Status:
COMPLETED
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Lead Sponsor:
Shaare Zedek Medical Center
Conditions:
Gaucher Disease, Type 1
Eligibility:
All Genders
18-75 years
Phase:
PHASE2
Brief Summary
Ambroxol hydrochloride, an over-the-counter antitussive available in many markets , was identified as an interesting pharmacological chaperone. In addition to a mucolytic action, ambroxol has antioxid...
Detailed Description
Introduction: Description of the Problem: Enzyme replacement therapy (ERT) makes a significant impact on the clinical manifestations and quality of life of patients with Gaucher disease (GD) . The g...
Eligibility Criteria
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.
Key Trial Info
Start Date :
March 1 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 30 2022
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT03950050
Start Date
March 1 2019
End Date
December 30 2022
Last Update
February 14 2023
Active Locations (1)
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1
Shaare Zedek Medical Center
Jerusalem, Israel