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
Product Name: Eteplirsen
Product Code: AVI-4658
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: eteplirsen
CAS Number: 1173755-55-9
Current Sponsor code: AVI-4658
Other
Sponsors
Sarepta Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Be a male between 6 months to 48 months of age, inclusive. 2. Have an established clinical diagnosis of DMD with a deletion mutation amenable to exon 51 skipping (eg, deletions of exons 45-50, 47-50, 48-50, 49-50, 50, 52, 52-63). 3. Have a parent(s) or legal guardian(s) who is able to understand and comply with the study requirements and is willing to provide written informed consent for the patient to participate in the study. 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. Has received any pharmacologic treatment that might have an effect on muscle strength or function within 12 weeks prior to dosing at Week 1 (eg, growth hormone, anabolic steroids). 2. Has received previous or current treatment with any experimental treatment. Prior drisapersen therapy is permitted if a patient has not received drisapersen for 6 months prior to the Week 1 dose. 3. Has a clinically significant illness other than DMD, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, behavioural disease, or malignancy likely to impair the patient’s ability to participate in this study. 4. Has a clinically significant laboratory abnormality that is either not expected or is of a greater severity than what is expected in DMD patients. 5. Has any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the study.
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): PK of eteplirsen by population PK methods, including assessment of the following PK parameters (if evaluable): • Maximum plasma concentration (Cmax) • Time of Cmax (Tmax) • AUC • Apparent volume of distribution at steady state (Vss) • Clearance (CL) • Elimination half-life (t½) • Amount of drug eliminated in urine (Ae%);Timepoint(s) of evaluation of this end point: PK plasma and uring sampling: Weeks 2, 6, 8, 10, 24 | — |
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: To determine the pharmacokinetics (PK) of eteplirsen at the 2-, 10-, 20 and 30-mg/kg dose levels, administered once weekly by IV infusion in male DMD patients ages 6 months to 48 months, inclusive;Primary end point(s): Safety and tolerability of eteplirsen as measured by: • Incidence of adverse events (AEs) • Abnormal changes from Baseline or clinically significant worsening of clinical safety laboratory abnormalities (hematology, chemistry, coagulation, and urinalysis) • Abnormal changes from Baseline or worsening of vital signs • Abnormal changes from Baseline or worsening of physical examination findings • Abnormal changes from Baseline or clinically significant worsening of ECGs and ECHOs;Timepoint(s) of evaluation of this end point: Incidence of AEs: continous Clinical safety laboratory sample: Screening, Weeks 2, 4, 8, 12, 24, 36, 48, 60, 72, 84, 96 and End-of-Study Follow-up Visit Vital signs: Screening, baseline, on weekly infusion days, Week 96 and End-of-Study Follow-up Visit Physical examination: Screening, baseline, Weeks 4, 8, 12, 16, 20-48, 60, 72, 84, 96 and End-of-Study Follow-up Visit ECG: Screening, Weeks 8, 12, 24, 36, 48, 60, 72, 84, 96 ECHO: Screening, Weeks 8, 12, 24, 36, 48, 60, 72, 96;Main Objective: To evaluate the safety and tolerability of eteplirsen administered once weekly by intravenous (IV) infusion in male Duchenne muscular dystrophy (DMD) patients ages 6 months to 48 months, inclusive | — |
Countries
Belgium, France, Germany, Italy, United Kingdom
Contacts
Public ContactClinical Trial Information
Voisin Consulting
Outcome results
None listed