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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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20 February 2023 |
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Main ID: |
NCT03950050 |
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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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Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
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Scientific title:
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Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy |
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Date of first enrolment:
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March 1, 2019 |
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Target sample size:
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40 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT03950050 |
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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 2
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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 |
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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:
*Adult patients = 18 years with type 1 GD and suboptimal response to ERT defined as one or
more than one of the following: platelet count < 100 x 103/mm3 bone mineral density < -2 T
score Lyso-GB1 > 200 ng/ml.
*No change in dose or preparation of ERT in the last 12 months (Except for Naive patients)
Exclusion Criteria:
- Patients with comorbidity that may impact on the primary and/or secondary endpoint.
- Pregnant women will be excluded from the study.
- Inability to cooperate with the study procedure
- Hypersensitivity or any other contraindication listed in the local labeling of
ambroxol
- Refusal of patients to participate in the study.
Age minimum:
18 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: Ambroxol
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Primary Outcome(s)
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Platelets count
[Time Frame: 12 months.]
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Lyso-GB1 biomarker for Gaucher disease
[Time Frame: 12 months]
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bone mineral density evaluated by Dual Energy X-ray Absorptiometry (DEXA)
[Time Frame: 12 months.]
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Secondary Outcome(s)
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Patient-reported outcomes (PRO)
[Time Frame: 12 months]
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Fatigue Severity Scale (FSS)
[Time Frame: 12 months]
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Secondary ID(s)
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0005-18-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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