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

May 8, 2024 updated by: Minicircle

A Phase I, Open Label, Single Dose Study to Evaluate the Safety and Efficacy of an Injectable Follistatin Plasmid Gene Therapy in Healthy Subjects

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

43

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Honduras
      • Roatan, Honduras
        • Global Alliance for Regenerative Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

Yes

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Basic Science
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental
Genetic: Follistatin plasmid
Follistatin-344 plasmid

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants With Treatment-Related Adverse Events as Assessed by CTCAE v4.0
Time Frame: 3 months
3 months
Change from Baseline in Follistatin Serum Concentration at 3 months
Time Frame: 3 months
3 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from Baseline in fat-free mass at 3 months via dual x-ray absorptiometry
Time Frame: 3 months
3 months
Change from Baseline in fat mass at 3 months via dual x-ray absorptiometry
Time Frame: 3 months
3 months
Change in intrinsic and extrinsic epigenetic clock age
Time Frame: 3 months
3 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Minicircle

Investigators

  • Principal Investigator: Glenn Terry, MD, Principal Investigator

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 18, 2022

Primary Completion (Actual)

August 31, 2023

Study Completion (Actual)

August 31, 2023

Study Registration Dates

First Submitted

May 8, 2024

First Submitted That Met QC Criteria

May 8, 2024

First Posted (Actual)

May 13, 2024

Study Record Updates

Last Update Posted (Actual)

May 13, 2024

Last Update Submitted That Met QC Criteria

May 8, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • FST-X7

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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