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

Primary Purpose

Gaucher Disease

Status

Completed

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

GA-GCB

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring Gaucher disease, Enzyme Replacement Therapy

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

Shaare Zedek Medical Center
Maria Sklodowska Curie Children's Hospital
Mother and Child Health Care Institute of Serbia

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

GA-GCB

Arm Description

15-60 U/kg every other week via intravenous infusion

Outcomes

Primary Outcome Measures

Evaluation of Long Term Safety

Overall Summary of Treatment-emergent Adverse Events-Safety Population

Secondary Outcome Measures

Percent Change From Baseline in Hemoglobin Concentration

Percent Change From Baseline in Platelet Counts

Percent Change From Baseline in Liver Volume

Percent Change From Baseline in Spleen Size

Full Information

NCT ID

NCT00391625

First Posted

October 20, 2006

Last Updated

May 30, 2021

Sponsor

Shire

1. Study Identification

Unique Protocol Identification Number

NCT00391625

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

May 2021

Overall Recruitment Status

Completed

Study Start Date

September 13, 2004 (Actual)

Primary Completion Date

January 31, 2008 (Actual)

Study Completion Date

January 31, 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Shire

4. Oversight

Data Monitoring Committee

5. Study Description

Brief 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.

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Gaucher Disease

Keywords

Gaucher disease, Enzyme Replacement Therapy

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Actual)

8. Arms, Groups, and Interventions

Arm Title

GA-GCB

Arm Type

Experimental

Arm Description

15-60 U/kg every other week via intravenous infusion

Intervention Type

Drug

Intervention Name(s)

GA-GCB

Other Intervention Name(s)

VPRIV®, velaglucerase alfa, gene-activated glucocerebrosidase, DRX008

Intervention Description

15-60 U/kg every other week via intravenous infusion

Primary Outcome Measure Information:

Title

Evaluation of Long Term Safety

Description

Overall Summary of Treatment-emergent Adverse Events-Safety Population

Time Frame

Up to 84 months

Secondary Outcome Measure Information:

Title

Percent Change From Baseline in Hemoglobin Concentration

Time Frame

Baseline, then every 12 months

Title

Percent Change From Baseline in Platelet Counts

Time Frame

Baseline, then every 12 months

Title

Percent Change From Baseline in Liver Volume

Time Frame

Baseline, Month 24, then every 9 or 12 months

Title

Percent Change From Baseline in Spleen Size

Time Frame

Baseline, Month 24, then every 9 or 12 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Study Director

Organizational Affiliation

Takeda

Official's Role

Study Director

Facility Information:

Facility Name

Shaare Zedek Medical Center

City

Jerusalem

Country

Israel

Facility Name

Maria Sklodowska Curie Children's Hospital

City

Bucharest

ZIP/Postal Code

75544

Country

Romania

Facility Name

Mother and Child Health Care Institute of Serbia

City

Belgrade

Country

Serbia

12. IPD Sharing Statement

Citations:

PubMed Identifier

20299511

Citation

Zimran A, Altarescu G, Philips M, Attias D, Jmoudiak M, Deeb M, Wang N, Bhirangi K, Cohn GM, Elstein D. Phase 1/2 and extension study of velaglucerase alfa replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood. 2010 Jun 10;115(23):4651-6. doi: 10.1182/blood-2010-02-268649. Epub 2010 Mar 18.

Results Reference

derived

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

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