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

Treating Anemia in Myelofibrosis With Repurposed Drugs (Nelfinavir) That Restore Iron Delivery to the Bone Marrow

This trial tests whether the antiviral drug nelfinavir can help people with myelofibrosis and anemia by raising hemoglobin and lowering blood markers of fibrosis.

Quick facts

• Phase: Phase1; Phase2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: University of California, Irvine (Academic / other)
• Drugs / interventions: ruxolitinib, fedratinib, Momelotinib, Pacritinib
• Locations: 1 site (Orange, California)
• Trial ID: NCT07281781 on ClinicalTrials.gov

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.

Who should consider this trial

Good fit: Adults (≥18) with WHO‑diagnosed myelofibrosis who have hemoglobin ≤10 g/dL, ferritin ≥100, peripheral blasts <10%, can undergo blood draws and agree to contraception are the intended candidates.

Not a fit: Patients who are pregnant, have poor performance status (ECOG ≥3), active metastatic malignancy, or who do not meet the laboratory or safety criteria are unlikely to be eligible or to benefit.

Why it matters

Potential benefit: If effective, nelfinavir could raise hemoglobin and reduce fibrosis-related blood markers, potentially decreasing transfusion dependence.

How similar studies have performed: Preclinical work and small clinical reports have suggested anticancer and hematologic effects of nelfinavir, but its use specifically to treat anemia in myelofibrosis has little prior clinical evidence.

Eligibility criteria

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 this trial is running

Orange, California

• 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

Conditions Myelofibrosis; Anemia; Nelfinavir