Safety and Efficacy of Klotho and Follistatin Gene Therapy

NCT07285629 | Recruiting Early Phase 1

• 1 other identifier: GIFIRB202507313
• Study Type: interventional
• Target: 30
• Locations: 2 countries
• Sites: 2

Brief Summary

The purpose of this study is to investigate the safety and efficacy of a combination klotho and follistatin gene therapy, delivered via a nonviral plasmid in healthy adult volunteers. Additionally, this study seeks to understand the cognitive and health benefits of this gene therapy.

Conditions

Healthy Adults

Keywords

klotho; follistatin; nonviral; gene therapy; plasmid

Outcome Measures

Primary Outcomes (3)

• Concentration of Serum α-Klotho Measured by Enzyme-Linked Immunosorbent Assay (ELISA) (pg/mL)

  Serum α-Klotho protein concentration will be quantified using a validated ELISA. Results will be determined from picograms per milliliter (pg/mL) for each participant at each time point. Higher or lower values have no inherent directionality and will be interpreted in study context.

  From 1 month prior to treatment to 3 months after treatment (5 timepoints)

• Concentration of Serum Follistatin Measured by Enzyme-Linked Immunosorbent Assay (ELISA) (pg/mL)

  Serum follistatin concentration will be quantified using a validated ELISA. Results will be reported as picograms per milliliter (pg/mL) for each participant at each time point. Higher or lower values have no inherent directionality and will be interpreted in study context.

  From 1 month prior to treatment to 3 months after treatment (5 timepoints)

• Number and Percentage of Participants Experiencing Treatment-Emergent Adverse Events as Assessed by Patient-Reported Outcomes Version of Common Terminology Criteria for Adverse Events (PRO-CTCAE)

  Assessed through a checklist version of the PRO-CTCAE with each symptom options being none, mild, moderate, or severe. Items will be scored with 0, 1, 2, 3 respectively. Item responses will be summarized as number and percentage of participants experiencing each adverse event by system/organ class. High scores indicate highest severity of symptoms and low scores indicate no symptoms.

  Within 1 week after treatment and then 1 month, 2 months, and 3 months after treatment

Secondary Outcomes (32)

• Change From Baseline in World Health Organization Quality of Life Brief Version (WHOQOL-BREF) Domain Scores (0-100)

  From 1 month prior to treatment to 3 months after treatment (5 timepoints)

• Change From Baseline in Pattern Comparison Processing Speed Test T-Score (Mean 50 ± 10)

  From 1 month prior to treatment to 3 months after treatment (4 timepoints)

• Change From Baseline in Picture Sequence Memory Test T-Score, Forms A and B (Mean 50 ± 10)

  From 1 month prior to treatment to 3 months after treatment (4 timepoints)

• Change From Baseline in Flanker Inhibitory Control and Attention Test T-Score (Mean 50 ± 10)

  From 1 month prior to treatment to 3 months after treatment (4 timepoints)

• Change From Baseline in Dimensional Change Card Sort Test T-Score (Mean 50 ± 10)

  From 1 month prior to treatment to 3 months after treatment (4 timepoints)

Study Arms (1)

Intervention with Cognitive/Health battery before and after

EXPERIMENTAL

This arm includes cognitive and health battery pre- and post-intervention of the gene therapy

Genetic: Follistatin and klotho gene therapy

Interventions

Follistatin and klotho gene therapy GENETIC

Injection of nonviral plasmid-delivered follistatin and klotho gene therapy

Intervention with Cognitive/Health battery before and after

Eligibility Criteria

• Age: 50 Years - 80 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adults aged 50 to 80 years
• General good health
• Willing to comply with all study-related procedures and visits
• Participant is open to morphological change
• If female, participant agrees to maintain contraception
• If female, participant agrees to take a pregnancy test
• If female, participant agrees to a pregnancy waiver

You may not qualify if:

• Currently enrolled in another clinical trial
• History of cancer, autoimmune disease, or chronic kidney/liver disease
• Use of immunosuppressive therapy
• Pregnant or breastfeeding
• Women of childbearing potential who are unwilling or unable to use effective contraception for the duration of the study.
• Regular use of NMDA (N-methyl-D-aspartate) antagonists (i.e., memantine, ketamine, etc.)
• Regular use of antiplatelet medications (i.e., aspirin)
• Any medical or psychiatric condition that could interfere with participation or pose safety concerns
• Unwilling or unable to provide informed consent

Sponsors & Collaborators

• Minicircle: lead

Study Sites (2)

Apeiron Center

Austin, Texas, 78701, United States

RECRUITING

Global Alliance of Regenerative Medicine (GARM) Clinic

Roatán, Bay Islands, Honduras

NOT YET RECRUITING

MeSH Terms

Interventions

Follistatin

Intervention Hierarchy (Ancestors)

Carrier Proteins; Proteins; Amino Acids, Peptides, and Proteins

Central Study Contacts

Mac Davis

CONTACT

512-630-0882; coordinator@minicircle.io

Study Design

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

Model Details: Non-placebo-controlled

Study Record Dates

• First Submitted: November 27, 2025
• First Posted: December 16, 2025
• Study Start: December 16, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2026
• Last Updated: January 8, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Time Frame: Data will be made available upon publication and will be available indefinitely.
• Access Criteria: Data will be made available to anyone who wishes to access it in an online repository at Zenodo (https://zenodo.org/). The types of data shared will include de-identified data of pre- and post-treatment values for the following measures: serum Klotho levels; serum Follistatin levels; kidney and blood safety panels; questionnaires and adverse event reporting data (all check box questions; however, any open-ended answer data will reviewed to ensure they cannot be used to identify an individual participant - final judgments will be made by the study central coordinator); quantitative muscle function outcomes; all NIH-toolbox measures; epigenetic age.

Locations

HO (1); US (1)