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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)
EU CTIS ID: 2024-517499-39-00
What this study is testing
Part A and OL Period: To evaluate the safety and tolerability of ENTR-601-45 in participants with Duchenne muscular dystrophy (DMD)
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene that is amenable to exon 45 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
- Ambulatory Status Part A: ambulatory with a specific Performance of the Upper Limb v2.0 (PUL 2.0) Entry item at Screening
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
- Other protocol-defined criteria apply
You likely can't join if
- Any significant concomitant medical condition that 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: a. Prior or current treatment with any exon skipping therapy within the previous 12 months b. Prior or current treatment with any gene therapy c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than systemic or oral corticosteroids for chronic non-DMD conditions) e. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
- 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.
See the full eligibility criteria
- Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene that is amenable to exon 45 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
- Ambulatory Status Part A: ambulatory with a specific Performance of the Upper Limb v2.0 (PUL 2.0) Entry item at Screening
- Adequate muscle for obtaining tissue biopsy as assessed by the investigator.
- Other protocol-defined criteria apply
- Any significant concomitant medical condition that 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: a. Prior or current treatment with any exon skipping therapy within the previous 12 months b. Prior or current treatment with any gene therapy c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than systemic or oral corticosteroids for chronic non-DMD conditions) e. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat
- 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).
- Use of any pharmacologic treatment, other than stable corticosteroids, that might have had an effect on muscle strength or function during the study (eg, growth hormone, testosterone).
- Other protocol-defined criteria apply.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.