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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 October 2017 |
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Main ID: |
NCT00391625 |
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Date of registration:
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20/10/2006 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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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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Scientific title:
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An Open-Label Extension of Study TKT025 Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A Enzyme Replacement Therapy |
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Date of first enrolment:
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February 2005 |
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Target sample size:
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10 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00391625 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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Former Serbia and Montenegro
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Israel
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Romania
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Serbia
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Contacts
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Name:
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Florea Iordachescu, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Maria Sklodowska Curie Children's Hospital |
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Name:
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Maja Djordjevic, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Mother and Child Health Care Institute of Serbia |
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Name:
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Ari Zimran, M.D. |
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Address:
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Telephone:
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Email:
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Affiliation:
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Gaucher Clinic, Shaare Zedek Medical Center |
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Key inclusion & exclusion criteria
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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.
Age minimum:
18 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Gaucher Disease
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Intervention(s)
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Drug: GA-GCB
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Primary Outcome(s)
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Evaluation of Long Term Safety
[Time Frame: Up to 84 months]
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Secondary Outcome(s)
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Percent Change From Baseline in Hemoglobin Concentration
[Time Frame: Baseline, then every 12 months]
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Percent Change From Baseline in Platelet Counts
[Time Frame: Baseline, then every 12 months]
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Percent Change From Baseline in Liver Volume
[Time Frame: Baseline, Month 24, then every 9 or 12 months]
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Percent Change From Baseline in Spleen Size
[Time Frame: Baseline, Month 24, then every 9 or 12 months]
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Secondary ID(s)
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TKT025EXT
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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