Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Gaucher Disease, Type 1
About this trial
This is an interventional treatment trial for Gaucher Disease, Type 1
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.
Sites / Locations
- Shaare Zedek Medical Center
Arms of the Study
Arm 1
Experimental
Ambroxol
Ambroxol therapy will be dosed up to 600 mg/day divided to twice a day starting 150 mg for the first month, 300 mg for the following month and 600 mg for the following month. The study was conducted in accordance with the provisions of the Declaration of Helsinki, Good Clinical Practice guidelines, and local laws and regulations.