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

Description

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.

Conditions

Cerebral Amyloid Angiopathy

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

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)

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

Condition
Cerebral Amyloid Angiopathy
Intervention / Treatment

-

Contacts and Locations

Naples

Clinical Trial Site, Naples, Florida, United States, 34105

New Orleans

Clinical Trial Site, New Orleans, Louisiana, United States, 70121

Plymouth

Clinical Trial Site, Plymouth, Massachusetts, United States, 02360

Saint Louis

Clinical Trial Site, Saint Louis, Missouri, United States, 63110

New York

Clinical Trial Site, New York, New York, United States, 10021

New York

Clinical Trial Site, New York, New York, United States, 10029

Abington

Clinical Trial Site, Abington, Pennsylvania, United States, 19001

Dallas

Clinical Trial Site, Dallas, Texas, United States, 75243

Houston

Clinical Trial Site, Houston, Texas, United States, 77030

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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

Ages Eligible for Study

30 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Alnylam Pharmaceuticals,

Medical Director, STUDY_DIRECTOR, Alnylam Pharmaceuticals

Study Record Dates

2029-11-01