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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06153966 | PrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717 | PHASE1 | RECRUITING | 76 | — | — | Jan 4, 2024 | Jun 1, 2030 | Apr 24, 2026 | 13 | United States, Australia +7 |
| Arm | Type | Description |
|---|---|---|
| ION717 + Placebo, Regimen 1 | EXPERIMENTAL | Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period. |
| ION717 + Placebo, Regimen 2 | EXPERIMENTAL | Participants will receive multiple doses of study drug (ION717 and placebo) during the 30-week treatment period; the order of doses is blinded. Participants will then receive multiple doses of ION717 during the 142-week open-label extension period. |
| ION717, Regimen 3 | EXPERIMENTAL | Participants will receive multiple doses of ION717 during the 30-week treatment period and the 142-week open-label extension period. |
| Name | Type | Description |
|---|---|---|
| ION717 | DRUG | ION717 will be administered by IT injection. |
| Placebo | DRUG | Placebo-matching ION717 will be administered by IT injection. |
Key Inclusion Criteria * A confirmed diagnosis of probable or definite prion disease. * Early-stage prion disease at the time of Screening. * Willing to meet all study requirements, including travel to Study Center, procedures, measurements and visits. * Patients must have a caregiver who is ≥ 18 y...