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A Phase 2 Trial of ALN-APP in Patients With Cerebral Amyloid Angiopathy

Sponsored by Alnylam Pharmaceuticals

About this trial

Last updated 10 days ago

Study ID

ALN-APP-002

Status

Recruiting

Type

Interventional

Phase

Phase 2

Placebo

Yes

Accepting

18-75 Years
30+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Started a year ago

What is this trial about?

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.

What are the Participation Requirements?

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:

- Moderate or severe stage Alzheimer's disease (AD) or significant cognitive
impairment (CI)

- Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less
than 90 days prior to anticipated randomization in the study

- 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

- Has recently received an investigational agent

- Has had treatment with amyloid-targeting antibody

Note: other protocol defined inclusion / exclusion criteria apply

Locations

Location

Status

Recruiting
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Recruiting
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Recruiting
Recruiting
Recruiting
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Recruiting

For more information, view the full study details:

NCT06393712