A Study of the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers

Phase 1

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: Placebo; Drug: ISU302

• Registration Number: NCT01881633
• Lead Sponsor: ISU Abxis Co., Ltd.

Brief Summary

The purpose of this study is to assess the safety, tolerability, and pharmacokinetics of single dosing study with three ascending dose cohorts of ISU302 in healthy subjects.

Detailed Description

Not available

Study Timeline

• Study Start: 2010-10
• Primary Completion: 2010-11
• Study Completion: 2010-11
• Status Verified: 2013-06
• Study First Submitted: 2013-06-14
• Study First Submitted QC: 2013-06-18
• Last Update Submitted: 2013-06-18
• Study First Posted: 2013-06-19
• Last Update Posted: 2013-06-19

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 24

Inclusion Criteria

• Healthy male volunteers, aged between ≥ 20 and ≤ 45 years old

• Weight ≥ 50, with calculated body mass index of 17 and 25 kg/m2

  • BMI = (Weight [kg])/(height [m])2

• Subject is informed of the investigational nature of this study and voluntarily agrees to participate in this study and comply with the relevant instructions in written

• Considered ineligible through screening test (such as medical history, physical examination, ECG, safety laboratory test) performed within 35 days prior to study start (dosing of investigational products)

Exclusion Criteria

• With symptoms indicating acute diseases within 28 days prior to start of study (dosing of investigational product)

• History or presence of clinically significant and active cardiovascular, respiratory, renal, endocrine, hematological, gastrointestinal, central nervous system, psychiatric disorder, autoimmune disease, or malignant tumor

• Any medical history that may affect drug absorption, distribution, metabolism and excretion(e.g., inflammatory gastric disease, gastric or intestinal ulcer, hepatic or renal disease)

• With presence of clinically significant allergic disease (including mild allergic rhinitis or allergic dermatitis which does not need medication)

• With presence of clinically significant hypersensitivity to any drugs

• With hemolytic anemia, anemia due to blood loss (Hb < 14g/dL and Hct <42%)

• With the results of safety laboratory test

  1. AST (Aspartate Transaminase) or ALT (Alanine Transaminase) > 1.5 times of upper normal limit
  2. Total bilirubin > 1.5 times of upper normal limit

• Subject who has immune deficiency or medication with immune suppressants

• Participation in other clinical study within 60 days prior to start of study (dosing of investigational products)

• Use of any drugs, possibly affecting drug metabolizing enzymes, within 1 month prior dosing, or any drugs, possibly affecting the results of clinical trial within 10 days or use of drug was not passes 5 x half-life of drug

• Donated whole blood within 60 days, or transfused within 20 days before the study

• History of alcohol abuse (> 14 units/week) and the subject could not stop drinking alcohol beverage during study period

• Heavy smoker (>10 cigarettes/day) or the subject could not stop smoking during study period

• Unwillingness or inability to follow the procedures outlined in the protocol

• Positive in pregnancy test in urine and unwilling to follow contraception during study period and following 3 months (for female subjects).

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	ISU302 Placebo I.V. injection
ISU302	ISU302	15 U/kg I.V. injection
ISU302 30 U/kg	ISU302	Drug ISU302 I.V. injection
ISU302 60 U/kg	ISU302	Drug ISU302 I.V. injection

Primary Outcome Measures

Name	Time	Method
Safety and tolerability	From Screening to Day 5 post-dose	Evaluation for safety data by treatment group/ Confirmative evaluation for serious adverse drug reaction/ Descriptive statistics (arithmetic mean and standard deviation) for quantitative analysis and evaluation of change from baseline/ Frequency counts for qualitative categorization of safety data; Parameters: * Adverse events including subjective/objective symptoms; * Physical examination; * 12-lead ECG; * Vital signs; * Local tolerability test; * Clinical laboratory test: Hematology, Coagulation, Blood Chemistry, Urinalysis; * Immunogenicity

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics	Day1	Pharmacokinetic Parameter assessment using non-compartmental analysis from the concentration-time data/ Summary of Pharmacokinetic parameters by treatment group using descriptive statistics/ Dose proportionality; Parameters: Cmax, AUCt, AUCinf, Tmax, t1/2, Clearance, Vd, MRTr