ECG)
  • Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening and while participating in the study for any of the following: angiotensin converting enzyme (ACE) inhibitors, angiotensin receptor blocking agents (ARBs), beta-blockers, potassium and steroids*
  • Requires antiarrhythmic and/or diuretic therapy for heart failure
  • Forced vital capacity (FVC) <40% of predicted value within 3 months of screening or at the Screening visit
  • Known kidney disease or had an acute kidney injury within 6 months
  • Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any experimental gene therapy for the treatment of DMD at any time

Other inclusion/exclusion criteria apply.

*The dose of steroids must remain constant except for modifications to accommodate changes in weight.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03375255

Contact: Medical Information +1-888-727-3782

United States, California
David Geffen School of Medicine at UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Clara Sam    310-825-3264   
Principal Investigator: Perry Shieh, MD, PhD         
United States, Florida
University of Florida Recruiting
Gainesville, Florida, United States, 32610
Contact: Norane Shehab    352-294-8705   
Principal Investigator: Barry Byrne, MD         
NW FL Clinical Research Group, LLC Recruiting
Gulf Breeze, Florida, United States, 32561
Contact: Shae Colbert    850-934-1299   
Principal Investigator: Weldon Mauney, MD         
United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30318
Contact: Han C Phan, MD    678-883-6897   
Principal Investigator: Han C Phan, MD         
United States, Illinois
Ann & Robert H. Lurie Children's Hospital of Chicago Recruiting
Chicago, Illinois, United States, 60611
Contact: Theresa Oswald    312-227-3019   
Principal Investigator: Nancy Kuntz, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
Contact: Kiley Higss    913-945-9922   
Contact: Katherine Roath    913-945-9928   
Principal Investigator: Jeffrey Statland, MD         
United States, Ohio
The Research Institute at Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Lauren Bird, RN,BSN,CCRC    614-722-2699   
Principal Investigator: Jerry R Mendell, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Jennifer M Monahan    412-692-5176   
Principal Investigator: Hoda Z Abdel-Hamid, MD         
United States, Texas
Children's Medical Center Dallas Recruiting
Dallas, Texas, United States, 75207
Contact: Holly Lawrence    214-456-2463   
Principal Investigator: Diana Castro, MD         
Sponsors and Collaborators
Sarepta Therapeutics
Study Director: Sanjay Rakhade, MD,PHD Sarepta Therapeutics, Inc.

Responsible Party: Sarepta Therapeutics Identifier: NCT03375255     History of Changes
Other Study ID Numbers: 5051-101
First Posted: December 18, 2017    Key Record Dates
Last Update Posted: August 7, 2018
Last Verified: August 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Sarepta Therapeutics:
Duchenne Muscular Dystrophy
Exon Skipping
Exon 51
Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO)

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked