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

Phase 1

• Conditions: Alzheimer Disease
• Interventions: Drug: AAV-hTERT

• Registration Number: NCT04133454
• Lead Sponsor: Libella Gene Therapeutics

Brief 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.

Study Timeline

• Study First Submitted: 2017-12-28
• Status Verified: 2019-10
• Study Start: 2019-10-10
• Study First Submitted QC: 2019-10-17
• Study First Posted: 2019-10-21
• Last Update Submitted: 2019-10-24
• Last Update Posted: 2019-10-28
• Primary Completion: 2021-01
• Study Completion: 2021-01

Recruitment & Eligibility

• Status: UNKNOWN
• Sex: All
• Target Recruitment: 5

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
subjects treated with LGT	AAV-hTERT	subjects will be treated with a single dose of LGT (AAV-hTERT)

Primary Outcome Measures

Name	Time	Method
Incidence of adverse events	12 months	Incidence of serious adverse events and adverse events throughout the study

Secondary Outcome Measures

Name	Time	Method
hTERT expression and telomerase activity	12 months	Change from baseline of the telomere length

Trial Locations

Locations (1)

IPS Arcasalud SAS; 🇨🇴 Zipaquirá, Cundinamarca, Colombia