View clinical trials related to Gaucher Disease.
Filter by:This is a multicenter, multinational long-term follow-up study to assess the long-term safety and durability of effect of AVR-RD-02 treatment in subjects who previously received AVR-RD-02 treatment (single dose administration).
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).
The main aim of this study is to describe the safety profile of velaglucerase alfa (VPRIV) in participants with Gaucher disease type 1. Participants will be transitioning from other enzyme replacement therapies or substrate reduction therapies to VPRIV. Some participants may have already transitioned to treatment with VPRIV before this study started. In this study, data on VPRIV will be collected from the medical records of participants who already transitioned to VPRIV before this study started. Other participants will join this study when they transition to VPRIV. All participants will be followed to allow for 12 months of observation from time of transition to VPRIV. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study.
The investigated cohort will examine liver and spleen fibrosis in patients with Gaucher Disease(GD) by using Shear Wave Elastography- SWE to evaluation fibrosis of the tissue and to check the correlation of fibrosis with the biomarkers of disease severity.
Multicenter, doubleblinded, randomized placebo-controlled study of arimoclomol in patients with Gaucher Disease Type 1 or 3
This is an open-label study designed to assess if AT2101 is safe in patients with Gaucher disease and how AT2101 gets through the body after it is taken by mouth. The study is being offered to adult patients with type 1 Gaucher disease who are currently receiving a stable dose of enzyme replacement therapy (ERT) with imiglucerase. During the study, subjects will not be receiving ERT (up to 35 days). The study consists of a screening period (~14 days), a treatment period (12 days) and a follow-up period (7 days after last dose). At two points in the study, subjects will be housed in an in-patient treatment facility for 3 days/2 nights to accommodate all necessary blood draws.