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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
Not yet recruiting
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2025-523087-20-00
Enrollment
26
Registered
2026-05-20
Start date
Unknown
Completion date
Unknown
Last updated
2026-05-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Duchenne muscular dystrophy

Brief summary

Change from baseline to Week 54 in TTR velocity

Detailed description

1. Change from baseline to Week 54 as per protocol, 2.1 Change from Baseline as per protocol, 2.2 • Change from Baseline (all visits except Week 54) o creatine kinase o 4 stair climb velocity o 10mWRT velocity o SV95C o TTR velocity o NSAA o DMD-QoL o Patient/Caregiver Global Impression of Severity o QMT • Patient/Caregiver Global Impression of Change (all visits except Week 54), 3. • Incidence of TEAEs • Changes in vital signs • Changes in laboratory parameters • Changes in ECGs

Interventions

DRUGSodium Chloride 0.9% w/v
DRUGAOC 1044

Sponsors

Avidity Biosciences Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
Male
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
Change from baseline to Week 54 in TTR velocity—

Secondary

MeasureTime frame
1. Change from baseline to Week 54 as per protocol, 2.1 Change from Baseline as per protocol, 2.2 • Change from Baseline (all visits except Week 54) o creatine kinase o 4 stair climb velocity o 10mWRT velocity o SV95C o TTR velocity o NSAA o DMD-QoL o Patient/Caregiver Global Impression of Severity o QMT • Patient/Caregiver Global Impression of Change (all visits except Week 54), 3. • Incidence of TEAEs • Changes in vital signs • Changes in laboratory parameters • Changes in ECGs—

Outcome results

None listed

Source: EU CTIS · Data processed: May 21, 2026