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Single-Dose Escalation Study Followed by a Multiple-Dose Escalation Study of SKL24741 in Healthy Subjects

Phase 1
Completed
Conditions
Healthy
Interventions
Drug: Placebo
Registration Number
NCT04505397
Lead Sponsor
SK Life Science, Inc.
Brief Summary

This study evaluates SKL24741 safety and tolerability in healthy subjects. Subjects will be randomized to receive oral doses of SKL24741 or placebo. This is a two-part, double-blinded, randomized study of SKL24741.

Detailed Description

The study includes a sequential cohort design that is intended to optimize subject safety and assess tolerability, safety, and PK of SKL24741. In the single-dose escalation part of the study (Part A), tolerability, safety, and PK of SKL24741 will be assessed in healthy male subjects under fasting conditions. At an appropriate dose based on PK results, additional cohorts will be repeated with the subjects under fed conditions, in order to evaluate the magnitude on the disposition of SKL24741. To assess the gender effect on the disposition of SKL24741, female subjects will be treated at one dose level under fasting conditions. A preliminary formulation effect may be assessed in a cohort of subjects to compare the capsule versus tablet formulation on the disposition of SKL24741.

Part A Primary Objective: To evaluate the safety and tolerability of single oral ascending doses of SKL24741 administered to healthy male subjects

Part A Secondary Objectives:

* To evaluate the PK of SKL24741 (R-enantiomer), SKL24742 (S-enantiomer) (if appropriate), and its possible metabolites (if deemed necessary) following administration of single oral ascending doses of SKL24741 administered to healthy male subjects

* To assess the food effect on the PK of SKL24741 and SKL24742 (if appropriate) following administration of a single oral dose of SKL24741 administered to healthy male subjects

* To assess the gender effect on the PK of SKL24741 and SKL24742 (if appropriate) following administration of a single oral dose of SKL24741 administered to healthy female subjects

Part A Exploratory Objective: To assess the formulation effect on the PK of SKL24741 and SKL24742 (if appropriate) following administration of a single oral dose of SKL24741 administered to healthy male subjects

Part B Primary Objective: To evaluate safety and tolerability of multiple oral ascending doses of SKL24741 administered for 14 days to healthy male subjects

Part B Secondary Objective: To evaluate the PK of SKL24741, SKL24742 (if appropriate), and its possible metabolites (if deemed necessary) following administration of multiple oral ascending doses of SKL24741 administered to healthy male subjects

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
111
Inclusion Criteria
  1. Male subjects of 18 to 50 years of age (inclusive) except for the gender effect cohort
  2. Able to read, understand, sign, and date a written informed consent form (ICF) before study participation at screening
  3. Agree to use 2 highly effective methods of contraception, including at least one barrier method (Section 10.6.7 for details)
  4. Body mass index (BMI) between 18.0 and 30.0 kg/m2 (inclusive) at screening
  5. Judged to be in good health on the basis of medical history, physical examination, and routine laboratory measurements (i.e., without clinically relevant pathology)
  6. Normal electrocardiogram (ECG) (12-lead), arterial blood pressure, and heart rate within the normal range of the study center or considered not clinically significant by the investigator and in agreement with the Sponsor
  7. Able to understand and comply with protocol requirements and instructions and likely to complete the study as planned
  8. For Part A (gender effect cohort): Female of non-childbearing potential (18 to 50 years of age (inclusive)), who have undergone a sterilization procedure at least 6 months prior to dosing with official documentation (e.g., hysteroscopic sterilization, bilateral tubal ligation or bilateral salpingectomy, hysterectomy, or bilateral oophorectomy), or be postmenopausal with amenorrhea for at least 1 year prior to dosing and follicle-stimulating hormone (FSH) serum levels consistent with postmenopausal status as per Principal Investigator's judgment
Exclusion Criteria
  1. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subjects.
  2. Smokers (subjects who have smoked within 6 months at screening or those with positive results from smoking screening).
  3. Cholecystectomy and/or surgery of the gastrointestinal tract that could interfere with pharmacokinetics of the trial medication (except appendectomy and simple hernia repair).
  4. Regular treatment with prescription medications. Subjects should have ended any prescription medications at least 14 days before the first dosing of the study drug. Potential subjects should only stop any prescribed medication at the direction of a physician.
  5. Regular treatment with nonprescription medications. Subjects should have ended any nonprescription medications at least 14 days before the first dosing of the study drug. Potential subjects should consult a physician before stopping any regular treatment with nonprescription medication.
  6. Consumption of herbal medications, dietary supplements, and specific fruit products. Subjects should have stopped consumption of herbal medications or dietary supplements (e.g., St. John's Wort, ginkgo biloba, and garlic supplements), vitamins, and grapefruit or grapefruit juice, or Seville oranges at least 14 days before the first dosing of study drug.
  7. History of drug or alcohol abuse or addiction within 2 years before the start of study drug dosing, or a positive test results for alcohol or drugs of abuse, such as amphetamine, barbiturate, benzodiazepine, cocaine, methadone, opiates, oxycodone, phencyclidine, propoxyphene, cannabinoid (THC), MDMA (Ecstasy), methaqualone, and tricyclic antidepressant (TCA).
  8. Regular consumption of more than 2 units of alcoholic beverages per day or more than 14 units per week (1 unit of alcohol equals 1 pint [473 mL] of beer or lager, 1 glass [125 mL] of wine, 25 mL shot of 40% spirit) before screening. Subjects may not consume any alcohol from 72 hours before the first dosing of study drug through the completion of the last PK sampling.
  9. Consumption of an average of more than 5 servings (8 ounces per serving) per day of coffee, cola, or other caffeinated beverage before screening. Subjects may not consume any caffeinated beverages from 48 hours prior to dosing until the collection of the last PK sample.
  10. Participation in a clinical study involving administration of either an investigational or a marketed drug within 2 months or 7 half-lives (whichever is longer) before screening.
  11. Blood donation or a significant loss of blood within 60 days of the start of study drug dosing or donation of more than 1 unit of plasma within 7 days before screening.
  12. Positive result at screening for any of the following infectious disease tests: hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab), human immunodeficiency virus antigen and antibody (HIV Ag, HIV Ab)
  13. Illness within 5 days before the start of study drug dosing ("illness" is defined as an acute [serious or non-serious] condition [e.g., the flu or the common cold])
  14. History of any known relevant allergy/hypersensitivity (including allergy to the trial medication or its excipients)
  15. Subject who is judged not eligible for study participation by investigator

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Single Ascending Dose Level 2SKL247414 Subjects will receive one dose of 25 mg and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 3Placebo6 Subjects will receive one dose of 400 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Single Ascending Dose Level 2Placebo4 Subjects will receive one dose of 25 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 6Placebo4 Subjects will receive one dose of 300 mg and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 2Placebo6 Subjects will receive one dose of 200 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Single Ascending Dose Level 1SKL247414 Subjects will receive one dose of 10 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 1Placebo4 Subjects will receive one dose of 10 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 3Placebo4 Subjects will receive one dose of 50 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 4Placebo4 Subjects will receive one dose of 100 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 5Placebo4 Subjects will receive one dose of 200 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 7SKL247414 Subjects will receive one dose of 400 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 8Placebo4 Subjects will receive one dose of 800 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 10SKL247414 Subjects will receive one dose on the outcome of preceding dose levels and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 1Placebo6 Subjects will receive one dose of 50 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose).
Multiple Ascending Dose Level 3SKL247416 Subjects will receive one dose of 400 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Single Ascending Dose Level 7Placebo4 Subjects will receive one dose of 400 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 9Placebo4 Subjects will receive one dose of 1200 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 10Placebo4 Subjects will receive one dose on the outcome of preceding dose levels and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 8Placebo6 Subjects will receive one dose each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Multiple Ascending Dose Level 4Placebo6 Subjects will receive one dose of 800 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Multiple Ascending Dose Level 5Placebo6 Subjects will receive one dose of 1200 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Multiple Ascending Dose Level 6Placebo6 Subjects will receive one dose of 1200 mg each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Food Effect CohortSKL24741After completing the fasting Single Ascending Dose escalation, the same 6 subjects who received one dose of 50 mg under fasting conditions will return to the clinic after a washout period for a second confinement to receive a second dose (same as fasting) under fed conditions.
Multiple Ascending Dose Level 7Placebo6 Subjects will receive one dose of 2000 mg each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Multiple Ascending Dose Level 5SKL247416 Subjects will receive one dose of 1200 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Multiple Ascending Dose Level 6SKL247416 Subjects will receive one dose of 1200 mg each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Optional Formulation Effect CohortSKL24741Up to 12 male subjects will be treated with the maximum daily dose tested in Part A using open-label, two period, two-sequence crossover design.
Multiple Ascending Dose Level 8SKL247416 Subjects will receive one dose each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Single Ascending Dose Level 4SKL247414 Subjects will receive one dose of 100 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 5SKL247414 Subjects will receive one dose of 200 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 9SKL247414 Subjects will receive one dose of 1200 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 3SKL247414 Subjects will receive one dose of 50 mg and 2 subjects will receive one dose of placebo
Single Ascending Dose Level 6SKL247414 Subjects will receive one dose of 300 mg and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 2SKL247416 Subjects will receive one dose of 200 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Single Ascending Dose Level 8SKL247414 Subjects will receive one dose of 800 mg and 2 subjects will receive one dose of placebo
Multiple Ascending Dose Level 1SKL247416 Subjects will receive one dose of 50 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose).
Multiple Ascending Dose Level 4SKL247416 Subjects will receive one dose of 800 mg each day for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose).
Gender Effect CohortSKL247416 female subjects of non-childbearing potential will receive one dose of 50 mg under fasting conditions.
Optional Food Effect CohortSKL24741Up to 12 male subjects will undergo an open-label, two period, two-sequence crossover design at the maximum daily dose tested in Part A.
Multiple Ascending Dose Level 7SKL247416 Subjects will receive one dose of 2000 mg each day (using tablet formulation instead of capsule formulation\*) for 14 days and 2 subjects will receive one dose placebo every day for 14 days. The dose will be determined based on the outcome of Part A (Single Ascending Dose) and/or preceding dose levels of Part B (Multiple Ascending Dose). \*Tablet formulation is utilized, instead of capsule formulation, in the marked dose levels.
Primary Outcome Measures
NameTimeMethod
Adverse events (Incidence of treatment-emergent adverse events)From Screening until follow-up (14-16 days after dosing day or final dosing day)

The number \[count\] and percentage \[%\] of subjects experiencing an adverse event

Vital signsFrom Screening until follow-up, including time-point assessments (In Part A, Day1: pre-dose until 12 hours post-dose. In Part B, Day1: pre-dose until 12 hours post-dose; Day 2-14: pre-dose until 8 hours post-dose)

Arterial oxygen saturation (SaO2) (using pulse oximetry) \[%\]

Physical examinationFull (complete) physical examination at screening only. From Screening until follow-up (14-16 days after dosing day or final dosing day)

Abbreviated (symptom-directed) physical examination.

Clinically significant laboratory assessmentsFrom Screening until follow-up (14-16 days after dosing day or final dosing day)

Hematology, coagulation, clinical chemistry, urinalysis, and cholesterol

Electrocardiogram outcomesFrom Screening until follow-up, including time-point assessments (In Part A, pre-dose until 24 hours post-dose. In Part B, Day1: pre-dose until 8 hours post-dose; Day 2-13: pre-dose only; Day 14: pre-dose until 24 hours post-dose)

Standard electrocardiograms (ECGs) parameters will be measured, including heart rate (HR) \[beats/min\], RR \[millisecond\], PR \[millisecond\], QT \[millisecond\], QTc intervals \[millisecond\], and QRS duration \[millisecond\]. Extensive triplicate 12-lead ECGs, except for the food effect cohort, will be collected at various time points.

Vital signFrom Screening until follow-up, including time-point assessments (In Part A, Day1: pre-dose until 12 hours post-dose. In Part B, Day1: pre-dose until 12 hours post-dose; Day 2-14: pre-dose until 8 hours post-dose)

Respiratory rate \[breaths/min\]

Peak expiratory flow rateAssessed at admission (Day -1), pre-dose (baseline), 2 and 24 hours post-dose after the first dose (Day 1 dosing) and the last dose (Day 14 dosing for Part B), and follow-up (14-16 days after dosing day or final dosing day)

Peak expiratory flow rate \[L/min\], the maximum rate that a person can exhale during a short maximal expiratory effort after a full inspiration

Columbia-Suicide Severity Rating Scale (Part B only)From Screening until follow-up (14-16 days after dosing day or final dosing day). Baseline ("Lifetime/Recent") at Screening and "Since Last Visit" versions on other visits

Columbia-Suicide Severity Rating Scale (C-SSRS) is a brief questionnaire that provides for the identification, quantification, and standardized assessment of the occurrences and severity of suicidal ideation and behavior. The changes from baseline and clinical abnormalities will be assessed (units on a scale).

Secondary Outcome Measures
NameTimeMethod
Area under the concentration-time curve (AUC) from 0 to infinity (AUC∞) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Area under the concentration-time curve (AUC) from 0 to infinity (AUC∞) \[ng\*h/mL\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles after a single dose administration

Maximum concentration (Cmax) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Maximum concentration (Cmax) \[ng/mL\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Time to maximum concentration (tmax) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Time to maximum concentration (tmax) \[hours, or h\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

AUC from time 0 to a given time t (AUCt) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

AUC from time 0 to a given time t (AUCt) \[ng\*h/mL\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

AUC over the dosing interval (AUCτ) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

AUC over the dosing interval (AUCτ) \[ng\*h/mL\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Apparent clearance (CL/F) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Apparent clearance (CL/F) \[L/h\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Half-life (t1/2) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Half-life (t1/2) \[hour\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Accumulation ratio (Racc) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Accumulation ratio (Racc) \[unit of ratio\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Apparent volume of distribution (Vd/F) of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) following the single and/or multiple dose administration of SKL24741In Part A, pre-dose and up to 240 hours post-dose plasma samples. In Part B, Days 1 and 7: pre-dose and up to 24 hours post-dose, Days 3-6 and 9-13: pre-dose only, and Day 14: Pre-dose and up to 240 hours post-dose plasma samples

Apparent volume of distribution (Vd/F) \[Liter, or L\] of SKL24741, SKL24742 (if appropriate), and possible metabolites (if deemed necessary) for the plasma concentration-time profiles

Trial Locations

Locations (1)

Spaulding Clinical Research

🇺🇸

West Bend, Wisconsin, United States

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