Selinexor for Myelofibrosis

(ESSENTIAL Trial)

Overseen By Priscilla Blosser, RN

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: University of Utah

Must be taking: Ruxolitinib, JAK1/2 inhibitors

Must not be taking: SINE compounds

Disqualifiers: Active infection, Hepatitis, Gastrointestinal dysfunction, others

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

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 5.5 months

17 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing selinexor, a medication that may help treat certain blood disorders. It focuses on patients with primary or secondary myelofibrosis who do not respond to or cannot tolerate common treatments like ruxolitinib. Selinexor works by blocking a protein that helps harmful cells grow and survive. Selinexor is the first oral selective inhibitor of nuclear export compound tested for cancer treatment.

Research Team

Srinivas Tantravahi

Principal Investigator

University of Utah

Eligibility Criteria

Adults diagnosed with primary or secondary myelofibrosis who haven't responded well to, or can't tolerate, JAK1/2 inhibitors like ruxolitinib. They should be relatively active (ECOG ≤ 2), have a spleen enlarged by at least 5 cm if symptomatic or 10 cm regardless of symptoms, and meet specific blood count and organ function criteria.

Inclusion Criteria

I have recovered from previous cancer treatment side effects, or they are mild and stable.

My blood, liver, and kidney functions are within normal ranges.

Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines

Exclusion Criteria

I have not had major surgery in the last 4 weeks.

My body surface area is less than 1.4 square meters.

I do not have an active infection needing IV drugs within the last week.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive selinexor for up to six cycles to assess efficacy and safety

5.5 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Long-term follow-up

Participants are monitored for adverse events and overall survival

Up to 24 months

Treatment Details

Interventions

• Selinexor

Trial Overview The trial is testing Selinexor's effectiveness and safety in patients with myelofibrosis who don't benefit from current treatments. It's an open-label study where all participants receive the drug; there's no comparison group.

Participant Groups

Group I: Selinexor, all patients Experimental Treatment 1 Intervention

Single Arm Study, all patients will get selinexor

Selinexor is already approved in United States, Canada for the following indications:

Approved in United States as Xpovio for:

• Multiple myeloma
• Diffuse large B-cell lymphoma

Approved in Canada as Xpovio for:

• Multiple myeloma

Who Is Running the Clinical Trial?

University of Utah

Lead Sponsor

Trials

1,169

Recruited

1,623,000+

Karyopharm Therapeutics Inc

Industry Sponsor

Trials

89

Recruited

7,200+

Richard Paulson

Karyopharm Therapeutics Inc

Chief Executive Officer since 2021

MBA from the University of Toronto's Rotman School of Management

Reshma Rangwala

Karyopharm Therapeutics Inc

Chief Medical Officer since 2023

MD, PhD