DM1Phase 2MARINA-OLENCT05479981
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
A Phase 2 study testing AOC 1001 in people with DM1 and Muscular Dystrophies, run by Avidity Biosciences, Inc. at 8 sites in United States.
Trial summary
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients Read more about this study
- Who is it for?
- Ages 18 to 66 years
- Conditions the study is for
- DM1, Muscular Dystrophies, Myotonic Dystrophy
- What is being tested?
- AOC 1001
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 37 participants
- Where
- 8 sitesUnited States
- Study dates
- 4 Aug 2022to 10 Sep 2025
- Phase
- Phase 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 18 to 66 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Who cannot join
- Pregnancy, intent to become pregnant, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
United States8 sites
Ohio State University
Columbus, Ohio, 43221
University of Colorado
Denver, Colorado, 80045
University of Florida
Gainesville, Florida, 32608
Kansas University Medical Center
Kansas City, Kansas, 66205
University of California Los Angeles
Los Angeles, California, 90095
Stanford University
Palo Alto, California, 94304
Virginia Commonwealth University
Richmond, Virginia, 23298
University of Rochester Medical Center
Rochester, New York, 14642
About this study
Official title: A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Read the detailed description
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) (NCT05027269) clinical study.
Participants from AOC 1001-CS1 (NCT05027269) are eligible to enroll in AOC 1001-CS2 (NCT05479981) if they have satisfactorily completed AOC 1001-CS1 (NCT05027269).
After completion of Q8W (once every 8 weeks) Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study - AOC 1001-CS4 (HARBOR-OLE) (NCT07008469). Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).
As of September 2024, the dosing regimen was updated to every 8 weeks.
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic
- Being tested
- Placebo, AOC 1001
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, MARINA-OLE, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy
- Medical subject headings
- Muscular Dystrophies, Myotonic Dystrophy, Myotonic Disorders, 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.
PlaceboDrug
Also called Saline
Placebo will be administered by intravenous (IV) infusion.
AOC 1001Drug
AOC 1001 will be administered by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| AOC 1001 | Experimental | AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. Receives: Drug: AOC 1001 |
| AOC 1001 (with Placebo at Day 43) | Experimental | AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. Receives: Drug: Placebo; Drug: AOC 1001 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number and severity of treatment-emergent adverse events (TEAEs)
Measured: Through study completion
Secondary outcomes (4)
AOC 1001 levels in muscle tissue
Measured: Through Day 183
Change and percentage change from baseline in DMPK mRNA knockdown
Measured: Through Day 183
Change and percentage change from baseline in Spliceopathy
Measured: Through Day 183
Plasma pharmacokinetic (PK) parameters
Maximum and trough plasma concentration
Measured: Up to Day 729
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Jul 2022
- Study start4 Aug 2022
- Primary completion5 Sep 2025
- Study completion10 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for DM1Studies 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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 37 participants (actual)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05479981
- Sponsor's protocol ID
- AOC 1001-CS2
- Acronym
- MARINA-OLE
- Status last verified
- Sep 2026
- First submitted
- 27 Jul 2022
- Last updated
- 11 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
- ClinicalTrials.gov ID
- NCT05479981
- Acronym / sponsor ID
- MARINA-OLE / AOC 1001-CS2
- Conditions
- DM1, Muscular Dystrophies, Myotonic Dystrophy, Myotonic Dystrophy 1, Myotonic Disorders, Muscular Disorders, Atrophic
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 37 participants (actual)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 4 Aug 2022 (actual)
- Primary completion
- 5 Sep 2025 (actual)
- Study completion
- 10 Sep 2025 (actual)
- First posted
- 29 Jul 2022 (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 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
Detailed description
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) (NCT05027269) clinical study.
Participants from AOC 1001-CS1 (NCT05027269) are eligible to enroll in AOC 1001-CS2 (NCT05479981) if they have satisfactorily completed AOC 1001-CS1 (NCT05027269).
After completion of Q8W (once every 8 weeks) Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study - AOC 1001-CS4 (HARBOR-OLE) (NCT07008469). Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).
As of September 2024, the dosing regimen was updated to every 8 weeks.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| AOC 1001 | Experimental | Drug: AOC 1001AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. |
| AOC 1001 (with Placebo at Day 43) | Experimental | Drug: Placebo, Drug: AOC 1001AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks. |
Who may qualify
- Ages
- 18 to 66 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
Key exclusion criteria (3 listed)
- Pregnancy, intent to become pregnant, or active breastfeeding
- Unwilling or unable to continue to comply with contraceptive requirements
- Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
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 |
|---|---|---|
| PlaceboAlso: SalinePlacebo will be administered by intravenous (IV) infusion. | Drug | AOC 1001 (with Placebo at Day 43) |
| AOC 1001AOC 1001 will be administered by intravenous (IV) infusion. | Drug | AOC 1001, AOC 1001 (with Placebo at Day 43) |
Primary outcome
Number and severity of treatment-emergent adverse events (TEAEs)
Time frame: Through study completion
4 secondary outcomes
AOC 1001 levels in muscle tissue
Time frame: Through Day 183
Change and percentage change from baseline in DMPK mRNA knockdown
Time frame: Through Day 183
Change and percentage change from baseline in Spliceopathy
Time frame: Through Day 183
Plasma pharmacokinetic (PK) parameters
Time frame: Up to Day 729
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 11 Sep 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
8 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 1001 in people with DM1 and Muscular Dystrophies, run by Avidity Biosciences, Inc. at 8 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 1 inclusion and 3 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 11 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 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?
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?
Number and severity of treatment-emergent adverse events (TEAEs) (time frame: Through study completion). 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 11 Sep 2026; AzTrials last refreshed its copy on 8 Nov 2026.