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

NCT06411366 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 43

Last updated 2024-05-13

No results posted yet for this study

Summary

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.

Conditions

Interventions

GENETIC

Follistatin plasmid

Follistatin-344 plasmid

Sponsors & Collaborators

  • Minicircle

    lead INDUSTRY

Principal Investigators

  • Glenn Terry, MD · Principal Investigator

Study Design

Allocation
NA
Purpose
BASIC_SCIENCE
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
22 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2022-08-18
Primary Completion
2023-08-31
Completion
2023-08-31

Countries

  • Honduras

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06411366 on ClinicalTrials.gov