Completed
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Led by Shaare Zedek Medical Center · Updated on 2023-02-14
40
Participants Needed
1
Research Sites
4 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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 antioxidant and anti-inflammatory properties. Importantly, ambroxol therapy was found safe when given to pregnant women for prevention of neonatal respiratory distress syndrome . Thus, ambroxol, an oral available drug on the market, may be a safe option for GD patients with potential disease-specific efficacy and should be expanded into a clinical trial using higher doses and placebo-controlled design. The investigators propose to start with a phase II study for patients with type 1 GD and suboptimal response to ERT. In addition the investigators plan to open an international registry of patients with GD currently receiving ambroxol (off study).
CONDITIONS
Brief Title
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Who Can Participate
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.
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