Type I Gaucher Disease Clinical Trial
Official title:
An Open-Label, Dose Escalation With 2 Dose Levels, Proof-of-Concept Clinical Trial of Ambroxol for the Treatment of Type I Gaucher Disease
Ambroxol is expected to improve the signs and symptoms of patients with Type I Gaucher Disease.
This is an Open-Label, Dose Escalation with 2 Dose Levels, Proof-of-Concept Clinical Trial
of Ambroxol for the Treatment of Patients with Type I Gaucher Disease.
This study is a randomized clinical trial involving 20 evaluable patients affected with Type
1 Gaucher disease who are responsive to Ambroxol in vitro. There are 2 treatment groups,
involving 2 dose levels of Ambroxol (187.5 and 225 mg/day), given once daily PO for 2 months
in both groups. The 187.5-mg/day dose level will be tested first. If there are no
significant adverse events, defined as >Grade 3 toxicity according to the latest version of
the National Cancer Institute (NCI) Common Toxicity Criteria (CTC), the 225-mg/day dose
level will be tested.
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Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment