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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06914609 | REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome | PHASE3 | RECRUITING | 158 | — | — | Jun 10, 2025 | Apr 1, 2030 | May 15, 2026 | 38 | United States, Australia +10 |
| NCT05127226 | HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome | PHASE1 | RECRUITING | 70 | — | — | Dec 22, 2021 | Mar 1, 2029 | Nov 14, 2025 | 11 | United States, Australia +4 |
The Bayley-4 is a performance-based assessment of developmental functioning across communication, cognition, and motor skills. The expressive communication subdomain of communication measures preverbal and verbal communication. The total raw score reflects the sum of all the item scores within the expressive communication subdomain, with higher scores reflecting greater expressive communication ability.
The safety and tolerability of ION582 will be assessed by determining the incidence, severity, and dose relationship of adverse effects and changes in the laboratory parameters by dose.
| Arm | Type | Description |
|---|---|---|
| Cohort 1 ION582 80 mg | EXPERIMENTAL | Participants (aged 2 to \<18 years old) will be administered ION582 80 mg via IT injection Q12W during the double blind and LTE treatment periods. |
| Cohort 1 Placebo | PLACEBO_COMPARATOR | Participants (aged 2 to \<18 years old) will be administered ION582 matching placebo via IT injection Q12W during the double-blind treatment period and then administered ION582 40 mg or 80 mg Q12W during the LTE treatment period. |
| Cohort 2 ION582 80 mg | EXPERIMENTAL | Participants (aged 18 to ≤50 years old) will be administered ION582 80 mg via IT injection Q12W during the double blind and LTE treatment periods. |
| Cohort 2 Placebo | PLACEBO_COMPARATOR | Participants (aged 18 to ≤50 years old) will be administered ION582 matching placebo via IT injection Q12W during the double-blind treatment period and then randomized to ION582 40 mg or 80 mg Q12W during the LTE treatment period. |
| Part 1 MAD: Cohort A | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort B | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort C | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort D | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort E | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 13 weeks, with a minimum of approximately 4 weeks between each dose administration. |
| Part 1 MAD: Cohort F | EXPERIMENTAL | ION582 will be administered as IT injection over a period of 13 weeks, with a minimum of approximately 12 weeks between each dose administration. |
| Part 2 Group 1 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals. |
| Part 2 Group 2 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 49 weeks, with additional dosing intervals. |
| Part 3 Group 1 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals. |
| Part 3 Group 2 | EXPERIMENTAL | ION582 will be administered as IT injection of over a period of 145 weeks, with additional dosing intervals. |
| Name | Type | Description |
|---|---|---|
| ION582 | DRUG | ION582 will be administered by IT injection. |
| Placebo | DRUG | ION582 matching placebo will be administered by IT injection. |
Key Inclusion Criteria: 1. The participants caregiver(s)/ legally authorized representative must have given written informed consent and any authorizations required by local law and be able to comply with all study requirements. 2. Medically stable and can undergo sedation and/or general anesthesia...