A Phase 1 Study to Assess the Safety and Effects of Single and Multiple Doses of KS101 in Healthy Volunteers

NCT07143331 | Not Yet Recruiting Phase 1 DMC

• 1 other identifier: KS101-CLIN-001
• Study Type: interventional
• Target: 84
• Locations: 1 country
• Sites: 1

Brief Summary

This study is the first clinical trial involving study drug KS101. The goal of this clinical trial is to investigate whether KS101 is safe, whether it causes side effects, and how KS101 is broken down in the body, in healthy participants. This information will be used to learn more about KS101 and to determine the most effective dose for age related diseases such as chronic kidney disease and Alzheimer's Disease, with the fewest unwanted side effects. There are 3 parts to this study. In Part 1, participants will take KS101 or a placebo once and will stay in the study centre for a 4-night inpatient stay. Participants will return for outpatient visits on Days 8 and 29. In Part 2, participants will take KS101 twice, once after a meal and once without a meal and will stay in the study centre for a 7-night inpatient stay. Participants will return for outpatient visits on Days 11 and 32. In Part 3, participants will take KS101 or a placebo once daily for 5 days and will stay in the study centre for an 8-night inpatient stay. Participants will return for outpatient visits on Days 12 and 33.

Conditions

Healthy Participants; Alzheimer Disease; Chronic Kidney Disease

Keywords

alzheimer disease; chronic kidney disease

Outcome Measures

Primary Outcomes (8)

• To assess the safety and tolerability of single and multiple ascending oral doses of KS101 in healthy adult volunteers.

  Number of participants reporting treatment emergent adverse events (TEAEs) including treatment-related TEAEs, serious adverse events (SAEs) and TEAEs of severity grade 3 or above.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes observed during the physical examination.

  Presence of clinically significant changes in participants physical examinations post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in haematology parameters.

  Presence of clinically significant changes in participants haematology parameters post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in serum biochemistry.

  Presence of clinically significant changes in participants serum biochemistry parameters post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in coagulation parameters.

  Presence of clinically significant changes in participants coagulation parameters post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in urinalysis parameters.

  Presence of clinically significant changes in participants urinalysis parameters post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in vital signs.

  Presence of clinically significant changes in participants vital signs post-first dose.

  Through to end of study, up to 63 days.

• Evaluation of the safety and tolerability of a single oral dose of KS101 taken after a high-fat meal, assessed by changes in ECG parameters.

  Presence of clinically significant changes in participants ECG parameters post-first dose.

  Through to end of study, up to 63 days.

Secondary Outcomes (22)

• Pharmacokinetics parameter - High-fat, high-calorie meal effects maximum observed concentration (Cmax).

  Through to 7 days post last dose.

• Pharmacokinetics parameter - High-fat, high-calorie meal effects on Area under the curve (AUC).

  Through to 7 days post last dose.

• Pharmacokinetics parameter - High-fat, high-calorie meal effects on time for maximum observed Concentration (Tmax).

  Through to 7 days post last dose.

• Pharmacokinetics parameter - single dose of KS101 effects maximum observed concentration (Cmax).

  Through to 7 days post last dose.

• Pharmacokinetics parameter - multiple doses of KS101 effects maximum observed concentration (Cmax).

  Through to 7 days post last dose.

Other Outcomes (1)

• Characterize the PK profile of KS101 in the urine of healthy adult participants after single ascending oral doses of KS101.

  Through to 24 hours post KS101 dose.

Study Arms (5)

Part 1 KS101

EXPERIMENTAL

Participants will be enrolled in 1 of 6 single ascending dose cohorts. Six participants in each cohort will receive KS101.

Drug: Part 1 KS101

Part 1 Placebo

PLACEBO COMPARATOR

6 doses of placebo will be administered to participants

Drug: Part 1 Placebo

Part 2 KS101 with Food

EXPERIMENTAL

KS101 with food

Drug: Part 2 with Food

Part 2 KS101 without Food

EXPERIMENTAL

KS101 without food

Drug: Part 2 without food

Part 3 KS101

EXPERIMENTAL

Multiple ascending doses of KS101

Drug: Part 3 KS101

Interventions

Part 1 KS101 DRUG

Six doses of oral KS101 will be administered to participants.

Part 1 KS101

Part 1 Placebo DRUG

Six doses of oral matching placebo will be administered to participants.

Part 1 Placebo

Part 2 with Food DRUG

KS101 administered with food

Part 2 KS101 with Food

Part 2 without food DRUG

KS101 administered without food

Part 2 KS101 without Food

Part 3 KS101 DRUG

3 multiple doses of KS101

Part 3 KS101

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Provided voluntary, written informed consent before any study-specific activities are performed.
• Male or female, aged 18 to 55 years inclusive at time of informed consent.
• Participants of childbearing potential must agree to the use of a double method of contraception of a highly effective method of birth control (refer to Appendix 11.1) combined with a barrier contraceptive (condom) when appropriate from screening visit to until 60 days after the last dose of IMP (covering a full menstrual cycle of 30 days starting after 5 half-lives of last dose of IMP). Childbearing potential is defined as fertile and following menarche until becoming post-menopausal, unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. Post-menopausal is defined as no menses for 12 months without an alternative medical cause and a Screening follicle stimulating hormone (FSH) level \>30 U/L (or at the local laboratory levels for post-menopause).
• Male participants with sexual partners of childbearing potential must agree to use a double method of contraception including condom with a highly effective method of birth control by the partner of childbearing potential (refer to Appendix 11.1), from the time of informed consent through to 90 days after the last dose of the IMP. Male participants who have undergone sterilisation (i.e., vasectomy ≥6 months before Screening) and have had testing to confirm the success of the sterilisation, may also be included and will not be required to use above described methods of contraception. Male participants must also agree not to donate sperm or plan to father children for up to 90 days after last dose of IMP.
• Non-smoker (or other nicotine use) (defined as ≤5 cigarettes or nicotine equivalent per month) for at least 32 months before Screening according to history, and urine cotinine \<500 ng/mL or carbon monoxide (CO) breath test \<10 ppm at Screening and Day -1.
• Total body weight ≥50 kg (male) or ≥45 kg (female) and body mass index (BMI) between 18.0 and 30.0 kg/m2 inclusive.
• Certified as healthy by comprehensive clinical assessment by the Investigator (detailed medical history, complete physical examination, vital signs, 12-lead electrocardiogram, and clinical laboratory evaluation) and with suitable veins for cannulation.
• Vital signs after 5 minutes resting in supine position (one repeat per timepoint permitted):
• Systolic blood pressure (SBP): 90 - 140 mmHg inclusive
• Diastolic blood pressure (DBP): 50 - 90 mmHg inclusive. DBP 40 - 49 mmHg inclusive is acceptable for eligibility if considered not clinically significant by the Investigator.
• Heart rate (HR): 50 - 90 bpm inclusive. HR 40 - 49 bpm inclusive are acceptable for eligibility if considered not clinically significant by the Investigator.
• Standard 12-lead electrocardiogram (ECG) parameters, after 10 minutes in supine position within the following ranges:
• QRS 50 - 120 msec
• QT ≤500 msec
• QTcF and QTcB ≤450 msec (male) ≤470 msec (female) Also: normal ECG tracing, or ECG tracing abnormality considered by the Investigator to be not clinically significant (not including parameters above).

You may not qualify if:

• Liver function tests at Screening and Day -1: aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\],) \>2 times upper limit of normal (ULN), serum alkaline phosphatase (ALP) or total bilirubin ≥2 times ULN. One re-test per parameter per timepoint is permitted, results must be available before randomization.
• Clinically significant abnormalities in laboratory tests (biochemistry, haematology, coagulation, urinalysis), physical examination, or 12-lead ECG during the Screening period or pre-first dose, that in the opinion of the Investigator, would affect the individual's ability to fully participate in the study and/or not be in the individual's best interest to participate in the study. One re-test per abnormality per scheduled timepoint is permitted
• Pregnant or breastfeeding, or planning to become pregnant or breastfeed, or planning to donate ova during study participation; or positive pregnancy test at screening/pre-first dose. Males planning to father children or unwilling to abstain from sperm donation within 90 days of the last dose of study product.
• History or presence of clinically relevant (as assessed by the Investigator) cardiovascular, broncho-pulmonary, gastrointestinal, hepatic/ gallbladder\*/ bile duct, renal, metabolic, hematological, neurological, musculoskeletal/rheumatological, systemic, ocular, gynaecological, or infectious disease or signs of acute illness. \*including medical history of asymptomatic gallbladder stones.
• Any gastrointestinal surgery or condition or disease that could affect drug absorption, distribution, metabolism or excretion (e.g., gastrectomy, cholecystectomy, diarrhea, recurrent nausea/vomiting).
• History of malignancy of any organ system (other than localized basal cell carcinoma of the skin or in situ cervical cancer considered treated and cured), treated or untreated, within 5 years before screening, regardless of if there is no evidence of local recurrence or metastases.
• History of schizophrenia, bipolar disorder psychoses, disorders requiring lithium, attempted or planned suicide, or any other severe (disabling) chronic psychiatric diagnosis including depression, generalised anxiety and obsessive-compulsive disorders. Hospitalization within 5 years before screening due to psychiatric illness or due to danger to self or others.
• Severe recurring headache (cluster or migrainous headaches) requiring prescription medication.
• Donation of \>450 mL blood or blood products within 8 weeks before first dose.
• Documented evidence of current or past cardiovascular disease including cardiac arrhythmias, clinical significant abnormal cardiac automaticity, or family history of congenital long QT syndrome, Brugada syndrome, or unexplained sudden cardiac death.
• Positive serology test at Screening for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibody, or human immune deficiency virus (HIV) antibody.
• History of alcohol or illicit drug misuse within 2 years before Screening, as determined by the Investigator and/or positive urine drug test at Screening or pre-first dose (unless there is an explanation deemed acceptable by the Investigator \[e.g., false positive\] and/or the participant tests negative upon re-test \[one re-test permitted per scheduled timepoint\]) and/or positive alcohol breath test at Screening or pre-first dose (no re-test permitted).
• Current tobacco smoker or other nicotine use \>5 cigarettes or nicotine equivalent per month within 32 months of Screening, or at Screening or upon admission to the clinical unit had urine cotinine of ≥500 ng/mL or CO breath test \>10 ppm.
• Use of prescription medicines, over-the-counter (OTC) medications (including vitamins), or herbal remedies within 14 days and/or vaccination within 28 days before Day -1. Contraceptives and occasional/non-chronic use (as judged by the Investigator) of non-prescription analgesia (e.g., ibuprofen, paracetamol) are permitted.
• In the judgement of the Investigator, individual is likely to be noncompliant or unable to comply with study requirements during the study for any reason (e.g., inability to return for follow-up visits, uncooperative attitude), and/or has a medical condition jeopardizing involvement in the study. For Part 2 FE, this includes individuals unwilling or unable to consume a high-fat meal within 30 minutes.

Sponsors & Collaborators

• Klotho Sciences Australia Pty Ltd.: lead

Study Sites (1)

Scientia Clinical Research

Sydney, New South Wales, 2031, Australia

Location

MeSH Terms

Conditions

Alzheimer Disease; Renal Insufficiency, Chronic

Interventions

Food

Condition Hierarchy (Ancestors)

Dementia; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Tauopathies; Neurodegenerative Diseases; Neurocognitive Disorders; Mental Disorders; Renal Insufficiency; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Diet, Food, and Nutrition; Physiological Phenomena; Food and Beverages

Central Study Contacts

Dr Youn Hoon Joo

CONTACT

+82 10 4879 6001; drjoo@klothosciences.com

Hyunjong Sung

CONTACT

shj@klothosciences.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2025
• First Posted: August 27, 2025
• Study Start: August 30, 2025
• Primary Completion: April 1, 2026
• Study Completion (Estimated): August 6, 2026
• Last Updated: August 27, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1)