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

Primary Purpose

Gaucher Disease, Type 1

Status

Completed

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

ISU302

About this trial

This is an interventional treatment trial for Gaucher Disease, Type 1 focused on measuring Type I Gaucher, ISU302, Imiglucerase

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Diagnosis of Type 1 GD.
Documented glucocerebrosidase deficiency.
GD-related anemia, defined as hemoglobin levels of at least 1 g/dL below the lower limit of normal for age and gender and one or more of the following 3 criteria:
- At least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation,
- GD-related thrombocytopenia, defined as a platelet count <90 x 109 platelets/L,
- GD-related readily palpable enlarged liver.
Not received treatment for GD (investigational products, miglustat, velaglucerase alfa, or imiglucerase) within 12 months prior to study entry.
Ability to comprehend and willing to sign the ICF.
Legal guardian (and patient if age appropriate) understood the nature of the procedure, was willing to comply with associated follow-up evaluations, and provided written informed consent and assent prior to the procedure.
Female patients of childbearing potential must had agreed to use a medically acceptable method of contraception at all the times during the study. Male patients must have used a medically acceptable method of birth control throughout their participation in the study and were required to report the pregnancy of a partner.

Exclusion Criteria:

Type 2 or 3 GD.
Splenectomy.
Antibody positive to ISU302 or imiglucerase during screening or the patient had experienced an anaphylactic reaction to ISU302 or imiglucerase. - Treatment with any non-GD-related investigational drug or medical device within 30 days prior to study entry; such use during the study was also not permitted.
Currently receiving red blood cell (RBC) growth factor (eg, erythropoietin) chronic systemic corticosteroids or received such treatment within the last 6 months.
Positive for human immunodeficiency virus (HIV) and hepatitis B or C.
Exacerbated anemia at screening (due to iron, folic acid, or vitamin B12 deficiency or infectious/immune-mediated cause).
Significant comorbidity(ies) that could affect study data or confounded the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease).
Pregnant or lactating female patients and those not willing to use highly effective barrier or medical method of contraception.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ISU302

Arm Description

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

Outcomes

Primary Outcome Measures

The Difference in Hemoglobin Concentration [g/dL]

Secondary Outcome Measures

Platelet Counts [10^3 Platelets/uL]

Spleen Volume

Liver Volume

Angiotensin-converting Enzyme Level

Chitotriosidase Level (Nmol/mL/hr)

Chemokine Ligand (CCL-18) Level [ng/mL]

Acid Phosphatase (ACP) Level (U/L)

Skeletal Status Improvement

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

Change in Bone Mineral Density

Full Information

NCT ID

NCT02770625

First Posted

May 7, 2016

Last Updated

June 13, 2017

Sponsor

ISU Abxis Co., Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT02770625

Brief Title

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

Official Title

A Multicenter, Open-Label Phase III Study to Evaluate the Safety and Efficacy of ISU302 (Imiglucerase for Injection) in Patients With Type 1 Gaucher Disease

Study Type

Interventional

2. Study Status

Record Verification Date

February 2017

Overall Recruitment Status

Completed

Study Start Date

September 2011 (undefined)

Primary Completion Date

March 2013 (Actual)

Study Completion Date

August 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

ISU Abxis Co., Ltd.

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.

Detailed Description

The objectives of this clinical study were to evaluate the efficacy and safety of every other week (EOW) dosing of ISU302 at a dose of 60 U/kg as an effective glucocerebrosidase enzyme replacement therapeutic product in patients with Type 1 Gaucher disease (GD). Primary efficacy endpoint was the difference in hemoglobin concentration between baseline and Week 24. Secondary efficacy endpoints included assessment of platelet counts, spleen and liver volume, and biomarker levels in plasma at Week 24 compared to baseline; skeletal change and bone mineral density (BMD); and single-dose pharmacokinetic (PK) analysis. Secondary safety endpoints included the assessment of adverse events (AEs), vital signs, physical examination, and electrocardiogram (ECG); clinical safety laboratory analyses included serum chemistry, urinalysis, hematology and coagulation, and the measurement of anti-ISU302 antibodies.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher Disease, Type 1

Keywords

Type I Gaucher, ISU302, Imiglucerase

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

ISU302

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

ISU302

Other Intervention Name(s)

Imiglucerase

Intervention Description

60 U/kg given intravenously

Primary Outcome Measure Information:

Title

The Difference in Hemoglobin Concentration [g/dL]

Time Frame

from baseline to Week 24

Secondary Outcome Measure Information:

Title

Platelet Counts [10^3 Platelets/uL]

Time Frame

from baseline to Week 24

Title

Spleen Volume

Time Frame

from baseline to Week 24

Title

Liver Volume

Time Frame

from baseline to Week 24

Title

Angiotensin-converting Enzyme Level

Time Frame

from baseline to Week 24

Title

Chitotriosidase Level (Nmol/mL/hr)

Time Frame

from baseline to Week 24

Title

Chemokine Ligand (CCL-18) Level [ng/mL]

Time Frame

from baseline to Week 24

Title

Acid Phosphatase (ACP) Level (U/L)

Time Frame

from baseline to Week 24

Title

Skeletal Status Improvement

Description

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

Time Frame

from baseline to Week 24

Title

Change in Bone Mineral Density

Time Frame

from baseline to Week 24

Other Pre-specified Outcome Measures:

Title

Assessment of AEs, Vital Signs, Physical Examination, and Electrocardiogram (ECG)

Time Frame

Screening to Visit14 (Week 26)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

2 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Diagnosis of Type 1 GD. Documented glucocerebrosidase deficiency. GD-related anemia, defined as hemoglobin levels of at least 1 g/dL below the lower limit of normal for age and gender and one or more of the following 3 criteria: At least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation, GD-related thrombocytopenia, defined as a platelet count <90 x 109 platelets/L, GD-related readily palpable enlarged liver. Not received treatment for GD (investigational products, miglustat, velaglucerase alfa, or imiglucerase) within 12 months prior to study entry. Ability to comprehend and willing to sign the ICF. Legal guardian (and patient if age appropriate) understood the nature of the procedure, was willing to comply with associated follow-up evaluations, and provided written informed consent and assent prior to the procedure. Female patients of childbearing potential must had agreed to use a medically acceptable method of contraception at all the times during the study. Male patients must have used a medically acceptable method of birth control throughout their participation in the study and were required to report the pregnancy of a partner. Exclusion Criteria: Type 2 or 3 GD. Splenectomy. Antibody positive to ISU302 or imiglucerase during screening or the patient had experienced an anaphylactic reaction to ISU302 or imiglucerase. - Treatment with any non-GD-related investigational drug or medical device within 30 days prior to study entry; such use during the study was also not permitted. Currently receiving red blood cell (RBC) growth factor (eg, erythropoietin) chronic systemic corticosteroids or received such treatment within the last 6 months. Positive for human immunodeficiency virus (HIV) and hepatitis B or C. Exacerbated anemia at screening (due to iron, folic acid, or vitamin B12 deficiency or infectious/immune-mediated cause). Significant comorbidity(ies) that could affect study data or confounded the study results (eg, malignancies, primary biliary cirrhosis, autoimmune liver disease). Pregnant or lactating female patients and those not willing to use highly effective barrier or medical method of contraception.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Amel El Beshlawy, Prof.

Organizational Affiliation

Abou El Reesh Children's Hospital

Official's Role

Principal Investigator

12. IPD Sharing Statement

Plan to Share IPD

Undecided

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

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