Duchenne Muscular DystrophyPhase 1/2EXPLORE44NCT05670730
Study of AOC 1044 in Healthy Adult Volunteers and Participants With Duchenne Muscular Dystrophy (DMD) Mutations Amenable to Exon 44 Skipping
A Phase 1/2 study testing AOC 1044 in people with Duchenne Muscular Dystrophy and Exon 44, run by Avidity Biosciences, Inc. at 10 sites in United States.
Trial summary
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping. Read more about this study
- Who is it for?
- Ages 7 to 55 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy, Exon 44
- 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
- 10 sitesUnited States
- Study dates
- 9 Nov 2022to 25 Nov 2024
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 55 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- Aged 18 to 55 years, inclusive, at the time of informed consent
- Body mass index (BMI) of 18.5 to 32.0 kg/m2
- 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
- Weight ≥ 23 kg
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- 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
- Clinically significant abnormalities in laboratory results, ECGs, or vitals
- Current or recent use of prescription or nonprescription drugs
- Positive drug/alcohol test at Screening or Day -1
- Elevated blood pressure (BP) >130/80 mmHg at Screening
- Participation in a clinical study in which an investigational product was received within 1 month of screening or 5 half-lives of the investigational product
- Blood or plasma donation within 16 weeks of planned AOC 1044 administration Note: Other protocol defined Inclusion/Exclusion criteria may apply
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- Part B:
- Biceps brachii muscles unsuitable for biopsy
- 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
- 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 Research Institute at Nationwide Children's Hospital
Columbus, Ohio, 43215
Rare Disease Research NC
Hillsborough, North Carolina, 27278
University of Iowa
Iowa City, Iowa, 52242
UCSD
La Jolla, California, 92037
Arkansas Children's
Little Rock, Arkansas, 72202
Stanford University
Palo Alto, California, 94304
Gillette Children's
Saint Paul, Minnesota, 55101
Worldwide Clinical Trials (Part A only)
San Antonio, Texas, 78217
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655
About this study
Official title: A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
AOC 1044-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.
Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
- Conditions
- Duchenne Muscular Dystrophy, Exon 44
- Being tested
- AOC 1044, Placebo
- Keywords
- EXPLORE44, Avidity Biosciences, Avidity, AOC 1044
- 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
PlaceboDrug
Also called Saline
Placebo will be administered via intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| AOC 1044-CS1 Part A - Single Dose Levels 1-5 | Experimental | AOC 1044 will be administered once. Receives: Drug: AOC 1044 |
| AOC 1044-CS1 Part A - Single Dose: Placebo | Placebo comparator | Placebo will be administered once. Receives: Drug: Placebo |
| AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3 | Experimental | AOC 1044 will be administered three times. Receives: Drug: AOC 1044 |
| AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo | Placebo comparator | Placebo will be administered three times. Receives: Drug: Placebo |
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, up to Day 85 (Part A) or Day 169 (Part B)
Secondary outcomes (8)
Plasma pharmacokinetic (PK) parameters
Maximum plasma concentration (Cmax) of AOC 1044
Measured: Through Week 8 (Part A); Through Week 12 (Part B)
Plasma pharmacokinetic (PK) parameters
Terminal half-life (T1/2) of AOC 1044
Measured: Through Week 8 (Part A); Through Week 12 (Part B)
Plasma pharmacokinetic (PK) parameters
Area under the concentration-time curve (AUC) of AOC 1044
Measured: Through Week 8 (Part A); Through Week 12 (Part B)
PMO44 levels in skeletal muscle tissue
Measured: Through Week 4 (Part A); Through Week 16 (Part B)
Urine pharmacokinetic parameters
Fraction of PMO44 excreted in urine
Measured: Day 1-2 (0-24 hours after first dose) (Part A); Day 1-2 (0-24 hours after first dose) (Part B)
Change from baseline in exon skipping as measured in skeletal muscle (Part B only)
Measured: Baseline, Week 16
Absolute change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Measured: Baseline, Week 16
Percentage change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Measured: Baseline, Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2023
- Study start9 Nov 2022
- Primary completion25 Nov 2024
- Study completion25 Nov 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- AOC 1044-CS1 (EXPLORE44) is a 2-part study: Part A: 5 cohorts with single ascending doses conducted in healthy adult volunteers Part B: 3 cohorts with multiple ascending doses in participants with DMD mutations amenable to exon 44 skipping
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 70 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
- NCT05670730
- Sponsor's protocol ID
- AOC 1044-CS1
- Acronym
- EXPLORE44
- Status last verified
- Jul 2025
- First submitted
- 6 Dec 2022
- Last updated
- 14 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Veerapandiyan A, Eskuri J, Laverty CG, McDonald CM, Phan H, Smith EC, Tesi Rocha C, Wong B, Morris M, Tami Y, Carmack T, Kovach P, Herzog JJ, Hughes SG, Zhu Y, Ackermann EJ, Flanigan KM; EXPLORE44 Study Investigators. Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial. Lancet Neurol. 2026 Nov;25(11):983-996. doi: 10.1016/S1474-4422(26)00337-6.
PubMed 42874619
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Randomized, Placebo-controlled, Double-blind, Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamic Effects of Single and Multiple Ascending Doses of AOC 1044 Administered Intravenously to Healthy Adult Volunteers and Participants With DMD Mutations Amenable to Exon 44 Skipping
- ClinicalTrials.gov ID
- NCT05670730
- Acronym / sponsor ID
- EXPLORE44 / AOC 1044-CS1
- Conditions
- Duchenne Muscular Dystrophy, Exon 44
- Keywords
- EXPLORE44, Avidity Biosciences, Avidity, AOC 1044
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 9 Nov 2022 (actual)
- Primary completion
- 25 Nov 2024 (actual)
- Study completion
- 25 Nov 2024 (actual)
- First posted
- 4 Jan 2023 (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-CS1 (EXPLORE44) is a Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of single and multiple ascending doses of AOC 1044 in healthy adult volunteers and participants with DMD mutations amenable to exon 44 skipping.
Part A is a single dose design with multiple cohorts (dose levels) in healthy adult volunteers.
Part B is a multiple-ascending dose design with 3 cohorts (dose levels) in participants with Duchenne.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
- Model description
- AOC 1044-CS1 (EXPLORE44) is a 2-part study: Part A: 5 cohorts with single ascending doses conducted in healthy adult volunteers Part B: 3 cohorts with multiple ascending doses in participants with DMD mutations amenable to exon 44 skipping
| Arm | Type | Receives |
|---|---|---|
| AOC 1044-CS1 Part A - Single Dose Levels 1-5 | Experimental | Drug: AOC 1044AOC 1044 will be administered once. |
| AOC 1044-CS1 Part A - Single Dose: Placebo | Placebo comparator | Drug: PlaceboPlacebo will be administered once. |
| AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3 | Experimental | Drug: AOC 1044AOC 1044 will be administered three times. |
| AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo | Placebo comparator | Drug: PlaceboPlacebo will be administered three times. |
Who may qualify
- Ages
- 7 to 55 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Aged 18 to 55 years, inclusive, at the time of informed consent
- Body mass index (BMI) of 18.5 to 32.0 kg/m2
- 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 (14 listed)
- Clinically significant abnormalities in laboratory results, ECGs, or vitals
- Current or recent use of prescription or nonprescription drugs
- Positive drug/alcohol test at Screening or Day -1
- Elevated blood pressure (BP) >130/80 mmHg at Screening
- Participation in a clinical study in which an investigational product was received within 1 month of screening or 5 half-lives of the investigational product
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-CS1 Part A - Single Dose Levels 1-5, AOC 1044-CS1 Part B - Multiple Ascending Dose Levels 1-3 |
| PlaceboAlso: SalinePlacebo will be administered via intravenous (IV) infusion. | Drug | AOC 1044-CS1 Part A - Single Dose: Placebo, AOC 1044-CS1 Part B - Multiple Ascending Dose: Placebo |
Primary outcome
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Through study completion, up to Day 85 (Part A) or Day 169 (Part B)
8 secondary outcomes
Plasma pharmacokinetic (PK) parameters
Time frame: Through Week 8 (Part A); Through Week 12 (Part B)
Plasma pharmacokinetic (PK) parameters
Time frame: Through Week 8 (Part A); Through Week 12 (Part B)
Plasma pharmacokinetic (PK) parameters
Time frame: Through Week 8 (Part A); Through Week 12 (Part B)
PMO44 levels in skeletal muscle tissue
Time frame: Through Week 4 (Part A); Through Week 16 (Part B)
Urine pharmacokinetic parameters
Time frame: Day 1-2 (0-24 hours after first dose) (Part A); Day 1-2 (0-24 hours after first dose) (Part B)
Change from baseline in exon skipping as measured in skeletal muscle (Part B only)
Time frame: Baseline, Week 16
Absolute change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Time frame: Baseline, Week 16
Percentage change from baseline in dystrophin protein level in skeletal muscle (Part B only)
Time frame: Baseline, Week 16
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Jul 2025.
- Derived from this study: PubMed 42874619Veerapandiyan A, Eskuri J, Laverty CG, McDonald CM, Phan H, Smith EC, Tesi Rocha C, Wong B, Morris M, Tami Y, Carmack T, Kovach P, Herzog JJ, Hughes SG, Zhu Y, Ackermann EJ, Flanigan KM; EXPLORE44 Study Investigators. Safety and efficacy of delpacibart zotadirsen in Duchenne muscular dystrophy mutations amenable to exon 44 skipping in the USA (EXPLORE44): a randomised, placebo-controlled, double-blind, phase 1/2 trial. Lancet Neurol. 2026 Nov;25(11):983-996. doi: 10.1016/S1474-4422(26)00337-6.
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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 1/2 study testing AOC 1044 in people with Duchenne Muscular Dystrophy and Exon 44, 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 11 inclusion and 14 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: finished, as of the sponsor's last update on 14 Jul 2025. 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. 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 combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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, up to Day 85 (Part A) or Day 169 (Part B)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05670730, which also lists 1 linked publication.
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 14 Jul 2025.