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

NCT02770625 | Completed Phase 3 Results

• 1 other identifier: ISU302-004
• Study Type: interventional
• Target: 8
• Locations: 0 countries
• Sites: N/A

Brief Summary

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

Conditions

Gaucher Disease, Type 1

Keywords

Type I Gaucher; ISU302; Imiglucerase

Outcome Measures

Primary Outcomes (1)

• The Difference in Hemoglobin Concentration [g/dL]

  from baseline to Week 24

Secondary Outcomes (9)

• Platelet Counts [10^3 Platelets/uL]

  from baseline to Week 24

• Spleen Volume

  from baseline to Week 24

• Liver Volume

  from baseline to Week 24

• Angiotensin-converting Enzyme Level

  from baseline to Week 24

• Chitotriosidase Level (Nmol/mL/hr)

  from baseline to Week 24

Other Outcomes (1)

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

  Screening to Visit14 (Week 26)

Study Arms (1)

ISU302

EXPERIMENTAL

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

Drug: ISU302

Interventions

ISU302 DRUG

60 U/kg given intravenously

Also known as: Imiglucerase

ISU302

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• ISU Abxis Co., Ltd.: lead

MeSH Terms

Conditions

Gaucher Disease

Interventions

imiglucerase

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: Taekseung Kim
• Organization: ISU ABXIS Co., Ltd.

kimts@isu.co.kr

+82.31.696.4715

Study Officials

• Amel El Beshlawy, Prof.

  Abou El Reesh Children's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 7, 2016
• First Posted: May 12, 2016
• Study Start: September 1, 2011
• Primary Completion: March 1, 2013
• Study Completion: August 1, 2014
• Last Updated: July 12, 2017
• Results First Posted: July 12, 2017

Record last verified: 2017-02