Duchenne muscular dystrophy (DMD) Genetic Diseases
Conditions
Interventions
Part A
Experimental Arm: ENTR-601-44.
• Participants will receive a fixed number of doses at one of three dose levels. One dose will be given every six
weeks.
• Drug: ENTR-601-44: Given by IV infusion
or if they were on placebo, cross over to ENTR-601-44 at the dose level that was administered in their original cohort.
Sponsors
Entrada Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Age
4 Years to 20 Years
Inclusion criteria
Inclusion criteria: Principal inclusion criteria: 1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor. 2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator. 3. Part A: 4-20 years of age, inclusive. 4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening 5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator. 6. Other protocol-defined criteria apply.
Exclusion criteria
Exclusion criteria: Principal exclusion criteria: 1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements. 2. 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. 3. Use of the following medications: 3.1. Prior treatment with any exon skipping therapy at any time 3.2. Prior treatment with any gene therapy at any time 3.3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from at least 30 days prior to the start of the screening period until the end of the study 3.4. Use of an immunosuppressant for a non-DMD condition from 30 days prior to screening until the end of the study 3.5. 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 4. Laboratory abnormalities. 5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy. 6. 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. 7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer). 8. Other protocol-defined criteria apply.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability of ENTR-601-44 measured using incidence and severity of treatment-emergent adverse events (TEAEs); changes in vital sign measurements, clinical laboratory results, electrocardiogram (ECG) parameters, physical examination findings from baseline through End of Study visit | — |
Secondary
| Measure | Time frame |
|---|---|
| Current secondary outcomes as of 02/01/2026: 1. Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite at timepoints as specified in the study protocol 2. Change from baseline in dystrophin by Western blot from muscle biopsy at End of Study and Part A 3. Change from baseline in dystrophin expression and localization from muscle biopsy at End of Study and Part A 4. Percent change from baseline in exon 44 skipping measured in muscle biopsy at End of Study and Part A 5. Anti-drug antibody (ADA) and anti-dystrophin antibody in serum at Baseline, End of Study, and additional timepoints as specified in the study protocol 6. Change from baseline to End of OL Period in 10 Metre Walk/Run (10MWR) 7. Change from baseline to End of OL period in Timed Rise from Floor 8. Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) 9. Change from baseline to End of OL period in Stride Velocity 95th Centile (SV95C) 10. Change from baseline to End of OL Period in North Star 11. Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) Previous secondary outcomes: 1. Plasma, muscle, and urine concentration of ENTR-601-44 and its final metabolite at timepoints as specified in the study protocol 2. Change from baseline in dystrophin by Western blot from muscle biopsy at End of Study 3. Change from baseline in dystrophin expression and localization from muscle biopsy at End of Study 4. Percent change from baseline in exon 44 skipping measured in muscle biopsy at End of Study 5. Anti-drug antibody (ADA) and anti-dystrophin antibody in serum at Baseline, End of Study, and additional timepoints as specified in the study protocol | — |
Countries
Belgium, Italy, Spain, United Kingdom
Contacts
Public Contact. Entrada Therapeutics Clinical Trials
Outcome results
None listed