Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

NCT01842841 | Completed Phase 3 Results

• 1 other identifier: HGT-GCB-091
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 5

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. 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 long-term safety of every other week (EOW) dosing of velaglucerase alfa in Japanese patients with Gaucher disease who completed study HGT-GCB-087 and elected to continue treatment with velaglucerase alfa. Velaglucerase alfa has been developed and approved as an enzyme replacement therapy for Type 1 Gaucher disease.

Conditions

Gaucher Disease

Outcome Measures

Primary Outcomes (4)

• Number of Participants With Drug-related Adverse Events (AEs), Infusion-related AEs, and Serious AEs (SAEs)

  An AE was any noxious, pathologic, or unintended change in anatomical, physiologic, or metabolic function as indicated by physical signs, symptoms, or laboratory changes occurring in any phase of a clinical study, whether or not considered related to investigational product. A SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged in-patient hospitalization; life threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. An infusion-related AE was defined as an AE that started either during or within 12 hours after the start of the infusion and that was judged as possibly or probably related to investigational product.

  From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)

• Number of Participants Using Concomitant Medication

  From the day of first infusion (Week 53) up to 30 days after last infusion (approximately 107 weeks)

• Number of Participants With Abnormal and Clinically Significant Laboratory Test Results

  Laboratory test results were considered abnormal and clinically significant at the discretion of the investigator.

  From Week 65 until the end of study (Week 155)

• Number of Participants With Positive Anti-Velaglucerase Alfa Antibodies

  Serum samples were collected for all participants for determination of anti-velaglucerase alfa antibodies every 12 weeks.

  From Week 65 until the end of study (Week 155)

Secondary Outcomes (12)

• Change From Baseline in Hemoglobin Concentration at Week 101

  Baseline, Week 101

• Change From Baseline in Platelet Count at Week 101

  Baseline, Week 101

• Change From Baseline in Liver Volume Normalized to Body Weight at Week 103

  Baseline, Week 103

• Change From Baseline in Spleen Volume Normalized to Body Weight at Week 103

  Baseline, Week 103

• Change From Baseline in Bone Mineral Density (BMD) at Week 103: Z Score

  Baseline, Week 103

Study Arms (1)

velaglucerase alfa

EXPERIMENTAL

15 to 60 U/kg, EOW via intravenous infusion

Drug: velaglucerase alfa

Interventions

velaglucerase alfa DRUG

15-60 U/kg, EOW

Also known as: VPRIV

velaglucerase alfa

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 completed treatment with EOW velaglucerase alfa through Week 51 of study HGT-GCB-087.
• 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.

You may not qualify if:

• The patient has received treatment with any investigational drug, other than velaglucerase alfa, or investigational device within 30 days prior to study entry; such use during the study is not permitted.
• 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.
• The patient has a 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 as determined by the Investigator.

Sponsors & Collaborators

• Shire: lead
• Quintiles, Inc.: collaborator

Study Sites (5)

Hamamatsu University School of Medicine

Hamamatsu, Shizuoka, 431-3192, Japan

Location

Iwata City Hospital

Ōkubo, Shizuoka, 438-8550, Japan

Location

The Jikei University School of Medicine

Minato-ku, Tokyo, 105-8471, Japan

Location

Chiba Children's Hospital

Chiba, 266-0007, Japan

Location

Osaka City University Hospital

Osaka, 545-8586, Japan

Location

Related Publications (1)

• Ida H, Tanaka A, Matsubayashi T, Murayama K, Hongo T, Lee HM, Mellgard B. A multicenter, open-label extension study of velaglucerase alfa in Japanese patients with Gaucher disease: Results after a cumulative treatment period of 24months. Blood Cells Mol Dis. 2016 Jul;59:140-7. doi: 10.1016/j.bcmd.2015.10.002. Epub 2015 Oct 16.

  PMID: 27241455 DERIVED

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

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: April 11, 2013
• First Posted: April 30, 2013
• Study Start: March 13, 2013
• Primary Completion: October 8, 2014
• Study Completion: October 8, 2014
• Last Updated: June 14, 2021
• Results First Posted: December 14, 2015

Record last verified: 2021-05

Locations

JP (5)