Expired Study
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Salt Lake City, Utah 84132


Purpose:

The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.


Study summary:

Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases and does not involve the 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 (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 contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.


Criteria:

Inclusion Criteria: 1. The patient has completed study TKT032 or TKT034, or study HGT-GCB-039. 2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study. 3. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator. 4. 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). 5. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator Exclusion Criteria: 1. The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted. 2. The patient is pregnant or lactating. 3. 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. 4. The patient has a significant comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, etc.). 5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator


Study is Available At:


Original ID:

HGT-GCB-044


NCT ID:

NCT00635427


Secondary ID:

2008-001965-27


Study Acronym:


Brief Title:

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease


Official Title:

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease


Overall Status:

Completed


Study Phase:

Phase 3


Genders:

Both


Minimum Age:

2 Years


Maximum Age:

N/A


Quick Facts

Healthy Volunteers
Oversight Has DMC
Study Is FDA Regulated
Study Is Section 801
Has Expanded Access

Study Source:

Shire


Oversight Authority:

  • United States: Food and Drug Administration
  • Paraguay: Ministerio de Salud Pública y Bienestar Social
  • Israel: Ministry of Health
  • Argentina: Administracion Nacional de Medicamentos, Alimentos y Tecnologia Medica
  • Poland: Ministry of Health
  • United Kingdom: Medicines and Healthcare Products Regulatory Agency
  • India: Drugs Controller General of India
  • South Korea: Korea Food and Drug Administration (KFDA)
  • Russia: Ministry of Health of the Russian Federation
  • Spain: Ministry of Health and Consumption
  • Tunisia: Ministry of Public Health


Reasons Why Stopped:


Study Type:

Interventional


Study Design:

Allocation: Non-Randomized, Endpoint Classificatio


Number of Arms:

3


Number of Groups:

0


Total Enrollment:

95


Enrollment Type:

Actual


Overall Contact Information

Official Name:Eric Crombez, M.D.
Study Director
Shire Human Genetic Therapies, Inc.

Study Dates

Start Date:May 2008
Completion Date:December 2012
Completion Type:Actual
Primary Completion Date:December 2012
Primary Completion Type:Actual
Verification Date:March 2014
Last Changed Date:May 15, 2015
First Received Date:March 6, 2008
First Results Date:December 11, 2013

Study Outcomes

Outcome Type:Primary Outcome
Measure:Overall Summary of Treatment Emergent Adverse Events
Time Frame:Baseline to termination of study
Safety Issues:True
Description:Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons
Outcome Type:Secondary Outcome
Measure:Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group
Time Frame:Baseline to 24 months
Safety Issues:False
Outcome Type:Secondary Outcome
Measure:Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group
Time Frame:Baseline to 24 months
Safety Issues:False
Outcome Type:Secondary Outcome
Measure:Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group
Time Frame:Baseline to 24 months
Safety Issues:False
Outcome Type:Secondary Outcome
Measure:Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group
Time Frame:Baseline to 24 months
Safety Issues:False

Study Interventions

Intervention Type:Biological
Name:VPRIV®
Description:Intravenous infusion, every other week (EOW)
Arm Name:VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TK
Other Name:velaglucerase alfa

Study Arms

Study Arm Type:Experimental
Arm Name:VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039)
Description:This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)
Study Arm Type:Experimental
Arm Name:VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039)
Description:imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044
Study Arm Type:Experimental
Arm Name:VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034)
Description:VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034

Study Agencies

Agency Class:Industry
Agency Type:Lead Sponsor
Agency Name:Shire

Sample and Retention Information

There are no available Sample and Retention Information

Study References

There are no available Study References

Data Source: ClinicalTrials.gov

Date Processed: January 21, 2020

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