Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu

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.
ClinicalTrials.gov Identifier: NCT03375255
Recruitment Status : Recruiting
First Posted : December 18, 2017
Last Update Posted : April 22, 2019
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

Tracking Information
First Submitted Date  ICMJE December 12, 2017
First Posted Date  ICMJE December 18, 2017
Last Update Posted Date April 22, 2019
Actual Study Start Date  ICMJE February 5, 2018
Estimated Primary Completion Date September 1, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 12, 2018)
Number of Participants with Adverse Events (AEs) [ Time Frame: From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks) ]
An AE is any untoward medical occurrence in a clinical trial participant, 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.
Original Primary Outcome Measures  ICMJE
 (submitted: December 14, 2017)
Number of Participants 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.
Change History Complete list of historical versions of study NCT03375255 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 12, 2018)
  • Maximum Plasma concentration (Cmax) of SRP-5051 [ Time Frame: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
  • Area under the plasma concentration versus time curve (AUC) of SRP-5051 [ Time Frame: Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours) ]
    Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Muscular Dystrophy, Duchenne
Intervention  ICMJE Drug: SRP-5051
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
Study Arms  ICMJE Experimental: SRP-5051

Patients will be sequentially assigned to receive 1 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.

Intervention: Drug: SRP-5051
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: December 14, 2017)
30
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE September 1, 2019
Estimated Primary Completion Date September 1, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 QT interval corrected with Fridericia's method (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, or potassium
  • 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 prior to Screening
  • Treatment with eteplirsen or drisapersen within 6 months prior to Screening, or any experimental gene therapy for the treatment of DMD at any time
  • Use of any herbal medication/supplement containing aristolochic acid

Other inclusion/exclusion criteria apply.

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

Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information +1-888-727-3782 clinicaltrials@sarepta.com
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03375255
Other Study ID Numbers  ICMJE 5051-101
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Sarepta Therapeutics
Study Sponsor  ICMJE Sarepta Therapeutics
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Medical Director Sarepta Therapeutics, Inc.
PRS Account Sarepta Therapeutics
Verification Date December 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP