Clinical trial

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

Name

AOC 1044-CS2

Description

AOC 1044-CS2 (EXPLORE44-OLE) is an 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.

Trial arms

Trial start

2024-01-22

Estimated PCD

2025-12-31

Trial end

2026-12-31

Phase

Early phase I

Treatment

AOC 1044

AOC 1044 will be administered via intravenous (IV) infusion

Arms:

AOC 1044 Multiple Dose Levels

Size

Primary endpoint

Change from baseline to biopsy visit in dystrophin protein level as measured in skeletal muscle by western blot

Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2

Eligibility 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 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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'NA', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE', 'maskingDescription': 'Randomization from the parent study, AOC 1044-CS1 will remain blinded.'}}, 'enrollmentInfo': {'count': 24, 'type': 'ESTIMATED'}}

Updated at

2024-02-06

1 organization

1 product

4 indications

Organization

Avidity Biosciences

Product

AOC 1044

Indication

Duchenne Muscular Dystrophy

Indication

Duchenne muscular dystrophy

Indication

Duchenne

Indication

Exon 44