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Polycythemia VeraPhase 1NCT07012109

Study of BEBT-507 Injection in Subjects With Polycythemia Vera (PV)

A Phase 1 study testing BEBT-507 Injection in people with Polycythemia Vera, run by BeBetter Med Inc at 1 site in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

This is a multicenter, open-label Phase I clinical trial of BEBT-507 in subjects with polycythemia vera(PV). Phase Ia is a single-agent dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), preliminary efficacy, and pharmacodynamics of BEBT-507 in subjects with PV . Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Polycythemia Vera
What is being tested?
BEBT-507 Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
90 participants
Where
1 siteChina
Study dates
17 Jun 2025to 31 Jul 2028
Phase
Phase 1
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

  • Male and female subjects aged 18-75 years (inclusive);
  • Subjects diagnosed with polycythemia vera (PV) according to the 2016 world health organization (WHO) criteria;
  • Hydroxyurea-resistant/intolerant and/or interferon α-resistant/intolerant:
  • 1)Hydroxyurea-resistant or -intolerant Must meet the definition of hydroxyurea (HU) resistance or intolerance in the 2024 chinese society of clinical oncology (CSCO) Guidelines for the diagnosis and treatment of malignant hematological diseases and satisfy at least one of the following criteria:a) Resistance: Despite ≥3 months of HU treatment at a dose of ≥2 g/d, phlebotomy is still required to maintain HCT <45%; after ≥3 months of HU treatment at a dose of ≥2 g/d, bone marrow proliferation remains uncontrolled (e.g., platelets >400×10⁹/L and WBC >10×10⁹/L); after ≥3 months of HU treatment at a dose of ≥2 g/d, a palpable massive splenomegaly fails to reduce by >50% or splenomegaly-related clinical symptoms do not fully resolve;b) Intolerance: At the minimum hydroxyurea (HU) dose required to achieve a complete or partial clinical hematologic response for the disease, absolute neutrophil count (ANC) <1.0×10⁹/L or PLT count <100×10⁹/L or Hemoglobin (HGB) <100 g/L occurs; during HU treatment at any dose, lower extremity ulcers or other intolerable non-hematological toxicities emerge, such as skin and mucous membrane manifestations (skin, teeth, or nail darkening; oral ulcers, mucositis; skin ulcers, rash, etc.), gastrointestinal symptoms (nausea, anorexia, indigestion, vomiting, abdominal pain, constipation, etc.), pneumonia, fever, etc.; 2)Interferon α-resistant or -intolerant Must satisfy at least one of the following criteria:a) Resistance: After achieving at least 12 weeks of interferon α therapy and a dose of at least 25×10⁶ U/week (or the subject's maximum tolerated dose if it is less than 25×10⁶ U/week), phlebotomy is still required to maintain HCT <45%, or PLT >400×10⁹/L and WBC >10×10⁹/L, or palpable splenomegaly (starting >10 cm from the left costal margin) fails to reduce by >50%;b) Intolerance: At the minimum interferon α dose required for complete or partial clinical hematologic remission, ANC <1.0×10⁹ or PLT <100×10⁹ or hemoglobin <100 g/L (<10 g/dL) occurs, or depression, influenza-like symptoms, neuropsychiatric symptoms, autoimmune issues, or other unacceptable non-hematological toxicities related to interferon-alpha (IFN-α) emerge, defined as common terminology criteria for adverse events (CTCAE) V5.0 grade 3-4 events, or CTCAE V5.0 grade 2 events lasting over 1 week, or permanent discontinuation of interferon α, or interruption of interferon α until toxicity resolves, or hospitalization due to interferon α toxicity.
  • 4.The subject has intact skin at the injection site, and the investigator deems it is suitable for subcutaneous injection; 5.Eastern cooperative oncology group (ECOG) performance status score is 0, 1, or 2; 6.The subject has undergone bone marrow biopsy within 12 months prior to enrollment; 7.The subject or the subject's legal guardian has signed a written informed consent, and the subject is able to comply with the study requirements.

Who cannot join

  • A history of intolerance to oligonucleotides, N-Acetylgalactosamine (GalNAc), or excipient components, or a history of intolerance to subcutaneous injections.
  • Clinically significant thrombosis (e.g., deep vein thrombosis or splenic vein thrombosis) within 12 weeks before screening.
  • Major bleeding event or blood transfusion for bleeding within 6 months before screening.
  • Meets the international working group for myeloproliferative neoplasms research and treatment criteria for post-PV myelofibrosis.
  • Received any investigational drug within 6 weeks before the first study drug administration, or not recovered from the effects of prior investigational drugs.
  • Received any clinical studies or marketed products of GalNAc-targeted drugs within 48 weeks before the first study drug administration.
Show all 14Show fewer
  • Laboratory abnormalities:a) Liver function tests: alanine aminotransferase (ALT) or aspartate aminotransferase (AST)>2.0×Upper Limit of Normal (ULN), total bilirubin(TBIL)>1.5×ULN;b) Renal function tests: estimated glomerular filtration rate (eGFR)<60 mL/min/1.73㎡ (Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation);c) Blood routine tests: platelet count>1000×10⁹/L, WBC count>25×10⁹/L;d) Coagulation tests: international normalized ratio (INR)>1.5×ULN;e) Others: presence of peripheral blood blasts.
  • Subjects with symptomatic splenomegaly (e.g., splenic infarction, left upper quadrant fullness or pain, early satiety, portal hypertension).
  • Due to potential genotoxicity, mutagenicity, and teratogenicity of the study drug, exclude:a) Men and women planning to conceive within 5 years without prior in vitro sperm/ovum preservation;b) Pregnant or breastfeeding women;c) Women within 2 years of menopause unwilling to use acceptable contraception until 6 months after the last study drug dose.
  • Presence of other active malignancies requiring treatment that may interfere with the study.
  • Comorbid conditions:a) Poorly controlled diabetes (random glucose≥11.1mmol/L or HbA1c≥8.5% despite antidiabetic treatment) assessed by the investigator;b) Severe pulmonary disease (CTCAE V5.0 grade III-IV);c) Severe cardiac disease (defined as any of the following:Left ventricular ejection fraction (LVEF) <50% as determined by multigated acquisition scan (MUGA) or Echocardiogram (ECHO);QT interval corrected using the Fridericia method (QTcF) interval >450 ms for males or >470 ms for females;Unstable angina;Symptomatic pericarditis;Myocardial infarction within the past 6 months with persistent elevation of cardiac enzymes or evidence of persistent regional wall abnormalities on LVEF assessment;History of congestive heart failure (new york heart association functional class III-IV) or cardiomyopathy;d) Significant renal or hepatic impairment;e) Poorly controlled active hepatitis B or C, or other diseases with clinically significant active infections, including hepatitis B (HBV), hepatitis C (HCV), or syphilis. Active HBV is defined as Hepatitis B Surface Antigen (HBsAg) or Hepatitis B e Antigen (HBeAg) positivity with HBV DNA≥2000 IU/ml (10⁴ copies/ml). If HBV DNA is below this level, antiviral therapy is required until one year post - study. Active HCV is defined as HCV RNA above the assay's upper limit. For syphilis, a positive non-treponemal test requires confirmation with a treponemal antibody test. If the latter is negative and the investigator confirms past infection with syphilitic cure, the subject can be included;f) Known human immunodeficiency virus (HIV) positivity or primary immunodeficiency;g) Deemed unsuitable for study participation due to a history of psychosis, family history of psychiatric illness, or mood disorders, as determined by the investigator or psychiatrist;h) Uncontrolled hypertension (systolic BP≥180mmHg and/or diastolic BP≥110mmHg);i) Severe medical conditions with a risk of major bleeding or history of major bleeding.
  • Concomitant use of drugs that may prolong the QT interval or cause torsades de pointes.
  • History of alcohol or drug abuse within the past year.
  • Subjects deemed unsuitable for this treatment regimen by the investigator.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Kegang Jiang, Master contact

+86-18664786382

kjiang@bebettermed.com

China1 site, 1 recruiting
  • Blood Diseases Hospital, Chinese Academy of Medical Sciences

    Tianjin, Tianjin Municipality, 311100

    Meijing Feng, Bachelor, +86-18920391612, meijing.feng@bebettermed.com

    Recruiting

Other trials in China

About this study

Official title: A Multicenter, Open-Label Phase I Clinical Trial of BEBT-507 Injection in Subjects With Polycythemia Vera (PV)

This is a multicenter, open-label Phase I clinical trial of BEBT-507 in subjects with polycythemia vera(PV). Phase Ia is a single-agent dose-escalation study designed to evaluate the safety, tolerability, pharmacokinetics (PK), preliminary efficacy, and pharmacodynamics of BEBT-507 in subjects with PV . Based on the results of Phase Ia, two doses will be selected for further evaluation in Phase Ib to assess the efficacy, safety, and PK profile of BEBT-507 in subjects with PV , and to recommend a dose for Phase III clinical trials.

Read the detailed description

Phase Ia Study:Phase Ia plans to set up 5 dose groups (Cohorts A1-A5), with 3-6 subjects planned for enrollment in each dose group. The 5 dose groups are 1.25 mg/kg, 2.5mg/kg, 5mg/kg, 10mg/kg, and 15mg/kg, respectively. Subcutaneous injection is administered every 12 weeks, for a total of 2 doses. A "3+3" dose-escalation design will be used. If no dose-limiting toxicity (DLT) is observed in Cohort A5, further dose escalation will be determined by investigators and sponsors based on PK and safety data. Additional dose groups can be added if necessary.

Phase Ib Study:Based on the results of the Phase Ia study, two doses will be selected for the phase Ib study. Each dose cohort will enroll approximately 10-30 eligible subjects.

Being tested
BEBT-507 Injection
Keywords
BEBT-507, Safety, Pharmacokinetics, Efficacy
Medical subject headings
Polycythemia Vera

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BEBT-507 InjectionDrug

    The initial dose of BEBT-507 injection is 1.25mg/kg, administered subcutaneously at 1.25mg/kg, 2.5mg/kg, 5mg/kg, 10mg/kg or 15mg/kg every 12 weeks for two doses in total.

Study groups in NCT07012109
Study groupRoleWhat they receive
Phase Ia-Dose EscalationExperimentalPhase Ia plans to set up 5 dose groups. If no DLT is observed in cohort A5, further dose escalation will be determined by investigators and sponsors based on PK and safety data. Additional dose groups can be added if necessary.
Receives: Drug: BEBT-507 Injection
Phase Ib-Dose ExpansionExperimentalBased on the results of the phase Ia study, two doses will be selected for the phase Ib study.
Receives: Drug: BEBT-507 Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. MTD

    Maximum Tolerated Dose

    Measured: Up to 52 weeks

  2. DLT

    Dose-Limiting Toxicity

    Measured: Up to 52 weeks

  3. Proportion of Subjects With Hematocrit (HCT) < 45%

    The proportion of subjects with HCT<45% following at least 21 days without or with specified therapies (phlebotomy or erythrocytapheresis).

    Measured: Up to 100 weeks

Secondary outcomes (18)
  1. AUC0-∞

    The area under the plasma concentration-time curve from time zero to infinity.

    Measured: Pre-dose to 168h post-dose on day 1; Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  2. AUC0-last

    The area under the plasma concentration-time curve from administration to the last measurable concentration time point.

    Measured: Pre-dose to 168h post-dose on day 1; Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  3. Cmax

    The maximum plasma drug concentration

    Measured: Pre-dose to 168h post-dose on day 1; Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  4. Tmax

    The time to reach maximum plasma drug concentration

    Measured: Pre-dose to 168h post-dose on day 1; Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  5. t1/2

    The time for plasma drug concentration to halve

    Measured: Pre-dose to 168h post-dose on day 1;Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  6. CL/F

    The ratio of an orally administered drug's absorbed amount to the administered dose

    Measured: Pre-dose to 168h post-dose on day 1; Pre-dose to 168h post-dose on day 85 (day 85±3 days).

  7. Changes in White Blood Cell (WBC)

    Changes in WBC over time relative to baseline.

    Measured: Up to 100 weeks

  8. Changes in Platelet (PLT)

    Changes in PLT over time relative to baseline.

    Measured: Up to 100 weeks

  9. Time to First HCT Response

    Time to first HCT response (days from study drug administration to HCT <45% without phlebotomy or erythrocytapheresis during this period).

    Measured: Up to 100 weeks

  10. Duration of Peripheral Blood HCT Response

    Duration of peripheral blood HCT response (days from achieving HCT <45% after study drug administration to HCT ≥45% without phlebotomy or erythrocytapheresis during this period).

    Measured: Up to 100 weeks

  11. Changes in Serum Iron

    Serum Iron changes at each dose level.

    Measured: Up to 100 weeks

  12. Changes in Hepcidin

    Hepcidin changes at each dose level.

    Measured: Up to 100 weeks

  13. Changes in Ferritin

    Ferritin changes at each dose level.

    Measured: Up to 100 weeks

  14. Changes in Transferrin Saturation

    Transferrin saturation changes at each dose level.

    Measured: Up to 100 weeks

  15. Changes in Spleen Volume Size

    Mean and percentage changes from baseline in spleen volume.

    Measured: Up to 100 weeks

  16. Symptom Improvement

    Symptom changes are assessed using the Myeloproliferative Neoplasms 10 (MPN10) questionnaire, with symptoms rated on a scale from 1 to 10 (0 if absent), where 1 indicates the mildest severity and 10 the most severe.

    Measured: Up to 100 weeks

  17. Thrombotic and Hemorrhagic Events

    The proportion of subjects without thrombotic or hemorrhagic events.

    Measured: Up to 100 weeks

  18. Occurrence of Adverse Events (AEs)

    Occurrence of AEs will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events Version 5.0 (NCI CTCAE V5.0).

    Measured: Up to 36 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Jun 2025
  2. Study start17 Jun 2025
  3. Primary completion31 Jan 2028 (estimated)
  4. Study completion31 Jul 2028 (estimated)

Resources

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 1
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Sequential
Masking
None (open label)
Enrolment
90 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07012109
Sponsor's protocol ID
GBMT-507-P01
Status last verified
Aug 2026
First submitted
29 May 2025
Last updated
18 Aug 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing BEBT-507 Injection in people with Polycythemia Vera, run by BeBetter Med Inc at 1 site in China.

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 5 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 18 Aug 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 China. 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. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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 Kegang Jiang, Master (+86-18664786382, kjiang@bebettermed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

MTD (time frame: Up to 52 weeks). The study lists 3 primary outcomes in all. 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 18 Aug 2026.

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