A Phase 3 interventional study of Eliglustat GZ385660 and Imiglucerase GZ437843 in Gaucher's Disease Type I and Gaucher's Disease Type III, sponsored by Sanofi. Completed at 21 sites in 10 countries. Open to participants aged 2 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-06-16.
Sponsored by Sanofi · Phase 3, Interventional, and Treatment
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Among 24 studies in Gaucher Disease by Sanofi — 2 open to participants now, 4 under way, 2 of them phase 3. Gaucher Disease · Sanofi.
Among the 24 studies in Gaucher Disease by Sanofi, this one is the 4th largest by enrollment of the 19 interventional ones that give a count, is one of 2 that started in 2018, is one of 7 in phase 3, and was the 14th registered.
177 studies on the registry are indexed under Gaucher Disease; 41 are open to participants now.
Browse Gaucher Disease studies →2 of the 24 studies in Gaucher Disease by Sanofi are open now, in 12 countries. Recruiting first, the newest first:
Genzyme, a Sanofi Company · Sites in United States, Argentina, Australia and 49 more countries
Sanofi
Ages 2–11
By country, each count listing its studies:
Of the 26 Gaucher Disease studies recruiting now, 11 (42%) set no upper age limit and 11 (42%) stop at 75 or younger; 15 take children under 18; 4 take healthy volunteers.
This study: 2 Years to 17 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
24 studies; the count open now beside each. This study's row is marked.
First in 1991; this study’s year is marked. 2 started in 2018.
This study's enrollment of 57 is above the median of 20 across 100 interventional studies indexed under Gaucher Disease.
Median enrollment 26 across the 19 interventional studies, 283 across the 4 observational. Of the 18 that completed or were terminated, 6 (33%) have results posted.
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Sanofi is the lead sponsor of 1,558 studies on the registry; 89 are open to participants now.
Of its 205 completed or terminated interventional studies of FDA-regulated products, 123 (60%) have results posted.
None of the studies under way reaches primary completion within a year.
Among the 24 studies in Gaucher Disease by Sanofi, from their registry updates:
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Primary Objective:
Evaluated the safety and pharmacokinetics of eliglustat in pediatric participants (≥2 to \<18 years old).
Secondary Objective:
Evaluated the efficacy of eliglustat and quality of life in pediatric participants (≥2 to \<18 years old).
The study included 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, participants were encouraged to enroll in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Cohort 1 (Eliglustat monotherapy):
Participants must had been receiving an enzyme replacement therapy (ERT) for a minimum of 24 months at a monthly dose equivalent to 30 U/kg to 130 U/kg of Cerezyme® (imiglucerase) with treatment ongoing at the time of enrollment. Participants had to be at pre-specified treatment goals, as defined by:
Cohort 2 (Eliglustat plus imiglucerase):
Participants must had been receiving an ERT for a minimum of 36 months at a dose equivalent to at least 60 U/kg of imiglucerase every 2 weeks, or at the maximum dose locally approved, at the time of enrollment with treatment ongoing at the time of enrollment and the dose stable for at least the 6 months preceding enrollment. Participants must had severe clinical manifestations of GD, as defined by the presence of at least one of the following:
Exclusion criteria:
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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
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 GZ385660 · Drug: Imiglucerase GZ437843
Pharmaceutical form: Capsule, Liquid Route of administration: Oral
Also known as: Cerdelga
Pharmaceutical form: Powder for solution for infusion Route of administration: Intravenous
Also known as: Cerezyme
Assessment of pharmacokinetic (PK) parameter of eliglustat: Cmax
Maximum concentration (Cmax) of eliglustat in plasma
Time frame: Weeks 2, 13, 26 and 52
Assessment of PK parameter of eliglustat: AUC
Area under the plasma eliglustat concentration-time curve (AUC)
Time frame: Weeks 2 and 52
Adverse Events
Number of adverse events in pediatric patients
Time frame: Up to Week 364
Change in hemoglobin level
Absolute change from baseline for hemoglobin (g/dL) (Cohort 1 patients)
Time frame: Baseline and Week 52
Change in platelet count
Percent change from baseline for platelet count (Cohort 1 patients)
Time frame: Baseline and Week 52
Change in liver volume
Percent change from baseline for liver volume (Cohort 1 patients)
Time frame: Baseline and Week 52
Change in spleen volume
Percent change from baseline for spleen volume (Cohort 1 patients)
Time frame: Baseline and Week 52
Pulmonary disease improvement
Proportion of patients with improvement in pulmonary disease (Cohort 2 patients)
Time frame: Baseline and Week 52
Bone disease improvement
Proportion of patients with improvement in bone disease (Cohort 2 patients)
Time frame: Baseline and Week 52
Thrombocytopenia
Proportion of patients with improvement in thrombocytopenia (Cohort 2 patients)
Time frame: Baseline and Week 52
Quality of Life
Health-related quality of life will be measured by the Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires
Time frame: Baseline and Week 52
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org
No publications or documents are linked to this record.
Ran from Apr 2018 to primary completion in Dec 2025, 92 months.
Of 11 phase 3 interventional studies under Gaucher Disease, half reached primary completion within 25 months of starting (the median), counting those still running; a quarter within 15 and three quarters within 57. This study's 92 months is above that.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Imiglucerase