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Eliglustat on Gaucher Disease Type IIIB

NCT03519646 · Status: COMPLETED · Phase: NA · Type: INTERVENTIONAL · Enrollment: 4

Last updated 2022-09-15

No results posted yet for this study

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.

Conditions

  • Gaucher Disease, Type III

Interventions

DRUG

Eliglustat

1. This is a 3-year study and the enrollment time of this study is 24 months. 2. The participants have to receive the investigational agent, Cerdelga 3. The participants have to go back to the hospital and receive the investigational agent and take the test before receving Cerdelga, and 2 weeks, 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga. 4. IMP Administration Method: Cerdelga have 21 mg、42 mg and 84 mg capsule. 5. No need for fasting before use, but can't take with grapefruit juice. 6. Five ml Blood and 10 ml urine shoud be taken before receiving Cerdelga, and 1, 3, 6, 12, 18 and 24 months after receiving Cerdelga for Gaucher related biomarkers test.

Sponsors & Collaborators

  • Sanofi

    collaborator INDUSTRY

  • National Taiwan University Hospital

    lead OTHER

Principal Investigators

  • Ni-Chung Lee, M.D., Ph.D · National Taiwan University Hospital

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
Yes

Timeline & Regulatory

Start
2018-04-23
Primary Completion
2020-09-11
Completion
2020-09-11

Countries

  • Taiwan

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03519646 on ClinicalTrials.gov