Muscular DystrophiesPhase 3SAFARI44NCT07587242
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping
A Phase 3 study testing AOC 1044 in people with Muscular Dystrophies and Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), run by Avidity Biosciences, Inc. at 18 sites in 8 countries.
Trial summary
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping Read more about this study
- Who is it for?
- Ages 7 to 16 years, men only
- Conditions the study is for
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic
- What is being tested?
- AOC 1044
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 70 participants
- Where
- 18 sitesin 8 countries
- Study dates
- 1 Sep 2026to Jul 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 7 to 16 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Ambulatory males with clinical and genetic diagnosis of DMD
- Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
- 7 to 16 years of age at time of consent
- TTR and NSAA assessment completed within the protocol specified parameters at Screening
- On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.
Who cannot join
- Previous treatment cell or gene therapy.
- Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
- Lab values outside of the protocol specified range at Screening
- If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
- Less than 1 month, for growth hormone and/or testosterone
- Less than 6 months for givinostat
Trial locations and contacts
18 sites in 8 countries. Contact the study team or a site near you to ask about taking part.
18 sites in 8 countries
Italy4 sites, 4 recruiting
- Recruiting
Istituto Giannina Gaslini
Genova
- Recruiting
Fondazione Serena ETS - Centro Clinico NeMO Milano
Milan
- Recruiting
IRCCS Ospedale San Raffaele
Milan
- Recruiting
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
Roma
Germany3 sites, 3 recruiting
- Recruiting
Universitaetsklinikum Essen
Essen
- Recruiting
Universitaetsklinikum Heidelberg
Heidelberg
- Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
München
Spain3 sites, 3 recruiting
- Recruiting
Hospital Sant Joan de Deu
Barcelona
- Recruiting
Hospital Universitario La Paz
Madrid
- Recruiting
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
Seville
Belgium2 sites, 2 recruiting
- Recruiting
UZ Leuven
Leuven
- Recruiting
Centre Hospitalier Régional de la Citadelle
Liège
France2 sites, 2 recruiting
- Recruiting
CHU de Nantes - Hotel-Dieu
Nantes
- Recruiting
AP-HP Hospital Armand-Trousseau
Paris
United Kingdom2 sites, 2 recruiting
- Recruiting
Royal Victoria Infirmary
Newcastle
- Recruiting
University of Oxford and Oxford University Hospitals NHS Foundation Trust
Oxford
Australia1 site, 1 recruiting
- Recruiting
The Children's Hospital at Westmead
Westmead, New South Wales
Netherlands1 site, 1 recruiting
- Recruiting
Academisch Ziekenhuis Leiden
Leiden
About this study
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
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
Read the detailed description
The study consists of a Screening Period of up to 44 Days, a Randomized 54 Week Treatment Period, a 54 Week Open Label Extension (OLE), and a Follow-Up Period of 6 weeks. Participants will be randomized to receive an intravenous infusion of either delpacibart-zotadirsen or placebo at the clinical study site every 6 weeks for a total of 9 doses. After completion of the randomized treatment period, all participants may enter the OLE portion of the study consisting of 9 doses of AOC 1044 regardless of group assignment in the randomized period. The final dose will occur at Week 102, followed by a final assessment at Week 108 and a safety follow-up visit at Week 114. An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease, Musculoskeletal Diseases, Neuromuscular Diseases (NMD)
- Being tested
- AOC 1044, Placebo
- Keywords
- AOC, AOC 1044, AOC 1044-CS3, AOC 1044-CS1, AOC 1044-CS2, EXPLORE44, EXPLORE44-OLE, SAFARI, SAFARI44, SAFARI 44, Avidity, Avidity Biosciences, Exon Skipping Therapy, Avidity Biosciences Inc., A Novartis Company, del-zota, DMD
- Medical subject headings
- Muscular Dystrophies, Muscular Dystrophy, Duchenne, Myotonic Dystrophy, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Diseases, Neuromuscular Diseases, Nervous System Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AOC 1044Drug
AOC 1044 will be administered by intravenous (IV) infusion
PlaceboDrug
Placebo will be administered by intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| AOC 1044 | Experimental | Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W Receives: Drug: AOC 1044 |
| Placebo | Placebo comparator | Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from Baseline in Time to Rise (TTR) Velocity at Week 54
Measured: Baseline, Week 54
Secondary outcomes (11)
Change from Baseline in Creatine Kinase (CK) at Week 54
Measured: Baseline, Week 54
Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54
Measured: Baseline, Week 54
Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54
Measured: Baseline, Week 54
Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54
Measured: Baseline, Week 54
Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54
Measured: Baseline, Week 54
Change from Baseline in DMD Quality of Life (DMD-QoL) Score at Week 54
Measured: Baseline, Week 54
Change from Baseline in Patient Global Impression of Severity (PGI-S) at Week 54
Measured: Baseline, Week 54
Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) at Week 54
Measured: Baseline, Week 54
Patient Global Impression of Change (PGI-C) at Week 54
Measured: Week 54
Caregiver Global Impression of Change (CaGI-C) at Week 54
Caregiver Global Impression of Change (CaGI-C) at Week 54
Measured: Week 54
Change from Baseline in Quantitative Muscle Testing (QMT) at Week 54
Measured: Baseline, Week 54
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2026
- Study start1 Sep 2026
- Primary completionMay 2029 (estimated)
- Study completionJul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DystrophiesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07587242
- Sponsor's protocol ID
- AOC 1044-CS3
- Acronym
- SAFARI44
- Status last verified
- Aug 2026
- First submitted
- 8 May 2026
- Last updated
- 13 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 13 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07587242
- Acronym / sponsor ID
- SAFARI44 / AOC 1044-CS3
- Conditions
- Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease, Musculoskeletal Diseases, Neuromuscular Diseases (NMD)
- Keywords
- AOC, AOC 1044, AOC 1044-CS3, AOC 1044-CS1, AOC 1044-CS2, EXPLORE44, EXPLORE44-OLE, SAFARI
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 1 Sep 2026 (actual)
- Primary completion
- May 2029 (estimated)
- Study completion
- Jul 2030 (estimated)
- First posted
- 14 May 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping
Detailed description
The study consists of a Screening Period of up to 44 Days, a Randomized 54 Week Treatment Period, a 54 Week Open Label Extension (OLE), and a Follow-Up Period of 6 weeks. Participants will be randomized to receive an intravenous infusion of either delpacibart-zotadirsen or placebo at the clinical study site every 6 weeks for a total of 9 doses. After completion of the randomized treatment period, all participants may enter the OLE portion of the study consisting of 9 doses of AOC 1044 regardless of group assignment in the randomized period. The final dose will occur at Week 102, followed by a final assessment at Week 108 and a safety follow-up visit at Week 114. An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AOC 1044 | Experimental | Drug: AOC 1044Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W |
Who may qualify
- Ages
- 7 to 16 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Ambulatory males with clinical and genetic diagnosis of DMD
- Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping
- 7 to 16 years of age at time of consent
- TTR and NSAA assessment completed within the protocol specified parameters at Screening
- On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.
Key exclusion criteria (6 listed)
- Previous treatment cell or gene therapy.
- Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).
- Lab values outside of the protocol specified range at Screening
- If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
- Less than 1 month, for growth hormone and/or testosterone
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AOC 1044AOC 1044 will be administered by intravenous (IV) infusion | Drug | AOC 1044 |
| PlaceboPlacebo will be administered by intravenous (IV) infusion | Drug | Placebo |
Primary outcome
Change from Baseline in Time to Rise (TTR) Velocity at Week 54
Time frame: Baseline, Week 54
11 secondary outcomes
Change from Baseline in Creatine Kinase (CK) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54
Time frame: Baseline, Week 54
Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54
Time frame: Baseline, Week 54
Change from Baseline in DMD Quality of Life (DMD-QoL) Score at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Patient Global Impression of Severity (PGI-S) at Week 54
Time frame: Baseline, Week 54
Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) at Week 54
Time frame: Baseline, Week 54
Patient Global Impression of Change (PGI-C) at Week 54
Time frame: Week 54
Caregiver Global Impression of Change (CaGI-C) at Week 54
Time frame: Week 54
Change from Baseline in Quantitative Muscle Testing (QMT) at Week 54
Time frame: Baseline, Week 54
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Oct 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
18 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing AOC 1044 in people with Muscular Dystrophies and Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), run by Avidity Biosciences, Inc. at 18 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Italy, Germany, Spain, Belgium, France and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Avidity Bioscience, Inc., A Novartis Company (1-888-669-6682, Novartis.email@novartis.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from Baseline in Time to Rise (TTR) Velocity at Week 54 (time frame: Baseline, Week 54). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Oct 2026.