Eliglustat on Gaucher Disease Type IIIB

Gaucher Disease, Type III Clinical Trial

Official title: Evaluation of the Safety in the Combination Usage of Cerdelga and Cerezyme in Type III Gaucher Disease Patients and the Efficacy on Soft Tissue Diseases.

Status Completed

Clinical Trial Summary

Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.

Clinical Trial Description

• This is a 3-year study and the enrollment time of this study is 24 months.

• The participants have to receive the investigational agent:Cerdelga.(Cerdelga have 21 mg、42 mg and 84 mg capsule.)

• The participants have to go back to the hospital and receive the investigational agent and take the Gaucher related biomarkers test before receiving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga.

• The participants have to inform if any adverse events happened.

• The investigators will follow up by phone if adverse events happened in the participants after one months start the treatment. ;

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type III

• NCT number: NCT03519646
• Study type: Interventional
• Source: National Taiwan University Hospital
• Status: Completed
• Phase: N/A
• Start date: April 23, 2018
• Completion date: September 11, 2020