Safety and Efficacy of Klotho and Follistatin Gene Therapy
ConditionHealthy Adults
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age50-80
SponsorMinicircle
About this trial
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.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
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
Disqualifiers
Currently enrolled in another clinical trial
History of cancer, autoimmune disease, or chronic kidney/liver disease
Use of immunosuppressive therapy
Pregnant or breastfeeding
Trial design
Design model
Single group
Treatments tested in this trial
Follistatin and klotho gene therapy
GeneticInjection of nonviral plasmid-delivered follistatin and klotho gene therapy
Treatment groups
30 Participants
are divided into 1 treatment groupGroup A: Intervention with Cognitive/Health battery before and afterExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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.
Time frame
From 1 month prior to treatment to 3 months after treatment (5 timepoints)2
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.
Time frame
From 1 month prior to treatment to 3 months after treatment (5 timepoints)3
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.
Time frame
Within 1 week after treatment and then 1 month, 2 months, and 3 months after treatmentSecondary outcomes
1
Change From Baseline in World Health Organization Quality of Life Brief Version (WHOQOL-BREF) Domain Scores (0-100)
The WHOQOL-BREF is a self-report questionnaire that includes four domains: Physical Health, Psychological, Social Relationships, and Environment. Each domain score is transformed to a 0-100 scale, with higher scores indicating better quality of life. Changes from baseline will be examined per domain.
Time frame
From 1 month prior to treatment to 3 months after treatment (5 timepoints)2
Change From Baseline in Pattern Comparison Processing Speed Test T-Score (Mean 50 ± 10)
The Pattern Comparison Processing Speed Test measures processing speed using age-adjusted T-scores (mean 50, SD 10). Higher scores reflect faster cognitive processing. Changes from baseline will be analyzed per participant and time point.
Time frame
From 1 month prior to treatment to 3 months after treatment (4 timepoints)3
Change From Baseline in Picture Sequence Memory Test T-Score, Forms A and B (Mean 50 ± 10)
Picture Sequence Memory Test evaluates episodic memory. Each form (A and B) yields an age-adjusted T-score (mean 50, SD 10). Higher scores indicate better memory performance. Forms A and B will be averaged.
Time frame
From 1 month prior to treatment to 3 months after treatment (4 timepoints)4
Change From Baseline in Flanker Inhibitory Control and Attention Test T-Score (Mean 50 ± 10)
The Flanker Inhibitory Control and Attention Test measures inhibitory control and attention. Scores are age-adjusted T-scores (mean 50, SD 10). Higher scores indicate better performance. Changes from baseline will be analyzed per participant and time point.
Time frame
From 1 month prior to treatment to 3 months after treatment (4 timepoints) Source ClinicalTrials.gov