Nelfinavir to improve anemia in people with myelofibrosis by restoring iron delivery to the bone marrow

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a Phase 1/2 single‑center protocol testing repurposed nelfinavir as a way to restore iron delivery to the bone marrow and improve anemia in patients with myelofibrosis. Adults with WHO‑defined myelofibrosis, hemoglobin ≤10 g/dL, ferritin ≥100, and peripheral blasts <10% may receive nelfinavir while continuing most standard MPN therapies. The study will monitor hemoglobin, transfusion needs, and serum biomarkers of fibrosis alongside routine safety labs and symptom assessments. Participants must be amenable to regular blood draws, symptom reporting, and effective contraception during and for 60 days after treatment.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* ≥18 years of age
* Have a diagnosis of myelofibrosis (MF) according to the 2016 WHO criteria
* Has a hemoglobin ≤ 10gm/dL at screening
* Serum ferritin ≥ 100
* May continue on current MPN treatment, including aspirin, hydroxyurea, interferon-alpha, ruxolitinib, fedratinib, or anagrelide.
* Peripheral blast count \<10% during Screening.
* Free of other known active or metastatic malignancies other than localized skin cancer.
* Amenable to blood draws and symptom assessments.
* Agree to the use of contraceptives. Female subjects of childbearing potential and their male partners, or male subjects who have female partners of childbearing potential, should both use an effective contraception method during the study and continue to use contraception for 60 days after the last dose of study drug.

Exclusion Criteria:

* Eastern Cooperative Oncology Group (ECOG) questionnaire score of ≥3
* Currently pregnant or planning on being pregnant within the study period.
* Currently taking Momelotinib or Pacritinib (these agents are purported to reduce hepcidin).
* Currently taking any of the contraindicated medications to Nelfinavir listed in section 13.2
* Currently breastfeeding.
* Known uncontrolled active viral or bacterial infection.
* Known HIV+
* Significant impairment of major organ or hematopoietic function defined as

  1. Serum creatinine clearance less than 30 ml/min (eGFR).
  2. Bilirubin more than 1.5 mg/dl except for Gilbert's disease. ALT or AST more than 2X upper normal limit or has radiologic evidence of liver cirrhosis.
  3. Platelets \< 50 × 10\^9/L without transfusions
  4. ANC \< 0.75 × 10\^9/L without growth factors
* Known history of allergic reaction to nelfinavir.

Where it's running

• Chao Family Comprehensive Cancer Center, University of California, Irvine
  Orange, California, United States Recruiting

Study contacts

• Principal investigator: Angela Flesichman, MD, PhD — Chao Family Comprehensive Cancer Center
• Study coordinator: Chao Family Comprehensive Cancer Center University of California, Irvine
• Email: ucstudy@uci.edu
• Phone: 1-877-827-8839

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• National registry for people with myelofibrosis and anemia in Germany.

  This project will collect health, treatment, and quality-of-life information from people in Germany with primary or secondary myelofibrosis and anemia to see how their care and symptoms change over time.

  Recruiting iOMEDICO AG · Multiple Locations · NCT06976918

• Evaluating DISC-0974 for treating anemia in myelofibrosis patients

  This study tests if a new treatment called DISC-0974 can help people with myelofibrosis who have anemia feel better and improve their condition.

  Recruiting at 33 sites Phase 1/2 Disc Medicine, Inc · Gilbert, Arizona and 34 other locations · NCT05320198

• Elritercept vs. placebo for adults with myelofibrosis and anemia on ruxolitinib

  This study tests whether elritercept can improve anemia in adults with myelofibrosis who are taking ruxolitinib, compared with placebo.

  Recruiting at 11 sites Phase 3 Takeda · Gilbert, Arizona and 213 other locations · NCT07623161

• DISC-0974-201 for adults with IBD-related anemia

  This Phase 2 trial will test whether DISC-0974-201 helps adults with inflammatory bowel disease who have anemia driven by inflammation.

  Recruiting at 15 sites Phase 2 Disc Medicine, Inc · Birmingham, Alabama and 14 other locations · NCT07368972

• MPN PROGRESSion Registry — tracking symptoms, treatments, and disease progression in people with MPNs.

  Track long-term symptoms, treatments, and disease progression in adults with myeloproliferative neoplasms to see how these conditions evolve over time.

  Recruiting at 2 sites MPN Research Foundation · Chicago, Illinois and 1 other locations · NCT07362225

• Iron status at ICU discharge in adults with anemia after critical illness

  We are testing whether iron deficiency is common at ICU discharge and whether it relates to how well patients recover after leaving the ICU.

  Recruiting Università degli Studi di Ferrara · Brussels and 3 other locations · NCT07781540

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.