DMDPhase 2EXPLORE44OLENCT06244082
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
A Phase 2 study testing AOC 1044 in people with DMD and Duchenne Muscular Dystrophy, run by Avidity Biosciences, Inc. at 10 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 7 to 27 years, men only
- Conditions the study is for
- DMD, Duchenne Muscular Dystrophy, Duchenne
- What is being tested?
- AOC 1044
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 39 participants
- Where
- 10 sitesUnited States
- Study dates
- 22 Jan 2024to 30 Apr 2027
- Phase
- Phase 2
- 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 27 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Rollover Participants:
- Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
- No significant tolerability issues with AOC 1044
- De novo Participants:
- Aged 7 to 27 years, inclusive, at the time of informed consent
- Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
Show all 13Show fewer
- Confirmation of DMD gene mutation amenable to exon 44 skipping
- Weight ≥ 23 kg
- Ambulatory or non-ambulatory
- Ambulatory participants: LVEF ≥50% and FVC≥50%
- Non-ambulatory participants: LVEF ≥45% and FVC≥40%
- PUL 2.0 entry item A ≥3
- If on corticosteroids, stable dose for 30 days before screening and throughout the study
Who cannot join
- Rollover Participants:
- Presence of any new condition or worsening of existing condition that could affect a participant\'s safety or ability to comply with study procedures
- De novo Participants:
- Serum hemoglobin \< lower limit of normal
- Uncontrolled hypertension or diabetes
- Prior treatment with any cell or gene therapy
Show all 9Show fewer
- Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
- Recently treated with an investigational drug
- History of multiple drug allergies
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Rare Disease Research - Atlanta
Atlanta, Georgia, 30329
Abigail Wexner Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43215
Neurology Rare Disease Center
Denton, Texas, 76208
Rare Disease Research
Hillsborough, North Carolina, 27278
University of California, San Diego, Rady's Children's Hospital
La Jolla, California, 92037
Arkansas Children&#39;s Hospital
Little Rock, Arkansas, 72202
UC Davis Medical Center
Sacramento, California, 95817
Gillette Children's Specialty Healthcare
Saint Paul, Minnesota, 55101
Lucille Packard Children&#39;s Hospital at Stanford
San Carlos, California, 94070
University of Massachusetts Medical School
Worcester, Massachusetts, 01655
About this study
Official title: A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and 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 Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
Read the detailed description
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.
- Conditions
- DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
- Being tested
- AOC 1044
- Keywords
- EXPLORE44, EXPLORE44-OLE, EXPLORE44 OLE, Avidity Biosciences, Avidity, AOC 1044, AOC 1044-CS1, AOC 1044-CS2, AOC
- Medical subject headings
- Muscular Dystrophy, Duchenne
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 via intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| AOC 1044 Multiple Dose Levels | Experimental | AOC 1044 will be IV infused every 6 weeks for approximately 2 years. Receives: Drug: AOC 1044 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment Emergent Adverse Events (TEAEs)
Measured: Through study completion (approximately 2 years)
Secondary outcomes (1)
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102
Measured: Through study completion (approximately 2 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Feb 2024
- Study start22 Jan 2024
- Primary completion30 Apr 2027 (estimated)
- Study completion30 Apr 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DMDStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carmen Castrillo, MD, Study director, Avidity Biosciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06244082
- Sponsor's protocol ID
- AOC 1044-CS2
- Acronym
- EXPLORE44OLE
- Status last verified
- Jul 2026
- First submitted
- 26 Jan 2024
- Last updated
- 30 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping
- ClinicalTrials.gov ID
- NCT06244082
- Acronym / sponsor ID
- EXPLORE44OLE / AOC 1044-CS2
- Conditions
- DMD, Duchenne Muscular Dystrophy, Duchenne, Exon 44
- Keywords
- EXPLORE44, EXPLORE44-OLE, EXPLORE44 OLE, Avidity Biosciences, Avidity, AOC 1044, AOC 1044-CS1, AOC 1044-CS2
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 22 Jan 2024 (actual)
- Primary completion
- 30 Apr 2027 (estimated)
- Study completion
- 30 Apr 2027 (estimated)
- First posted
- 6 Feb 2024 (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.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AOC 1044 Multiple Dose Levels | Experimental | Drug: AOC 1044AOC 1044 will be IV infused every 6 weeks for approximately 2 years. |
Who may qualify
- Ages
- 7 to 27 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (13 listed)
- Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
- No significant tolerability issues with AOC 1044
- Aged 7 to 27 years, inclusive, at the time of informed consent
- Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
- Confirmation of DMD gene mutation amenable to exon 44 skipping
Key exclusion criteria (9 listed)
- Presence of any new condition or worsening of existing condition that could affect a participant\'s safety or ability to comply with study procedures
- Serum hemoglobin \< lower limit of normal
- Uncontrolled hypertension or diabetes
- Prior treatment with any cell or gene therapy
- Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
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 via intravenous (IV) infusion | Drug | AOC 1044 Multiple Dose Levels |
Primary outcome
Incidence of Treatment Emergent Adverse Events (TEAEs)
Time frame: Through study completion (approximately 2 years)
1 secondary outcome
Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102
Time frame: Through study completion (approximately 2 years)
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 30 Jul 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Carmen Castrillo, MD study director
Avidity Biosciences, Inc.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AOC 1044 in people with DMD and Duchenne Muscular Dystrophy, run by Avidity Biosciences, Inc. at 10 sites in United States.
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 13 inclusion and 9 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: under way, not looking for participants, as of the sponsor's last update on 30 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment Emergent Adverse Events (TEAEs) (time frame: Through study completion (approximately 2 years)). 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 30 Jul 2026.