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.
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.
- 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. ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Recruiting |
NCT03240653 -
Gaucherite - A Study to Stratify Gaucher Disease
|