Primary MyelofibrosisPhase 2ESSENTIALNCT03627403

Selinexor in Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors

A Phase 2 study testing Selinexor in people with Primary Myelofibrosis and Post-essential Thrombocythemia Myelofibrosis, run by University of Utah at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

Search all trials

Trial summary

This is a phase II, open label, prospective, single-arm study evaluating the efficacy and safety of selinexor in patients with PMF or secondary MF (PPV-MF or PET-MF) who are refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors. Read more about this study

Who is it for?: Ages 18 years and older
Conditions the study is for: Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
What is being tested?: Selinexor
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 17 participants
Where: 1 siteUnited States
Study dates: 10 May 2019to 5 Mar 2025
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 years and older
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Male or female subject aged ≥ 18 years.
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
Diagnosis of primary myelofibrosis (PMF), post-essential thrombocytosis (PET-MF) or post-polycythemia vera (PPV-MF).
Life expectancy ≥ 6 months.
Prior treatment with ruxolitinib or any experimental JAK1/2 inhibitor with any one or more of the following:
Inadequate response after being on ≥ 3 months of treatment defined by: i. Palpable spleen ≥ 10 cm below the left subcostal margin on physical examination at the screening visit OR ii. Palpable spleen ≥ 5cm below the left subcostal margin on physical examination at the screening visit AND active symptoms of MF at the screening visit defined presence of 1 symptom score of ≥ 5 or two symptom scores each of ≥ 3 using the Screening Symptoms Form (Appendix 6) b. Intolerant to ruxolitinib and/or other JAK1/2 inhibitors due to any grade ≥ 3 non-hematologic AEs of or any grade ≥ 2 AEs requiring treatment discontinuation AND palpable spleen ≥ 5cm below the left subcostal margin on physical examination at the screening visit.

Show all 25Show fewer

Adequate organ function as defined as:
Hematologic (≤ 28 days prior to C1D1):
Total white blood cell (WBC) count ≥ 1000/mm3
Absolute neutrophil count (ANC) ≥ 500/mm3
Hemoglobin ≥ 7 g/dL
Platelet count ≥ 30,000/mm3
For patients receiving transfusion and growth factor support, the following delays must be observed between the last administration and hematologic laboratory screening assessments:
For hematopoietic growth factor support (including erythropoietin, darbepoetin, granulocyte-colony stimulating factor [G-CSF], granulocyte macrophage-colony stimulating factor [GM-CSF], and platelet stimulators [e.g., eltrombopag, romiplostim, or interleukin-11]): at least 2 weeks.
Growth factor support, RBC and/or platelet transfusions are allowed as clinically indicated per institutional guidelines during the study.
Hepatic (≤ 28 days prior to C1D1):
Total bilirubin < 1.5 × ULN except in patients with indirect hyperbilirubinemia due to hemolysis or with Gilbert's syndrome where total bilirubin should be < 5 × ULN
Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5 × ULN.
Renal (within 28 days prior to C1D1):
Estimated creatinine clearance (CrCl) ≥ 20 mL/min using the Cockcroft and Gault formula [(140-Age) × Mass (kg)/(72 × creatinine mg/dL), multiply by 0.85 if the patient is female] OR
Female patients of childbearing potential must have a negative serum pregnancy test (≤ 3 days prior to C1D1).
Female patients of childbearing potential must agree to use 2 methods of contraception throughout the study and for 3 months following the last dose of study treatment (including 1 highly effective and 1 effective method of contraception as defined in section 7.4)
Male patients must use an effective barrier method of contraception if sexually active with a female of childbearing potential.
Recovery to baseline or ≤ Grade 1 CTCAE v5.0 from toxicities related to any prior treatments including ruxolitinib or other experimental agents unless AE(s) are clinically nonsignificant and/or stable on supportive therapy.
Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines.

Who cannot join

Prior exposure to a SINE compound, including selinexor.
Patients who are below their ideal body weight and would be unduly impacted by changes in their weight, in the opinion of the investigator, will be excluded
Uncontrolled active infection requiring parenteral antibiotics, antivirals, or antifungals ≤ 1 week prior to C1D1. Patients on prophylactic antibiotics or with a controlled infection ≤ 1 week prior to C1D1 are acceptable.
Radiation, chemotherapy, immunotherapy, or any other anticancer therapy (including investigational therapies) ≤ 2 weeks.
Ruxolitinib or other JAK1/2 inhibitors ≤ at least 3 days or 5 half-lives prior to C1D1.
Major surgery ≤ 4 weeks prior to C1D1.

Show all 12Show fewer

Known active hepatitis A, B, or C infection; or known to be positive for hepatitis C virus ribonucleic acid (RNA) or hepatitis B virus surface antigen.
Any active gastrointestinal dysfunction interfering with the patient's ability to swallow tablets, or any active gastrointestinal dysfunction that could interfere with absorption of study treatment.
Any life-threatening illness, organ system dysfunction, or serious psychiatric, medical, or other conditions/situations which, in the investigator's opinion, could compromise a patient's ability to give informed consent, safety, or compliance with the protocol.
Contraindication to any of the required concomitant drugs or supportive treatments.
Subjects taking prohibited medications as described in Section 6.3. Following discontinuation of prohibited medications, a washout period is required prior to initiating study treatment (the duration of the washout must be as clinically indicated, e.g. at least five half-lives).
Subjects who are breastfeeding and unwilling to stop while on study

Trial locations and contacts

1 site in 1 country.

United States1 site

Huntsman Cancer Institute/University of Utah
Salt Lake City, Utah, 84112

Other trials in United States

About this study

Official title: A Phase II Study to Evaluate the Efficacy and Safety of Selinexor in Patients With Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors

Conditions: Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
Being tested: Selinexor
Medical subject headings: Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

SelinexorDrug
Selinexor will be administered orally at a dose of 80 mg once weekly until IWG-MR disease progression, intolerable toxicity, or no clinical benefit per treating physician's discretion whichever occurs first.
For patients enrolled after Protocol v7, Selinexor will be administered by oral route beginning at 40mg once weekly. Prior to protocol version 7, the starting dose of sSelinexor was 60 mg and 80 mg once weekly.

Study groups in NCT03627403
Study group	Role	What they receive
Selinexor, all patients	Experimental	Single Arm Study, all patients will get selinexor Receives: Drug: Selinexor

What is being measured

The primary outcome is the main question the study is designed to answer.

Count of Participants With Reduction in Spleen Volume
To assess the efficacy of selinexor on spleen volume reduction in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
This outcome will report the number of subjects with ≥ 35% reduction in spleen volume as measured by MRI or CT abdomen from baseline to after six cycles of treatment or end of treatment. Patients who did not complete six cycles of treatment received an MRI at discontinuation of therapy. Patients who died prior to completing six cycles of treatment or a follow-up scan were considered non-responders.
Measured: Up to 5.5 months

Secondary outcomes (5)

Adverse Events That Occur
To assess the safety/tolerability and further characterize the safety profile of selinexor in PMF, PET-MF, or PPV-MF patients refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
To evaluate the rate of adverse events (AEs) and serious adverse events (SAEs). This outcome measure will report the count of participants who experienced a Grade ≥ 3 AE while receiving study treatment until 30 days after the last dose of protocol treatment.
Measured: while receiving study treatment until 30 days after the last dose of protocol treatment (up to 17 months)
Percent Change of Spleen Volume
To further assess the efficacy and clinical activity of selinexor (by means of overall response) in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
This outcome will report the mean percent change of spleen volume as measured by MRI or CT abdomen from baseline to after six cycles of treatment or end of treatment. Patients who did not complete six cycles of treatment received an MRI at discontinuation of therapy. Patients who died prior to completing six cycles of treatment or a follow-up scan were not included in this endpoint.
The mean and standard will be reported for the change in spleen volume.
Measured: Up to 5.5 months
Change in Symptoms Score
To further assess the efficacy and clinical activity of selinexor (by means of reduction in symptoms) in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
This outcome reports the count of patients with ≥ 50% reduction of total symptoms scores as measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) from baseline after 6 cycles of treatment. Patients who did not complete 6 cycles of treatment will receive the MPN-SAF assessment at discontinuation of therapy.
The MPN-SAF is a symptom assessment completed by the patients including fatigue, concentration, early satiety, inactivity, night sweats, itching, bone pain, abdominal discomfort, weight loss, and fevers. Scoring is from 0 (absent/as good as it can be) to 10 (worst imaginable/as bad as it can be) for each item. The MPN-SAF TSS is the sum of all the individual scores (0-100 scale).
Measured: Up to 5.5 months
Overall Response
To further assess the efficacy and clinical activity of selinexor (by means of overall response) in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors.
Overall response according to the 2013 International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) consensus criteria for treatment response in primary and secondary MF (post-PV and post-ET). The IWG-MRT response categories are CR (Complete Response), PR (Partial Response), CI (Clinical Improvement), Anemia Response, Spleen Response, Symptoms Response, Progressive Disease, Stable Disease, and Relapse.
This outcome measure will report the number of participants who have progressed or died at 24 months after initiation of study therapy.
Measured: Up to 24 months after initiation of study therapy.
Overall Survival
Overall survival at 24 months from the initiation of study therapy.
This outcome measure will report the count of participants who were not deceased at 24 months after the initiation of study treatment.
Measured: Up to 24 months from the initiation of study therapy

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

10 May 20195 Mar 2025

First posted13 Aug 2018
Study start10 May 2019
Primary completion16 Aug 2023
Study completion5 Mar 2025

Why it stopped: PI and Sponsor decision. Accrual was low and there was sufficient data to detect a response.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Primary MyelofibrosisStudies of the same condition, open and under way.
More trials run by University of UtahThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Model description: Open label, non-randomized, prospective, single-arm study
Masking: None (open label)
Enrolment: 17 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03627403
Sponsor's protocol ID: 114354
Acronym: ESSENTIAL
Status last verified: May 2026
First submitted: 7 Aug 2018
Last updated: 26 Oct 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
RecruitingPhase 3
A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
Myelofibrosis, Anemia
Active, not recruitingPhase 3
Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
Myelofibrosis
RecruitingPhase 2
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Not yet recruitingPhase 1
Maintenance Treatment With Imetelstat in Patients With High-Risk Myeloid Neoplasms After Allo-SCT
High-risk Myeloid Malignancy, Allogenic Stem Cell Transplantation

All Primary Myelofibrosis trials

Study at a glance

From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II Study to Evaluate the Efficacy and Safety of Selinexor in Patients With Myelofibrosis Refractory or Intolerant to JAK1/2 Inhibitors
ClinicalTrials.gov ID: NCT03627403
Acronym / sponsor ID: ESSENTIAL / 114354
Conditions: Primary Myelofibrosis, Post-essential Thrombocythemia Myelofibrosis, Post-polycythemia Vera Myelofibrosis
Phase: Phase 2
Study type: Interventional
Recruitment status: TerminatedReason stopped: PI and Sponsor decision. Accrual was low and there was sufficient data to detect a response.
Enrolment: 17 participants (actual)
Lead sponsor: University of UtahUniversity, hospital or other
Collaborators: Karyopharm Therapeutics Inc
Study start: 10 May 2019 (actual)
Primary completion: 16 Aug 2023 (actual)
Study completion: 5 Mar 2025 (actual)
First posted: 13 Aug 2018 (actual)
Results: Posted 9 Oct 2024

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1
Model description: Open label, non-randomized, prospective, single-arm study

Arm	Type	Receives
Selinexor, all patients	Experimental	Drug: SelinexorSingle Arm Study, all patients will get selinexor

Who may qualify

Ages: 18 years and older
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (25 listed)

Male or female subject aged ≥ 18 years.
Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2
Diagnosis of primary myelofibrosis (PMF), post-essential thrombocytosis (PET-MF) or post-polycythemia vera (PPV-MF).
Life expectancy ≥ 6 months.
Prior treatment with ruxolitinib or any experimental JAK1/2 inhibitor with any one or more of the following:

Key exclusion criteria (12 listed)

Prior exposure to a SINE compound, including selinexor.
Patients who are below their ideal body weight and would be unduly impacted by changes in their weight, in the opinion of the investigator, will be excluded
Uncontrolled active infection requiring parenteral antibiotics, antivirals, or antifungals ≤ 1 week prior to C1D1. Patients on prophylactic antibiotics or with a controlled infection ≤ 1 week prior to C1D1 are acceptable.
Radiation, chemotherapy, immunotherapy, or any other anticancer therapy (including investigational therapies) ≤ 2 weeks.
Ruxolitinib or other JAK1/2 inhibitors ≤ at least 3 days or 5 half-lives prior to C1D1.

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SelinexorSelinexor will be administered orally at a dose of 80 mg once weekly until IWG-MR disease progression, intolerable toxicity, or no clinical benefit per treating physician's discretion whichever occurs first. For patients enrolled after Protocol v7, Selinexor will be administered by oral route beginning at 40mg once weekly. Prior to protocol version 7, the starting dose of sSelinexor was 60 mg and 80 mg once weekly.	Drug	Selinexor, all patients

Primary outcome

Count of Participants With Reduction in Spleen Volume
Time frame: Up to 5.5 months
To assess the efficacy of selinexor on spleen volume reduction in subjects with myelofibrosis (PMF, PET-MF, or PPV-MF) refractory or intolerant to ruxolitinib and/or any other experimental JAK1/2 inhibitors. This outcome will report the number of subjects with ≥ 35% reduction in spleen volume as measured by MRI or CT abdomen from baseline to after six cycles of treatment or end of treatment. Patients who did not complete six cycles of treatment received an MRI at discontinuation of therapy. Patients who died prior to completing six cycles of treatment or a follow-up scan were considered non-responders.

5 secondary outcomes

Adverse Events That Occur
Time frame: while receiving study treatment until 30 days after the last dose of protocol treatment (up to 17 months)
Percent Change of Spleen Volume
Time frame: Up to 5.5 months
Change in Symptoms Score
Time frame: Up to 5.5 months
Overall Response
Time frame: Up to 24 months after initiation of study therapy.
Overall Survival
Time frame: Up to 24 months from the initiation of study therapy

Results and safety

Results posted to the registry on 9 Oct 2024, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Selinexor, All Patients	17	17

Primary outcome: Count of Participants With Reduction in Spleen Volume

Time frame: Up to 5.5 months. Count of participants. Unit: Participants

Measure	Selinexor, All Patients n = 17
Value	3

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Event and Serious Adverse Event data was collected from the first dose of the study until 30 days after the last dose of the study drug (up to 17 months). Survival Data was collected from the initiation of study therapy until 2 years after the initiation of study therapy (up to 2 years).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Selinexor, All Patients	4 of 17 (23.5%)	17 of 17 (100%)	5 of 17

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Primary Myelofibrosis

RecruitingPhase 3
A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
Primary Myelofibrosis (PMF), Post-polycythemia Vera Myelofibrosis (PPV-MF)
RecruitingPhase 3
A Study to Compare Elritercept to Placebo in Adults With Myelofibrosis and Anemia Who Are Taking Ruxolitinib
Myelofibrosis, Anemia
Active, not recruitingPhase 3
Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis
Myelofibrosis
RecruitingPhase 2
Study of Momelotinib in Combination With Luspatercept in Participants With Transfusion Dependent Myelofibrosis
Primary Myelofibrosis, Myelofibrosis; Primary Myelofibrosis; Post-polycythemia Vera Myelofibrosis; Post-essential Thrombocythemia Myelofibrosis
Not yet recruitingPhase 1
Maintenance Treatment With Imetelstat in Patients With High-Risk Myeloid Neoplasms After Allo-SCT
High-risk Myeloid Malignancy, Allogenic Stem Cell Transplantation

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Srinivas Tantravahi, MD principal investigator

University of Utah

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Selinexor in people with Primary Myelofibrosis and Post-essential Thrombocythemia Myelofibrosis, run by University of Utah at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 9 Oct 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Count of Participants With Reduction in Spleen Volume (time frame: Up to 5.5 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03627403.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.