Overview

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:
0

Participant gender:
All

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).

Phase:

Phase 2

Accepts Healthy Volunteers?

Details

Lead Sponsor:

Shaare Zedek Medical Center

Treatments:

Ambroxol

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.