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Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3

NCT06523517 · Status: NOT_YET_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 5

Last updated 2024-07-26

No results posted yet for this study

Summary

Primary Objective:

Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3.

Secondary Objective:

Evaluate the quality of life in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3 treated with eliglustat.

Conditions

  • Gaucher Disease

Interventions

DRUG

Eliglustat Tartrate Capsules

The initial dose is 42 mg taken orally twice a day. After 2 weeks of treatment, if the blood trough concentration is less than 5 ng/mL, the dose will be increased to 84 mg taken orally twice daily.

Sponsors & Collaborators

  • Peking Union Medical College Hospital

    lead OTHER

Principal Investigators

  • Bing Han · Peking Union Medical College

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-08-01
Primary Completion
2025-07-31
Completion
2025-07-31

Countries

  • China

Study Locations

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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 NCT06523517 on ClinicalTrials.gov