Summary

Eligibility
for males ages 7-27 (full criteria)
Location
at UC Davis UCSD
Dates
study started
study ends around

Description

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.

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

Details

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.

Keywords

DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44, EXPLORE44, EXPLORE44-OLE, EXPLORE44 OLE, Avidity Biosciences, Avidity, AOC 1044, AOC 1044-CS1, AOC 1044-CS2, AOC

Eligibility

You can join if…

Open to males ages 7-27

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

You CAN'T join if...

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

Locations

  • University of California, San Diego, Rady&#39;s Children&#39;s Hospital
    La Jolla 5363943 California 5332921 92037 United States
  • UC Davis Medical Center
    Sacramento 5389489 California 5332921 95817 United States
  • Lucille Packard Children&amp;#39;s Hospital at Stanford
    San Carlos 5391760 California 5332921 94070 United States

Details

Status
in progress, not accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Avidity Biosciences, Inc.
ID
NCT06244082
Phase
Phase 2 research study
Study Type
Interventional
Participants
About 39 people participating
Last Updated