Overview

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

Status:
Unknown status
Trial end date:
2021-01-01
Target enrollment:
0
Participant gender:
All
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.
Phase:
Phase 1
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Libella Gene Therapeutics
Criteria
Inclusion Criteria

Subjects must meet the following inclusion criteria at the screening visit.

1. Age of 45 years or older.

2. Clinical diagnosis of Alzheimer's disease or early signs of dementia.

3. 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).

4. Women must undergo a negative serum pregnancy test at the screening assessment.

5. Subjects, or their representatives, must be able to communicate effectively with the
study staff.

6. 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.

1. 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.

2. Female subjects who are pregnant, expected to become pregnant, or lactating/nursing.

3. 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.

4. Subjects who are intolerant of, or do not wish to receive, IV or IT injections.

5. Subjects who are currently alcoholics and/or use psychoactive substances.

6. Subjects who cannot tolerate venipuncture and/or venous access.

7. 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.

8. Subjects who have received blood or blood products within 30 days prior to screening.

9. 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.

10. Subjects who have a history or evidence of active infection or febrile illness within
7 days prior to the screening assessment.

11. 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.