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

Duchenne Muscular Dystrophy Clinical Trial

— EXPLORE44OLE  

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT06244082
• Other study ID #: AOC 1044-CS2
• Status: Enrolling by invitation
• Phase: Phase 2
• Start date: January 22, 2024
• Est. completion date: December 31, 2026

Study information

• Verified date: February 2024
• Source: Avidity Biosciences, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

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 pharmacodynamic effect of AOC 1044 on dystrophin protein production in skeletal muscle as well as the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.

Participants from AOC 1044-CS1 Part B are eligible to enroll in AOC 1044-CS2 if they have satisfactorily completed AOC 1044-CS1.

All participants who enroll in AOC 1044-CS2 will receive active treatment regardless of whether they received active treatment or placebo in AOC 1044-CS1. The treatment period is 2 years with IV dosing every 6 or 8 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 24
• Est. completion date: December 31, 2026
• Est. primary completion date: December 31, 2025
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 7 Years to 27 Years

Eligibility

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

Related Conditions & MeSH terms

• DMD
• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Drug:
• AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion

Locations

Country	Name	City	State
United States	Rare Disease Research - Atlanta	Atlanta	Georgia

Sponsors (1)

Lead Sponsor	Collaborator
Avidity Biosciences, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Secondary	Incidence of treatment emergent adverse events (TEAEs)		Through study completion (approximately 2 years)
Secondary	Change from baseline to biopsy visit in dystrophin protein levels as measured in skeletal muscle by mass spectrometry		Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2.
Secondary	Change from baseline to biopsy visit in exon skipping as measured in skeletal muscle		Baseline collection in AOC 1044-CS1 to 8 months after targeted dose starts in AOC 1044-CS2.