Safety and Efficacy of Klotho and Follistatin Gene Therapy
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Follistatin and klotho gene therapy.
- Who it may be relevant to
- Registry conditions: Healthy Adults. Basic parameters: 50 years — 80 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Honduras
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Evaluating the Safety and Efficacy of Injectable Combination Klotho and Follistatin Plasmid Gene Therapy in Humans -- An Interventional, Non-Placebo Controlled Pilot Phase Study
Overview
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.
Detailed description
Healthy participants will take part in cognitive and health testing before and after administration of plasmid-delivered nonviral klotho and follistatin gene therapy. The method of administration will be subcutaneous injection into abdominal fat deposits. Klotho and follistatin plasmid gene therapy have the potential to improve physical function, cognitive function, kidney function, body composition, epigenetic age, and subjective well being.
Note that the investigational product will be administered at a site outside of the U.S. which is not under FDA jurisdiction, and only non-treatment pre/post outcome assessments (e.g., cognitive assessments or blood sample collection) occur at the U.S. site.
Interventions
- Genetic Follistatin and klotho gene therapy
Injection of nonviral plasmid-delivered follistatin and klotho gene therapy
Primary outcome measures
- Concentration of Serum α-Klotho Measured by Enzyme-Linked Immunosorbent Assay (ELISA) (pg/mL) [Time frame: 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) [Time frame: 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) [Time frame: Within 1 week after treatment and then 1 month, 2 months, and 3 months after treatment]
Secondary outcome measures (12)
- Change From Baseline in World Health Organization Quality of Life Brief Version (WHOQOL-BREF) Domain Scores (0-100) [Time frame: 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) [Time frame: 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) [Time frame: 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) [Time frame: 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) [Time frame: From 1 month prior to treatment to 3 months after treatment (4 timepoints)]
- Change From Baseline in Picture Vocabulary Test T-Score (Mean 50 ± 10) [Time frame: From 1 month prior to treatment to 3 months after treatment (5 timepoints)]
- Epigenetic Biological Age Estimated From Whole-Genome Deoxyribonucleic Acid (DNA) Methylation Profiles (change from baseline) (years) [Time frame: From 1 month prior to treatment to 3 months after treatment (5 timepoints)]
- Timed one leg stance change from baseline (seconds) [Time frame: From 1 month prior to treatment to 3 months after treatment (4 timepoints)]
- Change from baseline in number of squats performed (count) [Time frame: From 1 month prior to treatment to 3 months after treatment (4 timepoints)]
- Change from baseline in number of push-ups performed (count) [Time frame: From 1 month prior to treatment to 3 months after treatment (4 timepoints)]
- Number of sit-ups performed (change from baseline) (count) [Time frame: From 1 month prior to treatment to 3 months after treatment (4 timepoints)]
- Change from Baseline: Concentration of High-Sensitivity C-Reactive Protein (hs-CRP) Measured by Immunoturbidimetric Assay (mg/L) [Time frame: From 1 month prior to treatment to 3 months after treatment (5 timepoints)]
Eligibility criteria
Inclusion criteria
- 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
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Prevention
Study locations
United States · 1 center
- Apeiron Center — Austin
Honduras · 1 center
- Global Alliance of Regenerative Medicine (GARM) Clinic — Roatán
Identifiers
NCT: NCT07285629 · GIFIRB202507313