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Taking people in ItalyPhase 3

Tests AOC 1044 in boys with Duchenne muscular dystrophy and exon 44 mutations

Tests AOC 1044 infusions against placebo in young people with Duchenne muscular dystrophy.

Key facts

Who runs it
Avidity Biosciences Inc.
Where
Genoa, Milan, and Rome, ItalySee the 4 sitesAlso in 6 other countries, 13 sites
Trial code
Registry title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
Status
Recruiting
Phase
Phase 3
People taking part
42 people, planned
Started
28 Jul 2026
Expected to end
31 Oct 2029
Type of study
Interventional
Record updated
27 May 2026

Who can take part

AI summary

From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.

The trial is looking for boys and young men with Duchenne muscular dystrophy who can still walk and whose gene change can be treated by skipping exon 44.

Age
7 to 16 years old (inclusive), at the time of consent.
Sex
Male only.
Disease stage
Can walk (ambulatory). Diagnosed with Duchenne muscular dystrophy, or clear symptoms starting at or before age 6.
Earlier treatment
Must have been on a stable dose of corticosteroids for at least 6 months before Day 1. Must not have had a cell or gene therapy, or another oligonucleotide treatment within 6 months of consent.
Biomarker or test result
A genetic test showing an out-of-frame dystrophin gene change that can be treated by skipping exon 44.

Main reasons for not taking part

  • Previous cell or gene therapy
  • Another oligonucleotide treatment within 6 months of consent
  • Growth hormone or testosterone started less than 1 month before consent
  • Givinostat started less than 6 months before consent

The registry criteria, word for word

Show all 8 criteriaShow less

Who can take part

  • Participant and participant’s legally designated representative have given written informed consent and/or assent (signed and dated)
  • Ambulatory males with a documented clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
  • Acceptable genetic test confirming out-of-frame dystrophin gene mutation amenable to exon 44 skipping
  • 7 to 16 years of age (inclusive) at the time of informed consent
  • Participants must be on a stable regimen of corticosteroids for at least 6 months prior to Day 1. The regimen must be anticipated to remain stable for the duration of the study

Who cannot take part

  • Previous treatment with a cell or gene therapy product
  • Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines)
  • If on any of the following treatments, participants must be on a stable regimen and must plan to maintain the regimen for the duration of the study (appropriate changes to account for body weight and composition). Participants will be excluded if regimen stability prior to informed consent is as follows: a. For growth hormone and/or testosterone: <1 month. b. For givinostat: <6 months

Where in Italy

Also in 6 other countries, 13 sites

  • IRCCS Istituto Giannina GasliniGenoa, ItalyTaking people
  • Centro Clinico NemoMilan, ItalyTaking people
  • Ospedale San Raffaele S.r.l.Milan, ItalyTaking people
  • Fondazione Policlinico Universitario Agostino Gemelli IRCCSRome, ItalyTaking people

About the trial

What the trial wants to find out

To evaluate the efficacy of AOC 1044 on muscle function

Questions to ask the study team

  1. How often do I need to come in, and for how long each time?
  2. Are travel costs paid?
  3. Can I keep my own doctor?
  4. What happens if I want to stop?

Contact the study team: see the registry record

Print a page for my doctor

Data and sources

Registry record
Listed on the EU's official list of trials (CTIS).
Official title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (delpacibart zotadirsen) for the Treatment of DMD with Gene Mutations Amenable to Exon 44 Skipping
Plain-language title
Tests AOC 1044 in boys with Duchenne muscular dystrophy and exon 44 mutationsGenerated from the registry record. The registry title is the official one.
AI summary
From the trial's eligibility criteria. Read by machine on 9 November 2026.
Record updated
27 May 2026