Healthy Participants
Conditions
Keywords
YH35995, Gaucher Disease
Brief summary
This is a randomized, double-blind, first-in-human study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of single and multiple oral doses of YH35995
Detailed description
YH35995 is being developed as a treatment for the neurological symptoms of Gaucher Disease type 3. This study is a first-in-human (FIH), phase 1, randomized, double-blind, placebo-controlled study of YH35995, which consists of two parts. In Part A (SAD), single ascending dose of YH35995 is administered to healthy male participants to assess its safety, tolerability, PK, and PD. In Part B (MAD), multiple ascending dose of YH35995 is administered to healthy male participants to assess its safety, tolerability, PK, and PD.
Interventions
DRUGYH35995
Oral administration of YH35995
DRUGPlacebo
Oral administration of Placebo
Sponsors
Yuhan Corporation
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
This is a double-blind study, meaning that YH35995 tablets and placebo tablets will be identical in appearance and will be packaged in the same way. Information about the treatment arm to which participants are assigned must remain blinded for the duration of the study unless information about the causal relationship between the adverse event and the Investigational Product that meets the drug withdrawal criteria becomes essential or, in an emergency, if the information about the Investigational Product becomes critical for treating the participant.
Eligibility
Sex/Gender
MALE
Age
19 Years to 45 Years
Healthy volunteers
Yes
Inclusion criteria
* Male between the ages of 19 and 45 at the time of providing written consent * Participants who weigh at least 50 kg at screening and have a body mass index (BMI) of at least 18.0 kg/m2 and less than 30 kg/m2 * Participants who have been fully informed about and fully understand this study, have voluntarily decided to participate, and have agreed in writing to comply with the guidelines of the study during the duration of the study
Exclusion criteria
* Participation in a bioequivalence trial or any other clinical trials within 6 months prior to the first scheduled dose of the IP (within 1 month of the first scheduled dose for participants who have taken part in a dietary supplement clinical trial) * Individuals with clinically significant abnormal results that do not match any other inclusion/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| [Part A, B] Treatment-emergent adverse events (TEAEs) | Part A: Day1-150, Part B: Day1-232 | To assess the safety and tolerability of a single dose and multiple dose administration of YH35995 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| [Part A] Maximum observed plasma concentration (Cmax) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] Time to reach Cmax (Tmax) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] Area under the plasma concentration-time curve from time 0 to the last quantifiable concentration (AUClast) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] AUC from time 0 to infinity (AUCinf) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] Apparent terminal elimination half-life (t1/2) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] Total plasma clearance (CL/F) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part A] Apparent volume of distribution (Vz/F) | Day1-150 | To characterize the pharmacokinetics (PK) of YH35995 |
| [Part B] Cmax during the first dosing interval | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Tmax during the first dosing interval | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] AUC during the first dosing interval (AUCsingle) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] AUC during the dosing interval at steady state (AUCtau,ss) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Cmax at steady state (Cmax,ss) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Tmax at steady state (Tmax,ss) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Accumulation ratio using AUC (Rac(AUC)) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Accumulation ratio using Cmax (Rac(Cmax)) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Plasma concentration at the last observed time point during the dosing interval at steady state (Ctrough) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Average plasma concentration (Cavg) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Clearance at steady state (CLss/F) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Volume of distribution at steady state (Vss) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Effective half-life (t1/2,Rac) | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Cerebrospinal fluid to plasma concentration ratio(C/P ratio) of YH35995 | Day1-232 | To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration |
| [Part B] Properly derived PD parameters for YH35995, including the area under the effect curve (AUEC) and maximum effect (Emax) | Day1-232 | To assess the pharmacodynamics (PD) of YH35995 after multiple dose administration |
Countries
South Korea
Contacts
PRINCIPAL_INVESTIGATORHyounggyoon Yoo
CHA Bundang Medical Center
Outcome results
None listed