A Phase II, open-label long-term extension study in participants with Duchenne muscular dystrophy amenable to exon skipping to assess the long-term safety, tolerability, pharmacokinetics, and efficacy of endosomal escape vehicle phosphorodiamidate morpholino oligomer platform products (ELEVATE-LTE)

Registry ID
ISRCTN31690502
Source registry
ISRCTN
Status
Not yet recruiting
Phase
PHASE2
Study type
INTERVENTIONAL
Sponsor
Entrada Therapeutics, Inc
Enrollment
80
Start date
2026-10-28
Completion date
2032-03-31
Last update
2026-08-17

Conditions

Summary

Duchenne muscular dystrophy

Detailed description

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 for participants continuing from Study ENTR-601-44-201 and ENTR-601-45 for participants continuing from Study ENTR-601-45-201). Participants enrolling in ENTR-601-DMD-202 will begin the 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. After review of the cohort’s benefit-risk profile, participants may escalate to the higher dose that is deemed favourable compared with the preceding dose.

Interventions

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

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.

Locations

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