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Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Primary Objective:

Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (2 to <18 years old).

Secondary Objective:

Evaluate the efficacy of eliglustat and quality of life in pediatric patients (2 to <18 years old).

Detailed Description

The study will include a screening period of up to 60 days (Day -60 to -1), a primary analysis treatment period (Day 1 to Week 52), a long-term treatment period (Week 53 to Week 104), and an extension period continuing up to Week 364 (for patients who continue to demonstrate the clinical benefit from eliglustat monotherapy at Week 104). After study completion, patients will be encouraged to enroll in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.

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Call 1800-9860-568 now to find out if you are eligible.

Age

2 - 17

Gender

All

NCT ID

NCT03485677

Phase

3

Status

Recruiting Now

Medical Condition

Gaucher Disease

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Medical Condition

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The Study

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About Clinical Trials

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Your Journey

01
Receiving the medication

You would receive etanercept (Enbrel) twice a week for 12 weeks and then once a week for 12 weeks. Etanercept, as well as study related medical care, is provided at no cost.

02
Visiting the study site

Study participation involves approximately 8 visits to your local study center over 6 to 7 months.

03
Follow-up

There would also be a follow-up telephone call 30days after completing the study. No visits are required after participation is complete.

Call 1800-9860-568 now to find out if you are eligible.