Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06411288 | Global Study of Del-desiran for the Treatment of DM1 | PHASE3 | ACTIVE NOT_RECRUITING | 159 | — | — | May 30, 2024 | Sep 1, 2026 | Jan 22, 2026 | 34 | United States, Canada +8 |
| NCT05479981 | Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients | PHASE2 | COMPLETED | 37 | — | — | Aug 4, 2022 | Sep 10, 2025 | Oct 8, 2025 | 8 | United States |
| NCT05027269 | Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients | PHASE1 | COMPLETED | 39 | — | — | Oct 28, 2021 | Feb 14, 2023 | Mar 12, 2026 | 8 | United States |
video Hand Opening Time (vHOT)
| Arm | Type | Description |
|---|---|---|
| Del-desiran | EXPERIMENTAL | Del-desiran (AOC 1001) will be administered seven times |
| Placebo | PLACEBO_COMPARATOR | Saline will be administered seven times |
| 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. |
| 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. |
| Part A Single Dose: AOC 1001 Dose Level 1 | EXPERIMENTAL | AOC 1001 will be administered once. |
| Part A Single Dose: Placebo | PLACEBO_COMPARATOR | Saline will be administered once. |
| Part B Multiple Ascending Dose: AOC 1001 Dose Levels 2 & 3 | EXPERIMENTAL | AOC 1001 will be administered three times. |
| Part B Multiple Ascending Dose: Placebo | PLACEBO_COMPARATOR | Saline will be administered three times. |
| Name | Type | Description |
|---|---|---|
| AOC 1001 (del-desiran) | DRUG | Del-desiran will be administered by intravenous (IV) infusion. |
| Placebo | DRUG | Placebo will be administered by intravenous (IV) infusion. |
| AOC 1001 | DRUG | AOC 1001 will be administered by intravenous (IV) infusion. |
Key Inclusion Criteria: * Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1 * Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening Key Exclusion Criteria: * Breastfeeding, pregnancy, or intent to become pregnant during the study * Unwilling ...