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

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

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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Placebo; Drug: ISU302

• Study Type: Interventional
• Enrollment (Actual): 24
• Phase: Phase 1

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 43 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

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 Plan

How is the study designed?

Design Details

• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

4

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ISU302; 15 U/kg I.V. injection	Drug: ISU302
Experimental: ISU302 30 U/kg; Drug ISU302 I.V. injection	Drug: ISU302
Experimental: ISU302 60 U/kg; Drug ISU302 I.V. injection	Drug: ISU302
Placebo Comparator: Placebo; ISU302 Placebo I.V. injection	Drug: Placebo

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: ISU Abxis Co., Ltd.

Study record dates

Study Major Dates

• Study Start: October 1, 2010
• Primary Completion (Actual): November 1, 2010
• Study Completion (Actual): November 1, 2010

Study Registration Dates

• First Submitted: June 14, 2013
• First Submitted That Met QC Criteria: June 18, 2013
• First Posted (Estimate): June 19, 2013

Study Record Updates

• Last Update Posted (Estimate): June 19, 2013
• Last Update Submitted That Met QC Criteria: June 18, 2013
• Last Verified: June 1, 2013

More Information

Terms related to this study

• Keywords: gaucher disease, imiglucerase, ISU302
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: ISU-002