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Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

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ClinicalTrials.gov Identifier: NCT02770625
Recruitment Status : Completed
First Posted : May 12, 2016
Results First Posted : July 12, 2017
Last Update Posted : July 12, 2017
Sponsor:
Information provided by (Responsible Party):
ISU Abxis Co., Ltd.

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gaucher Disease, Type 1
Intervention Drug: ISU302
Enrollment 8

Recruitment Details  
Pre-assignment Details A total of 9 patients were planned to be enrolled in the study; 8 patients were enrolled into the study and all 8 patients completed the study.
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Period Title: Overall Study
Started 8
Completed 8
Not Completed 0
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Baseline Participants 8
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[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 8 participants
5.8  (4.74)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
Female
0
   0.0%
Male
8
 100.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 8 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
0
   0.0%
More than one race
8
 100.0%
Unknown or Not Reported
0
   0.0%
Body Mass Index (BMI)  
Mean (Standard Deviation)
Unit of measure:  Kilogram per square meter (kg/m^2)
Number Analyzed 8 participants
17.244  (1.9236)
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 8 participants
17.9  (7.64)
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 8 participants
100.40  (22.852)
1.Primary Outcome
Title The Difference in Hemoglobin Concentration [g/dL]
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: g/dL
Baseline 9.49  (0.857)
Visit 13 (Week 24) 11.44  (0.870)
2.Secondary Outcome
Title Platelet Counts [10^3 Platelets/uL]
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: 10^3 platelets/uL
Baseline 132.6  (72.27)
Visit 13 (Week 24) 180.3  (47.10)
3.Secondary Outcome
Title Spleen Volume
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Milliliters
Baseline 29.047  (18.9140)
Visit 13 (Week 24) 15.207  (9.4730)
4.Secondary Outcome
Title Liver Volume
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: Milliliters
Baseline 1.530  (0.6078)
Week 24 1.537  (0.5059)
5.Secondary Outcome
Title Angiotensin-converting Enzyme Level
Hide Description [Not Specified]
Time Frame from baseline to Week 24
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline 195.71  (108.952)
Week 24 159.31  (58.253)
6.Secondary Outcome
Title Chitotriosidase Level (Nmol/mL/hr)
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: nmol/mL/hr
Baseline 11093.67  (10354.369)
Visit 13 (Week 24) 3409.63  (3103.006)
7.Secondary Outcome
Title Chemokine Ligand (CCL-18) Level [ng/mL]
Hide Description [Not Specified]
Time Frame from baseline to Week 24
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Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: ng/mL
Baseline 927.05  (595.4552)
Week 24 577.331  (310.0457)
8.Secondary Outcome
Title Acid Phosphatase (ACP) Level (U/L)
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline 25.44  (7.517)
Week 24 15.44  (4.313)
9.Secondary Outcome
Title Skeletal Status Improvement
Hide Description The number of participant who have the skeletal status diagnosed as Osteosclerosis
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline
7
 100.0%
Week 24
7
 100.0%
10.Secondary Outcome
Title Change in Bone Mineral Density
Hide Description [Not Specified]
Time Frame from baseline to Week 24
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

Overall Number of Participants Analyzed 7
Mean (Standard Deviation)
Unit of Measure: g/cm^2
Baseline -0.954  (1.5967)
Week 24 -0.104  (2.4558)
11.Other Pre-specified Outcome
Title Assessment of AEs, Vital Signs, Physical Examination, and Electrocardiogram (ECG)
Hide Description [Not Specified]
Time Frame Screening to Visit14 (Week 26)
Outcome Measure Data Not Reported
Time Frame 2 years and 4 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title ISU302
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60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously

All-Cause Mortality
ISU302
Affected / at Risk (%)
Total   0/8 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
ISU302
Affected / at Risk (%) # Events
Total   3/8 (37.50%)    
Cardiac disorders   
Ludwig angina * [1]  1/8 (12.50%)  1
Respiratory, thoracic and mediastinal disorders   
Bronchitis * [2]  1/8 (12.50%)  1
Viral pneumonia * [3]  1/8 (12.50%)  1
*
Indicates events were collected by non-systematic assessment
[1]
Ludwig angina / Infections and infestations
[2]
Bronchitis / Infections and infestations
[3]
Pneumonia viral / Infections and infestations
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
ISU302
Affected / at Risk (%) # Events
Total   0/8 (0.00%)    
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Taekseung Kim
Organization: ISU ABXIS Co., Ltd.
Phone: +82.31.696.4715
Responsible Party: ISU Abxis Co., Ltd.
ClinicalTrials.gov Identifier: NCT02770625     History of Changes
Other Study ID Numbers: ISU302-004
First Submitted: May 7, 2016
First Posted: May 12, 2016
Results First Submitted: February 10, 2017
Results First Posted: July 12, 2017
Last Update Posted: July 12, 2017