- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02770625
Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease
June 13, 2017 updated by: ISU Abxis Co., Ltd.
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
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.
Study Overview
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.
Study Type
Interventional
Enrollment (Actual)
8
Phase
- Phase 3
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
2 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: ISU302
60 U/kg (once every 2 weeks for 6 months)
|
60 U/kg given intravenously
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
The Difference in Hemoglobin Concentration [g/dL]
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Platelet Counts [10^3 Platelets/uL]
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Spleen Volume
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Liver Volume
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Angiotensin-converting Enzyme Level
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Chitotriosidase Level (Nmol/mL/hr)
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Chemokine Ligand (CCL-18) Level [ng/mL]
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Acid Phosphatase (ACP) Level (U/L)
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
|
Skeletal Status Improvement
Time Frame: from baseline to Week 24
|
The number of participant who have the skeletal status diagnosed as Osteosclerosis
|
from baseline to Week 24
|
Change in Bone Mineral Density
Time Frame: from baseline to Week 24
|
from baseline to Week 24
|
Other Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Assessment of AEs, Vital Signs, Physical Examination, and Electrocardiogram (ECG)
Time Frame: Screening to Visit14 (Week 26)
|
Screening to Visit14 (Week 26)
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Amel El Beshlawy, Prof., Abou El Reesh Children's Hospital
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
September 1, 2011
Primary Completion (Actual)
March 1, 2013
Study Completion (Actual)
August 1, 2014
Study Registration Dates
First Submitted
May 7, 2016
First Submitted That Met QC Criteria
May 10, 2016
First Posted (Estimate)
May 12, 2016
Study Record Updates
Last Update Posted (Actual)
July 12, 2017
Last Update Submitted That Met QC Criteria
June 13, 2017
Last Verified
February 1, 2017
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- Genetic Diseases, Inborn
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Gaucher Disease
Other Study ID Numbers
- ISU302-004
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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