Safety and Efficacy of Klotho and Follistatin Gene Therapy

What Was Tested

In this early phase 1 trial, researchers set out to test Follistatin-344.

It had 1 arm (groups): Intervention with Cognitive/Health battery before and after (experimental).

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

The registry classes the interventions as genetic.

More on the Plan

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.

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.

Who Took Part

14 actual participants took part.

It was open to people of any sex aged from 50 years up to 80 years.

Healthy volunteers could join, not only people with the condition.

Examples of who could join: 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.

The full record lists 7 inclusion and 9 exclusion criteria; among the exclusions: currently enrolled in another clinical trial; history of cancer, autoimmune disease, or chronic kidney/liver disease; use of immunosuppressive therapy.

How the Study Was Run

The set-up was single-group and open-label.

Nothing was hidden in this open-label Follistatin-344 study: the participants and staff both knew the treatment.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2025-12-16 with completion listed for 2026-04-30.

It ran at 2 sites in United States (1) and Honduras (1).

Its stated primary purpose is prevention.

What Was Measured

Primary outcome: Concentration of Serum α-Klotho Measured by Enzyme-Linked Immunosorbent Assay (ELISA) (pg/mL) (time frame: From 1 month prior to treatment to 3 months after treatment (5 timepoints)). Concentration of Serum Follistatin Measured by Enzyme-Linked Immunosorbent Assay (ELISA) (pg/mL) (time frame: From 1 month prior to treatment to 3 months after treatment (5 timepoints)). Number and Percentage of Participants Experiencing Treatment-Emergent Adverse Events as Assessed by Patient-Reported Outcomes Version of Common Terminology Criteria for Adverse Events (PRO-CTCAE) (time frame: Within 1 week after treatment and then 1 month, 2 months, and 3 months after treatment).

It also tracked 32 secondary outcomes, for example Change From Baseline in World Health Organization Quality of Life Brief Version (WHOQOL-BREF) Domain Scores (0-100), Change From Baseline in Pattern Comparison Processing Speed Test T-Score (Mean 50 ± 10), Change From Baseline in Picture Sequence Memory Test T-Score, Forms A and B (Mean 50 ± 10), Change From Baseline in Flanker Inhibitory Control and Attention Test T-Score (Mean 50 ± 10), Change From Baseline in Dimensional Change Card Sort Test T-Score (Mean 50 ± 10) and Change From Baseline in Picture Vocabulary Test T-Score (Mean 50 ± 10).

What This Means in Plain Language

Put simply, this is a early phase 1 trial of Follistatin-344 in 14 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Follistatin-344 compound page, all human trials.