ENTR-601-DMD-202 (ELEVATE-LTE)

• Research type

  Research Study

• Full title

  A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)

• IRAS ID

  1013685

• Contact name

  Meghan MacNally

• Contact email

  mmacnally@entradatx.com

• Sponsor organisation

  Entrada Therapeutics, Inc.

• ISRCTN Number

  ISRCTN31690502

• Research summary

  This Long-Term Extension (LTE) is an open-label study following the initial ENTR-601-44-201 and ENTR-601-45-201 studies. There is no placebo. All participants will receive the active study drug at the same dose they were receiving upon completion of the initial study.
  This is a long-term extension study of the investigational medicines ENTR-601-44 and ENTR-601-45 in participants with a rare genetic condition: Duchenne muscular dystrophy (DMD).
  Changes in the genes, called variants, can alter production of certain proteins. As a result of one or more changes to the DMD gene, the body produces little or a non-working dystrophin, a protein needed to help muscle function correctly. ENTR-601-44 and ENTR-601-45 are exon skipping therapies that aim to help the body ‘skip over’ the relevant variant so that the body can produce a shorter but working form of dystrophin.

  The researchers want to: Test over a period of time, how safe ENTR-601-44 and ENTR-601-45 are, learn about any side effects, and look at the potential positive effects of ENTR-601-44 and ENTR-601-45. In this summary, ENTR-601-44 and ENTR-601-45 are both called study treatments.

  This Long-Term Extension (LTE) is an open-label study following the initial ENTR-601-44-201 and ENTR-601-45-201 studies. There is no placebo. All participants will receive the active study drug at the same dose they were receiving upon completion of the initial study.

  The study treatment will be given as an intravenous infusion (slow injection) into a vein. They will receive 18 a set number of doses of the study drug.

• REC name

  South Central - Berkshire Research Ethics Committee

• REC reference

  26/SC/0088

• Date of REC Opinion

  27 Apr 2026

• REC opinion

  Further Information Favourable Opinion