RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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)

Quick Facts

Study Start:2024-05-17
Study Completion:2029-11-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06393712

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.

Ages Eligible for Study:30 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. Age 18 years or older
  2. Willing and able to provide informed consent
  3. Able to understand and follow study procedures
  4. Stable medical condition
  1. * Moderate or severe stage Alzheimer's disease (AD) or significant cognitive impairment (CI)
  2. * Has a history of previous clinical intracerebral hemorrhage (ICH) with onset less than 90 days prior to anticipated randomization in the study
  3. * Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \>3×upper limit of normal (ULN) at Screening
  4. * Has estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73m\^2 at Screening
  5. * Has recently received an investigational agent
  6. * Has had treatment with amyloid-targeting antibody

Contacts and Locations

Study Contact

Alnylam Clinical Trial Information Line
CONTACT
1-877-ALNYLAM
clinicaltrials@alnylam.com
Alnylam Clinical Trial Information Line
CONTACT
1-877-256-9526
clinicaltrials@alnylam.com

Principal Investigator

Medical Director
STUDY_DIRECTOR
Alnylam Pharmaceuticals

Study Locations (Sites)

Clinical Trial Site
Naples, Florida, 34105
United States
Clinical Trial Site
New Orleans, Louisiana, 70121
United States
Clinical Trial Site
Plymouth, Massachusetts, 02360
United States
Clinical Trial Site
Saint Louis, Missouri, 63110
United States
Clinical Trial Site
New York, New York, 10021
United States
Clinical Trial Site
New York, New York, 10029
United States
Clinical Trial Site
Abington, Pennsylvania, 19001
United States
Clinical Trial Site
Dallas, Texas, 75243
United States
Clinical Trial Site
Houston, Texas, 77030
United States

Collaborators and Investigators

Sponsor: Alnylam Pharmaceuticals

  • Medical Director, STUDY_DIRECTOR, Alnylam Pharmaceuticals

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-17
Study Completion Date2029-11-01

Study Record Updates

Study Start Date2024-05-17
Study Completion Date2029-11-01

Terms related to this study

Keywords Provided by Researchers

  • CAA

Additional Relevant MeSH Terms

  • Cerebral Amyloid Angiopathy