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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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12 December 2020 |
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Main ID: |
NCT01411228 |
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Date of registration:
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04/08/2011 |
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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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A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
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Scientific title:
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A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease |
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Date of first enrolment:
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September 2011 |
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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/NCT01411228 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Care Provider, Investigator).
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Phase:
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Phase 3
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Countries of recruitment
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Israel
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Paraguay
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South Africa
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Successful completion of Protocol PB-06-002 or PB-06-005
- The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent
Exclusion Criteria:
- Currently taking another investigational drug for any condition.
- Presence of neurological signs and symptoms characteristic of Gaucher disease with
complex neuronopathic features other than longstanding oculomotor gaze palsy.
- Presence of any medical, emotional, behavioral or psychological condition that in the
judgment of the Investigator would interfere with the subject's compliance with the
requirements of the study.
Age minimum:
2 Years
Age maximum:
18 Years
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: Taliglucerase alfa
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Primary Outcome(s)
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Hemoglobin
[Time Frame: Baseline, months 9, 12 and 24]
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Secondary Outcome(s)
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Platelet Count
[Time Frame: Baseline, months 9, 12, 24 and 33-36]
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Spleen Volume
[Time Frame: Baseline, months 12 and 24]
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Chitotriosidase
[Time Frame: Baseline, months 9, 12 and 24]
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Liver Volume
[Time Frame: Baseline, months 12 and 24]
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Secondary ID(s)
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PB-06-006
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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