Nelfinavir for Myelofibrosis

Overseen By Chao Family Comprehensive Cancer Center University of California, Irvine

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: University of California, Irvine

Must not be taking: Momelotinib, Pacritinib, others

Disqualifiers: Pregnancy, HIV, Organ impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 4 weeks

10 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores whether Nelfinavir, an oral medication, can improve anemia (a condition characterized by insufficient healthy red blood cells) and reduce fibrosis markers in individuals with myelofibrosis, a type of bone marrow cancer. Researchers aim to determine if this treatment can effectively manage the disease. Participants should have myelofibrosis, experience low hemoglobin levels (indicative of anemia), and be willing to undergo regular blood tests. As a Phase 1/Phase 2 trial, this research seeks to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to contribute to early-stage advancements in myelofibrosis treatment.

Will I have to stop taking my current medications?

You can continue taking your current medications for myeloproliferative neoplasms (MPN) like aspirin, hydroxyurea, interferon-alpha, ruxolitinib, fedratinib, or anagrelide. However, you cannot take Momelotinib, Pacritinib, or any medications that are contraindicated with Nelfinavir.

Is there any evidence suggesting that Nelfinavir is likely to be safe for humans?

Research has shown that Nelfinavir, a drug typically used to treat HIV, is now under study for its safety in treating myelofibrosis, a type of bone marrow disorder. Nelfinavir acts as a protease inhibitor, blocking a protein that aids HIV growth. In HIV treatments, most people tolerate it without major issues.

As it undergoes testing for myelofibrosis, understanding its effects on this new condition is crucial. The study is in early stages (phase 1/2), focusing on safety and determining the correct dose. Early-phase trials like this one monitor side effects and ensure the treatment's safety for participants.

While direct safety data for Nelfinavir in myelofibrosis is not yet available, its previous use in HIV suggests it might be well-tolerated. However, each condition differs, and new side effects could emerge. Trial participants will be closely monitored to ensure safety, with any side effects recorded and addressed. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for myelofibrosis, which often involve drugs like ruxolitinib or fedratinib that target specific enzymes related to the disease, Nelfinavir is originally an antiviral medication used for HIV. Researchers are excited about Nelfinavir because it may work by a different mechanism, potentially disrupting cancer cell survival pathways not targeted by current treatments. Its oral administration offers convenience, and it might provide an alternative option for patients who do not respond well to existing therapies.

What evidence suggests that Nelfinavir might be an effective treatment for Myelofibrosis?

Research has shown that Nelfinavir, a drug used to treat HIV, might help with myelofibrosis by improving anemia and reducing certain blood markers related to fibrosis. Participants in this trial will receive Nelfinavir, which early findings suggest can block a protein called hepcidin, involved in controlling iron levels in the body. This could improve anemia by ensuring the body gets enough iron. Although more research is needed, these early results offer hope for patients seeking new treatment options. ^{1 5 6 7 8}

Who Is on the Research Team?

Angela Flesichman, MD, PhD

Principal Investigator

Chao Family Comprehensive Cancer Center

Are You a Good Fit for This Trial?

This trial is for individuals with myelofibrosis, a type of bone marrow cancer that causes severe anemia. Participants should have symptoms related to anemia and must not have had certain treatments before joining.

Inclusion Criteria

I am currently on treatment for myeloproliferative neoplasm (MPN) that may include medications like aspirin or hydroxyurea.

Peripheral blast count <10% during Screening

Hemoglobin ≤ 10gm/dL at screening

Exclusion Criteria

Currently pregnant or planning on being pregnant within the study period

I need help with my daily activities due to my health condition.

Known history of allergic reaction to nelfinavir

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive Nelfinavir orally to improve anemia and lower serum fibrosis biomarkers

4 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Nelfinavir

Trial Overview

The study is testing Nelfinavir, a drug being repurposed to see if it can improve anemia in patients with myelofibrosis by restoring iron delivery to the bone marrow.

How Is the Trial Designed?

Group I: Nelfinavir Experimental Treatment 1 Intervention

Given Orally

Who Is Running the Clinical Trial?

University of California, Irvine

Lead Sponsor

Trials

580

Recruited

4,943,000+

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07281781

Treating Anemia in Myelofibrosis With Repurposed Drugs ...

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

2.

cureswithinreach.org

cureswithinreach.org/2023/10/16/treating-anemia-in-myelofibrosis-with-nelfinavir/

Treating Anemia in Myelofibrosis with Nelfinavir

This Phase IIA pilot trial will treat 15 myelofibrosis patients with oral nelfinavir to block the activation of hepcidin under inflamed ...

3.

ctv.veeva.com

ctv.veeva.com/study/treating-anemia-in-myelofibrosis-with-repurposed-drugs-nelfinavir-that-restore-iron-delivery-to-th

Treating Anemia in Myelofibrosis With Repurposed Drugs ...

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

4.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC6707020/

Clinical Outcomes of the HIV Protease Inhibitor Nelfinavir With ...

This phase 1/2 clinical trial found that nelfinavir with concurrent chemoradiotherapy was well tolerated and had promising long-term local control and survival.

5.

accessdata.fda.gov

accessdata.fda.gov/drugsatfda_docs/nda/2011/202192Orig1s000Lbl.pdf

202192Orig1s000 | FDA

Of the total number of myelofibrosis patients in clinical studies with Jakafi, 51.9% were 65 years of age and older. No overall differences in ...

6.

go.drugbank.com

go.drugbank.com/drugs/DB00220

Nelfinavir: Uses, Interactions, Mechanism of Action

Nelfinavir is a potent HIV-1 protease inhibitor. It is used in combination with other antiviral drugs in the treatment of HIV in both adults and children.

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT01732445

Ruxolitinib Phosphate and Danazol in Treating Anemia ...

This phase II pilot trial studies how well ruxolitinib phosphate and danazol work in treating anemia in patients with myelofibrosis.

8.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC4882345/

Overcoming treatment challenges in myelofibrosis and ...

In this review, we summarize the efficacy and safety data for ruxolitinib in both indications and discuss specific pharmacologic properties relevant for its ...