ENTR-601-44-201 (ELEVATE-44)
Research type
Research Study
Full title
A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)
IRAS ID
1010840
Contact name
Meghan MacNally
Contact email
Sponsor organisation
Entrada Therapeutics, Inc.
ISRCTN Number
ISRCTN15088415
Research summary
The study aims to determine if ENTR-601-44 is safe, identify any side effects, and see how well it works on DMD by possibly increasing dystrophin protein production. Dystrophin helps muscles function properly. The study medication is an investigational medication, meaning it hasn’t been approved by The Medicines and Healthcare products Regulatory Agency (MHRA), the health authority that gives approval for new medications in the United Kingdom.
This is a a 2-part, randomized, double-blind, placebo-controlled study in participants with Duchenne muscular dystrophy amenable to exon 44 skipping. Part A will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ENTR-601-44 and Part B will evaluate the safety and efficacy of ENTR-601-44.
Up to 24 people will take part in this part of the study at multiple sites in different countries. Patients will be in this study for about 25 weeks, with approximately 11 study visits to the study site. Patients will be assigned randomly by chanceto receive either ENTR-601-44 or placebo (inactive ingredient).
Patients will have a 75% (3 in 4) chance of receiving ENTR-601-44 and a 25% (1 in 4) chance of receiving placebo.
Study participants may or may not benefit from their participation in this study. However, the results that are obtained from this study may benefit others in the future. Entrada Therapeutics is the Sponsor that is responsible for funding this clinical study.REC name
South Central - Oxford A Research Ethics Committee
REC reference
24/SC/0403
Date of REC Opinion
27 Jan 2025
REC opinion
Further Information Favourable Opinion