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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT07891533




Registration number
Help: Registration number
NCT07891533
Ethics application status
Help: Ethics application status
Date submitted
Help: Date submitted
17/10/2026
Date registered
Help: Date registered
23/10/2026
Date last updated
Help: Date last updated
23/10/2026

Titles & IDs
Public title
Phase 2 Study of Oral Epetraborole in Adults With Polycythemia Vera
Scientific 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
Additional trial identifiers [1] 0 0
EBO-PV-201
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Polycythemia Vera (SNOMED CT: 109992005) 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Bone
Cancer 0 0 0 0
Other cancer types
Cancer 0 0 0 0
Any cancer
Blood 0 0 0 0
Other blood disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

Intervention/exposure
Study type
Interventional
Description of the intervention or exposure
Treatment: Drugs - Epetraborole
Treatment: Drugs - Placebo

Experimental: Sentinel Cohort - Open Label Fixed Dose Epetraborole

Experimental: Part 1: Dose Selection Cohort - Open Label Epetraborole Dose Selection

Experimental: Part 2: Epetraborole (Double Blind) - Fixed Dose Epetraborole

Placebo comparator: Part 2: Placebo (Double Blind Randomized Withdrawal) - Placebo Control

Experimental: Part 3: Open Label Extension Cohort (Optional) - Open Label Epetraborole


Treatment: Drugs: Epetraborole
Oral tablets

Treatment: Drugs: Placebo
Oral tablets
Intervention code [1] 0 0
Treatment: Drugs
Description of the comparator or control
Control group

Outcomes
Primary outcome [1] 0 0
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
Timepoint [1] 0 0
Week 29 - Week 40

Eligibility
Key inclusion criteria
Inclusion Criteria

1. 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
2. Aged 18 to 75 years (inclusive) at Screening
3. 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
4. Documented positive JAK2 V617F (exon 14) or JAK2 exon 12 mutation
5. Hct <45% at Screening
6. Has records of all phlebotomies performed for at least 28 weeks (preferably up to 52 weeks) prior to Day 1
7. 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
8. Either receiving OR not receiving cytoreductive therapy for PV, as follows:

1. 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
2. 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
9. ECOG score of 0, 1, or 2
10. If FOCP

1. Must not be currently breastfeeding
2. 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
3. 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.
11. 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
12. Agrees to not donate sperm (males) or ova (females) from Screening through at least 1 week after the last dose of trial intervention
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
Exclusion Criteria

1. History of clinically significant arterial or venous thrombosis (eg, deep vein thrombosis or splenic vein thrombosis) within 24 weeks prior to Day 1
2. History of major bleeding events and/or a requirement for blood transfusion therapy due to bleeding in the 6 months prior to Day 1
3. Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (Tefferi, 2023)
4. Platelets >1000 × 109/L, WBC count >25 × 109/L; or presence of any blast cells in peripheral blood at Screening
5. Diagnosis of secondary polycythemia
6. Prior, concomitant, or planned therapy with ruxolitinib
7. 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
8. Prior exposure to epetraborole; drug intolerance or hypersensitivity to any compound from the same class as epetraborole
9. History of prior drug-induced pneumonitis
10. Severe hepatic impairment at Screening as evidenced by clinical signs of cirrhosis or end-stage hepatic disease
11. Liver disease or liver injury at Screening as evidenced by:

1. ALT or AST >3 × ULN
2. Total bilirubin >2 × ULN
12. eGFR <30 mL/min/1.73 m2 at Screening
13. Current skin malignancy or history of clinically significant skin cancer within 5 years prior to Day 1
14. Known history of:

1. Hepatitis B or C infection
2. HIV infection plus either an active AIDS-defining illness or a known CD4 count <200/mm3 within 12 months prior to Day 1
15. Known primary or secondary immunodeficiency defined as the following:

1. Primary immunodeficiency: rare, inherited genetic disorders, leading to frequent, severe, or unusual infections, autoimmune issues, and a higher cancer risk
2. 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.
16. 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:

1. Uncontrolled hypertension >160/100 mmHg
2. Uncontrolled diabetes mellitus (HbA1c level of =9%)
3. NYHA Class III/IV heart failure
4. 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
5. Symptomatic CVD or recent stroke within 12 months prior to Screening or active CVD with symptomatic transient events
6. Non-cutaneous malignancy requiring treatment
17. Any major surgical procedure requiring general anesthesia within 1 month prior to Day 1 or planned elective surgery during the trial
18. Prior or concomitant therapy within 4 weeks prior to Day 1, or expected during the trial, with the following:

1. Drugs known to consistently produce myelosuppression: oxazolidinone antibiotics , chloramphenicol, mTORC inhibitors
2. Drugs that can inhibit ADH: some H2-receptor antagonists (eg, cimetidine, famotidine, ranitidine), pyrazoles (fomepizole)
3. Drugs that inhibit ALDH: disulfiram, isoniazid, ketoconazole, certain cephalosporins , first-generation sulfonylureas

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/11/2026
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2030
Actual
Sample size
Target
75
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
501001 - Perth
Recruitment postcode(s) [1] 0 0
- Perth

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
AN2 Therapeutics, Inc
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Cameron Trenor
Address 0 0
Country 0 0
Phone 0 0
650-331-9090
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT07891533