Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
Status:
Unknown status
Trial end date:
2021-03-01
Target enrollment:
Participant gender:
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 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).