A Study of Rusfertide in Adults With Polycythemia Vera in China

NCT07765602 | Not Yet Recruiting Phase 2

• 1 other identifier: TAK-121-2002
• Study Type: interventional
• Target: 24
• Locations: 0 countries
• Sites: N/A

Brief Summary

Polycythemia vera (PV) is a long-term condition in which the bone marrow makes too many red blood cells (RBCs). Bone marrow is the soft tissue inside the large bones where blood cells are made. When there are too many RBCs, called erythrocytosis, the blood can become thicker and move less easily through blood vessels and organs. This can raise the risk of blood clots (thrombosis) in veins or arteries. Sometimes, these clots can be life-threatening and may cause a stroke or heart attack. Treatment aims to keep the percentage of RBCs in the blood (hematocrit) in a safe range. For adults with PV, this means keeping the hematocrit below 45%. This helps lower the blood thickness and the risk of thrombosis. PV can also be linked to low iron levels (iron deficiency), because the body uses iron to make more RBCs. Many people with PV are treated with blood removal (phlebotomy) to lower RBC levels in the blood. This can worsen the iron deficiency, because each blood removal also takes iron out of the body. Rusfertide is a medicine that lowers the amount of iron available in the blood. It works like hepcidin, a natural hormone that helps control iron levels in the body. By limiting iron available for RBC production, rusfertide may help keep hematocrit below 45% and may improve symptoms. It may also reduce, or even remove, the need for phlebotomy in people. The main aim of this study is to check how well rusfertide works to lower the number of phlebotomies needed in Chinese adults with PV. Other aims are to understand how well rusfertide works to keep hematocrit levels under control and how safe it is. The study also wants to learn how rusfertide moves through the body (pharmacokinetics) and if it causes the body's defense system to react to it (immunogenicity). During the study, participants will receive rusfertide for up to 1 year (52 weeks) and will have to visit their study clinic several times. Blood samples will be taken several times during the study.

Conditions

Polycythemia Vera

Keywords

Drug Therapy

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants Achieving a Response Starting at Week 20 Through Week 32

  Response is defined as the absence of phlebotomy eligibility. Phlebotomy eligibility is defined as: A confirmed hematocrit ≥45%, which is ≥3% (absolute) higher than the baseline hematocrit or hematocrit ≥48%. Confirmation is defined as 2 consecutive hematocrit assessments that are ≥45% and at least 3% higher than the baseline hematocrit.

  Week 20 through Week 32

Secondary Outcomes (8)

• Change From Baseline in Hematocrit Over Time

  From Baseline to Week 32

• Percentage of Participants Maintaining Hematocrit <45% During Week 0 to Week 32 and Week 0 to Week 52

  From Baseline to Weeks 32 and 52

• Median Time to First Hematocrit ≥45% After Enrollment

  From Baseline to Week 52

• Percentage of Participants With at Least One Hematocrit ≥48% During Week 0 to Week 32

  From Baseline to Week 32

• Percentage of Participants With Treatment Emergent Adverse Events (TEAEs)

  From Baseline to Week 56

Study Arms (1)

Rusfertide

EXPERIMENTAL

Participants will receive rusfertide (TAK-121), subcutaneously (SC) once weekly for 52 weeks.

Drug: Rusfertide

Interventions

Rusfertide DRUG

Rusfertide injections administered SC.

Also known as: TAK-121, PTG-300

Rusfertide

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Chinese male and female participants aged 18 years or older at the time of signing of informed consent.
• Participant understands the trial procedures, is willing and able to adhere to trial requirements, and agrees to participate in the trial by providing written informed consent.
• Meets the revised 2016 World Health Organization criteria for the diagnosis of polycythemia vera (PV).
• Participant with inadequate hematocrit control who meets all of the following criteria:
• Greater than or equal to (≥) 3 times documented hematocrit ≥45 percent (%) within 28 weeks prior to trial intervention or ≥5 times documented hematocrit ≥ 45% hematocrit within 1 year prior to trial intervention.
• Documented hematocrit ≥45% within 3 months prior to trial intervention.
• Phlebotomy or erythrocytapheresis, if performed, must not have occurred within 6 days of trial intervention administration (the day of phlebotomy or erythrocytapheresis and the day of trial intervention administration should not be included in the 6-day count).
• Complete blood count immediately prior to trial intervention administration must meet the following criteria:
• Hematocrit less than (\<) 45%.
• White blood cell count within the range of 4000/microliter (µL) to 20,000/µL (inclusive), and
• Platelet count within the range of 100,000/µL to 1,000,000/µL (inclusive).
• Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2.
• Participants receiving cytoreductive therapy (CRT) at the time of trial intervention administration must be on a stable PV treatment regimen, including:
• Hydroxyurea: at least 8 weeks.
• JAK inhibitor: at least 8 weeks.

You may not qualify if:

• Clinically significant laboratory abnormalities at screening, including but not limited to:
• Estimated glomerular filtration rate (eGFR): \<15 milliliters per minute per 1.73 square meters (mL/min/1.73 m\^2) according to the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation 2021. Estimated Glomerular Filtration Rate (eGFR) =142 × (serum creatinine \[Scr\] / A)\^B × 0.9938\^age × (1.012 if female), where A and B are the following: Female: Scr less than equal to (≤) 0.7, A equals to (=) 0.7, B=-0.241; Scr greater than (\>) 0.7, A=0.7, B=-1.2. Male: Scr ≤0.9, A=0.9, B=-0.302; Scr \>0.9, A=0.9, B=-1.2. Creatinine unit conversion: milligrams per deciliter (mg/dL) =micromoles per liter (μmol/L)/88.4.
• Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5×upper limit of normal (ULN).
• Total bilirubin \>1.5×ULN.
• Those who require phlebotomy at hematocrit levels \<45%.
• Clinically significant thrombosis (for example, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to trial intervention.
• Active or chronic bleeding within 2 months prior to trial intervention.
• Those who meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment.
• In situ or Stage 1 squamous cell carcinoma of the skin, in situ or Stage 1 basal cell carcinoma of the skin, or in situ melanoma of the skin, as determined by the dermatologic examination required at screening unless the cancer is adequately treated prior to trial intervention.
• Any infection requiring systemic therapy within 1 month of dosing except controlled human immunodeficiency virus (HIV), hepatitis B, and hepatitis C. Prophylactic therapies are allowed.
• Any serious or unstable medical condition (for example, poorly controlled HIV infection) or uncontrolled psychiatric condition that, in the judgement of the investigator, would impair the participant's ability to participate in the trial.
• Major surgical procedure within 2 months prior to trial intervention, unless the participant has fully recovered from the surgery; or planned major elective surgery during the trial.
• History of invasive malignancies within the last 5 years, except
• Localized cured cancer (for example, prostate cancer and cervical cancer).
• Localized cured in situ or Stage 1 squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or in situ melanoma of the skin.

Sponsors & Collaborators

• Takeda: lead

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MeSH Terms

Conditions

Polycythemia Vera

Condition Hierarchy (Ancestors)

Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases; Myeloproliferative Disorders

Study Officials

• Study Director

  Takeda

  STUDY DIRECTOR

Central Study Contacts

Takeda Contact

CONTACT

+1-877-825-3327; medinfoUS@takeda.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2026
• First Posted: August 14, 2026
• Study Start (Estimated): March 1, 2027
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): August 1, 2028
• Last Updated: August 14, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.