Trial Outcomes & Findings for Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease (NCT NCT02770625)

NCT ID: NCT02770625

Last Updated: 2017-07-12

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 8 participants
• Primary outcome timeframe: from baseline to Week 24
• Results posted on: 2017-07-12

Participant Flow

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.

Participant milestones

Measure	ISU302 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Measure	ISU302 n=8 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Age, Continuous	5.8 years STANDARD_DEVIATION 4.74 • n=5 Participants
Sex: Female, Male Female	0 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	0 Participants n=5 Participants
Race (NIH/OMB) More than one race	8 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Body Mass Index (BMI)	17.244 Kilogram per square meter (kg/m^2) STANDARD_DEVIATION 1.9236 • n=5 Participants
Weight	17.9 kg STANDARD_DEVIATION 7.64 • n=5 Participants
Height	100.40 cm STANDARD_DEVIATION 22.852 • n=5 Participants

PRIMARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
The Difference in Hemoglobin Concentration [g/dL] Baseline	9.49 g/dL Standard Deviation 0.857
The Difference in Hemoglobin Concentration [g/dL] Visit 13 (Week 24)	11.44 g/dL Standard Deviation 0.870

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Platelet Counts [10^3 Platelets/uL] Baseline	132.6 10^3 platelets/uL Standard Deviation 72.27
Platelet Counts [10^3 Platelets/uL] Visit 13 (Week 24)	180.3 10^3 platelets/uL Standard Deviation 47.10

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Spleen Volume Baseline	29.047 Milliliters Standard Deviation 18.9140
Spleen Volume Visit 13 (Week 24)	15.207 Milliliters Standard Deviation 9.4730

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Liver Volume Baseline	1.530 Milliliters Standard Deviation 0.6078
Liver Volume Week 24	1.537 Milliliters Standard Deviation 0.5059

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Angiotensin-converting Enzyme Level Baseline	195.71 U/L Standard Deviation 108.952
Angiotensin-converting Enzyme Level Week 24	159.31 U/L Standard Deviation 58.253

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Chitotriosidase Level (Nmol/mL/hr) Baseline	11093.67 nmol/mL/hr Standard Deviation 10354.369
Chitotriosidase Level (Nmol/mL/hr) Visit 13 (Week 24)	3409.63 nmol/mL/hr Standard Deviation 3103.006

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Chemokine Ligand (CCL-18) Level [ng/mL] Baseline	927.05 ng/mL Standard Deviation 595.4552
Chemokine Ligand (CCL-18) Level [ng/mL] Week 24	577.331 ng/mL Standard Deviation 310.0457

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Acid Phosphatase (ACP) Level (U/L) Baseline	25.44 U/L Standard Deviation 7.517
Acid Phosphatase (ACP) Level (U/L) Week 24	15.44 U/L Standard Deviation 4.313

SECONDARY outcome

Timeframe: from baseline to Week 24

The number of participant who have the skeletal status diagnosed as Osteosclerosis

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Skeletal Status Improvement Baseline	7 Participants
Skeletal Status Improvement Week 24	7 Participants

SECONDARY outcome

Timeframe: from baseline to Week 24

Outcome measures

Measure	ISU302 n=7 Participants 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Change in Bone Mineral Density Baseline	-0.954 g/cm^2 Standard Deviation 1.5967
Change in Bone Mineral Density Week 24	-0.104 g/cm^2 Standard Deviation 2.4558

OTHER_PRE_SPECIFIED outcome

Timeframe: Screening to Visit14 (Week 26)

Outcome measures

Outcome data not reported

Adverse Events

ISU302

Serious events: 3 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	ISU302 n=8 participants at risk 60 U/kg (once every 2 weeks for 6 months) ISU302: 60 U/kg given intravenously
Respiratory, thoracic and mediastinal disorders Bronchitis	12.5% 1/8 • Number of events 1 • 2 years and 4 months
Respiratory, thoracic and mediastinal disorders Viral pneumonia	12.5% 1/8 • Number of events 1 • 2 years and 4 months
Cardiac disorders Ludwig angina	12.5% 1/8 • Number of events 1 • 2 years and 4 months

Other adverse events

Adverse event data not reported

Additional Information

Taekseung Kim

ISU ABXIS Co., Ltd.

Phone: +82.31.696.4715

Email: kimts@isu.co.kr

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place