Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

NCT01614574 | Completed Phase 3 Results

• 1 other identifier: HGT-GCB-087
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 3

Brief Summary

Gaucher disease is an inherited deficiency of the lysosomal enzyme glucocerebrosidase (GCB) that leads to progressive accumulation of glucocerebroside within macrophages and subsequent tissue and organ damage; typically of the liver, spleen, bone marrow, and brain. The disease has been classified into 3 clinical subtypes based on the presence or absence of neurological symptoms and severity of neurological disease. Type 1 Gaucher disease affects an estimated 30,000 persons worldwide and is the most common. Type 1 Gaucher disease does not involve the central nervous system. Patients with type 2 Gaucher disease present with acute neurological deterioration, which leads to early death. Those with type 3 disease typically display a more sub-acute neurological course, with later onset and slower progression. The primary objective of this study is to evaluate the safety of every other week dosing of velaglucerase alfa in Japanese patients with Gaucher disease. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (5)

• Number of Severe Adverse Events (SAE)

  Baseline to week 51

• Number of Treatment Emergent Adverse Events (TEAE)

  Baseline to week 51

• Development of Anti-velaglucerase Alfa Antibody

  Baseline to week51

• Number of Infusion- Related Adverse Events

  Baseline to week 51

• Number of Patients With Concomitant Medication

  Baseline to week 51

Secondary Outcomes (6)

• Change From Baseline in Hemoglobin Concentration

  Baseline to week 51

• Change From Baseline in Platelet Count

  Baseline to week 51

• Change From Baseline in Liver Volume, Normalized to Body Weight

  Baseline to week 51

• Change From Baseline in Spleen Volume, Normalized to Body Weight

  Baseline to week 51

• Change From Baseline in Plasma Chitotriosidase Levels

  Baseline to week 51

Study Arms (1)

Investigational

EXPERIMENTAL

velaglucerase alfa

Biological: velaglucerase alfa

Interventions

velaglucerase alfa BIOLOGICAL

60 U/kg every other week intravenous infusion

Also known as: VPRIV, Gene activated human glucocerebrosidase

Investigational

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:

• The patient has a documented diagnosis of Gaucher disease
• The patient is at least 2 years of age
• Female patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study
• The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC)
• The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator
• Patients who are switched from imiglucerase ERT must meet the following additional criteria:
• Received treatment with imiglucerase for a minimum of 12 consecutive months
• Meet predefined limits for hemoglobin concentration and platelet counts
• Patients naïve to treatment for Gaucher disease must meet the following additional criteria:
• Not received treatment for Gaucher disease (investigational or approved products) within 12 months prior to study entry
• Have Gaucher disease related anemia and at least one of the following: moderate splenomegaly or, Gaucher disease-related thrombocytopenia or Gaucher disease-related enlarged liver

You may not qualify if:

• Treatment with any investigational drug or device within the 30 days prior to study entry (time of informed consent); such use during the study is not permitted
• Positive for hepatitis B or hepatitis C.
• Non-Gaucher disease related anemia
• The patient, patient's parent(s), or patient's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study
• Significant comorbidity, as determined by the Investigator that might affect study data or confound the study results
• The patient is unable to comply with the protocol or is unlikely to complete the study, as determined by the Investigator
• The patient has experienced a severe (grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any ERT (approved or investigational)
• Currently receiving red blood cell growth factor, (eg, erythropoietin) or chronic systemic corticosteroids in the last 6 months
• Patient has had a splenectomy or the patient has an active, clinically significant spleen infarction within 12 months of screening
• Patient has worsening bone necrosis within 12 months of screening
• The patient is pregnant or lactating.

Sponsors & Collaborators

• Shire: lead

Study Sites (3)

Hamamatsu University School of Medicine

Hamamatsu, Shizuoka, 431-3192, Japan

Location

The Jikei University School of Medicine

Minatoku, Toyko, 105-8461, Japan

Location

Osaka City University Hospital

Osaka, 545-0051, Japan

Location

MeSH Terms

Conditions

Gaucher Disease

Interventions

Glucosylceramidase

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

Intervention Hierarchy (Ancestors)

Glucosidases; Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Limitations and Caveats

All 6 enrolled patients received previous long-term treatment with the enzyme replacement therapy (ERT), imiglucerase. Key therapeutic parameters were expected to indicate stability after switching from imiglucerase.

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• 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: June 6, 2012
• First Posted: June 8, 2012
• Study Start: March 2, 2012
• Primary Completion: May 25, 2013
• Study Completion: May 25, 2013
• Last Updated: June 28, 2021
• Results First Posted: August 4, 2014

Record last verified: 2021-06

Locations

JP (3)