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

NCT01881633 | Completed Phase 1

• 1 other identifier: ISU-002
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

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.

Conditions

Gaucher Disease

Keywords

gaucher disease, imiglucerase, ISU302

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Pharmacokinetics

  Day1

Study Arms (4)

ISU302

EXPERIMENTAL

15 U/kg I.V. injection

Drug: ISU302

ISU302 30 U/kg

EXPERIMENTAL

Drug ISU302 I.V. injection

Drug: ISU302

ISU302 60 U/kg

EXPERIMENTAL

Drug ISU302 I.V. injection

Drug: ISU302

Placebo

PLACEBO COMPARATOR

ISU302 Placebo I.V. injection

Drug: Placebo

Interventions

ISU302 DRUG

ISU302; ISU302 30 U/kg; ISU302 60 U/kg

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 20 Years - 45 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• ISU Abxis Co., Ltd.: lead

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2013
• First Posted: June 19, 2013
• Study Start: October 1, 2010
• Primary Completion: November 1, 2010
• Study Completion: November 1, 2010
• Last Updated: June 19, 2013

Record last verified: 2013-06