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

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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)

key information

study id #: NCT03375255

condition: Muscular Dystrophy, Duchenne

status: recruiting

purpose:

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.

intervention: SRP-5051

mechanism of action: Exon-skipping to promote dystrophin production

results: https://clinicaltrials.gov/ct2/show/results/NCT03375255

last updated: May 06, 2019

study details

start date: February 5, 2018

estimated completion: September 1, 2019

phase of development: Phase 1

size / enrollment: 30

primary outcomes:

  • 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.

secondary outcomes:

  • 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.

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

study contacts

sponsor: Sarepta Therapeutics

contacts: Medical Information, +1-888-727-3782, clinicaltrials@sarepta.com

investigators: Medical Director; Sarepta Therapeutics, Inc.

locations: United States, Canada

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