Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping

NCT05670730 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: AOC 1044-CS1
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 10

Brief Summary

AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers. Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.

Conditions

Duchenne Muscular Dystrophy; Exon 44

Keywords

EXPLORE44; Avidity Biosciences; Avidity; AOC 1044

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (TEAEs)

  Through study completion, up to Day 85 (Part A) or Day 169 (Part B)

Secondary Outcomes (8)

• Plasma pharmacokinetic (PK) parameters

  Through Week 8 (Part A); Through Week 12 (Part B)

• Plasma pharmacokinetic (PK) parameters

  Through Week 8 (Part A); Through Week 12 (Part B)

• Plasma pharmacokinetic (PK) parameters

  Through Week 8 (Part A); Through Week 12 (Part B)

• PMO44 levels in skeletal muscle tissue

  Through Week 4 (Part A); Through Week 16 (Part B)

• Urine pharmacokinetic parameters

  Day 1-2 (0-24 hours after first dose) (Part A); Day 1-2 (0-24 hours after first dose) (Part B)

Study Arms (4)

AOC 1044-CS1 Part A - Single Dose Levels 1-5

EXPERIMENTAL

AOC 1044 will be administered once.

Drug: AOC 1044

AOC 1044-CS1 Part A - Single Dose: Placebo

PLACEBO COMPARATOR

Placebo will be administered once.

Drug: Placebo

AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3

EXPERIMENTAL

AOC 1044 will be administered three times.

Drug: AOC 1044

AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo

PLACEBO COMPARATOR

Placebo will be administered three times.

Drug: Placebo

Interventions

AOC 1044 DRUG

AOC 1044 will be administered via intravenous (IV) infusion

AOC 1044-CS1 Part A - Single Dose Levels 1-5; AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3

Placebo DRUG

Placebo will be administered via intravenous (IV) infusion.

Also known as: Saline

AOC 1044-CS1 Part A - Single Dose: Placebo; AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo

Eligibility Criteria

• Age: 7 Years - 55 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Aged 18 to 55 years, inclusive, at the time of informed consent
• Body mass index (BMI) of 18.5 to 32.0 kg/m2

You may not qualify if:

• 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
• Part B:
• 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

Sponsors & Collaborators

• Avidity Biosciences, Inc.: lead

Study Sites (10)

Arkansas Children's

Little Rock, Arkansas, 72202, United States

Location

UCSD

La Jolla, California, 92037, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Rare Disease Research - Atlanta

Atlanta, Georgia, 30329, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Massachusetts Chan Medical School

Worcester, Massachusetts, 01655, United States

Location

Gillette Children's

Saint Paul, Minnesota, 55101, United States

Location

Rare Disease Research NC

Hillsborough, North Carolina, 27278, United States

Location

Abigail Research Institute at Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

Location

Worldwide Clinical Trials (Part A only)

San Antonio, Texas, 78217, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Sodium Chloride

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Carmen Castrillo, MD

  Avidity Biosciences, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: AOC 1044-CS1 (EXPLORE44) is a 2-part study: Part A: 5 cohorts with single ascending doses conducted in healthy adult volunteers Part B: 3 cohorts with multiple ascending doses in participants with DMD mutations amenable to exon 44 skipping

Study Record Dates

• First Submitted: December 6, 2022
• First Posted: January 4, 2023
• Study Start: November 9, 2022
• Primary Completion: November 25, 2024
• Study Completion: November 25, 2024
• Last Updated: July 14, 2025

Record last verified: 2025-07

Locations

US (10)