Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
- Conditions
- Gaucher's Disease Type IGaucher's Disease Type III
- Interventions
- Registration Number
- NCT03485677
- Lead Sponsor
- Sanofi
- Brief Summary
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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 57
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1: Eliglustat monotherapy Eliglustat GZ385660 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. Cohort 2: Eliglustat plus imiglucerase Imiglucerase GZ437843 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. Cohort 2: Eliglustat plus imiglucerase Eliglustat GZ385660 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.
- Primary Outcome Measures
Name Time Method Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax Weeks 2, 13, 26 and 52 Maximum concentration (Cmax) of eliglustat in plasma
Adverse Events Up to Week 364 Number of adverse events in pediatric patients
Assessment of PK parameter of eliglustat: AUC Weeks 2 and 52 Area under the plasma eliglustat concentration-time curve (AUC)
- Secondary Outcome Measures
Name Time Method Change in hemoglobin level Baseline and Week 52 Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients)
Pulmonary disease improvement Baseline and Week 52 Proportion of patients with improvement in pulmonary disease (Cohort 2 patients)
Change in liver volume Baseline and Week 52 Percent change from baseline for liver volume (Cohort 1 patients)
Quality of Life Baseline and Week 52 Health-related quality of life will be measured by the Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires
Change in platelet count Baseline and Week 52 Percent change from baseline for platelet count (Cohort 1 patients)
Change in spleen volume Baseline and Week 52 Percent change from baseline for spleen volume (Cohort 1 patients)
Bone disease improvement Baseline and Week 52 Proportion of patients with improvement in bone disease (Cohort 2 patients)
Thrombocytopenia Baseline and Week 52 Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients)
Trial Locations
- Locations (21)
Investigational Site Number : 1240002
🇨🇦Calgary, Alberta, Canada
Investigational Site Number : 0320001
🇦🇷Capital Federal, Buenos Aires, Argentina
Investigational Site Number : 1240003
🇨🇦Vancouver, British Columbia, Canada
Investigational Site Number : 3920001
🇯🇵Minato-ku, Tokyo, Japan
Investigational Site Number : 7240002
🇪🇸Barakaldo, Bizkaia, Spain
Investigational Site Number : 7920004
🇹🇷Adana, Turkey
Investigational Site Number : 7920003
🇹🇷Istanbul, Turkey
Investigational Site Number : 1240001
🇨🇦Toronto, Ontario, Canada
Investigational Site Number : 6430004
🇷🇺Moscow, Russian Federation
Investigational Site Number : 3800002
🇮🇹Roma, Italy
Investigational Site Number : 2500002
🇫🇷BRON Cedex, France
Investigational Site Number : 3920002
🇯🇵Koshigaya-shi, Saitama, Japan
Investigational Site Number : 6430005
🇷🇺St-Petersburg, Russian Federation
Investigational Site Number : 6430001
🇷🇺Moscow, Russian Federation
Investigational Site Number : 6430002
🇷🇺Tomsk, Russian Federation
Investigational Site Number : 7240003
🇪🇸Zaragoza, Spain
Investigational Site Number : 7520002
🇸🇪Göteborg, Sweden
Investigational Site Number : 7240001
🇪🇸Esplugues de Llobregat, Catalunya [Cataluña], Spain
Investigational Site Number : 7920002
🇹🇷Izmir, Turkey
Investigational Site Number : 7520001
🇸🇪Luleå, Sweden
Investigational Site Number : 8260002
🇬🇧Birmingham, United Kingdom