Phase 3 Study on Long-Term Safety and Efficacy of Intravenous AOC 1001 in Myotonic Dystrophy Type 1

The primary objective of this phase 3 open-label extension study is to evaluate the long-term safety and tolerability of intravenous AOC 1001 in patients diagnosed with myotonic dystrophy type 1. This investigation is intended to monitor the continued physiological response and risk profile associated with the administration of a humanised IgG1 monoclonal antibody conjugated to a double stranded siRNA oligonucleotide targeting DMPK. The study also aims to assess the long-term efficacy of the therapeutic intervention.

The study population consists of 171 participants diagnosed with myotonic dystrophy type 1. The cohort includes both male and female subjects, encompassing vulnerable populations. Based on the provided age range codes, the participants include children and adolescents. Selection is contingent upon the ability to provide written informed consent or obtain consent from a legally authorized representative for minors. Additionally, participants must have successfully completed any prior studies involving AOC 1001 with satisfactory dosing, follow-up assessments, and protocol compliance.

This Phase 3 open-label extension study is designed to evaluate the long-term safety, tolerability, and efficacy of intravenous AOC 1001 in patients diagnosed with myotonic dystrophy type 1. The research methodology focuses on assessing the incidence of treatment-emergent adverse events as the primary endpoint. Participants must have completed any prior AOC 1001 studies with satisfactory dosing and follow-up assessments. The trial is estimated to conclude by October 30, 2030. The study protocol involves monitoring patients over an extended period to observe the long-term effects of the humanised igg1 monoclonal antibody against tfr1 conjugated to double stranded sirna oligonucleotide against dmpk via a non-cleavable linker.

The investigational product is AOC 1001, a humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DMPK via a non-cleavable linker. This substance is prepared as a solution for infusion and is administered via intravenous route at a dosage of 4 mg/kg.

The assessment of efficacy for the treatment of Myotonic Dystrophy Type 1 involves the evaluation of clinical parameters in patients receiving intravenous administration of AOC 1001. The study is designed as a Phase 3 open-label extension to monitor long-term outcomes.