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ENTR-601-45-201

Entrada - ELEVATE-45

A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45 ( ELEVATE-45 )

Locations:
recruiting
Leeds
,
recruiting
Oxford
,
recruiting
London - GOSH
,
recruiting
Alder Hey
,

This 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 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:
•    Receive study treatment in the form of an intravenous (IV) infusion (slow injection) into a vein for several weeks 
•    Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise tests

Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.

Participants may be eligible to enter an open label extension study (OLE). An open label extension study allows participants to continue receiving the study drug, which helps researchers better understand the safety, tolerability and efficacy of ENTR-601-45 over a longer period of time. All participants in the open label extension will receive ENTR-601-45.

 
Primary Outcome Measures

Overall safety and tolerability of ENTR-601-45

 
Secondary Outcome Measures

To see how safe ENTR-601-45 is compared to placebo

To look at what ENTR-601-45 does to the body (pharmacodynamics).

To look at how the body interacts with ENTR-601-45 (pharmacokinetics).

Determine the optimal dose for further study in Part B. Additional details on Part B will become available as we approach its expected start.

 
Can I take part?

 
Inclusion Criteria

  • Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counsellor.
  • 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.

 
Exclusion Criteria

  • Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.
  • Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant’s safety.
  • Use of the following medications:
    • Prior treatment with any exon skipping therapy within the previous 12 months (the PI must approve with agreement from the sponsor)
    • Prior treatment with any gene therapy at any time
    • Use of anti-coagulants, anti-thrombotics, or anti-platelet agents
    • Use of immunosuppressants (including systemic or oral corticosteroids for chronic non-DMD conditions) from at least 30 days prior to the start of the screening period until the end of the study
    • Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from at least 30 days prior to the start of the screening period until the end of the study
  • Laboratory abnormalities.
  • Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.
  • Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and/or a QT interval with Fridericia correction method (QTcF) >450 msec at Screening or prior to the first dose of study drug on Day 1.
  • Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).

Other protocol-defined criteria apply.

 

Age

4 - 20 years

Mutation Specific

Mutation Specific

Muscle Biopsy

Required

Ambulation

ambulant