Evaluation of Safety and Tolerability of Libella Gene Therapy for Alzheimer's Disease: AAV- hTERT

Alzheimer Disease Clinical Trial

Official title: Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for Alzheimer's Disease: AAV- hTERT

Status Recruiting

Clinical Trial Summary

Using gene therapy to express active telomerase (hTERT) in human cells has the potential to treat many neurodegenerative diseases related to aging, including Alzheimer's disease (AD).

This study will entail treating subjects with hTERT delivered via transduction using AAV. The goal is to extend the telomeres to prevent, delay, or even reverse the development of the pathology of AD. It is expected to have a direct consequence on cognitive function and quality of life in patients with neurodegenerative diseases, such as AD.

Clinical Trial Description

Patients diagnosed with AD who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravenously (IV) and intrathecally (IT).

Baseline will be performed within 8 weeks of beginning the treatment regimen. The treatment regimen will begin with IV delivery of AAV-hTERT, defined as "Day 0." Safety and efficacy analyses will be conducted at Weeks 1, 4, 13, 26, 39, and 52 post-treatment.

Study objectives

Primary: Safety and Tolerability

1. Investigate the safety and tolerability of AAV-hTERT by IV and IT administration.

Secondary: Provisional Efficacy

1. Investigate LGT's ability to deliver hTERT to human cells and lengthen telomeres.

2. Investigate the effects of lengthening telomeres on AD.

3. Investigate other benefits provided by lengthening telomeres. ;

Related Conditions & MeSH terms

• Alzheimer Disease

• NCT number: NCT04133454
• Study type: Interventional
• Source: Libella Gene Therapeutics
• Contact: Jeff Mathis, PhD
• Phone: 1 785 4100223
• Email: jmathis@libellagt.com
• Status: Recruiting
• Phase: Phase 1
• Start date: October 10, 2019
• Completion date: January 2021