Myelofibrosis, Myelofibrosis; Anemia
Conditions
Keywords
Myelofibrosis, Anemia, Nelfinavir
Brief summary
This is a phase I/II protocol investigating whether Nelfinavir can improve anemia and lower serum fibrosis biomarkers in Myelofibrosis patients.
Interventions
DRUGNelfinavir
Taken twice daily
Sponsors
University of California, Irvine
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Hepcidin Levels | 30 days | Comparison of Hepcidin Levels prior to and after Nelfinavir treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Hemoglobin Levels | 30 days | Comparison of hemoglobin levels to determine whether there is restoration of iron to the bone marrow. |
| Change From Baseline in Serum Fibrosis Biomarker Concentrations | 30 days | To evaluate if nelfinavir lowers fibrosis biomarkers in serum. |
Countries
United States
Contacts
CONTACTChao Family Comprehensive Cancer Center University of California, Irvine
CONTACTUniversity of California Irvine Medical
PRINCIPAL_INVESTIGATORAngela Flesichman, MD, PhD
Chao Family Comprehensive Cancer Center
Outcome results
None listed