Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers

NCT03056209 | Completed Phase 1 DMC

• 1 other identifier: KL1333_101
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this First In Human study is to investigate the safety and tolerability of KL1333 after a single oral dose and to investigate the pharmacokinetic characteristics of KL1333 after a single oral dose.

Conditions

MELAS Syndrome; Mitochondrial Respiratory Chain Deficiencies

Keywords

Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation

Outcome Measures

Primary Outcomes (1)

• Number of reported adverse events

  from day 1 to day 15

Secondary Outcomes (3)

• Maximum plasma concentration (Cmax) of KL1333

  from day 1 to day 15

• Area Under the Curve (AUC) of KL1333

  from day 1 to day 15

• Half-life (T1/2) of KL1333

  from day 1 to day 15

Study Arms (7)

KL1333 25mg

EXPERIMENTAL

Group 1

Drug: KL1333 25 mg; Drug: Placebo

KL1333 50mg

EXPERIMENTAL

Group 2

Drug: KL1333 50 mg; Drug: Placebo

KL1333 100mg

EXPERIMENTAL

Group 3

Drug: KL1333 100 mg; Drug: Placebo

KL1333 200mg

EXPERIMENTAL

Group 4

Drug: KL1333 200 mg; Drug: Placebo

KL1333 400mg

EXPERIMENTAL

Group 5

Drug: KL1333 400 mg; Drug: Placebo

KL1333 600mg

EXPERIMENTAL

Group 6

Drug: KL1333 600 mg; Drug: Placebo

KL1333 800mg

EXPERIMENTAL

Group 7

Drug: KL1333 800 mg; Drug: Placebo

Interventions

KL1333 25 mg DRUG

oral administration, single dose, 25 mg 1 tab

KL1333 25mg

KL1333 50 mg DRUG

oral administration, single dose, 25 mg 2 tabs

KL1333 50mg

KL1333 100 mg DRUG

oral administration, single dose, 100 mg 1 tab

KL1333 100mg

KL1333 200 mg DRUG

oral administration, single dose, 100 mg 2 tabs

KL1333 200mg

KL1333 400 mg DRUG

oral administration, single dose, 100 mg 4 tabs

KL1333 400mg

KL1333 600 mg DRUG

oral administration, single dose, 100 mg 6 tabs

KL1333 600mg

KL1333 800 mg DRUG

oral administration, single dose, 100 mg 8 tabs

KL1333 800mg

Placebo DRUG

oral administration, placebo

KL1333 100mg; KL1333 200mg; KL1333 25mg; KL1333 400mg; KL1333 50mg; KL1333 600mg; KL1333 800mg

Eligibility Criteria

• Age: 19 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• years of age at the time of screening
• Subjects weighing ≥55 and ≤90 kg with BMI between 18 and 27 kg/m2
• Subjects who agreed to voluntarily participate in this study and comply with all the study requirements by signing informed consent form after being informed of the nature of this study and understanding all aspects of this study

You may not qualify if:

• History of clinically significant hepatic, renal, neurologic, immunologic, respiratory, endocrine disease or hematologic•oncologic disease, cardiovascular, psychiatric disease
• History of disease or surgery of the gastrointestinal tract that could interfere with kinetics of the study drug. Simple hernia repair or appendectomy are excepted.
• History of clinically significant or relevant allergy/hypersensitivity
• Blood AST (SGOT), ALT (SGPT) \>1.5 of upper limit
• eGFR value of ≤90mL/min/1.73m2
• Systolic blood pressure of \<100 mmHg or \>160 mmHg
• Diastolic blood pressure of \<60 mmHg or \>100 mmHg
• Any abnormalities in 12-lead ECG at screening visit
• Subjects who showed positive result in drug abuse tests, or who has history of drug abuse within 60 days prior to the time of screening
• Subjects who took prescribed medications or oriental medicine within 14 days or over-the-counter (OTC) medications or vitamins within 7 days prior to the dose of the study drug (however, the subject can be included if other criteria are met according to the discretion of the investigator)
• Subjects who were administered any investigational products within 3 months from the first dose of the study drug
• Subjects who have donated whole blood (60 days) or partial blood (30 days), or received blood transfusion
• Subjects who have had alcohol consistently (\>21units/week, 1unit=10 g of pure alcohol) or who is not able to stop drinking alcohol throughout the study period
• Subjects who have smoked until 90 days prior to the study initiation or who are not able to stop smoking throughout the study period
• Subjects who are not able to stop taking grapefruit/caffeine from 3 days prior to the first dose of the study drug throughout the study period

Sponsors & Collaborators

• Yungjin Pharm. Co., Ltd.: lead

Study Sites (1)

Seoul National University Hospital

Seoul, Jongno-gu, 110744, South Korea

Location

MeSH Terms

Conditions

MELAS Syndrome; Mitochondrial Diseases

Condition Hierarchy (Ancestors)

Mitochondrial Encephalomyopathies; Mitochondrial Myopathies; Muscular Diseases; Musculoskeletal Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Neuromuscular Diseases; Vascular Diseases; Cardiovascular Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Kyung-Sang Yu, MD., MBA

  Seoul National University Hospital

  STUDY CHAIR

Study Design

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

Study Record Dates

• First Submitted: February 5, 2017
• First Posted: February 17, 2017
• Study Start: June 26, 2017
• Primary Completion: April 16, 2018
• Study Completion: April 16, 2018
• Last Updated: April 27, 2018

Record last verified: 2018-04

Data Sharing

• IPD Sharing: Will not share

Locations

KR (1)