A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Intrathecally Administered ALN-APP in Patients with Cerebral Amyloid Angiopathy (CAA)

causing problems such as bleeding into the brain, Cerebral Amyloid Angiopathy (CAA) is a neurological condition in which amyloid proteins build up in the walls of blood vessels in the brain

Annualized rate of new lobar Cerebral Microbleeds as detected by MRI in patients with Cerebral Amyloid Angiopathy (sCAA)

Global rank of patients based on clinical or MRI imaging outcomes by severity, count, presence of symptoms, and timing of: ­ New ICH ­ New cSS or cSAH ­ New lobar CMBs, Change from baseline over time in BOLD slope (amplitude over time-to-peak), amplitude, and time-to-peak detected, in a subset of enrolled patients, by visual-evoked fMRI, • Total number of new lobar CMBs over time detected by MRI • New ICH over time as detected by MRI or clinical diagnosis • Time to first ICH recurrence • New/extended cSS or new cSAH over time as detected by MRI or clinical diagnosis • Proportion of patients with hemorrhagic disease progression over time detected by MRI or clinical diagnosis where appropriate. • Total number of new symptomatic hemorrhages over time • Change from baseline over time in WMH volume, Change from baseline over time in total CAA SVD score as detected by MRI based on: ­ Lobar CMBs ­ CSO PVS ­ WMH ­ cSS, Change from baseline over time in sAPPα and in sAPPβ concentration in CSF

No related papers found yet.

Netherlands