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| ID | Type | Description | Link |
|---|---|---|---|
| 2025-525124-10-00 | EU Trial (CTIS) Number | ||
| U1111-1334-9287 | Other Identifier | WHO |
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This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).
The investigational medicines are currently being investigated in multiple ascending dose parent studies. After participants complete their respective parent study, there is a need to understand the effects of long-term administration of ENTR-601-44 and ENTR-601-45.
Participants enrolling in this study will begin this long-term extension (LTE) study at the dose level they received upon completion of the parent study with possible dose escalation in the LTE study based on emerging safety and efficacy data from the parent studies.
Participants will:
Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ENTR-601-44 | Experimental | ENTR-601-44 intravenous infusion every 6 weeks |
|
| ENTR-601-45 | Experimental | ENTR-601-45 intravenous infusion every 6 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ENTR-601-44 | Drug | ENTR-601-44 intravenous infusion |
| |
| ENTR-601-45 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period) | Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests. | From baseline through End of Study (up to 2 years). |
| Measure | Description | Time Frame |
|---|---|---|
| Plasma concentration of study drug compounds and their final metabolite | From baseline through End of Study (up to 2 years). | |
| Change from parent study Part A and OL Period baselines to LTE EOS in 10-Meter Walk/Run (10MWR) | From baseline through End of Study (up to 2 years). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Entrada Therapeutics Clinical Trials | Entrada Therapeutics, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospital Gent | Ghent | 9000 | Belgium | |||
| UZ Leuven |
The datasets generated during and/or analyzed during the current study are not expected to be made available due to the data´s high commercial sensitivity.
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Participants with DMD who completed either clinical study ENTR-601-44-201 or ENTR-601-45-201 will begin the Long Term Extension (LTE) at the dose level they received upon completion of the open-label portion of the parent study.
Dose escalation in the LTE study may be permitted based on emerging safety and efficacy data from the parent studies.
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| Drug |
ENTR-601-45 intravenous infusion |
|
| Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF) | From baseline through End of Study (up to 2 years). |
| Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC) | From baseline through End of Study (up to 2 years). |
| Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C) | From baseline through End of Study (up to 2 years). |
| Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA) | From baseline through End of Study (up to 2 years). |
| Change from parent study Part A and OL Period baselines to LTE EOS in Performance of the Upper Limb v2.0 (PUL 2.0) | From baseline through End of Study (up to 2 years). |
| Anti-drug antibody (ADA) and anti-dystrophin antibody in serum | From baseline through End of Study (up to 2 years). |
| Leuven |
| 3000 |
| Belgium |
| Centre Hospitalier Régional de la Citadelle | Liège | 4000 | Belgium |
| IRCCS Ospedale San Raffaele | Milan | 20132 | Italy |
| Fondazione Serena Onlus - Centro Clinico NeMO Milano | Milan | 20162 | Italy |
| Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore | Roma | 00168 | Italy |
| Ospedale Pediatrico Bambino Gesu | Rome | 00165 | Italy |
| Leids Universitair Medisch Centrum | Leiden | 2333 ZA | Netherlands |
| Stichting Radboud Universitair Medisch Centrum | Nijmegen | 6525 GA | Netherlands |
| Hospital Universitario Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital Sant Joan de Deu | Barcelona | 08950 | Spain |
| Leeds General Infirmary | Leeds | LS1 3EX | United Kingdom |
| Alder Hey Children's NHS Foundation Trust | Liverpool | L122AP | United Kingdom |
| Great Ormond Street Hospital for Children | London | WC1N 3JH | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | M13 9WL | United Kingdom |
| Freeman Hospital | Newcastle upon Tyne | NE1 3BZ | United Kingdom |
| Oxford University Hospitals NHS Foundation Trust | Oxford | OX3 9DU | United Kingdom |
| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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