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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07891533
- Acronym / sponsor ID
- EBO-PV-201
- Conditions
- Polycythemia Vera (SNOMED CT: 109992005)
- Keywords
- Hematologic disease, Bone marrow disease, Myeloproliferative disorder, Bone Marrow Neoplasms, Hematologic Neoplasms, Neoplasms by Site, Neoplasms, Hemic and Lymphatic Diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- AN2 Therapeutics, IncIndustry (pharma, biotech, devices)
- Study start
- Nov 2026 (estimated)
- Primary completion
- Apr 2029 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 23 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Sentinel Cohort | Experimental | Drug: EpetraboroleOpen Label Fixed Dose Epetraborole |
| Part 1: Dose Selection Cohort | Experimental | Drug: EpetraboroleOpen Label Epetraborole Dose Selection |
| Part 2: Epetraborole (Double Blind) | Experimental | Drug: EpetraboroleFixed Dose Epetraborole |
| Part 2: Placebo (Double Blind Randomized Withdrawal) | Placebo comparator | Drug: PlaceboPlacebo Control |
| Part 3: Open Label Extension Cohort (Optional) | Experimental | Drug: EpetraboroleOpen Label Epetraborole |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- 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
Key exclusion criteria (33 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EpetraboroleOral tablets | Drug | Part 1: Dose Selection Cohort, Part 2: Epetraborole (Double Blind), Part 3: Open Label Extension Cohort (Optional), Sentinel Cohort |
| PlaceboOral tablets | Drug | Part 2: Placebo (Double Blind Randomized Withdrawal) |
Primary outcome
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
Time frame: Week 29 - Week 40
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2026.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Epetraborole in people with Polycythemia Vera (SNOMED CT: 109992005), run by AN2 Therapeutics, Inc at 1 site in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 17 inclusion and 33 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 23 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cameron Trenor (650-331-9090 ext. 700, trials@an2therapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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 (time frame: Week 29 - Week 40). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Oct 2026.