Duchenne muscular dystrophy Genetic Diseases
Conditions
Interventions
Participants with DMD who completed either clinical study ENTR-601-44-201 or ENTR-601-45-201 will receive study medication administered intravenously (IV) every 6 weeks for 24 months (ie, ENTR-601-44
Sponsors
Entrada Therapeutics, Inc
Eligibility
Sex/Gender
Male
Age
0 Years to 120 Years
Inclusion criteria
Inclusion criteria: 1. Willing and able to provide consent (if at the age of majority) or assent (if a minor), as required by local regulations or the institutional review board (IRB)/independent ethics committee (IEC) after the nature of the study has been explained and prior to the performance of any study-related procedures 2. For participants under the legal age of consent, parent(s) or guardian(s) must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to the performance of any study-related procedures. Adult participants must be willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to the performance of any study-related procedures. Participants who reach the age of majority in their country while the study is ongoing will be asked to provide their own written consent again upon reaching the legal age of majority. 3. Participant completed clinical study ENTR-601-44- 201 or ENTR-601-45-201 4. Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual intercourse.
Exclusion criteria
Exclusion criteria: 1. Any change from the applicable parent study eligibility criteria, including safety events during the parent study, that in the opinion of the investigator in consultation with the medical monitor and/or sponsor designee precludes safe use of study drug. 2. Participant has a condition or circumstance that in the view of the investigator places the subject at high risk of poor treatment compliance or for not completing the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| To evaluate the long-term safety and tolerability of study drug in participants with DMD assessed by monitoring adverse events, physical examination, electrocardiogram (ECG) parameters, vital signs and clinical laboratory tests. From parent study baseline through End of Study (up to 2 years). | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. To characterise the pharmacokinetics (PK) of study medicine in participants with DMD after long-term dosing by measuring the plasma concentration of study drug compounds and their final metabolite from parent study baseline through End of Study (up to 2 years) 2. To evaluate the impact of study medicine on measures of function in participants with DMD after long-term dosing by measuring: 2.1. Change from parent study Part A and OL (open-label) Period baselines to LTE EOS (long term extension end of study) in 10-Meter Walk/Run (10MWR) from parent study baseline through End of Study (up to 2 years) 2.2. Change from parent study Part A and OL Period baselines to LTE EOS in timed rise from floor (TRF) from parent study baseline through End of Study (up to 2 years) 2.3. Change from parent study Part A and OL Period baselines to LTE EOS in Timed 4-Stair Climb (4SC) from parent study baseline through End of Study (up to 2 years) 2.4. Change from parent study Part A and OL Period baselines to LTE EOS in stride velocity 95th centile (SV95C) from parent study baseline through End of Study (up to 2 years) 2.5. Change from parent study Part A and OL Period baselines to LTE EOS in North Star Ambulatory Assessment (NSAA) from parent study baseline through End of Study (up to 2 years) 2.6. 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 parent study baseline through End of Study (up to 2 years) 3. To evaluate the immune response to study medicine in participants with DMD after long-term dosing by measuring anti-drug antibody (ADA) and anti-dystrophin antibody in serum from parent study baseline through End of Study (up to 2 years) | — |
Countries
Belgium, England, Italy, Netherlands, Spain, United Kingdom
Contacts
Public Contact.;- Entrada Therapeutics Clinical Trials;Medpace Regulatory Submissions
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Outcome results
None listed