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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 November 2022 |
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Main ID: |
NCT04120506 |
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Date of registration:
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10/08/2018 |
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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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Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
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Scientific title:
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Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Adult Patients With Type 1 Gaucher Disease, Previously on a Stable Dose of VPRIV for at Least 3 Months: an Extension of the Investigator-initiated Study |
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Date of first enrolment:
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January 10, 2016 |
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Target sample size:
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15 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT04120506 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 4
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Contacts
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Name:
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Ari Zimran |
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Address:
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Telephone:
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Email:
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Affiliation:
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Ari Zimran - Shaare Zedek |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Aged 18-75 years, non-splenectomized Enzymatic diagnosis & molecular analysis
indicative of type 1 Gaucher disease Receiving VPRIV for at least 6 infusions (3
months) prior to Baseline at a constant dose and frequency and without clinically
significant AEs including allergic reactions
Exclusion Criteria:
- Experience of a clinically significant AE to VPRIV at any time in the past Existence
of a clinically significant co-morbidity
Age minimum:
6 Years
Age maximum:
75 Years
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: VPRIV
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Primary Outcome(s)
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Incidence of change from baseline in temperature for rapid infusion-1
[Time Frame: 9 months]
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Incidence of change from baseline in heart rate for rapid infusion-1
[Time Frame: 9 months]
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Incidence of change from baseline in blood pressure for rapid infusion-1
[Time Frame: 9 months]
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Secondary Outcome(s)
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Non deterioration in Gaucher manifestations- measured by lack of elevated biomarker- Lyso-GB1
[Time Frame: 9 months]
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Non deterioration in Gaucher manifestations- measured by liver volume
[Time Frame: 9 months]
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Non deterioration in Gaucher manifestations- measured by spleen volumes
[Time Frame: 9 months]
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Non deterioration in Gaucher manifestations- stability in platelet counts
[Time Frame: 9 months]
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Non deterioration in Gaucher manifestations- stability in hemaglobin count
[Time Frame: 9 months]
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Secondary ID(s)
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0075-15-SZMC
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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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