A Study to Evaluate AB-1001 Striatal Administration in Adults With Early Manifest Huntington's Disease

Huntington Disease Clinical Trial

Official title: An Open-Label Phase I/II Dose Finding Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Striatal Administration of AB-1001 in Adult Subjects With Early Manifest Huntington's Disease (HD)

Status Active, not recruiting

Clinical Trial Summary

A Phase I/II Dose-Finding Study to Evaluate Striatal Administration of AB-1001 (previously BV-101) in Adults with Early Manifest Huntington's Disease

Clinical Trial Description

This is a Phase I/II, first-in-human, open-label study to evaluate the safety, tolerability, and preliminary efficacy signals in subjects with early manifest HD following treatment with one-time intracerebral bilateral injections of AB-1001 within the striatum (caudate and putamen).

This study consists of 2 parts: Dose-Finding Part and Expansion Part; each part consists of 3 phases: Screening Phase (8 weeks, with extension to 12 weeks to accommodate scheduling if needed), Treatment and Initial Follow-Up Phase (52 weeks) and Long-Term Follow-Up Phase (4 years). In the Dose-Finding Part, 2 dose titers will be tested in 3-6 subjects in each cohort. Once a dose is selected based on Dose-Limiting Toxicities, an additional 6 subjects will be enrolled into the Dose Expansion Part. ;

Related Conditions & MeSH terms

• Huntington Disease

• NCT number: NCT05541627
• Study type: Interventional
• Source: Asklepios Biopharmaceutical, Inc.
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: October 12, 2022
• Completion date: December 31, 2029