Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Gaucher Disease Clinical Trial

Official title:

An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00391625
• Other study ID #: TKT025EXT
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: September 13, 2004
• Est. completion date: January 31, 2008

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the long term safety of enzyme replacement therapy with DRX008A (VPRIV®, GA-GCB; velaglucerase alfa) in patients with type 1 Gaucher disease.

Description:

Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the central nervous system (CNS). Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (the long term safety of enzyme replacement therapy with DRX008A (GA-GCB; velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB (velaglucerase alfa) contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to evaluate the long term safety of GA-GCB (velaglucerase alfa) in patients with Type 1 Gaucher disease

Recruitment information / eligibility

• Status: Completed
• Enrollment: 10
• Est. completion date: January 31, 2008
• Est. primary completion date: January 31, 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Patients who have completed through Week 41 visit in the TKT025 study.

• Patients must have voluntarily signed an IRB/EC approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.

• Patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

• Female and male patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Female patients must have a negative serum pregnancy test on enrollment.

Exclusion Criteria:

• Patient has received treatment with non-Gaucher disease related investigational drug or device within the past 30 days prior to study entry; such use during the study is not permitted.

• Patient has a clinically relevant medical condition (e.g., HIV, hepatitis B or C) that would make implementation of the protocol difficult and/or confound an assessment of the effects of the experimental therapy and its adverse events.

• Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope and possible consequences of the study.

• Patient is unable to comply with the protocol, e.g. uncooperative attitude, medical condition, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• GA-GCB
15-60 U/kg every other week via intravenous infusion

Locations

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem
Romania	Maria Sklodowska Curie Children's Hospital	Bucharest
Serbia	Mother and Child Health Care Institute of Serbia	Belgrade

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

Israel,  Romania,  Serbia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Evaluation of Long Term Safety	Overall Summary of Treatment-emergent Adverse Events-Safety Population	Up to 84 months
Secondary	Percent Change From Baseline in Hemoglobin Concentration		Baseline, then every 12 months
Secondary	Percent Change From Baseline in Platelet Counts		Baseline, then every 12 months
Secondary	Percent Change From Baseline in Liver Volume		Baseline, Month 24, then every 9 or 12 months
Secondary	Percent Change From Baseline in Spleen Size		Baseline, Month 24, then every 9 or 12 months