A new phase 1/2 clinical trial, EXPLORE44™ (NCT05670730), to investigate the use of AOC 1044 in healthy adult volunteers and patients with Duchenne muscular dystrophy (DMD) is now recruiting participants at a study location in San Antonio, Texas.

The trial, sponsored by Avidity Biosciences Inc, will enroll patients during 2 different parts of the trial. During part A, healthy adult participants will receive either a single intravenous (IV) infusion at 1 of 5 dose levels or a saline placebo infusion. In part B, patients with DMD with mutations amenable to exon 44 skipping will receive either 3 IV infusions of AOC 1044 at 1 of 3 dose levels or 3 placebo infusions containing saline.

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The study’s primary outcome will be to measure the incidence of treatment-emergent adverse events for both parts of the study. Participants will be monitored for up to 85 days during part A and up to 169 days for part B.

Secondary outcomes will incorporate the measurement of plasma pharmacokinetic parameters including the maximum plasma concentration, terminal half-life, and area under the concentration curve for AOC 1044. Levels of phosphorodiamidate morpholino oligomer for skipping exon 44 (PMO44) will also be measured in the skeletal muscle tissue and urine. Changes from baseline in exon skipping and dystrophin protein levels will also be measured in skeletal muscle samples.

For part A of the study, healthy volunteers must be between 18 and 45 years of age with a BMI between 18.5 and 32.0 kg/m2. Patients with DMD during part B must be between 7 to 27 years of age, with a weight greater than or equal to 30.5 kg, and confirmation of a DMD gene mutation amenable to exon 44 skipping.

The study aims to enroll 5 cohorts of healthy individuals for part A and 3 cohorts of patients with DMD for part B for an estimated enrollment of 64 participants. The study has an estimated completion date of March 2025.

Reference

Study of AOC 1044 in healthy adult volunteers and participants with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping (EXPLORE44). ClinicalTrials.gov. January 4, 2023. Accessed January 25, 2023.