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

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ClinicalTrials.gov Identifier: NCT03375255
Recruitment Status : Recruiting
First Posted : December 18, 2017
Last Update Posted : November 5, 2018
Sponsor:
Information provided by (Responsible Party):
Sarepta Therapeutics

December 12, 2017
December 18, 2017
November 5, 2018
February 5, 2018
September 1, 2019   (Final data collection date for primary outcome measure)
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.
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.
Complete list of historical versions of study NCT03375255 on ClinicalTrials.gov Archive Site
  • 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.
Not Provided
Not Provided
Not Provided
 
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 in Patients With Duchenne Muscular Dystrophy (DMD)
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
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.
Not Provided
Interventional
Phase 1
Intervention Model: Sequential Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Muscular Dystrophy, Duchenne
Drug: SRP-5051
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
30
Same as current
September 1, 2019
September 1, 2019   (Final data collection date for primary outcome measure)

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.

Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
12 Years and older   (Child, Adult, Older Adult)
No
Contact: Medical Information +1-888-727-3782 clinicaltrials@sarepta.com
Canada,   United States
 
 
NCT03375255
5051-101
No
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
Sarepta Therapeutics
Sarepta Therapeutics
Not Provided
Study Director: Medical Director Sarepta Therapeutics, Inc.
Sarepta Therapeutics
November 2018

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