Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping (EXPLORE44)
Duchenne Muscular Dystrophy, Exon 44
About this trial
This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring EXPLORE44, Avidity Biosciences, Avidity, AOC 1044
Eligibility Criteria
Part A: Key Inclusion Criteria: Aged 18 to 55 years, inclusive, at the time of informed consent Body mass index (BMI) of 18.5 to 32.0 kg/m2 Key Exclusion Criteria: Clinically significant abnormalities in laboratory results, ECGs, or vitals Current or recent use of prescription or nonprescription drugs Positive drug/alcohol test at Screening or Day -1 Elevated blood pressure (BP) >130/80 mmHg at Screening Participation in a clinical study in which an investigational product was received within 1 month of screening or 5 half-lives of the investigational product Blood or plasma donation within 16 weeks of planned AOC 1044 administration Note: Other protocol defined Inclusion/Exclusion criteria may apply Part B: Key Inclusion Criteria: Aged 7 to 27 years, inclusive, at the time of informed consent Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years Confirmation of DMD gene mutation amenable to exon 44 skipping Weight ≥ 23 kg Ambulatory or non-ambulatory Ambulatory participants: LVEF ≥50% and FVC≥50% Non-ambulatory participants: LVEF ≥45% and FVC≥40% PUL 2.0 entry item A ≥3 If on corticosteroids, stable dose for 30 days before screening and throughout the study Key Exclusion Criteria: Biceps brachii muscles unsuitable for biopsy Serum hemoglobin < lower limit of normal Uncontrolled hypertension or diabetes Prior treatment with any cell or gene therapy Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent Recently treated with an investigational drug History of multiple drug allergies
Sites / Locations
- Stanford UniversityRecruiting
- Rare Disease Research - AtlantaRecruiting
- Worldwide Clinical Trials (Part A only)Recruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Experimental
Placebo Comparator
Experimental
Placebo Comparator
AOC 1044-CS1 Part A - Single Dose Levels 1-5
AOC 1044-CS1 Part A - Single Dose: Placebo
AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3
AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo
AOC 1044 will be administered once.
Placebo will be administered once.
AOC 1044 will be administered three times.
Placebo will be administered three times.