Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
Primary Purpose
MELAS Syndrome, Mitochondrial Respiratory Chain Deficiencies
Status
Completed
Phase
Phase 1
Locations
Korea, Republic of
Study Type
Interventional
Intervention
KL1333 25 mg
KL1333 50 mg
KL1333 100 mg
KL1333 200 mg
KL1333 400 mg
KL1333 600 mg
KL1333 800 mg
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for MELAS Syndrome focused on measuring Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Eligibility Criteria
Inclusion Criteria:
- 19 - 45 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
Exclusion Criteria:
- 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
- Subjects who plan for pregnancy during the study period or who are not able to use established contraceptive method
- Subject who judged not eligible for study participation by investigator
Sites / Locations
- Seoul National University Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm 5
Arm 6
Arm 7
Arm Type
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Experimental
Arm Label
KL1333 25mg
KL1333 50mg
KL1333 100mg
KL1333 200mg
KL1333 400mg
KL1333 600mg
KL1333 800mg
Arm Description
Group 1
Group 2
Group 3
Group 4
Group 5
Group 6
Group 7
Outcomes
Primary Outcome Measures
Number of reported adverse events
Secondary Outcome Measures
Maximum plasma concentration (Cmax) of KL1333
Area Under the Curve (AUC) of KL1333
Half-life (T1/2) of KL1333
Full Information
NCT ID
NCT03056209
First Posted
February 5, 2017
Last Updated
April 25, 2018
Sponsor
Yungjin Pharm. Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03056209
Brief Title
Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
Official Title
A Dose Block-randomized, Double-blind, Placebo-controlled, Single-dose, Dose-escalation, Phase I Clinical Study
Study Type
Interventional
2. Study Status
Record Verification Date
April 2018
Overall Recruitment Status
Completed
Study Start Date
June 26, 2017 (Actual)
Primary Completion Date
April 16, 2018 (Actual)
Study Completion Date
April 16, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Yungjin Pharm. Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
MELAS Syndrome, Mitochondrial Respiratory Chain Deficiencies
Keywords
Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
60 (Actual)
8. Arms, Groups, and Interventions
Arm Title
KL1333 25mg
Arm Type
Experimental
Arm Description
Group 1
Arm Title
KL1333 50mg
Arm Type
Experimental
Arm Description
Group 2
Arm Title
KL1333 100mg
Arm Type
Experimental
Arm Description
Group 3
Arm Title
KL1333 200mg
Arm Type
Experimental
Arm Description
Group 4
Arm Title
KL1333 400mg
Arm Type
Experimental
Arm Description
Group 5
Arm Title
KL1333 600mg
Arm Type
Experimental
Arm Description
Group 6
Arm Title
KL1333 800mg
Arm Type
Experimental
Arm Description
Group 7
Intervention Type
Drug
Intervention Name(s)
KL1333 25 mg
Intervention Description
oral administration, single dose, 25 mg 1 tab
Intervention Type
Drug
Intervention Name(s)
KL1333 50 mg
Intervention Description
oral administration, single dose, 25 mg 2 tabs
Intervention Type
Drug
Intervention Name(s)
KL1333 100 mg
Intervention Description
oral administration, single dose, 100 mg 1 tab
Intervention Type
Drug
Intervention Name(s)
KL1333 200 mg
Intervention Description
oral administration, single dose, 100 mg 2 tabs
Intervention Type
Drug
Intervention Name(s)
KL1333 400 mg
Intervention Description
oral administration, single dose, 100 mg 4 tabs
Intervention Type
Drug
Intervention Name(s)
KL1333 600 mg
Intervention Description
oral administration, single dose, 100 mg 6 tabs
Intervention Type
Drug
Intervention Name(s)
KL1333 800 mg
Intervention Description
oral administration, single dose, 100 mg 8 tabs
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
oral administration, placebo
Primary Outcome Measure Information:
Title
Number of reported adverse events
Time Frame
from day 1 to day 15
Secondary Outcome Measure Information:
Title
Maximum plasma concentration (Cmax) of KL1333
Time Frame
from day 1 to day 15
Title
Area Under the Curve (AUC) of KL1333
Time Frame
from day 1 to day 15
Title
Half-life (T1/2) of KL1333
Time Frame
from day 1 to day 15
10. Eligibility
Sex
Male
Minimum Age & Unit of Time
19 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
Inclusion Criteria:
19 - 45 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
Exclusion Criteria:
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
Subjects who plan for pregnancy during the study period or who are not able to use established contraceptive method
Subject who judged not eligible for study participation by investigator
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Kyung-Sang Yu, MD., MBA
Organizational Affiliation
Seoul National University Hospital
Official's Role
Study Chair
Facility Information:
Facility Name
Seoul National University Hospital
City
Seoul
State/Province
Jongno-gu
ZIP/Postal Code
110744
Country
Korea, Republic of
12. IPD Sharing Statement
Plan to Share IPD
No
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Safety, Tolerability and Pharmacokinetic Study of KL1333 in Healthy Male Volunteers
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