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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02632786 | The PRONTO Study, a Global Phase 2b Study of NEOD001 in Previously Treated Subjects With Light Chain (AL) Amyloidosis | PHASE2 | COMPLETED | 129 | — | — | Mar 1, 2016 | Mar 1, 2018 | Apr 5, 2019 | 40 | United States, Australia +8 |
N-terminal pro-brain natriuretic peptide (NT-proBNP ) best response (Response or Non-Response \[Stable, Progression\]) from baseline through 12 months of treatment. Cardiac best response, as assessed by NT-proBNP alone, is defined as the most favorable category among response (ie, decrease in NT-proBNP from baseline of \>30% and \>300 ng/L), stable (ie, neither response nor progression), and progression (ie, increase in NT-proBNP from baseline of \>30% and \>300 ng/L) across all visits after the first infusion of study drug up to and through the end of the study. Subjects are considered non-responders until a response is achieved. Non-response is defined as either stable or progression.
| Arm | Type | Description |
|---|---|---|
| NEOD001 | EXPERIMENTAL | Study Drug given IV every 28 days at 24mg/kg |
| Placebo | PLACEBO_COMPARATOR | Placebo |
| Name | Type | Description |
|---|---|---|
| NEOD001 | DRUG | NEOD001 is a monoclonal antibody directed at soluble and insoluble light chain aggregates |
| Placebo | DRUG | Saline Bag |
Inclusion Criteria: 1. Age ≥18 years 2. Confirmed diagnosis of systemic AL amyloidosis 3. ≥1 prior systemic plasma cell dyscrasia therapy with at least a partial hematologic response 4. Cardiac involvement 5. NT-proBNP ≥650 Exclusion Criteria: 1. Non-AL amyloidosis 2. Meets the International Myel...