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OpenTrials
Completed

NCT Number: NCT06411366

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

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.

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Key information

Conditions

Frailty Aging Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

22 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Global Alliance for Regenerative Medicine

Roatán, Honduras

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Follistatin plasmid

Genetic

Follistatin-344 plasmid

Primary outcomes

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

    Time frame: 3 months

  2. Change from Baseline in Follistatin Serum Concentration at 3 months

    Time frame: 3 months

Secondary outcomes

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

    Time frame: 3 months

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

    Time frame: 3 months

  3. Change in intrinsic and extrinsic epigenetic clock age

    Time frame: 3 months

Sponsors and collaborators

Lead sponsor

Minicircle

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2023
Study completion
2023
First posted
May 13, 2024
Registry last updated
May 13, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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