Trial results
AI generated results summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
According to the results reported on ClinicalTrials.gov, this trial tested a medicine called eliglustat in people with Gaucher disease (a rare condition where a fatty substance builds up in organs and bones). The trial ran in several stages. First, 170 participants went through a lead-in period of up to 78 weeks where they all received eliglustat. Then 131 of those participants moved into a main comparison period of up to 52 weeks, where they were randomly assigned to receive eliglustat either once a day (65 people) or twice a day (66 people). After that, 121 participants continued into a longer treatment phase, and 25 went on to an extended phase of up to 42 months. The main thing being measured in the comparison period was whether participants stayed "stable" — meaning their bone health, blood counts, and organ sizes did not worsen beyond set limits over 52 weeks. The reported data shows that in the main 52-week comparison period, 80.4% of participants in the once-daily group and 83.1% in the twice-daily group met the criteria for remaining stable. For the secondary measurements, haemoglobin levels (a measure of red blood cells) stayed broadly similar across both groups from the start to weeks 26 and 52, ranging between roughly 13.6 and 13.9 g/dL. Platelet counts (cells that help blood clot) also remained in a similar range throughout. Spleen volume, measured as a multiple of what is considered a normal spleen size, appeared to show small reductions from the starting point in both groups over the 52 weeks. Liver volume figures also remained relatively steady. A blood marker called chitotriosidase — which can be elevated in Gaucher disease — showed lower reported values at weeks 26 and 52 compared to the starting values in both groups. The reported data shows the numbers across both dosing groups were broadly comparable on these measures, though exact differences between groups were not broken down further in the submitted results. Some participants did not complete each phase of the trial, and the reasons were not detailed in the data provided here. These are the results as reported to ClinicalTrials.gov. They are not medical advice — always discuss what they mean for you with your doctor.
View full results on ClinicalTrials.gov ↗ · Read the linked publication on PubMed (PMID 29358012) ↗
These are the results as reported to ClinicalTrials.gov, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
Phase 3 Gaucher Disease Trial, Completed
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are 18 years of age or older.
- You have been diagnosed with Type 1 Gaucher disease, confirmed by a blood or tissue test showing low levels of a specific enzyme called acid β-glucosidase.
- Your blood test results at screening meet certain minimum levels: hemoglobin (a measure of red blood cells) of at least 9 g/dL, platelet count of at least 70,000/mm³, and your spleen and liver are not enlarged beyond specific limits (confirm with trial site).
- If you are a woman who could become pregnant, you have a negative pregnancy test before starting the study and agree to use an approved form of contraception throughout the study.
- You are willing to provide a blood sample for genetic testing related to Gaucher disease and how your body processes certain medications, if this has not already been done.
- You agree not to eat or drink grapefruit or grapefruit products for at least 72 hours before your first dose and for the entire duration of the study.
- You are willing and able to give signed consent before any study-related procedures begin.
Who may not be able to join:
- You are currently enrolled in, or eligible and able to join, one of two specific related clinical trials for the same condition (confirm with trial site).
- You have taken a medication called miglustat within the past 6 months.
- You have had part or all of your spleen removed within the past 3 years.
- You have taken certain other experimental treatments (pharmacological chaperones) within the past 6 months.
- You have signs of Gaucher disease affecting your nervous system (such as tremors, seizures, or memory problems) or your lungs (such as high blood pressure in the lungs).
- You depend on regular blood transfusions to stay well.
- You have a known deficiency of iron, vitamin B-12, or folate that is either untreated or has been treated for less than 3 months.
- You have a history of swollen veins in the esophagus, significant liver damage, or current liver blood test results that are more than twice the normal range (unless you have a specific harmless condition called Gilbert Syndrome).
- You have another significant medical condition — such as a serious heart, kidney, liver, lung, nerve, hormone, or mental health condition — that the study doctor believes would make participation unsafe.
- You have a significant heart condition, including a history of heart attack, signs of poor blood flow to the heart, heart failure, or certain abnormal heart rhythms or electrical conduction problems (confirm with trial site).
- You have tested positive for HIV, Hepatitis C, or Hepatitis B.
- You have taken another experimental drug (other than the study drug itself) within the past 30 days.
- You are scheduled for a planned hospital stay or elective surgery during the study period.
- You have had cancer (other than a common, easily treated skin cancer called basal cell carcinoma) within the past 5 years.
- You are currently pregnant or breastfeeding.
- You have taken any medication known to affect the heart's electrical activity (QTc interval prolongation) within the past 30 days, with limited exceptions for certain pre-medications used during enzyme replacement therapy infusions.
- You have recently started (within the past 30 days) taking certain medications that strongly affect how your body breaks down drugs, specifically strong inhibitors or inducers of liver enzymes called CYP2D6 or CYP3A4 (confirm with trial site).
- Depending on how your body naturally processes medications (based on your genetic profile), you may not be eligible if you are already regularly taking certain combinations of strong drug-interaction medications with no safe alternative available (confirm with trial site).
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Monitor, Genzyme, a Sanofi Company
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
3 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
PAP: Percentage of Participants Who Remained Stable for 52 Weeks During the PAP
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.