A Study of Rusfertide in Adults With Polycythemia Vera (PV)
Condition(s) studied
Investigational drug(s) / intervention(s)
Rusfertide: Rusfertide SC injection
Placebo: Rusfertide-matching placebo SC injection
Study 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 compared with placebo to lower the number of phlebotomies. A placebo looks like rusfertide but does not have any medicine in it.
Other aims are to learn how safe rusfertide is compared with placebo and to understand how well it works to reduce the need for phlebotomy and to keep hematocrit levels under control. The study also wants to learn how rusfertide moves through the body (pharmacokinetics) and how it affects the body (pharmacodynamics or PD).
This study has 4 parts that follow each other:
* In part 1a, participants will receive either rusfertide or placebo.
* In part 1b, all participants will receive rusfertide.
* In part 2, participants can continue to receive rusfertide if they benefit from treatment with rusfertide.
* Part 3 will include participants who finish Part 2 and still benefit from rusfertide.
During the study, participants will visit the study clinical several times at fixed timepoints.
Eligibility
Primary outcome measure(s)
- Part 1a: Percentage of Participants Who Achieved Response — From Weeks 20 to 32
Response was defined as absence of phlebotomy eligibility. Phlebotomy eligibility was defined as either: 1) A confirmed hematocrit (HCT) greater than or equal to (≥) 45% that was at least 3% higher than the baseline HCT, where confirmation was defined as 2 consecutive HCT assessments that were ≥ 45% and at least 3% higher than the baseline HCT, or 2) HCT ≥ 48%.
Trial sites (184)
| Facility | City | Region | Status |
|---|---|---|---|
| Infirmary Cancer Care | Mobile | Alabama | |
| Palo Verde Hematology-Oncology | Glendale | Arizona | |
| City of Hope Medical Center, Duarte | Duarte | California | |
| California Cancer Associates for Research and Excellence - Fresno | Fresno | California | |
| Marin Cancer Care | Greenbrae | California | |
| City of Hope Medical Center, Lenner | Irvine | California | |
| University of California, San Diego (UCSD) - Moores Cancer Center | La Jolla | California | |
| University of California, Los Angeles (UCLA) - Medical Center | Los Angeles | California | |
| Stanford University | Palo Alto | California | |
| Pomona Valley Hospital Medical Center | Pomona | California | |
| Sharp Memorial Hospital | San Diego | California | |
| University of California, San Francisco | San Francisco | California | |
| City of Hope Medical Center, Upland | Upland | California | |
| Rocky Mountain Cancer Centers | Aurora | Colorado | |
| University of Colorado - Aurora Cancer Center | Aurora | Colorado | |
| Rocky Mountain Cancer Centers - Boulder | Boulder | Colorado | |
| Rocky Mountain Cancer Centers | Colorado Springs | Colorado | |
| Rocky Mountain Cancer Centers | Colorado Springs | Colorado | |
| Rocky Mountain Cancer Centers | Denver | Colorado | |
| Rocky Mountain Cancer Centers | Lakewood | Colorado | |
| Rocky Mountain Cancer Centers | Littleton | Colorado | |
| Rocky Mountain Cancer Centers | Lone Tree | Colorado | |
| Yale University School of Medicine - Yale Cancer Center | New Haven | Connecticut | |
| BRCR Medical Center | Plantation | Florida | |
| BayCare Suncoast Medical Clinic - Hematology/Oncology | St. Petersburg | Florida | |
| Moffitt Cancer Center | Tampa | Florida | |
| Northwestern Memorial Hospital | Chicago | Illinois | |
| Northwestern Medicine Lake Forest Hospital | Lake Forest | Illinois | |
| Northwest Oncology and Hematology | Rolling Meadows | Illinois | |
| Orchard Healthcare Research Inc | Skokie | Illinois | |
| Fort Wayne Medical Oncology and Hematology | Fort Wayne | Indiana | |
| Mission Cancer & Blood | Des Moines | Iowa | |
| University of Iowa Hospitals and Clinics | Iowa City | Iowa | |
| University of Kansas Cancer Center | Kansas City | Kansas | |
| Pontchartrain Cancer Center | Covington | Louisiana | |
| Pontchartrain Cancer Center | Hammond | Louisiana | |
| American Oncology Partners of Maryland PA (Center for Cancer & Blood Disorders) | Bethesda | Maryland | |
| University of Michigan | Ann Arbor | Michigan | |
| Cancer Care Specialists | Reno | Nevada | |
| Memorial Sloan Kettering Cancer Center - BASKING RIDGE | Basking Ridge | New Jersey |
+ 144 more sites — see the full list on the official registry below.
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05210790 on ClinicalTrials.gov ↗ ← All trials in the UK