NCT04133454

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

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

• Status: Unknown
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 5 (estimated)
• Sponsor: Libella Gene Therapeutics · Industry
• Sex: All
• Age: 45 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Alzheimer Disease

Interventions

Type	Name	Description
DRUG	AAV-hTERT	subjects will receive a single LGT (AAV-hTERT) treatment via IV and IT administration

Timeline

• Start date: 2019-10-10
• Primary completion: 2021-01-01
• Completion: 2021-01-01
• First posted: 2019-10-21
• Last updated: 2019-10-28

Locations

1 site across 1 country: Colombia