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

This is a phase I/II protocol investigating whether Nelfinavir can improve anemia and lower serum fibrosis biomarkers in Myelofibrosis patients.

Study Overview

• Status: Recruiting
• Conditions: Myelofibrosis; Myelofibrosis; Anemia
• Intervention / Treatment: Drug: Nelfinavir

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Chao Family Comprehensive Cancer Center University of California, Irvine; Phone Number: 1-877-827-8839; Email: ucstudy@uci.edu
• Study Contact Backup: Name: University of California Irvine Medical

Study Locations

• United States
  • California
    • Orange, California, United States, 92868
      • Recruiting
      • Chao Family Comprehensive Cancer Center, University of California, Irvine
      • Contact: Angela Fleischman, MD, PhD; Phone Number: 877-827-8839; Email: ucstudy@uci.edu

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Nelfinavir; Given Orally	Drug: Nelfinavir; Taken twice daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Hepcidin Levels	Comparison of Hepcidin Levels prior to and after Nelfinavir treatment	30 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Hemoglobin Levels	Comparison of hemoglobin levels to determine whether there is restoration of iron to the bone marrow.	30 days
Change From Baseline in Serum Fibrosis Biomarker Concentrations	To evaluate if nelfinavir lowers fibrosis biomarkers in serum.	30 days

Collaborators and Investigators

• Sponsor: University of California, Irvine
• Investigators: Principal Investigator: Angela Flesichman, MD, PhD, Chao Family Comprehensive Cancer Center

Study record dates

Study Major Dates

• Study Start (Actual): September 16, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: December 4, 2025
• First Submitted That Met QC Criteria: December 4, 2025
• First Posted (Actual): December 15, 2025

Study Record Updates

• Last Update Posted (Actual): May 4, 2026
• Last Update Submitted That Met QC Criteria: April 28, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: Anemia; Myelofibrosis; Nelfinavir
• Additional Relevant MeSH Terms: Hematologic Diseases; Bone Marrow Diseases; Myeloproliferative Disorders; Hemic and Lymphatic Diseases; Anemia; Primary Myelofibrosis; Heterocyclic Compounds; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Isoquinolines; Nelfinavir
• Other Study ID Numbers: 6408; UCI 22-118 (Other Identifier: UCI CFCCC)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No