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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT02770625
First Posted: May 12, 2016
Last Update Posted: July 12, 2017
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.
Information provided by (Responsible Party):
ISU Abxis Co., Ltd.
  Purpose
The purpose of this study is to evaluate the safety and efficacy of ISU302 in patients with Type 1 Gaucher disease.

Condition Intervention Phase
Gaucher Disease, Type 1 Drug: ISU302 Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by ISU Abxis Co., Ltd.:

Primary Outcome Measures:
  • The Difference in Hemoglobin Concentration [g/dL] [ Time Frame: from baseline to Week 24 ]

Secondary Outcome Measures:
  • Platelet Counts [10^3 Platelets/uL] [ Time Frame: from baseline to Week 24 ]
  • Spleen Volume [ Time Frame: from baseline to Week 24 ]
  • Liver Volume [ Time Frame: from baseline to Week 24 ]
  • Angiotensin-converting Enzyme Level [ Time Frame: from baseline to Week 24 ]
  • Chitotriosidase Level (Nmol/mL/hr) [ Time Frame: from baseline to Week 24 ]
  • Chemokine Ligand (CCL-18) Level [ng/mL] [ Time Frame: from baseline to Week 24 ]
  • Acid Phosphatase (ACP) Level (U/L) [ Time Frame: 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

  • Change in Bone Mineral Density [ Time Frame: from baseline to Week 24 ]

Other Outcome Measures:
  • Assessment of AEs, Vital Signs, Physical Examination, and Electrocardiogram (ECG) [ Time Frame: Screening to Visit14 (Week 26) ]

Enrollment: 8
Study Start Date: September 2011
Study Completion Date: August 2014
Primary Completion Date: March 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: ISU302
60 U/kg (once every 2 weeks for 6 months)
Drug: ISU302
60 U/kg given intravenously
Other Name: Imiglucerase

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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   2 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02770625


Sponsors and Collaborators
ISU Abxis Co., Ltd.
Investigators
Principal Investigator: Amel El Beshlawy, Prof. Abou El Reesh Children's Hospital
  More Information

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
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by ISU Abxis Co., Ltd.:
Type I Gaucher
ISU302
Imiglucerase

Additional relevant MeSH terms:
Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders