Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping

A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06244082

Acronym

EXPLORE44OLE

Enrollment

Registered

2024-02-06

Start date

2024-01-22

Completion date

2027-04-30

Last updated

2026-07-30

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

DMD, Duchenne, Duchenne Muscular Dystrophy, Exon 44

Keywords

EXPLORE44, EXPLORE44-OLE, EXPLORE44 OLE, Avidity Biosciences, Avidity, AOC 1044, AOC 1044-CS1, AOC 1044-CS2, AOC

Brief summary

AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.

Detailed description

AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044. All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks. Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.

Interventions

DRUGAOC 1044

AOC 1044 will be administered via intravenous (IV) infusion

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Masking description

Randomization from the parent study, AOC 1044-CS1 will remain blinded.

Eligibility

Sex/Gender

MALE

Age

7 Years to 27 Years

Healthy volunteers

Inclusion criteria

Key Inclusion Criteria Rollover Participants: * Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor * No significant tolerability issues with AOC 1044 De novo Participants: * 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

Rollover Participants: * Presence of any new condition or worsening of existing condition that could affect a participant\'s safety or ability to comply with study procedures De novo Participants: * 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

Design outcomes

Primary

Measure	Time frame
Incidence of Treatment Emergent Adverse Events (TEAEs)	Through study completion (approximately 2 years)

Secondary

Measure	Time frame
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102	Through study completion (approximately 2 years)

Countries

United States

Contacts

STUDY_DIRECTORCarmen Castrillo, MD

Avidity Biosciences, Inc.

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Jul 31, 2026