Klotho and Follistatin Gene Therapy for Healthy Adults

Recruiting at 1 trial location

Overseen By Mac Davis

Age: 18+

Sex: Any

Trial Phase: Phase < 1

Sponsor: Minicircle

Must not be taking: NMDA antagonists, Antiplatelets

Disqualifiers: Cancer, Autoimmune disease, Chronic kidney/liver disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 10

Duration: 1 day

30 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new gene therapy that combines klotho and follistatin to assess its safety and effectiveness. Researchers aim to determine if this treatment can enhance brain health and overall well-being in healthy adults. The trial seeks individuals aged 50 to 80 who are generally healthy and open to changes in their physical appearance. As an Early Phase 1 trial, participants will be among the first to receive this innovative treatment, aiding researchers in understanding its effects on people.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications, like NMDA antagonists (used for conditions like Alzheimer's) and antiplatelet medications (like aspirin). If you're on these, you would need to stop them to participate.

Is there any evidence suggesting that this treatment is likely to be safe for humans?

Previous studies found that patients receiving follistatin gene therapy sometimes experienced a small increase in LDL cholesterol. This occurred in about one-third of patients and could be managed with dietary changes. Research on the klotho protein suggests it protects cells from oxidative stress damage. However, detailed safety information for klotho in humans remains limited.

As an early-phase study, this trial represents one of the initial stages of human testing. Scientists are still learning about the treatment's tolerability. Available data suggests that follistatin might cause manageable cholesterol changes, while more information is needed to understand klotho's safety. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about Klotho and Follistatin gene therapy because it introduces a novel approach to enhancing cognitive and physical health. Unlike traditional treatments that may focus on managing symptoms, these therapies aim to modify the body's genes to promote healthier aging processes. Klotho and Follistatin are proteins that play roles in protecting brain cells and supporting muscle growth, respectively. By directly targeting these proteins, this gene therapy has the potential to offer more sustained and comprehensive benefits compared to current treatment options.

What evidence suggests that this gene therapy could be effective for cognitive and health benefits?

Research has shown that gene therapy using klotho and follistatin might offer significant benefits. In animal studies, klotho gene therapy increased the lifespan of adult mice by 20%, suggesting potential longevity benefits for humans. Research in humans indicates that follistatin can increase muscle mass and reduce fat in areas like the thighs. These findings suggest possible health and mental benefits. Although direct evidence in humans remains limited, the positive results in animals and early human indicators suggest that this therapy, currently under trial, could improve overall health. ^{1 3 4 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy adults aged 50 to 80 who agree to follow study procedures and are open to body changes. Women must not be pregnant, agree to contraception, and take pregnancy tests. Excluded are those with serious health conditions, cancer history, or on certain medications.

Inclusion Criteria

If female, participant agrees to a pregnancy waiver

I am between 50 and 80 years old.

General good health

Exclusion Criteria

Unwilling or unable to provide informed consent

Pregnant or breastfeeding

Any medical or psychiatric condition that could interfere with participation or pose safety concerns

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Pre-Treatment Assessment

Cognitive and health testing before administration of gene therapy

1 month

1 visit (in-person)

Treatment

Administration of klotho and follistatin gene therapy via subcutaneous injection

1 day

1 visit (in-person)

Post-Treatment Assessment

Cognitive and health testing after administration of gene therapy

3 months

4 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

3 months

4 visits (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• Klotho and Follistatin Gene Therapy

Trial Overview

The trial studies the safety and effectiveness of klotho and follistatin gene therapy in healthy volunteers. It involves a nonviral plasmid delivery system aiming at cognitive and overall health improvements.

How Is the Trial Designed?

Group I: Intervention with Cognitive/Health battery before and after Experimental Treatment 1 Intervention

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

Who Is Running the Clinical Trial?

Minicircle

Lead Sponsor

Trials

1

Recruited

40+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07285629?aggFilters=status%3Anot

Safety and Efficacy of Klotho and Follistatin Gene Therapy

The purpose of this study is to investigate the safety and efficacy of a combination klotho and follistatin gene therapy, delivered via a ...

2.

ctv.veeva.com

ctv.veeva.com/study/safety-and-efficacy-of-klotho-and-follistatin-gene-therapy

Safety and Efficacy of Klotho and Follistatin Gene Therapy

The purpose of this study is to investigate the safety and efficacy of a combination klotho and follistatin gene therapy, delivered via a ...

3.

nmn.com

nmn.com/news/gene-therapy-for-aging-new-advances-that-could-extend-lifespan

Gene Therapy for Aging: New Advances That Could ...

As far as the data on follistatin gene therapy goes, after treatment, her thighs exhibited gained muscle mass and loss of fat between muscles.

4.

fightaging.org

fightaging.org/archives/2025/05/klotho-gene-therapy-in-adult-mice-produces-a-20-increase-in-life-span/

Klotho Gene Therapy: 20% Life Span Increase in Mice

Researchers here report a 20% increase in life span for adult mice given a gene therapy to express the circulating factor klotho.

5.

sciencedirect.com

sciencedirect.com/science/article/abs/pii/S0006291X2501424X

Mini review: Gene therapy targets for aging-associated ...

TERT, KLOTHO, FoxOs, SIRTs show promise for age-related disease treatment. Key challenges: immunogenicity, limited capacity, off-targets, cancer risk, ethics.

6.

trial.medpath.com

trial.medpath.com/clinical-trial/23d6cb9927e8a04c/nct07285629-klotho-follistatin-gene-therapy-safety-efficacy

Safety and Efficacy of Klotho and Follistatin Gene Therapy | MedPath

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 ...

7.

minicircle.io

minicircle.io/our-therapies/follistatin/

Follistatin Gene Therapy (FST-344) | Build Muscle, Reduce ...

The most common side effect observed is a slight increase (8mg/dl) in LDL cholesterol in about a third of patients, which can be managed through diet and ...