Klotho Gene Therapy for Healthy Adults

Recruiting at 1 trial location

Overseen By Mac Davis

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Minicircle

Disqualifiers: Cancer, Gene therapy, others

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

Compensation: Varies

Number of Visits: 4

Duration: 1 day

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests the safety and effectiveness of a new gene therapy that uses a plasmid (a small DNA molecule) to deliver the Klotho gene in healthy adults. Researchers aim to determine if this gene therapy can improve cognition and overall health. Participants receive an injection and take part in health and memory tests before and after the treatment. Individuals who have not had cancer, gene therapy in the past year, or serious health problems, and are open to physical changes, might be suitable for this trial. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects on people.

Do I need to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research shows that the Klotho gene therapy, delivered using a nonviral plasmid, is currently being tested for safety in humans. Previous patients have generally tolerated this type of gene therapy well. In one study, a similar Klotho gene transfer did not cause any serious side effects. Another study using a different method with nanoparticles also demonstrated safe administration. While these results are encouraging, it is important to remember that this trial is in its early stages, focusing on safety and understanding potential effects. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments, which often aim at managing symptoms or slowing progression, Klotho Gene Therapy directly enhances the expression of the Klotho gene, which is associated with longevity and improved cognitive function. This therapy is unique because it uses a plasmid to deliver the Klotho gene into the body, potentially offering a more direct and lasting impact on the aging process. Researchers are excited because this method could lead to improvements in cognitive health and overall well-being, potentially offering a new way to counteract age-related decline.

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

Research has shown that Klotho gene therapy might offer several health benefits. Animal studies have found that this therapy can improve memory, kidney function, and overall health, and may even extend lifespan. In one study, mice treated with Klotho gene therapy lived almost 20% longer and showed improvements in age-related changes in bones, muscles, and the brain. Although research in humans remains limited, these findings suggest that the therapy could potentially enhance brain function and increase healthy years of life in people. Participants in this trial will receive Klotho gene therapy to further investigate these potential benefits in humans. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for healthy adults who want to participate in a study testing the safety and effectiveness of a new gene therapy. Specific eligibility criteria are not provided, so generally, participants should be in good health without any major medical conditions.

Inclusion Criteria

If female, participant agrees to maintain contraception

If female, participant agrees to a pregnancy waiver

I agree to take a pregnancy test if I am female.

Exclusion Criteria

Unwilling or unable to provide written informed consent

I am not using birth control and do not plan to start during the study.

I have been diagnosed with cancer before.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous injection of Klotho plasmid gene therapy

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including cognitive and health assessments

6 months

Multiple visits (in-person and virtual) at 3 days, 7 days, 1 month, 3 months, and 6 months

What Are the Treatments Tested in This Trial?

Interventions

• Injectable Plasmid Klotho Gene Therapy

Trial Overview

The trial is examining an injectable Klotho gene therapy delivered through a nonviral plasmid. Researchers aim to determine if it's safe for use and what potential cognitive and overall health benefits it may offer.

How Is the Trial Designed?

Group I: Schedule of Administration and Sample Selection Experimental Treatment 1 Intervention

This arm includes cognitive and health battery at multiple intervals pre- and post-administration of Klotho

Who Is Running the Clinical Trial?

Minicircle

Lead Sponsor

Trials

1

Recruited

40+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07216781

Safety and Efficacy of Injectable Klotho Plasmid Gene ...

The purpose of this study is to investigate the safety and efficacy of a gene therapy for Klotho, delivered via a nonviral plasmid in healthy adult volunteers.

2.

trialx.com

trialx.com/clinical-trials/listings/325279/safety-and-efficacy-of-injectable-klotho-plasmid-gene-therapy-in-humans/

Safety and Efficacy of Injectable Klotho Plasmid Gene ...

Klotho may improve cognitive function, kidney function, healthspan, and lifespan. Healthy volunteers will partake in a series of blood draws, ...

3.

patlynk.com

patlynk.com/trial/NCT07216781

Klotho Plasmid Gene Therapy for Prevention in Humans

The Klotho gene is thought to be beneficial for improving memory, kidney function, and overall health and longevity.

4.

withpower.com

withpower.com/trial/phase-1-healthy-adults-9-2025-80d9e

Klotho Gene Therapy for Healthy Adults

The purpose of this study is to investigate the safety and efficacy of a gene therapy for Klotho, delivered via a nonviral plasmid in healthy adult ...

5.

nad.com

nad.com/news/one-time-anti-aging-gene-therapy-treatment-prolongs-life-by-20-new-study-shows

One-Time Anti-Aging Gene Therapy Treatment Prolongs ...

Klotho gene therapy prolongs the lifespan of male mice by 19.7%. · The one-time gene therapy improves bone, muscle, and brain aging markers.

6.

sciencedirect.com

sciencedirect.com/science/article/pii/S0142961224004605

Nanoparticle-mediated Klotho gene therapy prevents acute ...

Our results showed that the PDA-PEI-l-serine-Klotho plasmid nanoparticles (PPSK NPs) could safely and efficiently deliver the Klotho gene to injured TECs ...

7.

maplespub.com

maplespub.com/article/Safety-Study-of-AAV-hTert-and-Klotho-Gene-Transfer-Therapy-for-Dementia

Safety Study of AAV hTERT and KLOTHO gene transfer ...

The therapy demonstrated a very high safety profile with no serious adverse effects identified. Clinical evaluation of the patients over the course of the one ...