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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Avidity Biosciences Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Decision date (initial)

2026-05-20

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Avidity Biosciences Inc.

External identifiers

EU CT number

2025-523087-20-00

WHO UTN

U1111-1325-1075

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacodynamic, Efficacy, Safety

To evaluate the efficacy of AOC 1044 on muscle function

Secondary objectives 3

1. 1. To evaluate the efficacy of AOC 1044 on a biomarker of muscle injury and measures of muscle function.
2. 2. To evaluate the efficacy of AOC 1044 on circulating biomarkers, cardiovascular mesures, patient-reported outcomes, and functional measures over time.
3. 3. To characterize the safety and tolerability profile of AOC 1044

Conditions and MedDRA coding

Duchenne muscular dystrophy

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration, European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 5

1. 1. Participant and participant’s legally designated representative have given written informed consent and/or assent (signed and dated)
2. 2. Ambulatory males with a documented clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
3. 3. Acceptable genetic test confirming out-of-frame dystrophin gene mutation amenable to exon 44 skipping
4. 4. 7 to 16 years of age (inclusive) at the time of informed consent
5. 5. 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

Exclusion criteria 3

1. 1. Previous treatment with a cell or gene therapy product
2. 2. Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
3. 3. 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline to Week 54 in TTR velocity

Secondary endpoints 4

1. 1. Change from baseline to Week 54 as per protocol
2. 2.1 Change from Baseline as per protocol
3. 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)
4. 3. • Incidence of TEAEs • Changes in vital signs • Changes in laboratory parameters • Changes in ECGs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Avidity Biosciences Inc.

Sponsor organisation

Avidity Biosciences Inc.

Address

3020 Callan Road

City

San Diego

Postcode

92121-1109

Country

United States

Scientific contact point

Organisation

Avidity Biosciences Inc.

Contact name

Avidity Biosciences Inc.

Public contact point

Organisation

Avidity Biosciences Inc.

Contact name

Avidity Biosciences Inc.

Third parties 9

Locations

7 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 102 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications