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

NCT ID: NCT07281781

Last Updated: 2026-02-04

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-09-16
• Study Completion Date: 2027-06-30

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; Myelofibrosis; Anemia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Nelfinavir

Given Orally

Group Type: EXPERIMENTAL

Nelfinavir

Intervention Type: DRUG

Taken twice daily

Interventions

Nelfinavir

Taken twice daily

Intervention Type: DRUG

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.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University of California, Irvine

OTHER

Sponsor Role: lead

Responsible Party

Angela G. Fleischman

Associate Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Angela Flesichman, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Chao Family Comprehensive Cancer Center

Locations

Chao Family Comprehensive Cancer Center, University of California, Irvine

Orange, California, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Chao Family Comprehensive Cancer Center University of California, Irvine

Role: CONTACT

ucstudy@uci.edu

1-877-827-8839

University of California Irvine Medical

Role: CONTACT

Facility Contacts

Angela Fleischman, MD, PhD

Role: primary

ucstudy@uci.edu

877-827-8839

Other Identifiers

UCI 22-118

Identifier Type: OTHER

Identifier Source: secondary_id

6408

Identifier Type: -

Identifier Source: org_study_id