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Trial record 26 of 103 for:    Gaucher Disease

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
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

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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.

Reporting Groups
  Description
ISU302

60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously


Participant Flow:   Overall Study
    ISU302
STARTED   8 
COMPLETED   8 
NOT COMPLETED   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
ISU302

60 U/kg (once every 2 weeks for 6 months)

ISU302: 60 U/kg given intravenously


Baseline Measures
   ISU302 
Overall Participants Analyzed 
[Units: Participants]
 8 
Age 
[Units: Years]
Mean (Standard Deviation)
 5.8  (4.74) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      0   0.0% 
Male      8 100.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of 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) 
[Units: Kilogram per square meter (kg/m^2)]
Mean (Standard Deviation)
 17.244  (1.9236) 
Weight 
[Units: Kg]
Mean (Standard Deviation)
 17.9  (7.64) 
Height 
[Units: Cm]
Mean (Standard Deviation)
 100.40  (22.852) 


  Outcome Measures

1.  Primary:   The Difference in Hemoglobin Concentration [g/dL]   [ Time Frame: from baseline to Week 24 ]

2.  Secondary:   Platelet Counts [10^3 Platelets/uL]   [ Time Frame: from baseline to Week 24 ]

3.  Secondary:   Spleen Volume   [ Time Frame: from baseline to Week 24 ]

4.  Secondary:   Liver Volume   [ Time Frame: from baseline to Week 24 ]

5.  Secondary:   Angiotensin-converting Enzyme Level   [ Time Frame: from baseline to Week 24 ]

6.  Secondary:   Chitotriosidase Level (Nmol/mL/hr)   [ Time Frame: from baseline to Week 24 ]

7.  Secondary:   Chemokine Ligand (CCL-18) Level [ng/mL]   [ Time Frame: from baseline to Week 24 ]

8.  Secondary:   Acid Phosphatase (ACP) Level (U/L)   [ Time Frame: from baseline to Week 24 ]

9.  Secondary:   Skeletal Status Improvement   [ Time Frame: from baseline to Week 24 ]

10.  Secondary:   Change in Bone Mineral Density   [ Time Frame: from baseline to Week 24 ]

11.  Other Pre-specified:   Assessment of AEs, Vital Signs, Physical Examination, and Electrocardiogram (ECG)   [ Time Frame: Screening to Visit14 (Week 26) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

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
e-mail: kimts@isu.co.kr



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