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)

Status: Recruiting
Location: See all (14) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-44 is, learn about any side effects, and look at the potential positive effects of ENTR-601-44, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-44 and placebo are both called study treatments. The study has 2 parts: Part A: to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-44 at the dose determined in Part A. 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, 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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 4
Maximum Age: 20
Healthy Volunteers: f
View:

• Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.

• Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.

• Part A: 4-20 years of age, inclusive.

• Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening

• Adequate muscle for obtaining tissue biopsy as assessed by the investigator.

• Other protocol-defined criteria apply.

Locations
Other Locations
Belgium
University Hospital Gent
RECRUITING
Ghent
UZ Leuven
RECRUITING
Leuven
Centre Hospitalier Régional de la Citadelle
NOT_YET_RECRUITING
Liège
Italy
Fondazione Serena Onlus - Centro Clinico NeMO Milano
NOT_YET_RECRUITING
Milan
IRCCS Ospedale San Raffaele
NOT_YET_RECRUITING
Milan
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
NOT_YET_RECRUITING
Rome
Ospedale Pediatrico Bambino Gesu
NOT_YET_RECRUITING
Rome
Spain
Hospital Sant Joan de Deu
NOT_YET_RECRUITING
Barcelona
Hospital Universitario Vall d'Hebron
RECRUITING
Barcelona
United Kingdom
Leeds General Infirmary
NOT_YET_RECRUITING
Leeds
Alder Hey Children's NHS Foundation Trust
NOT_YET_RECRUITING
Liverpool
Great Ormond Street Hospital for Children
NOT_YET_RECRUITING
London
Freeman Hospital
NOT_YET_RECRUITING
Newcastle Upon Tyne
Oxford University Hospitals NHS Foundation Trust
NOT_YET_RECRUITING
Oxford
Time Frame
Start Date: 2025-06-30
Estimated Completion Date: 2029-03-28
Participants
Target number of participants: 24
Treatments
Experimental: ENTR-601-44
intravenous infusion every 6 weeks
Placebo_comparator: Placebo
intravenous infusion every 6 weeks
Sponsors
Leads: Entrada Therapeutics, Inc.

This content was sourced from clinicaltrials.gov

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