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 involved people with Gaucher disease who were already being treated with a medicine called imiglucerase. The trial had two main phases. In the first phase (the Primary Analysis Period, lasting 52 weeks), 106 participants were switched to a tablet medicine called eliglustat and 54 continued on imiglucerase, for comparison. In the second, longer phase (up to 5 years), 152 participants received eliglustat. The trial was primarily measuring how many participants stayed "stable" — meaning their blood measures (haemoglobin levels and platelet counts) and organ sizes (spleen and liver) did not worsen beyond set thresholds. The reported data shows that during the 52-week period, 84.8% of participants taking eliglustat and 93.6% of participants taking imiglucerase met the criteria for remaining stable. During the longer 5-year phase, the percentage of eliglustat participants reported as remaining stable each year was 83.6% at year one, 75.65% at year two, 60.53% at year three, and 26.97% at year four. The trial also measured bone density using a scoring system that compares a person's bone strength to that of a healthy young adult (called a T-score and a Z-score). The reported data shows that changes in these bone scores from the start of the trial to week 52 were very small in both groups — generally less than a tenth of a point in either direction — across measurements of the spine and femur (thigh bone). 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 28167660) ↗
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 (and/or your parent or legal guardian) are willing and able to sign a consent form before any study activities begin
- You are at least 18 years old at the time of joining the study
- You have a confirmed diagnosis of Gaucher disease type 1
- You have been receiving enzyme replacement therapy (ERT) for at least 3 years, and for at least 6 of the 9 months before joining, your monthly dose was within a specific range (confirm with trial site)
- You have already met the standard treatment goals for Gaucher disease before joining the study
- If you are a woman who could become pregnant, you must have a negative pregnancy test before starting and must use an accepted form of birth control throughout the study
Who may not be able to join:
- You have had part or all of your spleen removed within the last 3 years
- You have taken a type of Gaucher disease medication called substrate reduction therapy in the last 6 months
- You have Gaucher disease type 2 or type 3, or there is any suspicion you may have type 3
- You have another significant health condition — such as heart, kidney, liver, lung, nerve, digestive, hormonal, or mental health problems — that could affect the study results or your safety (confirm with trial site)
- You have tested positive for HIV, Hepatitis C, or Hepatitis B
- You have taken part in another clinical trial or received an experimental treatment within the last 30 days
- You are currently pregnant or breastfeeding
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
1 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Percentage of Participants Who Remained Stable for 52 Weeks During the Primary Analysis Period; Percentage of Participants Who Remained Stable Annually for 4 Years During the LTTP
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.