aKLmRNA-mediated Protein Replacement Therapy

NCT07544420 | Not Yet Recruiting Phase 1

• 1 other identifier: AKL003
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 1b randomized, double-blind, placebo-controlled study to assess the safety and tolerability of a proprietary aKLmRNA formulated in lipid nanoparticles. Approximately 21 subjects will be enrolled. Each subject will receive a total of two injections during the study. The cohort will consist of approximately 21 subjects, with each receiving 0.5 mg aKLmRNA (AKL003) or placebo. Subjects will be randomized in a 2:1 ratio (active treatment:placebo). The placebo will be saline, matching the active treatment in both appearance and volume.

Conditions

Aging

Keywords

Longevity; Healthy Aging; Klotho; mRNA

Outcome Measures

Primary Outcomes (2)

• Overall incidence and severity of unsolicited AEs by treatment group

  • To characterize the overall safety and tolerability of multiple administrations of the Investigational Medicinal product (IMP), aKLmRNA (AKL003), in healthy volunteers

  From first dose through end of study (Day 60)

• • Overall incidence and severity of solicited reactogenicity events by treatment group

  • To characterize the overall safety and tolerability of multiple administrations of the Investigational Medicinal product (IMP), aKLmRNA (AKL003), in healthy volunteers

  From first dose through end of study (Day 60)

Secondary Outcomes (2)

• • Increase in serum aKL levels over the mean of the baseline values until end of study

  Baseline through Day 60

• • aKL serum level by treatment group

  Baseline through Day 60

Other Outcomes (11)

• • Change from baseline to day 60 in episodic memory performance measrued by Face-Name Associative Memory Exam

  Baseline through Day 60

• • Change from baseline to day 60 in attention and inhibitory control measured Flanker Test

  Baseline through Day 60

• • Change from baseline to day 60 in working memory performance measured by NIH Toolbox List Sorting Working Memory Test

  Baseline through Day 60

Study Arms (2)

aKLmRNA (AKL003)

EXPERIMENTAL

Each subject will be treated every 4 weeks for a total of 2 times with aKLmRNA (AKL003)

Drug: aKLmRNA (AKL003)

Placebo

PLACEBO COMPARATOR

Each subject will be treated every 4 weeks for a total of 2 times with TRIS-Buffer

Other: Placebo

Interventions

aKLmRNA (AKL003) DRUG

Each subject will be treated every 4 weeks for a total of 2 times with aKLmRNA via i.v.

aKLmRNA (AKL003)

Placebo OTHER

Each subject will be treated every 4 weeks for a total of 2 times with TRIS buffer

Placebo

Eligibility Criteria

• Age: 50 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• Subjects will be excluded from the trial if they present one or more of the following conditions:
• Known of suspected allergy to IMP or related procedure
• Known or suspected allergy, or history of anaphylaxis, to vaccines or their excipients, if considered relevant by the Investigator
• Contraindications for the use of emergency treatments
• Clinically relevant findings at screening, e.g., active diseases of any kind particularly infections
• History of chronic alcohol or drug abuse/ dependence within the past 5 yearsUse of any prescription or over-the-counter medication within 7 days prior to first dosing or planned use during the study period, with the exception of medications considered medically necessary and administered at a stable dose and regimen. Stable medication is defined as medication for which no initiation, discontinuation, or dose adjustment has occurred for at least 3 monthsprior to first dosing and for which no change is anticipated during the study period. Occasional use of paracetamol (acetaminophen) and/or ibuprofen is permitted at the discretion of the investigator.
• Employee at the study site, spouse/partner or relative of any study staff (e.g., investigator, sub investigators, or study nurse) or employee of the Sponsor
• Known or suspected pregnancy, planned pregnancy of lactation
• Clinically significant unstable psychiatric illness in the past 6 months
• Presence of autoimmune disease, autoinflammatory syndrome, or immunological deficiency syndrome (including human immunodeficiency virus (HIV) infection)
• Relevant cardiovascular, hepatic, gastroenterological, respiratory, endocrinological, hematologic disease, or any other condition that, in the Investigator's opinion, could interfere with the analyses of safety and efficacy in this study, unless patient has been on stable doses of medication for any of these concurrent illnesses for at least 3 months prior to study entry
• Presence of end stage kidney failure (on dialysis)
• Current or anticipated use of immunosuppressive drugs such as, but not limited to, azathioprine, cyclosporine, methotrexate, tacrolimus, or mycophenylate within 2 months or any myelosuppressive or cytotoxic chemotherapies within the 12 months prior to the screening visit. Use of systemic corticosteroids equivalent to ≥20mg/week prednisone within the past 4 weeks before screening and during the course of the study (intranasal or inhaled steroids for allergies/asthma is allowed)
• History within the last 2 years of a primary or recurrent malignant disease with the exception of resected cutaneous squamous cell carcinoma in situ, basal cell carcinoma, cervical carcinoma in situ, or in situ prostate cancer with a normal prostate-specific antigen posttreatment; or has a life expectancy of \<2 years.
• Patients with long covid-19 showing long-term neurological sequelae within the past 12 months at the time of screening

Sponsors & Collaborators

• Klothea Bio Inc: lead

Study Sites (1)

GARM

Roatán, Bay Islands, 34101, Honduras

Location

Study Officials

• Douglas A Tucker, MD

  GARM

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Agustin Fernandez III

CONTACT

786-542-5499; AFS@advantagetherapeutics.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2026
• First Posted: April 22, 2026
• Study Start (Estimated): May 15, 2026
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2026
• Last Updated: April 22, 2026

Record last verified: 2026-04

Locations

HO (1)