Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)

Condition(s) studied

Investigational drug(s) / intervention(s)

ENTR-601-44: ENTR-601-44 intravenous infusion

ENTR-601-45: ENTR-601-45 intravenous infusion

Study summary

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:

* Receive study treatment in the form of multiple intravenous (IV) infusions (slow injections) into a vein over the course of several weeks
* Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and excersice tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a results of the study drug.

Participants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.

Eligibility

Primary outcome measure(s)

• Number of participants with Treatment Emergent Adverse Events (TEAEs) according to study protocol (Part A and OL Period) — From baseline through End of Study (up to 2 years).
  Safety will be assessed by monitoring adverse events, physical examination, vital signs and clinical laboratory tests.

Trial sites (17)