Muscular Dystrophy, Duchenne
Conditions
Keywords
Duchenne Muscular Dystrophy, Exon Skipping, DMD, Exon 51, Ambulatory, Pediatric, Nonambulatory, Peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO), Duchenne
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.
Interventions
DRUGSRP-5051
Single dose of SRP-5051 administered as an intravenous (IV) infusion.
Sponsors
Sarepta Therapeutics, Inc.
Study design
Allocation
NA
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
12 Years to No maximum
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 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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants with Adverse Events (AEs) | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Maximum Plasma concentration (Cmax) of SRP-5051 | 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 | 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. |
Countries
Canada, United States
Outcome results
None listed