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

ConditionCerebral Amyloid Angiopathy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age30+
SponsorAlnylam Pharmaceuticals

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Disqualifiers

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/1.73m² at Screening

Trial design

Design model

Parallel

Treatments tested in this trial

  • Placebo

    Drug

    Placebo will be administered intrathecally

  • ALN-APP

    Drug

    ALN-APP will be administered intrathecally

Treatment groups

200 Participants
are divided into 2 treatment groups
Group A: ALN-APPExperimental treatment 1 intervention
Group B: Placebo/ALN-APPPlacebo comparator 2 interventions

Trial outcomes

Primary outcomes

1

Double-blind Treatment Period: Annualized Rate of New Lobar Cerebral Microbleeds (CMBs) Assessed on Magnetic Resonance Imaging (MRI) of Brain in Patients with Sporadic Cerebral Amyloid Angiopathy (sCAA)

Time frame
Up to 24 months

Secondary outcomes

1

Double-blind Treatment Period: Global Rank Based on Severity, Count, Symptom Burden, and Timing of New Clinical Hemorrhagic Events and Hemorrhagic Lesions Assessed on MRI of Brain

Time frame
Up to 24 months
2

Double-blind Treatment Period: Change from Baseline Over Time in Cerebrovascular Vasoreactivity Measured by Blood Oxygenation Level Dependent (BOLD) Parameters with Visual-evoked Functional MRI

Time frame
Up to 24 months
3

Double-blind Treatment Period: Incidence of New Cerebral Hemorrhagic Lesions and White Matter Hyperintensities Assessed on MRI of Brain

Time frame
Up to 24 months
4

Double-blind Treatment Period: Change from Baseline Over Time in the Total Cerebral Amyloid Angiopathy (CAA) Small Vessel Disease Score Assessed on MRI of Brain

Time frame
Up to 24 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alnylam Pharmaceuticals

Lead sponsor

Source ClinicalTrials.gov
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