This is a Phase 1, double-blind, placebo-controlled, single ascending dose cohort study to evaluate the safety, tolerability, and plasma concentrations of WVE-210201 in ambulatory and non-ambulatory male pediatric patients with DMD amenable to exon 51 skipping intervention.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
36
WVE-210201 is a stereopure antisense oligonucleotide (ASO)
Sodium Chloride
Rare Disease Research, LLC.
Atlanta, Georgia, United States
UZ Gent
Ghent, Belgium
Universitaire Ziekenhuizen Leuven
Leuven, Belgium
Safety: Number of patients with adverse events (AEs)
Time frame: Day 1 to Day 85 (end of study)
Safety: Severity of AEs
Time frame: Day 1 to Day 85 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Time frame: Day 1 to Day 85 (end of study)
Safety and Tolerability: Number of patients who withdraw due to AEs
Time frame: Day 1 to Day 85 (end of study)
Pharmacokinetics (PK): Maximum observed concentration (Cmax)
Time frame: Day 1, Day 2, and Day 8
PK: Time of occurrence of Cmax (tmax)
Time frame: Day 1, Day 2, and Day 8
PK: Area under the plasma concentration-time curve (AUC 0-t)
Time frame: Day 1, Day 2, and Day 8
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CHR de la Citadelle
Liège, Belgium
London Health Sciences Centre - Hospital
London, Ontario, Canada
Hôpital Armand Trousseau
Paris, France
U.O.C di Neurologia e Malattie Neuromuscolari Centro Clinico Nemo Sud
Messina, Italy
U.O. Immunologia Pediatrica
Milan, Italy
Radbound University Nijmegen Medical Care
Nijmegen, Netherlands
University Hospitals Bristol NHS Foundation Trust
Bristol, United Kingdom
...and 3 more locations