A Study of Rusfertide in Japanese Adults With Polycythemia Vera

NCT07648030 | Not Yet Recruiting Phase 2

• 1 other identifier: TAK-121-2001
• Study Type: interventional
• Target: 9
• Locations: 0 countries
• Sites: N/A

Brief Summary

Polycythemia vera (PV) is a rare blood cancer in which the body makes too many red blood cells. This can make the blood thicker and may increase the risk of serious health problems such as blood clots. Many people with PV need regular phlebotomy, which is a procedure to remove blood, to help keep their hematocrit level under control. Hematocrit is the proportion of red blood cells in the blood. The main aim of this study is to evaluate whether rusfertide helps Japanese participants with PV keep their hematocrit under control and avoid the need for phlebotomy. All participants in this study will receive rusfertide. This study is open-label, which means both the participants and the study team will know what treatment is being given. Participants will be followed for up to about 244 weeks, including a screening period, a 52-week initial treatment period, a long-term extension period, and a 4-week safety follow-up period.

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 absence of phlebotomy eligibility. Phlebotomy eligibility is defined as either: A confirmed hematocrit 45% or more and that is at least 3% (absolute) higher than the baseline hematocrit. Confirmation is defined as 2 consecutive hematocrit assessments that are 45% or more and at least 3% higher than the baseline hematocrit (if it is observed at Week 32, confirmation is not required). OR a hematocrit 48% or more.

  Week 20, up to Week 32

Secondary Outcomes (9)

• Mean Number of Phlebotomies through Week 32

  Baseline, up to Week 32

• Percentage of Participants with All Hematocrit Values Less Than 45%

  Baseline, up to Week 32

• Median Time to First Hematocrit Value 45% Or More After Enrollment

  Baseline, up to Week 32

• Percentage of Participants with At Least One Hematocrit Value 48% Or More

  Baseline, up to Week 32

• Percentage of Participants who Maintain Absence of Phlebotomy Eligibility

  Baseline, up to Week 32

Study Arms (1)

Rusfertide

EXPERIMENTAL

Participants will receive rusfertide (TAK-121) injections, subcutaneously once a week for 52 weeks (Part 1) followed by 182 weeks long-term extension (Part 2)

Drug: Rusfertide

Interventions

Rusfertide DRUG

Rusfertide injections administered subcutaneously.

Also known as: TAK-121

Rusfertide

Eligibility Criteria

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

You may qualify if:

• Japanese male and female participants aged 18 years or older at the time of signing informed consent.
• Participant understands the trial procedures, is willing and able to adhere to trial requirements and agrees to participate in the trial by giving written informed consent.
• Meet revised 2016 WHO criteria for the diagnosis of PV.
• Phlebotomy requiring defined as ALL of the following:
• At least 3 phlebotomies due to inadequate hematocrit control in 28 weeks before trial intervention or at least 5 phlebotomies due to inadequate hematocrit control in 1 year before trial intervention, and
• Last phlebotomy due to inadequate hematocrit control within 3 months before trial intervention, and
• No phlebotomy within 6 days prior to trial intervention (do not include day of phlebotomy and day of trial intervention in the 6-day count).
• Note: Phlebotomies performed within an 8-day period will be counted as a single phlebotomy.
• Hematology test values at screening:
• Hematocrit \<45%
• WBC 4,000/µL to 20,000/µL (inclusive), and
• Platelets 100,000/µL to 1,000,000/µL (inclusive).
• ECOG performance status 0, 1 or 2.
• WOCBP agree to use at least 1 form of highly effective contraception during the trial and for 30 days after the last dose of trial intervention.
• A female participant must agree not to donate eggs (ova, oocytes) for the purposes of assisted reproduction during the trial and for a period of 30 days after receiving the last dose of trial medication.

You may not qualify if:

• Clinically meaningful laboratory abnormalities at Screening including, but not limited to:
• eGFR \<15 mL/min/1.73 m\^2 as determined by Japanese Society of Nephrology. Calculated by the correction formula for Japanese\*
• \*eGFR=194×(serum creatinine value)-1.094×(age)-0.287×(sex correction factor), Sex correction factor: 0.739 in female (Japanese Society of Nephrology,2023).
• ALT or AST ≥2.5×ULN
• Total bilirubin \>1.5×ULN. Note: Screening laboratory tests with abnormal results (if considered by the investigator to be transient and inconsistent with the participant's clinical condition) may be repeated within the screening window to confirm abnormal results. If results return to protocol acceptable limits within the screening period, the participant may enter the trial. Use local labs for all eligibility lab tests.
• Participants who require phlebotomy at hematocrit levels lower than 45%.
• Pregnant females will be ineligible to participate in this trial if, despite a negative pregnancy test, the investigator determines that based on interview, clinical assessment, or other relevant information that the individual may be in the very early stages of pregnancy.
• Is capable of breastfeeding but does not agree to forego breastfeeding from the first dose of trial intervention through 30 days after the last dose of trial intervention.
• Clinically significant thrombosis (eg, 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.
• Meets the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT).
• Any infection requiring systemic therapy within 1 month of dosing except controlled: HIV, hepatitis B, and hepatitis C. Prophylactic therapies are allowed.
• Any serious or unstable medical condition (eg, poorly controlled HIV infection) or uncontrolled psychiatric condition as judged by the investigator that 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 surgery or planned major elective surgery during the trial.
• History of invasive malignancies within the last 5 years, except

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: June 10, 2026
• First Posted: June 15, 2026
• Study Start (Estimated): June 23, 2026
• Primary Completion (Estimated): October 28, 2027
• Study Completion (Estimated): May 17, 2030
• Last Updated: June 15, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share