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

NCT07281781 | Recruiting Phase 1 FDA Drug

• 2 other identifiers: 6408UCI 22-118
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Myelofibrosis; Anemia; Myelofibrosis

Keywords

Myelofibrosis; Anemia; Nelfinavir

Outcome Measures

Primary Outcomes (1)

• Change in Hepcidin Levels

  Comparison of Hepcidin Levels prior to and after Nelfinavir treatment

  30 days

Secondary Outcomes (2)

• Change in Hemoglobin Levels

  30 days

• Change From Baseline in Serum Fibrosis Biomarker Concentrations

  30 days

Study Arms (1)

Nelfinavir

EXPERIMENTAL

Given Orally

Drug: Nelfinavir

Interventions

Nelfinavir DRUG

Taken twice daily

Nelfinavir

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥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.

You may not qualify if:

• 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
• Serum creatinine clearance less than 30 ml/min (eGFR).
• 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.
• Platelets \< 50 × 10\^9/L without transfusions
• ANC \< 0.75 × 10\^9/L without growth factors
• Known history of allergic reaction to nelfinavir.

Sponsors & Collaborators

• University of California, Irvine: lead

Study Sites (1)

Chao Family Comprehensive Cancer Center, University of California, Irvine

Orange, California, 92868, United States

RECRUITING

MeSH Terms

Conditions

Primary Myelofibrosis; Anemia

Interventions

Nelfinavir

Condition Hierarchy (Ancestors)

Myeloproliferative Disorders; Bone Marrow Diseases; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Isoquinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds

Study Officials

• Angela Flesichman, MD, PhD

  Chao Family Comprehensive Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Chao Family Comprehensive Cancer Center University of California, Irvine

CONTACT

1-877-827-8839; ucstudy@uci.edu

University of California Irvine Medical

CONTACT

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: December 4, 2025
• First Posted: December 15, 2025
• Study Start: September 16, 2025
• Primary Completion (Estimated): June 1, 2026
• Study Completion (Estimated): June 1, 2027
• Last Updated: May 4, 2026

Record last verified: 2026-04

Locations

US (1)