Safety and Efficacy of Klotho and Follistatin Gene Therapy

Minicircle

Status and phase: Enrolling; Early Phase 1

Conditions: Healthy Adults

Treatments: Genetic: Follistatin and klotho gene therapy

Study type: Interventional

Funder types: Industry

Identifiers

NCT07285629

GIFIRB202507313

Details and patient eligibility

About

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

Full description

Healthy participants will take part in cognitive and health testing before and after administration of plasmid-delivered nonviral klotho and follistatin gene therapy. The method of administration will be subcutaneous injection into abdominal fat deposits. Klotho and follistatin plasmid gene therapy have the potential to improve physical function, cognitive function, kidney function, body composition, epigenetic age, and subjective well being.

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.

Enrollment: 30 estimated patients

Sex: All

Ages: 50 to 80 years old

Volunteers: Accepts Healthy Volunteers

Inclusion criteria

• Adults aged 50 to 80 years
• General good health
• Willing to comply with all study-related procedures and visits
• 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

• Currently enrolled in another clinical trial
• History of cancer, autoimmune disease, or chronic kidney/liver disease
• Use of immunosuppressive therapy
• Pregnant or breastfeeding
• Women of childbearing potential who are unwilling or unable to use effective contraception for the duration of the study.
• Regular use of NMDA (N-methyl-D-aspartate) antagonists (i.e., memantine, ketamine, etc.)
• Regular use of antiplatelet medications (i.e., aspirin)
• Any medical or psychiatric condition that could interfere with participation or pose safety concerns
• Unwilling or unable to provide informed consent

Trial design

Primary purpose: Prevention

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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30 participants in 1 patient group

Intervention with Cognitive/Health battery before and after

Experimental group

Description:

This arm includes cognitive and health battery pre- and post-intervention of the gene therapy

Treatment:

Genetic: Follistatin and klotho gene therapy