A Phase 1 interventional study of YH35995 and Placebo in Healthy Participants, sponsored by Yuhan Corporation. Active, not recruiting at 1 site in South Korea. Open to male participants aged 19 Years to 45 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-07-28.
Sponsored by Yuhan Corporation · Phase 1, Interventional, and Treatment
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
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.
171 studies on the registry are indexed under Gaucher Disease; 37 are open to participants now.
This study's enrollment of 94 is above the median of 20 across 98 interventional studies indexed under Gaucher Disease.
Browse Gaucher Disease studies →Yuhan Corporation is the lead sponsor of 107 studies on the registry; 6 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
\[Part A\] Participants will be orally administered a single dose of YH35995 in five dose groups, gradually escalating from lower to higher doses. Each cohort includes 10 participants (8 randomly assigned to the YH35995 arm and 2 randomly assigned to the placebo arm). \[Part B\] Participants will receive multiple oral doses of YH35995 once every 4 weeks in three dose groups. Each cohort includes 12 participants (9 randomly assigned to the YH35995 arm and 3 randomly assigned to the placebo arm).
Drug: YH35995
\[Part A\] Participants will be orally administered a single dose of Placebo in five dose groups, gradually escalating from lower to higher doses. Each cohort includes 10 participants (8 randomly assigned to the YH35995 arm and 2 randomly assigned to the placebo arm). \[Part B\] Participants will receive multiple oral doses of Placebo once every 4 weeks in three dose groups. Each cohort includes 12 participants (9 randomly assigned to the YH35995 arm and 3 randomly assigned to the placebo arm).
Drug: Placebo
Oral administration of YH35995
Oral administration of Placebo
[Part A, B] Treatment-emergent adverse events (TEAEs)
To assess the safety and tolerability of a single dose and multiple dose administration of YH35995
Time frame: Part A: Day1-150, Part B: Day1-232
[Part A] Maximum observed plasma concentration (Cmax)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] Time to reach Cmax (Tmax)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] Area under the plasma concentration-time curve from time 0 to the last quantifiable concentration (AUClast)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] AUC from time 0 to infinity (AUCinf)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] Apparent terminal elimination half-life (t1/2)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] Total plasma clearance (CL/F)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part A] Apparent volume of distribution (Vz/F)
To characterize the pharmacokinetics (PK) of YH35995
Time frame: Day1-150
[Part B] Cmax during the first dosing interval
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Tmax during the first dosing interval
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] AUC during the first dosing interval (AUCsingle)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] AUC during the dosing interval at steady state (AUCtau,ss)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Cmax at steady state (Cmax,ss)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Tmax at steady state (Tmax,ss)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Accumulation ratio using AUC (Rac(AUC))
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Accumulation ratio using Cmax (Rac(Cmax))
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Plasma concentration at the last observed time point during the dosing interval at steady state (Ctrough)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Average plasma concentration (Cavg)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Clearance at steady state (CLss/F)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Volume of distribution at steady state (Vss)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Effective half-life (t1/2,Rac)
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Cerebrospinal fluid to plasma concentration ratio(C/P ratio) of YH35995
To assess the pharmacokinetics (PK) of YH35995 after multiple dose administration
Time frame: Day1-232
[Part B] Properly derived PD parameters for YH35995, including the area under the effect curve (AUEC) and maximum effect (Emax)
To assess the pharmacodynamics (PD) of YH35995 after multiple dose administration
Time frame: Day1-232
Plan to share: Yes — De-identified individual participant data (including data dictionaries) that underline the results reported in study-related publications will be made available during the period beginning 1 year and ending 5 years after all trial primary and secondary endpoints were assessed. Only requests from researchers who provide a methodologically sound proposal will be reviewed and approved by the sponsor. The analysis type should be in accordance with aims in the proposal approved by the sponsor. Proposals should be directed to clinicaltrials@yuhan.co.kr. A summary of the study results will be posted in the publicly accessible database (i.e. clinicaltrials.gov) no later than 1 year after the study's primary completion date.
Supporting information: Study protocol, Sap, Csr
No publications or documents are linked to this record.
This study is active, not recruiting, as verified in Jul 2026. You cannot join it, but the record below documents what was studied.
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Yuhan Corporation