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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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29 March 2021 |
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Main ID: |
NCT03702361 |
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Date of registration:
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06/08/2018 |
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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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Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease
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Scientific title:
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Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease: An Investigator-initiated Study |
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Date of first enrolment:
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September 4, 2018 |
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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/NCT03702361 |
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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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Countries of recruitment
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Israel
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Contacts
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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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Ari Zimran - Shaare Zedek |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Males and females, 18 years or older for the first ten adult patients; 6 years or
older for the last five patients.
- Confirmed enzymatic diagnosis of Gaucher disease with a defined genotype and
elevated biomarker LysoGb1, performed at CentoGene using DBS methodology.
- Indications for ERT will be guided by fulfilling the MOH criteria.
- Female patients of child-bearing potential who agree to use a medically
acceptable method of contraception.
- Patients who have not received ERT or SRT in the past or Patients who have not
received ERT or SRT in the past 12 months and have a negative
anti-glucocerebrosidase antibody
Exclusion Criteria:
- Currently taking another experimental drug for any condition
- Presence of neurologic signs and symptoms characteristic of Type 2 or Type 3
Gaucher disease
- Pregnant or nursing
- Presence of any medical, emotional, behavioral or psychological condition that in
the judgment of the Investigator would interfere with the patient's compliance
with the study.
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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Primary Disease
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Intervention(s)
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Drug: VPRIV
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Primary Outcome(s)
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Percent change from baseline in spleen volume measured by MRI
[Time Frame: 12 months.]
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Secondary Outcome(s)
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Change from baseline 10% reduction in spleen volume
[Time Frame: 6 months]
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Change from baseline in liver volume
[Time Frame: 12 months]
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Change from baseline in platelet count
[Time Frame: 12 months]
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Change from baseline in Hemoglobin
[Time Frame: 12 months]
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Change from baseline in Lyso-GB1
[Time Frame: 12 months]
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Secondary ID(s)
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307-17-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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