Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping (EXPLORE44OLE)

Avidity Biosciences

Status and phase: Active, not recruiting; Phase 2

Conditions: Exon 44; DMD; Duchenne; Duchenne Muscular Dystrophy

Treatments: Drug: AOC 1044

Study type: Interventional

Funder types: Industry

Identifiers

NCT06244082

AOC 1044-CS2

Details and patient eligibility

About

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.

Full 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.

Enrollment: 39 patients

Sex: Male

Ages: 7 to 27 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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39 participants in 1 patient group

AOC 1044 Multiple Dose Levels

Experimental group

Description:

AOC 1044 will be IV infused every 6 weeks for approximately 2 years.

Treatment:

Drug: AOC 1044