Safety and Efficacy of Injectable Klotho Plasmid Gene Therapy in Humans

Minicircle

Status and phase: Enrolling; Phase 1

Conditions: Healthy Adults

Treatments: Genetic: Injectable Plasmid Klotho Gene Therapy

Study type: Interventional

Funder types: Industry

Identifiers

NCT07216781

MINICIRCLE-KLO

Details and patient eligibility

About

The purpose of this study is to investigate the safety and efficacy of a gene therapy for Klotho, delivered via a nonviral plasmid in healthy adult volunteers. Additionally, this study seeks to understand the cognitive and health benefits of the Klotho gene therapy.

Full description

Note that the investigational product will be administered at a site outside of the U.S. which is not under FDA jurisdiction, and only non-treatment pre/post outcome assessments (e.g., cognitive assessments or blood sample collection) occur at the U.S. site.

Participants will take part in cognitive and health testing before and after administration of plasmid-delivered Klotho gene therapy. The method of administration will be subcutaneous injection into abdominal fat deposits. Klotho may improve cognitive function, kidney function, healthspan, and lifespan. Healthy volunteers will partake in a series of blood draws, health screenings, questionnaires, brain perfusion/function measures, and cognitive testing multiple times before and after the intervention.

Enrollment: 24 estimated patients

Sex: All

Ages: 23 to 90 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Participant is open to morphological change
• If female, participant agrees to maintain contraception
• If female, participant agrees to take a pregnancy test
• If female, participant agrees to a pregnancy waiver

Exclusion criteria

• Women of childbearing potential who are unwilling or unable to use effective contraception for the duration of the study
• History of cancer diagnosis
• Preexisting medical issues that may be exacerbated by the treatment
• Has received any gene therapy within the past 12 months
• Unwilling or unable to provide written informed consent

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

24 participants in 1 patient group

Schedule of Administration and Sample Selection

Experimental group

Description:

This arm includes cognitive and health battery at multiple intervals pre- and post-administration of Klotho

Treatment:

Genetic: Injectable Plasmid Klotho Gene Therapy