ENTR-601-50-201 (ELEVATE-50-201)
Research type
Research Study
Full title
A Multiple Ascending Dose, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 50 Skipping to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-50 (ELEVATE-50-201)
IRAS ID
1012449
Contact name
Meghan MacNally
Contact email
Sponsor organisation
Entrada Therapeutics, Inc.
Research summary
The study aims to determine if ENTR-601-50 is safe, identify any side effects, and see how well it works on DMD by measuring how much dystrophin is produced after treatment. Dystrophin helps muscles function properly. This is a randomised, double-blind, placebo-controlled, multiple ascending dose study in participants with Duchenne muscular dystrophy amenable to exon 50 skipping.
Up to 24 people will take part in the study. Patients will be in this study for about 25 weeks.
The study is evaluating three separate dose levels of ENTR-601-50 compared to placebo. A placebo looks like a study drug but does not have any medicine in it.
Each participant will be randomised to receive one of the dose levels of ENTR-601-50 or the placebo. There is a 75% chance that a participant is assigned to receive the study drug and a 25% chance they are assigned to receive placebo.
The study doctor will administer the study drug or the placebo via intravenous (IV) infusion approximately once every six weeks. Additional visits will occur throughout the study to learn more about the safety and potential impact of ENTR-601-50 on the body's functions.
Participants will have a muscle biopsy at the beginning of their participation in the study and after their last dose. Muscle biopsies are important because they allow researchers to compare whether there have been changes in the muscle as a result of the study drug.
Participants who complete the study will have an opportunity to continue dosing in an open-label (OL) treatment period, in which the safety, tolerability, and clinical benefits of ENTR-601-50 will be further assessed over a longer period of time, provided treatment is tolerated. Information on an OL period will be provided as participants approach the end of participation in this study.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
25/NW/0310
Date of REC Opinion
18 Nov 2025
REC opinion
Further Information Favourable Opinion