Polycythemia Vera (SNOMED CT: 109992005)Phase 2NCT07891533
Phase 2 Study of Oral Epetraborole in Adults With Polycythemia Vera
A Phase 2 study testing Epetraborole in people with Polycythemia Vera (SNOMED CT: 109992005), run by AN2 Therapeutics, Inc at 1 site in Australia.
Trial summary
This is a Phase 2 trial to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of oral epetraborole tablets for the treatment of adults with phlebotomy-dependent polycythemia vera (PV). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Polycythemia Vera (SNOMED CT: 109992005)
- What is being tested?
- Epetraborole
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 75 participants
- Where
- 1 siteAustralia
- Study dates
- Nov 2026to Jun 2030
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Willing and able to read and provide written informed consent prior to the performance of any trial-specific procedures and willing to follow the protocol requirements
- Aged 18 to 75 years (inclusive) at Screening
- A confirmed diagnosis of PV according to ICC (Thiele, 2023), including Hgb/Hct level, EPO level, and JAK2 mutation status assessed by analysis of peripheral blood sampling
- Documented positive JAK2 V617F (exon 14) or JAK2 exon 12 mutation
- Hct <45% at Screening
- Has records of all phlebotomies performed for at least 28 weeks (preferably up to 52 weeks) prior to Day 1
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- Phlebotomy-dependent, defined as at least 3 phlebotomies during the 28 weeks prior to Day 1, including a phlebotomy within 8 weeks prior to Screening
- Either receiving OR not receiving cytoreductive therapy for PV, as follows:
- Participants who are not currently receiving cytoreductive therapy must have either never received it or been discontinued from it for at least 24 weeks prior to Day 1 and any AEs due to cytoreductive therapy have either recovered or are ≤ Grade 1 prior to Day 1
- Participants currently receiving cytoreductive therapy with HU, interferon, or busulfan must have received such therapy for at least 24 weeks prior to Day 1 and have been on a stable or decreasing dose for at least 8 weeks prior to Day 1, and with no planned change in dose during the trial
- ECOG score of 0, 1, or 2
- If FOCP
- Must not be currently breastfeeding
- If sexually active with a male partner: Must be either sexually abstinent or agree to use at least 2 medically accepted, effective methods of birth control from Screening through at least 1 week after the last dose of trial intervention
- Must have a negative urine pregnancy test at Screening and on Day 1; the Day 1 urine test will be confirmed by a serum pregnancy test.
- Male participants who are sexually active with an FOCP must agree to use an effective barrier method of contraception from Screening through at least 1 week after the last dose of trial intervention
- Agrees to not donate sperm (males) or ova (females) from Screening through at least 1 week after the last dose of trial intervention
Who cannot join
- History of clinically significant arterial or venous thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 24 weeks prior to Day 1
- History of major bleeding events and/or a requirement for blood transfusion therapy due to bleeding in the 6 months prior to Day 1
- Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (Tefferi, 2023)
- Platelets >1000 × 109/L, WBC count >25 × 109/L; or presence of any blast cells in peripheral blood at Screening
- Diagnosis of secondary polycythemia
- Prior, concomitant, or planned therapy with ruxolitinib
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- Received any investigational drug within 6 weeks prior to Day 1 or 5 half-lives (whichever is longer) prior to Day 1 or has not recovered from adverse effects of prior administration of any investigational agent
- Prior exposure to epetraborole; drug intolerance or hypersensitivity to any compound from the same class as epetraborole
- History of prior drug-induced pneumonitis
- Severe hepatic impairment at Screening as evidenced by clinical signs of cirrhosis or end-stage hepatic disease
- Liver disease or liver injury at Screening as evidenced by:
- ALT or AST >3 × ULN
- Total bilirubin >2 × ULN
- eGFR <30 mL/min/1.73 m2 at Screening
- Current skin malignancy or history of clinically significant skin cancer within 5 years prior to Day 1
- Known history of:
- Hepatitis B or C infection
- HIV infection plus either an active AIDS-defining illness or a known CD4 count <200/mm3 within 12 months prior to Day 1
- Known primary or secondary immunodeficiency defined as the following:
- Primary immunodeficiency: rare, inherited genetic disorders, leading to frequent, severe, or unusual infections, autoimmune issues, and a higher cancer risk
- Secondary immunodeficiency: acquired deficiencies, including those from chemotherapy/immunosuppressive drugs, certain cancers (like leukemia/lymphoma), severe malnutrition, major burns/trauma, and other chronic conditions. These are common and result from environmental influences or diseases, unlike primary ones that are genetic.
- Any other condition, prior therapy, or clinically significant co-morbidity at Screening that would, in the Investigator's opinion, interfere with the participant's ability to participate in the entire trial, pose a risk to the participant, or constitute a potentially confounding medical condition, including the following:
- Uncontrolled hypertension >160/100 mmHg
- Uncontrolled diabetes mellitus (HbA1c level of ≥9%)
- NYHA Class III/IV heart failure
- Severe coronary artery disease, including unstable angina or myocardial infarction in the 12 months prior to Screening, or likely to require invasive procedure(s) to manage their CAD during the trial
- Symptomatic CVD or recent stroke within 12 months prior to Screening or active CVD with symptomatic transient events
- Non-cutaneous malignancy requiring treatment
- Any major surgical procedure requiring general anesthesia within 1 month prior to Day 1 or planned elective surgery during the trial
- Prior or concomitant therapy within 4 weeks prior to Day 1, or expected during the trial, with the following:
- Drugs known to consistently produce myelosuppression: oxazolidinone antibiotics , chloramphenicol, mTORC inhibitors
- Drugs that can inhibit ADH: some H2-receptor antagonists (eg, cimetidine, famotidine, ranitidine), pyrazoles (fomepizole)
- Drugs that inhibit ALDH: disulfiram, isoniazid, ketoconazole, certain cephalosporins , first-generation sulfonylureas
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
About this study
Official title: A Phase 2 Study of the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Oral Epetraborole Tablets for the Treatment of Adults With Phlebotomy-Dependent Polycythemia Vera
This is a Phase 2 trial to evaluate the efficacy, safety, tolerability, pharmacokinetics, and pharmacodynamics of oral epetraborole tablets for the treatment of adults with phlebotomy-dependent polycythemia vera (PV). The trial is designed to confirm the safety profile of oral epetraborole in the PV patient population and to identify 1 or more oral epetraborole doses for the treatment of phlebotomy-dependent PV. The study consists of an open-label Sentinel Cohort, an open-label Dose Selection Cohort (Part 1), followed by a double-blind, randomized, Placebo-Controlled Cohort (Part 2), and an optional Open-Label Extension (OLE) Cohort (Part 3)
- Conditions
- Polycythemia Vera (SNOMED CT: 109992005)
- Being tested
- Epetraborole, Placebo
- Keywords
- Hematologic disease, Bone marrow disease, Myeloproliferative disorder, Bone Marrow Neoplasms, Hematologic Neoplasms, Neoplasms by Site, Neoplasms, Hemic and Lymphatic Diseases, Polycythemia Vera, Epetraborole, Benzoxaborole, Boron, Phase 2[
- Medical subject headings
- Polycythemia Vera, Hematologic Diseases, Bone Marrow Diseases, Myeloproliferative Disorders, Bone Marrow Neoplasms, Hematologic Neoplasms, Neoplasms by Site, Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EpetraboroleDrug
Oral tablets
PlaceboDrug
Oral tablets
| Study group | Role | What they receive |
|---|---|---|
| Sentinel Cohort | Experimental | Open Label Fixed Dose Epetraborole Receives: Drug: Epetraborole |
| Part 1: Dose Selection Cohort | Experimental | Open Label Epetraborole Dose Selection Receives: Drug: Epetraborole |
| Part 2: Epetraborole (Double Blind) | Experimental | Fixed Dose Epetraborole Receives: Drug: Epetraborole |
| Part 2: Placebo (Double Blind Randomized Withdrawal) | Placebo comparator | Placebo Control Receives: Drug: Placebo |
| Part 3: Open Label Extension Cohort (Optional) | Experimental | Open Label Epetraborole Receives: Drug: Epetraborole |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 2: Proportion of Participants per Treatment Group Maintaining Clinical Success defined as hematocrit control, not undergoing therapeutic phlebotomy and completing 12 weeks of treatment in Part 2
Measured: Week 29 - Week 40
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2026
- Study startNov 2026 (estimated)
- Primary completionApr 2029 (estimated)
- Study completionJun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Polycythemia Vera (SNOMED CT: 109992005)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- AN2 Therapeutics, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07891533
- Sponsor's protocol ID
- EBO-PV-201
- Status last verified
- Oct 2026
- First submitted
- 17 Oct 2026
- Last updated
- 23 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No