Trials Sponsors Conditions Drugs Pricing

Conditions

Gaucher's disease type I; Gaucher's disease type III Gaucher's Disease Type I Gaucher's Disease Type III

Outcome Measures

Primary Outcomes (3)

Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax
Maximum concentration (Cmax) of eliglustat in plasma
Weeks 2, 13, 26 and 52
Assessment of PK parameter of eliglustat: AUC
Area under the plasma eliglustat concentration-time curve (AUC)
Weeks 2 and 52
Adverse Events
Number of adverse events in pediatric patients
Up to Week 364

Secondary Outcomes (8)

Change in hemoglobin level
Baseline and Week 52
Change in platelet count
Baseline and Week 52
Change in liver volume
Baseline and Week 52
Change in spleen volume
Baseline and Week 52
Pulmonary disease improvement
Baseline and Week 52
+3 more secondary outcomes

Study Arms (2)

Cohort 1: Eliglustat monotherapy

EXPERIMENTAL

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.

Drug: Eliglustat GZ385660

Cohort 2: Eliglustat plus imiglucerase

EXPERIMENTAL

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.

Drug: Eliglustat GZ385660Drug: Imiglucerase GZ437843

Interventions

Eliglustat GZ385660Drug

Pharmaceutical form: Capsule, Liquid Route of administration: Oral

Also known as: Cerdelga

Cohort 1: Eliglustat monotherapyCohort 2: Eliglustat plus imiglucerase

Imiglucerase GZ437843Drug

Pharmaceutical form: Powder for solution for infusion Route of administration: Intravenous

Eligibility Criteria

Age2 Years - 17 Years

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17)

You may qualify if:

The participant were 2 to \<18 years old at the time of informed consent.
Male and female participants with a clinical diagnosis of Gaucher disease (GD) type 1 or type 3 with documented deficiency of acid beta-glucosidase activity by enzyme assay and glucocerebrosidase (GBA) genotype.

Sponsors & Collaborators

Genzyme Corp.
Industrylead
Sanofi
Industrylead

Study Sites (21)

Investigational Site Number : 0320001

Buenos Aires, 1428, Argentina

Investigational Site Number : 1240002

Calgary, Alberta, T3B 6A9, Canada

Investigational Site Number : 1240003

Vancouver, British Columbia, V6H 3N1, Canada

Investigational Site Number : 1240001

Toronto, Ontario, M5G 1X8, Canada

Investigational Site Number : 2500002

Bron, 69500, France

Investigational Site Number : 3800002

Rome, Roma, 00165, Italy

Investigational Site Number : 3920002

Koshigaya, Saitama, 343-0845, Japan

MeSH Terms

Conditions

Gaucher Disease

Interventions

eliglustatimiglucerase

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

Clinical Sciences & Operations
Sanofi
STUDY DIRECTOR

Enrollment

57 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: phase 3
Allocation: NON RANDOMIZED
Masking: NONE
Purpose: TREATMENT
Intervention Model: SEQUENTIAL
Sponsor Type: INDUSTRY
Responsible Party: SPONSOR

Study Record Dates

First Submitted

March 23, 2018

First Posted

April 2, 2018

Study Start

April 11, 2018

Actual (reported)

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

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (8)

Study Arms (2)

Cohort 1: Eliglustat monotherapy

Cohort 2: Eliglustat plus imiglucerase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (21)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

First Posted

Study Start

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (3)

Secondary Outcomes (8)

Study Arms (2)

Cohort 1: Eliglustat monotherapy

Cohort 2: Eliglustat plus imiglucerase

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (21)

Related Links

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Study Officials

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source records

Locations