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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01960348 | APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis | PHASE3 | COMPLETED | 225 | — | — | Nov 1, 2013 | Aug 1, 2017 | Apr 22, 2024 | 52 | United States, Argentina +19 |
| NCT01617967 | Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis | PHASE2 | COMPLETED | 29 | — | — | May 1, 2012 | Jan 1, 2014 | Apr 19, 2024 | 10 | United States, Brazil +5 |
The difference between the patisiran (ALN-TTR02) and placebo groups in the change from baseline in mNIS+7 at 18 months. The mNIS+7 is a composite score that quantitates motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
The number of participants experiencing at least one adverse event (AE), at least one serious adverse event (SAE) and study drug discontinuation (due to any reason).
| Arm | Type | Description |
|---|---|---|
| patisiran (ALN-TTR02) | ACTIVE_COMPARATOR | - |
| Sterile Normal Saline (0.9% NaCl) | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| patisiran (ALN-TTR02) | DRUG | administered by intravenous (IV) infusion |
| Sterile Normal Saline (0.9% NaCl) | DRUG | administered by intravenous (IV) infusion |
| Patisiran | DRUG | Participants received a single dose of patisiran as an intravenous (IV) infusion on Day 0 and Day 28 (Q4W). Optional cohorts received an alternative dosing regimen (once every 3 weeks \[Q3W\]: Day 0 and Day 21) and an alternative premedication regimen. |
Inclusion Criteria: * Male or female of 18 to 85 years of age (inclusive); * Have a diagnosis of FAP * Neuropathy Impairment Score requirement of 5-130 * Meet Karnofsky performance status requirements * Have adequate complete blood counts and liver function tests * Have adequate cardiac function * ...