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Can a hormone mimic curb the need for bloodletting in a blood cancer?

NCT ID NCT07765602

Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Aug 14, 2026 · Last updated Aug 28, 2026 · Updated 1 time

Summary

This trial tests whether rusfertide, a synthetic version of the hormone hepcidin, can help people with polycythemia vera—a condition where the bone marrow makes too many red blood cells—by lowering iron availability and keeping red blood cell counts in check. The study enrolls Chinese adults with polycythemia vera who need regular blood removal (phlebotomy) to manage their condition. Participants receive rusfertide injections for up to a year, and researchers track how many phlebotomies are needed, how well hematocrit levels are controlled, and the drug's safety.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
rusfertide (TAK-121), a hepcidin mimetic given as an injection
What this could lead to
If successful, rusfertide could offer a new way to control red blood cell levels in polycythemia vera, potentially reducing or eliminating the need for regular blood removal.
What could go wrong
This is an early-phase, small trial (24 participants) focused on Chinese patients, so results may not apply broadly. The drug's safety and effectiveness are not yet established, and it may not work as hoped.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Mar 2027

An estimate. Start dates often move.

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

* Inclusion Criteria 1. Chinese male and female participants aged 18 years or older at the time of signing of informed consent. 2. 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. 3. Meets the revised 2016 World Health Organization criteria for the diagnosis of polycythemia vera (PV). 4. Participant with inadequate hematocrit control who meets all of the following criteria: 1. 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. 2. Documented hematocrit ≥45% within 3 months prior to trial intervention. 3. 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). 5. Complete blood count immediately prior to trial intervention administration must meet the following criteria: 1. Hematocrit less than (\<) 45%. 2. White blood cell count within the range of 4000/microliter (µL) to 20,000/µL (inclusive), and 3. Platelet count within the range of 100,000/µL to 1,000,000/µL (inclusive). 6. Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2. 7. Participants receiving cytoreductive therapy (CRT) at the time of trial intervention administration must be on a stable PV treatment regimen, including: 1. Hydroxyurea: at least 8 weeks. 2. JAK inhibitor: at least 8 weeks. 3. Interferon: at least 24 weeks. 8. Women of childbearing potential (WOCBP) agrees to use at least 1 form of highly effective contraception during the trial and for 30 days after the last dose of trial intervention. 9. 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. 10. A fertile male participant agrees to use a condom, preferably combined with at least 1 form of acceptable contraception for any WOCBP partner(s) during the trial and for 90 days after the last dose of trial intervention. 11. A male participant must agree not to donate sperm for the purpose of reproduction during the trial and for a minimum of 90 days after receiving the last dose of trial medication. * Exclusion Criteria 1. Clinically significant laboratory abnormalities at screening, including but not limited to: 1. 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. 2. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) ≥2.5×upper limit of normal (ULN). 3. Total bilirubin \>1.5×ULN. 2. Those who require phlebotomy at hematocrit levels \<45%. 3. Clinically significant thrombosis (for example, deep vein thrombosis or splenic vein thrombosis) within 2 months prior to trial intervention. 4. Active or chronic bleeding within 2 months prior to trial intervention. 5. Those who meet the criteria for post-PV myelofibrosis as defined by the International Working Group-Myeloproliferative Neoplasms Research and Treatment. 6. 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. 7. 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. 8. 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. 9. 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. 10. History of invasive malignancies within the last 5 years, except 1. Localized cured cancer (for example, prostate cancer and cervical cancer). 2. 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. 11. Pregnant females. 12. Those capable of breastfeeding but do not agree to forego breastfeeding from the first dose of trial intervention through 30 days after the last dose. 13. Active alcohol or drug addiction that would interfere with their ability to comply with trial requirements. 14. Those who do not complete at least 4 days of Myelofibrosis Symptom Assessment Form (MFSAF) v4.0 assessments within 1 week prior to trial intervention. 15. Receipt of an investigational agent within 2 months or 5 half-lives, whichever is longer, prior to trial intervention. 16. Receipt of busulfan or \^ 32 phosphorus within 7 months prior to screening. 17. Known hypersensitivity to rusfertide or any of its excipients. 18. Any lesion or mass detected by physical examination or imaging during screening that is suspicious for malignancy, unless it has been evaluated and confirmed to be nonmalignant.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

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