A Phase 2 interventional study of AOC 1044 in DMD, Duchenne Muscular Dystrophy and Duchenne, sponsored by Avidity Biosciences, Inc.. Active, not recruiting at 10 sites in United States. Open to male participants aged 7 Years to 27 Years. Per ClinicalTrials.gov, last updated 2026-07-30.
Sponsored by Avidity Biosciences, Inc. (part of Novartis) · Phase 2, Interventional, and Treatment
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Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 35 open to participants now, 60 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 70th largest by enrollment of the 171 interventional ones that give a count, is one of 15 that started in 2024, is one of 57 in phase 2, reaches primary completion later than 22 of the 59 other live ones, and was the 203rd registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →35 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now. Recruiting first, the newest first:
Vanderbilt University Medical Center · Sites in United States
Ages 18+ · Men only
Sarepta Therapeutics, Inc. · Sites in United States
Ages 4+ · Men only
University of Florida · Sites in United States
Ages 6+ · Men only
Vanderbilt University Medical Center · Sites in United States
Ages 8–18 · Men only
University of Rochester · Sites in United States
Ages 0 days–3 yrs · Men only
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 59 take children under 18; 12 take healthy volunteers.
This study: 7 Years to 27 Years · male · no healthy volunteers.
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232 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 57 · Phase 2 90 · Phase 3 40 · Phase 4 5
First in 2000; this study’s year is marked. 15 started in 2024.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 39 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 78 (49%) have results posted.
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130 of 232 have an industry lead sponsor. The 10 most frequent:
This study is registered under Avidity Biosciences, Inc., one of the names counted as Novartis. Under that name alone:
Avidity Biosciences, Inc. is the lead sponsor of 11 studies on the registry; 4 are open to participants now.
Of its 5 completed or terminated interventional studies of FDA-regulated products, 1 (20%) have results posted.
Novartis has 6 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
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Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
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.
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.
Key Inclusion Criteria
Rollover Participants:
De novo Participants:
Ambulatory or non-ambulatory
Key Exclusion Criteria
Rollover Participants:
De novo Participants:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
AOC 1044 will be IV infused every 6 weeks for approximately 2 years.
Drug: AOC 1044
AOC 1044 will be administered via intravenous (IV) infusion
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Through study completion (approximately 2 years)
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102
Time frame: Through study completion (approximately 2 years)
Plan to share: No
No publications or documents are linked to this record.
Started Jan 2024 (actual). Primary completion is estimated for Apr 2027, 39 months after the start.
Of 40 industry-sponsored phase 2 interventional studies under Muscular Dystrophy, Duchenne, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 20 and three quarters within 42. This study's planned 39 months is above that.
Counting every sponsor, half of 47 studies reached it within 31 months; a quarter within 20 and three quarters within 42.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Delpacibart zotadirsen
Muscular Dystrophy, Duchenne →