Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients (NCT05027269) | Clinical Trial Compass
CompletedPhase 1/2
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
United States39 participantsStarted 2021-10-28
Plain-language 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).
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.
Who can participate
Age range
18 Years – 65 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key Inclusion Criteria:
* 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:
* 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this AOC 1001 trial for DM1 has already completed, has any safety or adverse event data been published yet that you could walk me through, so I understand what kinds of side effects were reported?
2Because this was a Phase 1/2 trial — meaning it was primarily designed to test safety rather than confirm effectiveness — what does that mean for how much we actually know about whether AOC 1001 helps with DM1 symptoms at this stage?
3Are there any follow-on trials of AOC 1001 for myotonic dystrophy type 1 that are now open or upcoming that I might be worth discussing as a next step, given this one has completed?
4Compared to what was studied in this trial, are there any currently available standard-of-care treatments for DM1 that might be a more established option for me while this research is still maturing?
5Given that the trial focused on adults with DM1, and considering my specific situation and symptom profile, does the patient population studied seem like a reasonable match for me — and is that something worth factoring into how we interpret the results?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Frequency of treatment emergent adverse events (TEAEs)
Timeframe: Through study completion, up to Day 183