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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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7 June 2021 |
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Main ID: |
NCT00954460 |
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Date of registration:
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05/08/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
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Scientific title:
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Multicenter Open-Label Treatment Protocol to Observe the Safety of Gene-Activated™ Human Glucocerebrosidase (GA-GCB, Velaglucerase Alfa) ERT in Newly Diagnosed or Previously Treated (With Imiglucerase) Patients With Type 1 Gaucher Disease |
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Date of first enrolment:
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October 2009 |
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Target sample size:
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Recruitment status: |
Approved for marketing |
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URL:
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https://clinicaltrials.gov/show/NCT00954460 |
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Study type:
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Expanded Access |
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Study design:
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Phase:
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Countries of recruitment
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United States
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Contacts
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Name:
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Study Director |
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Address:
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Telephone:
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Email:
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Affiliation:
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Takeda |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. The patient has a documented diagnosis of type 1 Gaucher disease
2. The patient is > 2 years of age
3. The patient has NOT previously experienced an anaphylactic or anaphylactoid reaction
to another ERT including imiglucerase
4. Women of child-bearing potential must agree to use a medically acceptable method of
contraception at all times during the study; and must have a negative result to a
pregnancy test as required throughout their participation in the study. Male patients
must use a medically acceptable method of birth control throughout their participation
in the study and must report their partner's pregnancy.
5. The patient is sufficiently cooperative to participate in this treatment plan as
judged by the Investigator
6. If the patient is naïve or new to treatment, the patient has one or more of the
following (in absence of the following criteria, please call the sponsor for treatment
justification):
- Gaucher disease-related anemia
- Moderate splenomegaly (2 to 3 cm below the left costal margin), by palpation
- Gaucher disease-related thrombocytopenia
- Gaucher disease-related palpable enlarged liver
Exclusion Criteria: None
Age minimum:
3 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Gaucher Disease, Type 1
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Intervention(s)
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Drug: velaglucerase alfa
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Secondary ID(s)
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HGT-GCB-058
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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