Eliglustat +/− Imiglucerase for Gaucher Disease

(ELIKIDS Trial)

Recruiting at 65 trial locations

Overseen By Trial Transparency email recommended (Toll free number for US & Canada)

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Waitlist Available

Sponsor: Sanofi

No Placebo Group

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

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).

Research Team

Clinical Sciences & Operations

Principal Investigator

Sanofi

Eligibility Criteria

This trial is for kids aged 2-17 with Gaucher Disease types 1 or 3. They must have been on enzyme replacement therapy (ERT) and meet certain health criteria. Girls post-puberty need a negative pregnancy test and agree to contraception or abstinence.

Inclusion Criteria

I am between 2 and 17 years old.

I've been on enzyme replacement therapy for at least 24 months and meet specific treatment goals.

I've been on enzyme replacement therapy for over 36 months and have severe Gaucher's disease symptoms.

Exclusion Criteria

I have been on substrate reduction therapy for Gaucher's disease within the last 6 months.

I have health issues like heart, liver problems, or other serious conditions.

I cannot or do not want to take imiglucerase due to allergies or personal choice.

Treatment Details

Interventions

• Eliglustat GZ385660 (Substrate Reduction Therapy)
• Imiglucerase GZ437843 (Enzyme Replacement Therapy)

Trial Overview The study tests the safety and effects of Eliglustat alone, or combined with Imiglucerase in children with Gaucher Disease. It looks at how their bodies handle the drugs (pharmacokinetics), changes in their condition, and life quality.

Participant Groups

Group I: Cohort 2: Eliglustat plus imiglucerase Experimental Treatment 2 Interventions

Eliglustat plus imiglucerase for three years, at the dose of enzyme replacement therapy received before enrollment. After Week 52, Cohort 2 patients will switch to eliglustat monotherapy for the remainder of the study if the desired clinical response has been achieved.

Group II: Cohort 1: Eliglustat monotherapy Experimental Treatment 1 Intervention

Eliglustat for at least two years. Cohort 1 patients that experience significant clinical decline will receive rescue treatment. Rescue Treatment Step 1: Switch from eliglustat to imiglucerase monotherapy. Rescue Treatment Step 2: Patients who after 6 months of rescue therapy with imiglucerase monotherapy do not show improvement in the parameter(s) that led to the switch from eliglustat to imiglucerase, will then receive combination therapy with eliglustat + imiglucerase.

Who Is Running the Clinical Trial?

Sanofi

Lead Sponsor

Trials

2,246

Recruited

4,085,000+

Paul Hudson

Sanofi

Chief Executive Officer since 2019

Degree in Economics from Manchester Metropolitan University

Christopher Corsico

Sanofi

Chief Medical Officer

MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University