ENTR-601-45 for Duchenne muscular dystrophy

Inclusion criteria

• {"criterion_text":"- 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."}
• {"criterion_text":"- Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator."}
• {"criterion_text":"- Part A: 4-20 years of age"}
• {"criterion_text":"- Ambulatory Status Part A: ambulatory with a specific Performance of the Upper Limb v2.0 (PUL 2.0) Entry item at Screening"}
• {"criterion_text":"- Adequate muscle for obtaining tissue biopsy as assessed by the investigator."}
• {"criterion_text":"- Other protocol-defined criteria apply"}

Exclusion criteria

• {"criterion_text":"- Any significant concomitant medical condition that interfere with the ability to comply with protocol requirements"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- 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"}
• {"criterion_text":"- Laboratory abnormalities"}
• {"criterion_text":"- Daytime ventilator dependence, or any use of invasive mechanical ventilation via tracheostomy."}
• {"criterion_text":"- 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."}
• {"criterion_text":"- Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer)."}
• {"criterion_text":"- 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)."}
• {"criterion_text":"- Other protocol-defined criteria apply."}

Primary endpoints

• {"endpoint_text":"- Incidence and severity of treatment emergent adverse events (TEAEs) (Part A and OL Period)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Changes in vital sign measurements (Part A and OL Period)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Changes in clinical laboratory results (Part A and OL Period)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Changes in electrocardiogram (ECG) parameters (Part A and OL Period)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Changes in physical examination findings (Part A and OL Period)","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Plasma, muscle, and urine concentration of ENTR-601-45 and its final metabolite (Part A and OL Period)","definition_or_measurement_approach":"Concentrations measured in plasma, muscle and urine (PK sampling in Part A and OL Period)."}
• {"endpoint_text":"- Change from baseline to End of Part A in dystrophin by Western blot from muscle biopsy (Part A)","definition_or_measurement_approach":"Dystrophin quantified by Western blot on muscle biopsy samples (baseline vs End of Part A)."}
• {"endpoint_text":"- Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)","definition_or_measurement_approach":"Assessment of dystrophin expression and localization from muscle biopsy (baseline vs End of Part A)."}
• {"endpoint_text":"- Percent change from baseline to End of Part A in exon 45 skipping measured in muscle biopsy (Part A)","definition_or_measurement_approach":"Exon 45 skipping measured in muscle biopsy; percent change from baseline to End of Part A."}
• {"endpoint_text":"- Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period)","definition_or_measurement_approach":"Serum ADA and anti-dystrophin antibodies measured (Part A and OL Period)."}
• {"endpoint_text":"- Change from baseline to End of OL Period in 10-Meter Walk/Run (10MWR) (Part A and OL Period)","definition_or_measurement_approach":"Change in 10-Meter Walk/Run time from baseline to End of OL Period."}
• {"endpoint_text":"- Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period)","definition_or_measurement_approach":"Change in Timed Rise from Floor from baseline to End of OL Period."}
• {"endpoint_text":"- Change from baseline to End of OL Period in Timed 4-Stair Climb (4SC) (Part A and OL Period)","definition_or_measurement_approach":"Change in Timed 4-Stair Climb time from baseline to End of OL Period."}
• {"endpoint_text":"- Change from baseline to End of OL Period in 95th centile Stride Velocity (SV95C) (Part A and OL Period)","definition_or_measurement_approach":"Change in SV95C from baseline to End of OL Period (gait/stride velocity metric)."}
• {"endpoint_text":"- Change from baseline to End of OL Period in North Star Ambulatory Assessment (NSAA) (Part A and OL Period)","definition_or_measurement_approach":"Change in NSAA score from baseline to End of OL Period."}
• {"endpoint_text":"- Change from baseline to End of OL Period in Performance of the Upper Limb v2.0 (PUL 2.0) (Part A and OL Period)","definition_or_measurement_approach":"Change in PUL 2.0 score from baseline to End of OL Period."}

Methods

• Brochure materials (country-specific brochures available; used to inform patients/families).
• Email blast for advocacy groups (documents titled 'Email blast for Advocacy groups' present - target: advocacy groups / patient organisations).
• FAQ sheet for advocacy groups (target: advocacy groups / patient organisations).
• Posters (study posters for patient/family outreach).
• Social media ads (digital outreach via social platforms).
• Website (study website materials and cookie banner / Usercentrics cookie banner).
• Video script / video material (scripted videos for recruitment).
• Direct outreach via advocacy groups (materials prepared to be shared with advocacy/registry networks).

Netherlands

Earliest CTIS Part Ii Submission Date

21-04-2025

Latest Decision Or Authorization Date

11-02-2026

Processing Time Days

296

Number Of Sites

2

Number Of Participants

3

Spain

Earliest CTIS Part Ii Submission Date

25-04-2025

Latest Decision Or Authorization Date

11-02-2026

Processing Time Days

292

Number Of Sites

2

Number Of Participants

3

Belgium

Earliest CTIS Part Ii Submission Date

16-04-2025

Latest Decision Or Authorization Date

11-02-2026

Processing Time Days

301

Number Of Sites

3

Number Of Participants

4

Italy

Earliest CTIS Part Ii Submission Date

21-04-2025

Latest Decision Or Authorization Date

11-02-2026

Processing Time Days

296

Number Of Sites

3

Number Of Participants

4

Primary sponsor

Full Name

Entrada Therapeutics Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Medpace Finland Oy

Responsibilities

sponsorDuties codes: [1,12,15 (Imaging),4,5,6,8]; contact email: RS-Advisor-Support@medpace.com

Name

PPD Development LP

Responsibilities

sponsorDuties codes: [4]; contact email: carrie.mootz@ppd.com

Name

QPS LLC

Responsibilities

sponsorDuties codes: [4]; contact email: John.Kolman@qps.com

Name

Flagship Biosciences Inc.

Responsibilities

sponsorDuties codes: [4]; contact email: bfrey@flagshipbio.com

Name

Agada Biosciences Inc.

Responsibilities

sponsorDuties codes: [4]; contact email: amackinnon@agadabio.com

Name

Trinds LLC

Responsibilities

sponsorDuties codes: [13]; contact email: sshella@trinds.com

Name

Illingworth Research Group Limited

Responsibilities

sponsorDuties codes: [15] value: Home Health Services; contact email: taylor.ross@syneoshealth.com

Name

ATOM International Limited

Responsibilities

sponsorDuties codes: [15] value: Physical Function Oversight; contact email: kyle.haas@atom-international.org

Third parties

• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"sponsorDuties codes: [1,12,15 (Imaging),4,5,6,8]; contact email: RS-Advisor-Support@medpace.com","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Agada Biosciences Inc.","duties_or_roles":"sponsorDuties codes: [4]; contact email: amackinnon@agadabio.com","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"sponsorDuties codes: [15] value: Home Health Services; contact email: taylor.ross@syneoshealth.com","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Flagship Biosciences Inc.","duties_or_roles":"sponsorDuties codes: [4]; contact email: bfrey@flagshipbio.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: [4]; contact email: carrie.mootz@ppd.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Trinds LLC","duties_or_roles":"sponsorDuties codes: [13]; contact email: sshella@trinds.com","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"sponsorDuties codes: [15] value: Physical Function Oversight; contact email: kyle.haas@atom-international.org","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"QPS LLC","duties_or_roles":"sponsorDuties codes: [4]; contact email: John.Kolman@qps.com","organisation_type":"Pharmaceutical company"}