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

Minicircle

Status and phase

Completed

Phase 1

Conditions

Aging

Frailty

Treatments

Genetic: Follistatin plasmid

Study type

Interventional

Funder types

Industry

Identifiers

NCT06411366

FST-X7

Details and patient eligibility

About

This study examines the safety and effects of an injectable plasmid gene therapy. Plasmids are circular pieces of DNA which have been widely studied as a non-permanent & non-heritable method for transferring genes and inducing gene expression. 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. Follistatin is a myostatin and activin inhibitor that has demonstrated improved functional outcomes in mouse models of neuromuscular disease.

Participants will undergo dual energy x-ray absorptiometry scans before and after the treatment's administration to compare change from baseline and rates of change in fat vs muscle tissue and bone density. Participants will also undergo metabolic and epigenetic blood panels to observe any changes. Participants will be monitored at the clinic site for a short time period after receiving the therapy and participants will be able to report any adverse events through an online form. Lastly, participants will have blood drawn just prior to, and three months after, the gene therapy's administration in order to assess circulating levels of follistatin. This study is administered at the Global Alliance for Regenerative Medicine clinical research site on the island of Roatan and is sponsored by Minicircle. The main contact for this study is Mac Davis.

Enrollment

43 patients

Sex

All

Ages

22+ years old

Volunteers

Accepts Healthy Volunteers

Inclusion criteria

Openness to morphological change
Will practice contraception for the duration of trial (if female)

Exclusion criteria

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
Systolic blood pressure (supine) ≤90 mmHg;
Resting heart rate > 100 bpm
Unwilling or unable to give written informed consent.