Phase 1 study for people with Essential Thrombocythaemia
Recruiting Phase 1
This trial at a glance
Where this trial is running
| Site | City | State or country | Status at this site |
|---|---|---|---|
| Macquarie University Hospital | Sydney | NSW | Recruiting |
| Royal Adelaide Hospital | Adelaide | SA | Recruiting |
| The Alfred Hospital | Melbourne | VIC | Recruiting |
| Linear Clinical Research | Nedlands | WA | Recruiting |
voxsanity.com.au · trial sites from public registries · registry record synced 11 October 2026 · not medical advice
Who can join
The official eligibility criteria
Registry text, reproduced exactly. These are the words the trial's sponsor published on ClinicalTrials.gov. They have not been edited, shortened or rewritten, and they are written for researchers rather than for patients.
Inclusion Criteria:
* Age 18 years or older at the time of signing the ICF
* ECOG performance status of 0 to 1 for the dose escalation (Part 1a) and 0 to 2 for the dose expansion (Part 1b)
* Documented CALR exon-9 mutation
* Confirmed diagnosis of MPN according to the 2022 ICC criteria:
* DIPSS+ intermediate-2/high-risk MF with prior JAKi, \<20% blasts, and measurable spleen
* High-risk ET with platelets \>450×10⁹/L
* Resistant, refractory, intolerant, or has lost response to ≥1 prior line of therapy for MF and ≥2 prior lines for ET (unless only a single standard-of-care option is approved in the participating country)
* No prior stem cell transplant and none planned within 6 months
* Minimum Laboratory Requirements:
* Platelet count ≥50 × 10⁹/L
* Absolute neutrophil count ≥1 × 10⁹/L
* International normalized ratio (INR) and activated partial thromboplastin time (aPTT) ≤1.5 × upper limit of normal (ULN), unless receiving vitamin K antagonists
* Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) \<2.5 × ULN
* Total bilirubin \<2 × ULN
* Estimated creatinine clearance \>45 or \>30 mL/min (depending on study part)
Exclusion Criteria:
* Major bleeding or thrombosis (e.g., stroke, DVT, PE) within the past 3 months
* Active or high-risk HBV, HCV, or HIV infection, or other chronic active infections requiring systemic treatment
* Active invasive cancer within the past 2 years, except certain early-stage or low-risk cancers (e.g., resected skin, cervical, thyroid, or prostate cancer)
* Pregnant or unwilling to avoid pregnancy or fathering a child during the study and for a defined period after the last dose.
Other protocol-defined Inclusion/Exclusion Criteria may apply.
Dates on the record
Results, publications and the official record
- No results are posted on the official record yet.
- No publications are listed on the official record yet.
- Official record and contacts on ClinicalTrials.gov – contact details for each site are kept there, and are not copied onto this page.
Sources and dates
- ClinicalTrials.gov – the record this page reformats
- ClinicalTrials.gov processing date: 6 August 2026.
- This page last checked by Voxsanity: 11 October 2026.
Voxsanity reformats data from public registries into this layout. It does not edit, interpret or add to what the registry published.
Not medical advice.
Individual trial records are refreshed on a rolling basis rather than all at once, so a record may be up to several weeks old. Each trial page and trial card shows the date its own record was last synced. Where a record has lagged, its status, sites and enrolment can differ from the current registry state in either direction, so check the registry link on the trial page before acting on them.
Letter for your doctor
A one-page summary of this trial to print or email to your GP. It carries the registry's own record of the trial and its eligibility criteria, and nothing Voxsanity has written about whether it suits you.