Evaluation of Safety and Tolerability of Libella Gene Therapy for the Treatment of Aging: AAV- hTERT

Study Overview
=================
Brief Summary
-----------------
Using gene therapy to express active telomerase (hTERT) in humans has the potential to treat many of the age related diseases, including Aging itself. 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 Aging.

Detailed Description
-----------------
Subjects willing to participate who meet with the inclusion - exclusion criteria, will be treated with a single dose of LGT delivered intravascularly (IV). 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 intravenous (IV) administration. Secondary: Provisional Efficacy To determine changes from baseline in hTERT expression, telomerase activity, and telomere length in cells after treatment with AAV-hTERT. To determine changes from baseline in health and aging-related biomarkers after treatment with AAV-hTERT. To determine changes from baseline in the immune system after treatment with AAV-hTERT. To determine changes from baseline in physical function measures after treatment with AAV-hTERT

Official Title
-----------------
Evaluation of Safety and Tolerability of Libella Gene Therapy (LGT) for the Treatment of Aging: AAV- hTERT

Conditions
-----------------
Aging

Intervention / Treatment
-----------------
* Drug: AAV-hTERT


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Subjects must meet the following inclusion criteria at the screening visit. Age of 45 years or older. Subjects, or their authorized representatives, must be adequately informed and understand the nature and risks of the study and must be able to provide a signature and date in the Informed Consent Form (ICF). Women must undergo a negative serum pregnancy test at the screening assessment. Subjects, or their representatives, must be able to communicate effectively with the study staff. Subjects, or their authorized representatives, must certify that they are able and willing to follow all protocol requirements and study restrictions. Exclusion Criteria: Subjects are not eligible for the study if they meet any of the following criteria at the screening visit, unless otherwise specified. Subjects who have a history of allergy, hypersensitivity, or intolerance to any medications, components, or excipients of the investigational product, which cannot be resolved by the staff conducting the study. Female subjects who are pregnant, expected to become pregnant, or lactating/nursing. Subjects who are sexually active and who are unwilling or unable to use a method of effective contraception (e.g., hormonal and/or barrier) during their participation in the study. Subjects who are intolerant of, or do not wish to receive, IV or IT injections. Subjects who are currently alcoholics and/or use psychoactive substances. Subjects who cannot tolerate venipuncture and/or venous access. Subjects who have donated or had a significant loss of whole blood (480 ml or more) within 30 days, or donated plasma or platelets within 14 days prior to screening. Subjects who have received blood or blood products within 30 days prior to screening. Subjects who have been treated with another research product 30 days prior to the screening assessment, or plan to participate in another clinical trial, while in this study if in the opinion of the principal investigator, may place the subject at risk due to participation in the study, or may influence the results of the study or the subject's ability to complete the study. If more than 30 days have passed since participation in another clinical trial, the study staff must ensure that the subject has recovered from any adverse event (AE) associated with the research product used. Subjects who have a history or evidence of active infection or febrile illness within 7 days prior to the screening assessment. Subjects who have a history of any other clinically significant disease or disorder that, in the opinion of the principal investigator, may place the subject at risk due to participation in the study, or may influence the results of the study or the subject's ability to complete the study. -

Ages Eligible for Study
-----------------
Minimum Age: 45 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
Accepts Healthy Volunteers

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: subjects treated with LGT<br>Subjects will be treated with a single IV dose of LGT (AAV-hTERT) | Drug: AAV-hTERT<br>* subjects will receive a single LGT (AAV-hTERT) treatment via IV administration<br>* Other names: LGT;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse events | Incidence of serious adverse events and adverse events throughout the study | 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| hTERT expression and telomerase activity | Change from baseline in telomere length | 12 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Aging, Gene therapy, AAV, hTert, telomere, Telomerase