Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
All Genders
ID06773195

A Phase 1/2 Study of Combined JAK/ERK Inhibition in Patients With Myelofibrosis

Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-04-03

37

Participants Needed

9

Research Sites

N/A

Total Duration

On this page

Sponsors

M

Memorial Sloan Kettering Cancer Center

Lead Sponsor

B

BioMed Valley Discoveries, Inc

Collaborating Sponsor

AI-Summary

What this Trial Is About

Researchers are evaluating the combination of ruxolitinib and ulixertinib as a potential treatment for people with myelofibrosis. This study aims to determine whether this combination is safe and effective, focusing on identifying the highest dose of ulixertinib that causes few or mild side effects when given alongside ruxolitinib. The trial includes patients with specific types of myelofibrosis who have persistent disease despite ruxolitinib alone. The study has two phases. In Phase 1, participants receive one of three doses of ulixertinib (450 mg, 300 mg, or 150 mg twice daily) combined with ruxolitinib in 28-day cycles. A dose escalation method will be used to find the recommended dose for Phase 2. During Phase 2, participants will receive ulixertinib at this recommended dose with ruxolitinib, continuing in 28-day cycles. Participants will be monitored for side effects and response to treatment, with safety assessed up to 28 days and therapeutic response evaluated at week 25. Assessments include blood tests and evaluations of disease symptoms and organ function. The study requires participants to use contraception during and after treatment and will continue through 2028. This involvement includes regular visits and close monitoring to evaluate the combination therapy's impact on myelofibrosis.

CONDITIONS

Brief Title

A Study of Ruxolitinib in Combination With Ulixertinib in People With Myelofibrosis

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients diagnosed with primary myelofibrosis, post-ET myelofibrosis, post-PV myelofibrosis, or post-pre-fibrotic myelofibrosis by WHO 2016 criteria.
  • Age 18 years or older.
  • Receiving stable ruxolitinib monotherapy (10 mg BID to 20 mg BID) for at least 3 months and stable dose for at least 4 weeks before first study drug dose.
  • Must have DIPSS+ intermediate 2 or greater risk or MIPSS70+ intermediate or greater risk disease.
  • Persistent disease despite ruxolitinib monotherapy, shown by grade 2 or 3 bone marrow fibrosis and either splenomegaly or active symptoms.
  • ECOG performance status of 2 or less.
  • Adequate organ and marrow function as defined by neutrophil count, platelets, bilirubin, liver enzymes, creatinine clearance, and blast count.
  • Agreeable to use effective contraception during and for 4 months after study participation.
Not Eligible

You will not qualify if you...

  • Use of experimental drugs for myelofibrosis or other standard drugs (except hydroxyurea or ruxolitinib) within 2 weeks before starting combination therapy or unresolved toxicities from previous therapy.
  • Prior or concurrent malignancy interfering with safety or efficacy assessment.
  • Unwillingness to receive red blood cell transfusions.
  • Receiving any other investigational agents.
  • History of allergic reactions to ruxolitinib or ulixertinib or similar compounds.
  • Use of strong inhibitors or inducers of certain enzymes or transporters unless stopped 14 days before study.
  • Pregnant or breastfeeding women.
  • Active infections requiring treatment.
  • Chronic HIV or hepatitis B or C infection unless controlled and viral load undetectable.
  • Active lung disease or pneumonitis.
  • Certain cardiovascular conditions including uncontrolled arrhythmias, QTc > 480 msec, recent acute coronary syndrome, heart failure class II or greater, or uncontrolled hypertension.
  • History or risk of retinal vein occlusion or central serous retinopathy.
  • Psychiatric illness or substance use interfering with compliance.
  • Conditions interfering with study participation or data interpretation.
  • Eligible for allogeneic stem cell transplant at enrollment.
  • Inability to understand or unwillingness to sign informed consent.
  • Transformation to accelerated or blast phase disease, including myeloid sarcoma.

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Treatment

Duration - Repeated 28-day cycles until disease progression or discontinuation

Participants receive ulixertinib in combination with ruxolitinib in repeated 28-day cycles. Ulixertinib dose levels will be adjusted during Phase 1 to determine the recommended dose for Phase 2.

Visits every 28 days for treatment and assessments

Trial Site Locations

Total: 9 locations

1

Massachusetts General Hospital (Data Collection Only)

Boston, Massachusetts, United States, 02114

Actively Recruiting

2

Memorial Sloan Kettering Basking Ridge (Limited Protocol Activities)

Basking Ridge, New Jersey, United States, 07920

Actively Recruiting

3

Memorial Sloan Kettering Monmouth (Limited Protocol Activities)

Middletown, New Jersey, United States, 07748

Actively Recruiting

4

Memorial Sloan Kettering Bergen (Limited Protocol Activities)

Montvale, New Jersey, United States, 07645

Actively Recruiting

5

Memorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities)

Commack, New York, United States, 11725

Actively Recruiting

6

Memorial Sloan Kettering Westchester (Limited Protocol Activities)

Harrison, New York, United States, 10604

Actively Recruiting

7

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

Actively Recruiting

8

Memorial Sloan Kettering Nassau (Limited Protocol Activities)

Uniondale, New York, United States, 11553

Actively Recruiting

9

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Not Yet Recruiting

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Research Team

R

Raajit Rampal, MD, PhD

P

Prioty Islam, MD, MS

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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