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

NCT06411366 | Completed Phase 1

• 1 other identifier: FST-X7
• Study Type: interventional
• Target: 43
• Locations: 1 country
• Sites: 1

Brief 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

Frailty; Aging

Outcome Measures

Primary Outcomes (2)

• Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0

  3 months

• Change from Baseline in Follistatin Serum Concentration at 3 months

  3 months

Secondary Outcomes (3)

• Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry

  3 months

• Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry

  3 months

• Change in intrinsic and extrinsic epigenetic clock age

  3 months

Study Arms (1)

Experimental

EXPERIMENTAL

Genetic: Follistatin plasmid

Interventions

Follistatin plasmid GENETIC

Follistatin-344 plasmid

Experimental

Eligibility Criteria

• Age: 22 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Minicircle: lead

Study Sites (1)

Global Alliance for Regenerative Medicine

Roatán, Honduras

Location

MeSH Terms

Conditions

Frailty

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Glenn Terry, MD

  Principal Investigator

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 8, 2024
• First Posted: May 13, 2024
• Study Start: August 18, 2022
• Primary Completion: August 31, 2023
• Study Completion: August 31, 2023
• Last Updated: May 13, 2024

Record last verified: 2024-05

Locations

HO (1)