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Safety, Tolerability and Efficacy of NPI-001 in Patients With Hereditary Cystatin C Amyloid Angiopathy (HCCAA)

NCT07716865 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 15

Last updated 2026-07-21

No results posted yet for this study

Summary

The study aims to measure safety, tolerability and biomarker-based efficacy of NPI-001 (AT-001) in Subjects with Hereditary Cystatin C Amyloid Angiopathy (HCCAA). In the study participants receive increasing dose or active treatment (250 mg vs 500mg vs 750 mg) or matched placebo in the form of tablets BID.

Conditions

  • Hereditary Cystatin C Amyloid Angiopathy (HCCAA)
  • Hemorrhage Brain
  • Hemorrhage Cerebral

Interventions

DRUG

NACA

250 mg tablets BID, increasing dosage from 250 mg BID to 750 mg BID

Sponsors & Collaborators

  • Arctic Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-03
Primary Completion
2026-12-29
Completion
2027-01-31

Countries

  • Iceland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07716865 on ClinicalTrials.gov