NCT06393712Active, not recruitingPhase 2
A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy
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)
Source: ClinicalTrials.gov · Last updated 20 Oct 2026
Brief summary (registry, in English)
The purpose of the study is to evaluate the effect of ALN-APP on measures of CAA disease progression and to characterize the safety, tolerability, and pharmacodynamics (PD) of ALN-APP in adult patients with sporadic CAA (sCAA) and Dutch-type CAA (D-CAA). The study will be conducted over 2 periods: a 24-month double-blind treatment period and an optional 18-month open-label extension (OLE) period. The estimated duration of study participation, inclusive of screening, treatment, and additional safety follow-up, is up to 50 months.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 210 (actual)
- First submitted
- 19 Apr 2024
- Start date
- 17 May 2024
- Primary completion date
- 9 Aug 2027
- Lead sponsor
- Alnylam Pharmaceuticals
- NCT number
- NCT06393712
- EU CT number
- 2023-510137-29-00
What you commit to
- Study type: Interventional · Phase 2
- Participants enrolled: 210
- From 17 May 2024 to the primary completion date, 9 Aug 2027
- 51 sites in 6 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study
- Has recently received an investigational agent
- Has had treatment with amyloid-targeting antibody
- Note: other protocol defined inclusion / exclusion criteria apply
Other illness
- Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI)
Lab values
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3×upper limit of normal (ULN) at Screening
- Has estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 at Screening
Who the study is looking for
- Is 50 years or older
- Has probable CAA per the Boston Criteria Version 2.0
- Is 30 years or older
- Has known E693Q amyloid precursor protein (APP) gene mutation for Dutch-type CAA
Age in the registry: 30 years to - · Sex: All · Does not accept healthy volunteers
Sites (51)
Sites in registry order. Pick your city on the disease page to sort them by distance.
This page is information, not medical advice. Discuss any trial with your doctor.