ENTR-601-45-201 (ELEVATE-45)

• Research type

  Research Study

• Full title

  A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants with Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping with an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)

• IRAS ID

  1010846

• Contact name

  Meghan MacNally

• Contact email

  mmacnally@entradatx.com

• Sponsor organisation

  Entrada Therapeutics, Inc.

• Eudract number

  2024-517499-39

• ISRCTN Number

  ISRCTN18511908

• Research summary

  The study aims to determine if ENTR-601-45 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 45 skipping. Part A will assess the safety, tolerability, pharmacokinetics and pharmacodynamics of ENTR-601-45 and Part B will evaluate the safety and efficacy of ENTR-601-45.
  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 chance to receive either ENTR-601-45 or placebo (inactive ingredient).
  Patients will have a 75% (3 in 4) chance of receiving ENTR-601-45 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

  London - Brent Research Ethics Committee

• REC reference

  25/LO/0074

• Date of REC Opinion

  3 Feb 2025

• REC opinion

  Further Information Favourable Opinion