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ENTR-601-45 · ELEVATE-45

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45

Ensayo de Entrada Therapeutics, Inc. en Distrofia muscular de Duchenne.

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 1/2

Estado

Reclutando pacientes

Participantes

24

previstos

Centros

15

Fin del objetivo primario

mar 2029

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en ago 2025.

Qué mide

Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period). (From baseline through End of Study (up to 62 weeks).) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments. The study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A. Participants will be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated. Participants will: * Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B * Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug. Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.

Más ensayos de TRDA

Todos los de TRDA, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT07038824), actualizada en ago 2026.