DM1Phase 1/2MARINANCT05027269
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
A Phase 1/2 study testing AOC 1001 in people with DM1 and Myotonic Dystrophy 1, run by Avidity Biosciences, Inc. at 8 sites in United States.
Trial summary
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA). Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- DM1, Myotonic Dystrophy 1, 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
- 39 participants
- Where
- 8 sitesUnited States
- Study dates
- 28 Oct 2021to 14 Feb 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Genetic diagnosis of DM1 (CTG repeat length ≥ 100)
- Clinician assessed signs of DM1
- Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Who cannot join
- Diabetes that is not adequately controlled
- BMI > 35 kg/m2
- Uncontrolled hypertension
- Congenital DM1
- History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
- Recently treated with an investigational drug
- Treatment with anti-myotonic medication within 14 days of Day 1
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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 Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).
Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period.
Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders
- Being tested
- AOC 1001, Placebo
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease, MARINA, Avidity Biosciences, Avidity, AOC 1001, Myotonic Muscular Dystrophy
- Medical subject headings
- Myotonic Dystrophy, Myotonic Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AOC 1001Drug
AOC 1001 will be administered by intravenous (IV) infusion.
PlaceboDrug
Placebo will be administered by intravenous (IV) infusion.
| Study group | Role | What they receive |
|---|---|---|
| Part A Single Dose: AOC 1001 Dose Level 1 | Experimental | AOC 1001 will be administered once. Receives: Drug: AOC 1001 |
| Part A Single Dose: Placebo | Placebo comparator | Saline will be administered once. Receives: Drug: Placebo |
| Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3 | Experimental | AOC 1001 will be administered three times. Receives: Drug: AOC 1001 |
| Part B Multiple Ascending Dose: Placebo | Placebo comparator | Saline 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.
Frequency of Treatment Emergent Adverse Events (TEAEs)
Cumulative number of participants with treatment emergent adverse events at the end of study.
Measured: Through study completion, up to Day 183
Secondary outcomes (8)
Plasma PK Parameters
Maximum concentration in Plasma (Cmax)
Measured: Day 1
Plasma Pharmacokinetic (PK) Parameters
Time to maximum plasma concentration (Tmax)
Measured: Day 1
Plasma Pharmacokinetic (PK) Parameters
Terminal Half-life (t1/2)
Measured: Day 1
Plasma Pharmacokinetic (PK) Parameters
Area Under the Concentration-time Curve (AUC) obtained until the last observed timepoint
Measured: Day 1
Urine Pharmacokinetic (PK) Parameters
fraction excreted (fe) in urine in first 24 hours
Measured: Day 1
Change From Baseline in DMPK mRNA Knockdown
percent change from baseline in DMPK mRNA level
Measured: Day 43 (participants treated with 1 mg/kg) and Day 92 (participants treated with 2 mg/kg and 4 mg/kg)
Absolute Change From Baseline in 4 Gene Spliceopathy
Change from baseline in 4 gene panel mean scaled percent spliced in value. The 4 gene panel includes the following genes: CLCN1, CACNA1S, ATP2A1, and BIN1
Measured: Day 43 (participants treated with 1 mg/kg) and Day 92 (participants treated with 2 mg/kg and 4 mg/kg)
AOC 1001 Concentration Levels in Plasma, Urine, and Muscle Tissue
AOC 1001 concentration measures in plasma, urine, and muscle tissue. Assessments below lower limit of quantification are set to zero.
Measured: Up to Day 43
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2021
- Study start28 Oct 2021
- Primary completion14 Feb 2023
- Study completion14 Feb 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Avidity Biosciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Li Tai, MD, Study director, Avidity Biosciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05027269
- Sponsor's protocol ID
- AOC 1001-CS1
- Acronym
- MARINA
- Status last verified
- Sep 2026
- First submitted
- 24 Aug 2021
- Last updated
- 28 Sep 2026
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Johnson NE, Tai LJ, Hamel JI, Day JW, Statland JM, Soltanzadeh P, Subramony SH, Thornton CA, Arnold WD, Wicklund M, Freimer ML, Eichinger K, Dekdebrun J, Chen CY, Goel V, McEvoy B, Zhu Y, Hughes SG, Ackermann EJ, Levin AA. An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1. N Engl J Med. 2026 Feb 19;394(8):763-772. doi: 10.1056/NEJMoa2407326.
PubMed 41707138
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized, Double-Blind, Placebo-Controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple-Doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
- ClinicalTrials.gov ID
- NCT05027269
- Acronym / sponsor ID
- MARINA / AOC 1001-CS1
- Conditions
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy, Myotonic Dystrophy Type 1 (DM1), Dystrophy Myotonic, Myotonic Disorders
- Keywords
- DM1, Myotonic Dystrophy 1, Myotonic Dystrophy Type 1 (DM1), Myotonic Dystrophy, DM, Dystrophy Myotonic, Myotonic Disorders, Steinert Disease
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Avidity Biosciences, Inc.Industry (pharma, biotech, devices)
- Study start
- 28 Oct 2021 (actual)
- Primary completion
- 14 Feb 2023 (actual)
- Study completion
- 14 Feb 2023 (actual)
- First posted
- 30 Aug 2021 (actual)
- Results
- Posted 28 Sep 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
AOC 1001-CS1 is a randomized, double-blind, placebo-controlled, Phase 1/2 study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients (MARINA).
Part A is a single dose design with 1 cohort (dose level). In Part A, the patient duration is 6 months as the treatment period is 1 day followed by a 6 month follow-up period.
Part B is a multiple-ascending dose design with 2 cohorts (dose levels). In Part B, the patient duration is 6 months as the treatment period is 3 months followed by a 3 month follow-up period.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Part A Single Dose: AOC 1001 Dose Level 1 | Experimental | Drug: AOC 1001AOC 1001 will be administered once. |
| Part A Single Dose: Placebo | Placebo comparator | Drug: PlaceboSaline will be administered once. |
| Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3 | Experimental | Drug: AOC 1001AOC 1001 will be administered three times. |
| Part B Multiple Ascending Dose: Placebo | Placebo comparator | Drug: PlaceboSaline will be administered three times. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Genetic diagnosis of DM1 (CTG repeat length ≥ 100)
- Clinician assessed signs of DM1
- Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
Key exclusion criteria (8 listed)
- Diabetes that is not adequately controlled
- BMI > 35 kg/m2
- Uncontrolled hypertension
- Congenital DM1
- History of tibialis anterior (TA) biopsy within 3 months of Day 1 or planning to undergo TA biopsies during study period
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 1001AOC 1001 will be administered by intravenous (IV) infusion. | Drug | Part A Single Dose: AOC 1001 Dose Level 1, Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3 |
| PlaceboPlacebo will be administered by intravenous (IV) infusion. | Drug | Part A Single Dose: Placebo, Part B Multiple Ascending Dose: Placebo |
Primary outcome
Frequency of Treatment Emergent Adverse Events (TEAEs)
Time frame: Through study completion, up to Day 183
Cumulative number of participants with treatment emergent adverse events at the end of study.
8 secondary outcomes
Plasma PK Parameters
Time frame: Day 1
Plasma Pharmacokinetic (PK) Parameters
Time frame: Day 1
Plasma Pharmacokinetic (PK) Parameters
Time frame: Day 1
Plasma Pharmacokinetic (PK) Parameters
Time frame: Day 1
Urine Pharmacokinetic (PK) Parameters
Time frame: Day 1
Change From Baseline in DMPK mRNA Knockdown
Time frame: Day 43 (participants treated with 1 mg/kg) and Day 92 (participants treated with 2 mg/kg and 4 mg/kg)
Absolute Change From Baseline in 4 Gene Spliceopathy
Time frame: Day 43 (participants treated with 1 mg/kg) and Day 92 (participants treated with 2 mg/kg and 4 mg/kg)
AOC 1001 Concentration Levels in Plasma, Urine, and Muscle Tissue
Time frame: Up to Day 43
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 41707138Johnson NE, Tai LJ, Hamel JI, Day JW, Statland JM, Soltanzadeh P, Subramony SH, Thornton CA, Arnold WD, Wicklund M, Freimer ML, Eichinger K, Dekdebrun J, Chen CY, Goel V, McEvoy B, Zhu Y, Hughes SG, Ackermann EJ, Levin AA. An Antibody-Oligonucleotide Conjugate for Myotonic Dystrophy Type 1. N Engl J Med. 2026 Feb 19;394(8):763-772. doi: 10.1056/NEJMoa2407326.
- Related InfoLink provided by the sponsor.
- 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.
Li Tai, MD study director
Avidity Biosciences, Inc.
8 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 1001 in people with DM1 and Myotonic Dystrophy 1, 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 3 inclusion and 8 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 28 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. 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?
Yes. Results are posted in the registry (first posted 28 Sep 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Frequency of Treatment Emergent Adverse Events (TEAEs) (time frame: Through study completion, up to Day 183). 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 NCT05027269, 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 28 Sep 2026.