What changed for Gaucher Disease
A plain English summary of what recently moved in the public data Voxsanity tracks for Gaucher Disease. Updated from government registries. Not medical advice.
How to read these changes: every figure on this page is the difference between two snapshots of what Voxsanity had tracked on each date. Voxsanity only began taking daily snapshots in July 2026 and is still ingesting the public registries, so a rise here is mostly our own coverage expanding rather than new research appearing. Read it as our tracked coverage growing, not as a claim about real-world research changing this fast: it overstates how much actually changed.
Comparing the two most recent snapshots, 27 July 2026 and 28 July 2026.
- Trials recruiting worldwide 24 to 25 (+1)
- Trials on record 154 to 155 (+1)
Newly subsidised on the PBS
No PBS-reviewed medicines are tracked for Gaucher Disease yet, so new PBS listings cannot be reported for it. This is a gap in what Voxsanity has mapped, not a statement that no medicines are subsidised — check the PBS directly.
Past updates
Tracking began in July 2026. Month on month changes will appear here once a second month of data has been recorded. Until then, the comparison above shows the most recent movement.
Recent trend
Weekly, most recent weeks (fills out to a monthly trend as history grows).
Recent trial results
Trials tracked for Gaucher Disease that have reported results to a public registry.
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
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NCT05529992 · results posted 26 February 2025
According to the results reported on ClinicalTrials.gov, this trial (NCT05529992) enrolled 20 people who received a treatment called VPRIV. Nineteen of the 20 participants completed the study, and one did not. The trial was primarily measuring how many participants experienced a serious unexpected medical event (called a serious treatment-emergent adverse event, or serious TEAE) — that is, an unwanted medical occurrence that happened after starting the treatment and was serious enough to cause death, be life-threatening, require hospitalisation, or result in lasting disability, among other cri...
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NCT03625882 · results posted 10 January 2025
According to the results reported on ClinicalTrials.gov, this trial enrolled 63 participants who received intravenous infusions of a medicine called velaglucerase alfa. Sixty participants completed the study, while three did not. The trial was measuring how many participants experienced unwanted medical events (called adverse events) after starting treatment, and also tracked several body measurements — including red blood cell levels (haemoglobin), platelet counts (tiny blood cells that help with clotting), liver and spleen size, and bone density — to see how these changed over the course of...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT04145037 · results posted 18 January 2024
According to the results reported on ClinicalTrials.gov, this trial (NCT04145037) looked at a gene therapy called AVR-RD-02, which was being studied in people with Gaucher disease — a condition where the body cannot properly break down certain fatty substances. The trial enrolled two groups: people who had been stable on existing enzyme replacement therapy ("Switch Stable") and people who had never received treatment ("Treatment-naïve"). Eight people started in the Switch Stable group and none started in the Treatment-naïve group. Of those eight, six went through the cell collection process, f...
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NCT04718779 · results posted 21 December 2023
According to the results reported on ClinicalTrials.gov, this trial (NCT04718779) enrolled a very small number of participants — just 2 people in a prospective group (followed forward in time) and 2 people in a retrospective group (reviewed using past medical records), giving a total of 4 participants. All 4 completed the study. The trial was measuring whether certain key blood and organ markers stayed stable over 12 months in people with Gaucher disease who were receiving treatment. Specifically, it tracked red blood cell levels (haemoglobin), platelet counts, and the size of the liver and sp...
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NCT00813865 · results posted 15 August 2018
According to the results reported on ClinicalTrials.gov, this trial (NCT00813865) enrolled 8 participants, all of whom received a medicine called afegostat tartrate. The trial was measuring the number of participants who experienced severe side effects (called severe treatment-emergent adverse events — meaning serious unwanted health events that appeared or got worse after starting the study drug), as well as changes in the size of the spleen and liver, measured using MRI scans. Only 1 of the 8 participants completed the study, while 7 did not complete it; the reasons for this are not detailed...
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NCT02770625 · results posted 12 July 2017
According to the results reported on ClinicalTrials.gov, this trial (NCT02770625) involved 8 participants, all of whom completed the study. All participants received the treatment being studied, called ISU302. The trial was measuring a range of things in the blood, liver, and spleen — including levels of haemoglobin (a protein in red blood cells that carries oxygen), platelet counts (platelets are tiny blood cells that help with clotting), spleen and liver size, and two substances in the blood called angiotensin-converting enzyme and chitotriosidase, which can act as markers of certain conditi...
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NCT01074944 · results posted 2 December 2016
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 long...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01842841 · results posted 14 December 2015
According to the results reported on ClinicalTrials.gov, this trial (NCT01842841) enrolled 5 participants who were already receiving velaglucerase alfa (VPRIV®), a treatment for Gaucher disease, in an earlier related study. All 5 participants completed the trial — none dropped out. The trial was measuring a range of things, including any unwanted health events (called adverse events), whether participants developed antibodies against the study drug, laboratory test results, and changes in two blood measurements: haemoglobin (a protein in red blood cells) and platelet count (tiny blood cells th...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01132690 · results posted 11 November 2014
According to the results reported on ClinicalTrials.gov, this trial enrolled 11 people in total — 6 received a lower dose of the study treatment (30 Units per kilogram of body weight) and 5 received a higher dose (60 Units per kilogram). All 11 participants completed the trial, with no drop-outs recorded. The trial was measuring several things in the blood and organs, including haemoglobin levels (a measure of red blood cells), platelet counts (tiny blood cells involved in clotting), the size of the spleen and liver (organs often affected in the condition being studied), and levels of two biol...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00943111 · results posted 4 September 2014
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 an...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00891202 · results posted 3 September 2014
According to the results reported on ClinicalTrials.gov, this trial involved 40 adults who took part in the main comparison phase (called the Primary Analysis Period, or PAP), with 20 receiving the study drug eliglustat and 20 receiving a placebo (a dummy treatment with no active ingredient). The trial was looking at whether eliglustat had any effect on several body measurements in people with Gaucher disease, including the size of the spleen and liver, haemoglobin levels (a measure of red blood cells in the blood), and platelet counts (tiny blood cells involved in clotting). After this phase...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00705939 · results posted 15 July 2014
According to the results reported on ClinicalTrials.gov, this trial enrolled 44 people across three groups. Twelve participants received a lower dose of the study treatment (30 units per kilogram of body weight) and had not previously been treated for their condition — this group is called "Naive 30 Units/kg." Fourteen participants received a higher dose (60 units per kilogram) and were also treatment-naïve — the "Naive 60 Units/kg" group. The remaining 18 participants had previously been on a different treatment and switched to the study treatment — the "Switchover" group. The trial tracked s...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00391625 · results posted 23 June 2014
According to the results reported on ClinicalTrials.gov, this trial enrolled 10 participants, all of whom received a treatment referred to as GA-GCB. The trial ran over a long period — up to 84 months (seven years) — and was designed to track two things: any unwanted health events (called adverse events) that occurred during that time, and changes in certain body measurements such as blood counts, liver size, and spleen size. The reported data shows that all 10 participants experienced at least one adverse event over the course of the trial. One participant experienced an adverse event consid...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00712348 · results posted 5 June 2014
According to the results reported on ClinicalTrials.gov, this trial enrolled 31 people who received a treatment called taliglucerase alfa. Thirty of the 31 participants completed the study, with one person not finishing. The trial was measuring several things in people's blood and organs, with the main focus being haemoglobin levels — haemoglobin is the protein in red blood cells that carries oxygen around the body. The study also tracked platelet counts (tiny blood cells that help with clotting), as well as the size of the spleen and liver, both of which can be affected in the condition being...
Read the full trial page · View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00635427 · results posted 28 January 2014
According to the results reported on ClinicalTrials.gov, this trial (NCT00635427) was a long-term follow-on study looking at a medicine called velaglucerase alfa (VPRIV), which is given by drip infusion to people with Gaucher disease — a condition where a fatty substance builds up in the body. A total of 95 participants were enrolled across five groups, each coming from a different earlier ("parent") study. The groups differed by the dose of VPRIV they had previously received, or whether some had previously been on a different medicine called imiglucerase. The trial's main focus was on trackin...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT01427517 · results posted 2 August 2013
According to the results reported on ClinicalTrials.gov, this trial enrolled nine people in total — three with Parkinson's disease (referred to as PD), three with Gaucher disease (referred to as GD), and three people without either condition who acted as a comparison group. All nine participants completed the study. The trial was measuring whether levels of a substance called glutathione (GSH) — a naturally occurring compound found in the brain — changed after participants received N-acetyl cysteine (NAC), a supplement. GSH levels were measured in the brain before and after NAC was given, roug...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00376168 · results posted 26 July 2012
According to the results reported on ClinicalTrials.gov, this trial looked at a medicine called ELELYSO given at two different doses — 30 units per kilogram of body weight, and 60 units per kilogram of body weight. A total of 32 adults took part, 16 in each dose group. The trial was measuring changes in spleen size (the spleen is an organ in the abdomen that can become enlarged in certain conditions), liver size, red blood cell levels, and platelet counts (platelets are tiny blood cells that help with clotting) over a period of 9 months. The reported data shows that for the main measurement —...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00430625 · results posted 10 September 2010
According to the results reported on ClinicalTrials.gov, this trial involved 25 adults or children with Gaucher disease (a rare condition where a fatty substance builds up in the body's organs). Participants were split into two groups: 13 people received a lower dose of the study medicine, VPRIV® (velaglucerase alfa), at 45 units per kilogram of body weight, and 12 people received a higher dose at 60 units per kilogram. Both groups received the medicine through a drip into a vein every two weeks for 12 months. The trial was measuring changes in several body markers over that period, including...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
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NCT00478647 · results posted 2 September 2010
According to the results reported on ClinicalTrials.gov, this trial enrolled 40 people, all of whom received a medicine called velaglucerase alfa. Thirty-eight participants completed the study, while two did not finish. The trial's main focus was on tracking unwanted or unexpected health events (called adverse events) experienced by participants over the course of the study, as well as monitoring things like heart tracings, blood tests, and whether participants developed antibodies to the medicine. The reported data shows that, out of 40 participants, 34 experienced at least one adverse event...
View reported results on ClinicalTrials.gov ↗ · Linked publication on PubMed ↗
These are the results as reported to the registry, not medical advice. Verify independently with the trial site and discuss what they mean for you with your doctor.
What is coming
AI generated pipeline 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 NIH funding data, public investment in Gaucher Disease research is at a moderate level and has been rising over time. This upward trend is an encouraging sign that researchers and funding bodies see value in continuing to investigate this condition. For patients, a growing research base generally means that scientific understanding is expanding, which can lay the groundwork for future...
See the full Gaucher Disease page
Not medical advice. Voxsanity republishes public government health data in plain English. These figures describe research and registry activity, not the suitability of any treatment for any person. Trial status can change; always verify current status directly with the trial site and confirm Australian availability with your doctor.