Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)
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ClinicalTrials.gov Identifier: NCT00391625 |
Recruitment Status :
Completed
First Posted : October 24, 2006
Results First Posted : June 23, 2014
Last Update Posted : June 17, 2016
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Condition or disease | Intervention/treatment | Phase |
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Gaucher Disease | Drug: GA-GCB | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 10 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
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 Start Date : | February 2005 |
Actual Primary Completion Date : | December 2011 |
Actual Study Completion Date : | December 2011 |

Arm | Intervention/treatment |
---|---|
Experimental: GA-GCB
15-60 U/kg every other week via intravenous infusion
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Drug: GA-GCB
15-60 U/kg every other week via intravenous infusion
Other Names:
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- Evaluation of Long Term Safety [ Time Frame: Up to 84 months ]Overall Summary of Treatment-emergent Adverse Events-Safety Population
- Percent Change From Baseline in Hemoglobin Concentration [ Time Frame: Baseline, then every 12 months ]
- Percent Change From Baseline in Platelet Counts [ Time Frame: Baseline, then every 12 months ]
- Percent Change From Baseline in Liver Volume [ Time Frame: 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 ]

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00391625
Israel | |
Shaare Zedek Medical Center | |
Jerusalem, Israel | |
Romania | |
Maria Sklodowska Curie Children's Hospital | |
Bucharest, Romania, 75544 | |
Serbia | |
Mother and Child Health Care Institute of Serbia | |
Belgrade, Serbia |
Principal Investigator: | Florea Iordachescu, MD | Maria Sklodowska Curie Children's Hospital | |
Principal Investigator: | Ari Zimran, M.D. | Gaucher Clinic, Shaare Zedek Medical Center | |
Principal Investigator: | Maja Djordjevic, M.D. | Mother and Child Health Care Institute of Serbia |
Responsible Party: | Shire |
ClinicalTrials.gov Identifier: | NCT00391625 |
Other Study ID Numbers: |
TKT025EXT |
First Posted: | October 24, 2006 Key Record Dates |
Results First Posted: | June 23, 2014 |
Last Update Posted: | June 17, 2016 |
Last Verified: | August 2015 |
Gaucher disease, Enzyme Replacement Therapy |
Gaucher Disease 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 Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |