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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06393712 | A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy | PHASE2 | RECRUITING | 200 | — | — | May 17, 2024 | Dec 14, 2029 | May 14, 2026 | 57 | United States, Australia +4 |
| Arm | Type | Description |
|---|---|---|
| ALN-APP | EXPERIMENTAL | Participants will be administered multiple doses of ALN-APP during the double-blind treatment period and optional open-label extension period. |
| Placebo/ALN-APP | PLACEBO_COMPARATOR | Participants will be administered multiple doses of placebo during the double-blind treatment period. Participants who continue into the optional open-label extension period will be administered multiple doses of ALN-APP. |
| Name | Type | Description |
|---|---|---|
| Placebo | DRUG | Placebo will be administered intrathecally |
| ALN-APP | DRUG | ALN-APP will be administered intrathecally |
Inclusion Criteria (sporadic CAA patients): * Is 50 years or older * Has probable CAA per the Boston Criteria Version 2.0 Inclusion Criteria (Dutch-type CAA patients): * Is 30 years or older * Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA Exclusion Criteria: *...