search
Back to results

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

Primary Purpose

Muscular Dystrophy, Duchenne

Status
Completed
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
SRP-5051
Sponsored by
Sarepta Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Muscular Dystrophy, Duchenne focused on measuring Duchenne Muscular Dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Nonambulatory, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Neuromuscular Research Center
  • NW FL Clinical Research Group, LLC
  • Rare Disease Research, LLC
  • Ann & Robert H. Lurie Children's Hospital of Chicago
  • University of Kansas Medical Center
  • Children's Hospital of Pittsburgh of UPMC
  • Children's Medical Center Dallas
  • London Health Sciences Centre

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

SRP-5051

Arm Description

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.

Outcomes

Primary Outcome Measures

Number of Participants with Adverse Events (AEs)
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.

Secondary Outcome Measures

Maximum Plasma concentration (Cmax) of SRP-5051
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
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.

Full Information

First Posted
December 12, 2017
Last Updated
June 30, 2022
Sponsor
Sarepta Therapeutics, Inc.
search

1. Study Identification

Unique Protocol Identification Number
NCT03375255
Brief Title
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of a Single Dose of SRP-5051 (Vesleteplirsen) in Patients With Duchenne Muscular Dystrophy (DMD)
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2022
Overall Recruitment Status
Completed
Study Start Date
February 5, 2018 (Actual)
Primary Completion Date
August 19, 2019 (Actual)
Study Completion Date
August 19, 2019 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Sarepta Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Muscular Dystrophy, Duchenne
Keywords
Duchenne Muscular Dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Nonambulatory, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
SRP-5051
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
SRP-5051
Other Intervention Name(s)
vesleteplirsen
Intervention Description
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
Primary Outcome Measure Information:
Title
Number of Participants with Adverse Events (AEs)
Description
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.
Time Frame
From signing of informed consent to 12 weeks after the last infusion of SRP-5051 (Up to 14 weeks)
Secondary Outcome Measure Information:
Title
Maximum Plasma concentration (Cmax) of SRP-5051
Description
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Time Frame
Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)
Title
Area under the plasma concentration versus time curve (AUC) of SRP-5051
Description
Plasma samples to be collected via peripheral venipuncture from the contralateral arm used for drug infusion.
Time Frame
Pre-dose, mid-infusion, end of infusion, post-dose (0.25, 0.5, 1, 2, 4, 8, 12 hours)

10. Eligibility

Sex
Male
Gender Based
Yes
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Sarepta Therapeutics, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Neuromuscular Research Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
NW FL Clinical Research Group, LLC
City
Gulf Breeze
State/Province
Florida
ZIP/Postal Code
32561
Country
United States
Facility Name
Rare Disease Research, LLC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30318
Country
United States
Facility Name
Ann & Robert H. Lurie Children's Hospital of Chicago
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Children's Hospital of Pittsburgh of UPMC
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Children's Medical Center Dallas
City
Dallas
State/Province
Texas
ZIP/Postal Code
75207
Country
United States
Facility Name
London Health Sciences Centre
City
London
State/Province
Ontario
ZIP/Postal Code
N6A 5W9
Country
Canada

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs