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

May 30, 2021 updated by: Shire

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed 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

Study Type

Interventional

Enrollment (Actual)

10

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Israel
      • Jerusalem, Israel
        • Shaare Zedek Medical Center
  • Romania
      • Bucharest, Romania, 75544
        • Maria Sklodowska Curie Children's Hospital
  • Serbia
      • Belgrade, Serbia
        • Mother and Child Health Care Institute of Serbia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: GA-GCB
15-60 U/kg every other week via intravenous infusion
Drug: GA-GCB
15-60 U/kg every other week via intravenous infusion
Other Names:
  • velaglucerase alfa
  • VPRIV®
  • gene-activated glucocerebrosidase
  • DRX008

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Evaluation of Long Term Safety
Time Frame: Up to 84 months
Overall Summary of Treatment-emergent Adverse Events-Safety Population
Up to 84 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Percent Change From Baseline in Hemoglobin Concentration
Time Frame: Baseline, then every 12 months
Baseline, then every 12 months
Percent Change From Baseline in Platelet Counts
Time Frame: Baseline, then every 12 months
Baseline, then every 12 months
Percent Change From Baseline in Liver Volume
Time Frame: Baseline, Month 24, then every 9 or 12 months
Baseline, Month 24, then every 9 or 12 months
Percent Change From Baseline in Spleen Size
Time Frame: Baseline, Month 24, then every 9 or 12 months
Baseline, Month 24, then every 9 or 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 13, 2004

Primary Completion (Actual)

January 31, 2008

Study Completion (Actual)

January 31, 2008

Study Registration Dates

First Submitted

October 20, 2006

First Submitted That Met QC Criteria

October 20, 2006

First Posted (Estimate)

October 24, 2006

Study Record Updates

Last Update Posted (Actual)

June 22, 2021

Last Update Submitted That Met QC Criteria

May 30, 2021

Last Verified

May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TKT025EXT

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Subscribe