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

Study Overview
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Brief Summary
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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.

Official Title
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A Dose Block-randomized, Double-blind, Placebo-controlled, Single Dosing, Dose-escalation Phase I Clinical Trial to Evaluate the Tolerability, Safety, and Pharmacokinetics of ISU302 in Healthy Volunteers

Conditions
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Gaucher Disease

Intervention / Treatment
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* Drug: ISU302
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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 AST (Aspartate Transaminase) or ALT (Alanine Transaminase) > 1.5 times of upper normal limit 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).

Ages Eligible for Study
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Minimum Age: 20 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ISU302<br>15 U/kg I.V. injection | Drug: ISU302<br> <br> |
| Experimental: ISU302 30 U/kg<br>Drug ISU302 I.V. injection | Drug: ISU302<br> <br> |
| Experimental: ISU302 60 U/kg<br>Drug ISU302 I.V. injection | Drug: ISU302<br> <br> |
| Placebo Comparator: Placebo<br>ISU302 Placebo I.V. injection | Drug: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability | 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 | From Screening to Day 5 post-dose | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Pharmacokinetics | 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 | Day1 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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gaucher disease, imiglucerase, ISU302