ENTR-601-44 for Duchenne muscular dystrophy

Inclusion criteria

• {"criterion_text":"- Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 44 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, inclusive"}
• {"criterion_text":"- Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol 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 might 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 treatment with any exon skipping therapy at any time b. Prior treatment with any gene therapy at any time From at least 30 days prior to the start of the screening period until the end of the study: c. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents d. Use of immunosuppressants (other than oral corticosteroids for DMD conditions) e. Has taken or is currently taking 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":"- 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-601-44 and its final metabolite (Part A and OL Period)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Change from baseline in dystrophin by Western blot from muscle biopsy (Part A)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Change from baseline to End of Part A in dystrophin expression and localization from muscle biopsy (Part A)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Percent change from baseline to End of Part A in exon 44 skipping measured in muscle biopsy (Part A)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Anti-drug antibody (ADA) and anti-dystrophin antibody in serum (Part A and OL Period))","definition_or_measurement_approach":""}
• {"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":""}
• {"endpoint_text":"- Change from baseline to End of OL Period in Timed Rise from Floor (Part A and OL Period)","definition_or_measurement_approach":""}
• {"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":""}
• {"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":""}
• {"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":""}
• {"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":""}

Methods

• Country-specific recruitment arrangements documents (K1) for Belgium, Spain, Italy.
• Brochures (K2) in English, French, Dutch targeted at potential participants/caregivers.
• Posters in multiple languages for site and public display.
• Social media advertisements in English, French, Dutch.
• Website pages (multi-language) and website cookie banner (Usercentrics) for online recruitment.
• Email blasts targeted to advocacy groups (materials available in English, French, Dutch).
• FAQ sheets for advocacy groups and video scripts for outreach.

Belgium

Earliest CTIS Part Ii Submission Date

28-03-2025

Latest Decision Or Authorization Date

19-01-2026

Processing Time Days

297

Number Of Sites

3

Number Of Participants

3

Spain

Earliest CTIS Part Ii Submission Date

24-02-2025

Latest Decision Or Authorization Date

19-01-2026

Processing Time Days

329

Number Of Sites

2

Number Of Participants

2

Italy

Earliest CTIS Part Ii Submission Date

17-01-2025

Latest Decision Or Authorization Date

08-04-2026

Processing Time Days

446

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

PPD Development LP

Responsibilities

sponsorDuties codes: 4

Name

Medpace Finland Oy

Responsibilities

sponsorDuties codes: 1,12,14,15 (Imaging),4,5,6,8

Name

QPS LLC

Responsibilities

sponsorDuties codes: 4

Name

Precision For Medicine Inc.

Responsibilities

sponsorDuties codes: 4

Third parties

• {"country":"United States","full_name":"Trinds LLC","duties_or_roles":"sponsorDuties codes: 13","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Alliance Pharma Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"ATOM International Limited","duties_or_roles":"Physical Function Oversight","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"sponsorDuties codes: 1, 12, 14, 15 (Imaging), 4, 5, 6, 8","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Flagship Biosciences Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Canada","full_name":"Agada Biosciences Inc.","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"Home Health Services","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"QPS LLC","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Pharmaceutical company"}