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

Korábbi kezelés

• 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

Más betegség

• 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:
• Uncontrolled hypertension or diabetes
• History of multiple drug allergies

Laborértékek

• Serum hemoglobin \< lower limit of normal

Egyéb

• Rollover Participants:

• 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