Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Exondys 51
Product Name: Eteplirsen
Product Code: AVI-4658
Pharmaceutical Form: Concentrate for solution for infusion
INN or
Sponsors
Sarepta Therapeutics, Inc
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Patient has successfully completed 96 weeks of treatment in Study 4658-102. 2. Parent(s) or legal guardian(s) who is/are able to understand and comply with the study requirements. 3. Patient and/or their parent(s)/legal guardian(s) are willing and able to provide signed informed consent. Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Patient has a prior or ongoing medical condition that, in the Investigator's opinion, could adversely affect the safety of the patient, or make it unlikely that the course of treatment or follow-up would be completed, or impair the assessment of study results.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: To evaluate the efficacy of additional treatment with eteplirsen administered once weekly by IV infusion in male DMD patients who have successfully completed Study 4658-102 ; Main Objective: To evaluate the ongoing safety and tolerability of additional treatment with eteplirsen administered once weekly by intravenous (IV) infusion in male Duchenne muscular dystrophy (DMD) patients who have successfully completed the 96-week eteplirsen study: Study 4658-102 ; Primary end point(s): - Incidence of AEs, SAEs, and discontinuation from treatment due to AEs - Incidence of deaths due to AEs - Incidence of adverse events of special interest (AESIs), including infusion-related reactions, hypersensitivity, and renal events - Clinically significant laboratory testing, including hematology, coagulation, serum chemistry, and urinalysis - Clinically significant cardiac function assessments, including electrocardiogram (ECG) - Clinically significant vital signs - Clinically significant physical examinations ;Timepoint(s) of evaluation of this end point: Baseline to end of study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The efficacy of eteplirsen will be assessed by the change from Baseline to the end of Week 144, Week 192, and Week 284 in the NSAA score. ;Timepoint(s) of evaluation of this end point: Baseline to the end of Week 144, Week 192, and Week 284 | — |
Countries
Belgium, France, United Kingdom
Contacts
Public ContactClinical Trial Information
Voisin Consulting
Outcome results
None listed