Active Studies

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.

The purpose of this trial is to compare the morbidity and mortality of CRS-HIPEC using
mitomycin-C versus melphalan for colorectal peritoneal carcinomatosis. Morbidity and
mortality will measured using the Comprehensive Complication Index (CCI) score, Common
terminology criteria for adverse events (CTCAE version 4.03), and the Clavien-Dindo

The purpose of this study is to test how different doses of a statin, Lipitor, affect muscle
health and function, and cardiovascular fitness.

The study is designed to compare two methods of delivering short (~10 min) physical activity
breaks that will occur during the regular school day for 3 years. Schools will be randomized
to deliver physical activity breaks led by either the regular classroom teacher or by a
remote instructor through a video in the classroom. Both delivery methods will promote a
variety of whole body movements using large muscle groups. The schools will not be able to
choose which group (regular teacher or remote instructor) they will be part of.

The purpose of this study is to compare relapse-free survival between participants with
FLT3/ITD AML in first morphologic complete remission (CR1) who undergo hematopoietic stem
cell transplant (HCT) and are randomized to receive gilteritinib or placebo beginning after
the time of engraftment for a two year period.