Phase I: Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Humans

What Was Tested

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

This study examines the safety and effects of an injectable plasmid gene therapy.

In this study the plasmid is a gene vector which contains the human FST344 gene intended to express and secrete bioidentical human follistatin into serum circulation.

It had 1 arm (groups): Experimental (experimental).

The registry classes the interventions as genetic.

Who Took Part

Enrollment: 43 actual participants took part.

It was open to people of any sex aged from 22 years with no upper age limit.

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

Examples of who could join: Openness to morphological change. Will practice contraception for the duration of trial (if female).

The full record lists 2 inclusion and 6 exclusion criteria; among the exclusions: women who are pregnant, nursing or of childbearing potential who are unwilling or unable to maintain contraceptive therapy for the duration of the study; life expectancy \< 6 months due to concomitant illness; pre-existing joint, ligament, or heart issues.

How the Study Was Run

It was run as a single-group and open-label study.

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 2022-08-18 with completion listed for 2023-08-31.

It ran at 1 site in Honduras.

Its stated primary purpose is basic science.

What Was Measured

Primary outcome: Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0 (time frame: 3 months). Change from Baseline in Follistatin Serum Concentration at 3 months (time frame: 3 months).

It also tracked 3 secondary outcomes, for example Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry, Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry and Change in intrinsic and extrinsic epigenetic clock age.

What This Means in Plain Language

Put simply, this is a phase 1 trial of Follistatin-344 in 43 people.

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.