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 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 spleen size and liver size (measured by MRI scan), as well as haemoglobin levels (a measure of red blood cells) and platelet counts (cells that help blood clot), across multiple time points. The reported data shows that spleen volume in the Naive 30 Units/kg group started at an average of 2,324 mL and was recorded at 1,708 mL, then 1,420 mL, and then 1,237 mL at later time points. For the Naive 60 Units/kg group, starting spleen volume was 2,120 mL, moving to 1,268 mL, 947 mL, and 761 mL over time. The Switchover group began at a lower average of 778 mL, with later readings of 884 mL, 609 mL, and 548 mL. For liver volume, the Naive 30 Units/kg group went from 3,000 mL to around 2,341 mL, the Naive 60 Units/kg group from 2,471 mL to 1,972 mL, and the Switchover group remained relatively stable, moving from 1,776 mL to 1,821 mL across time points. The reported data also shows that haemoglobin levels — which started at 12.5, 11.4, and 13.6 mg/dL respectively across the three groups — were recorded at 14.3, 14.1, and 13.3 mg/dL at the final time point. Platelet counts in the Naive 30 Units/kg group started at around 64,900 per cubic millimetre and were recorded at approximately 94,700 by the last time point; the Naive 60 Units/kg group went from about 69,000 to 144,400; and the Switchover group went from around 163,800 to 172,500. 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 27174694) ↗
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 have already successfully completed the related study called Protocol PB-06-001
- You are willing and able to give your written agreement (consent) to take part in this trial
Who may not be able to join:
- You are currently taking another experimental or investigational drug for any health condition
- You have severe nervous system symptoms such as complete loss of eye movement, obvious involuntary muscle jerking, or a history of seizures, which may be signs of a form of Gaucher disease that affects the brain and nervous system
- You are currently pregnant or breastfeeding
- You have any medical, emotional, behavioral, or mental health condition that the trial doctor believes would make it difficult for you to follow the study requirements (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
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
Spleen Volume
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.