MPN Clinical Trials

The MPN Research Foundation has compiled a list of trials below, some of which are currently recruiting patients. Clinical trials are an important step in the process of discovering new treatments for MPNs. The decision to participate in a clinical trial is one you should carefully consider. It is important to discuss clinical trial participation with your family and your physician. If you are considering participating in a clinical trial click here to read our FAQ. If you are interested in participating in research beyond clinical drug trials here is a list of surveys and studies looking for input from people living with Essential Thrombocythemia, Myelofibrosis, and Polycythemia Vera.
 
If you are a company or investigator who is running a trial and you need to make a change to the listing for your trial contact our office by sending an email.


Find a Clinical Trial

Click the boxes below to filter clinical trials by disease type.

All
Myelofibrosis (MF)
Essential Thrombocythemia (ET)
Polycythemia Vera (PV)
MF

CPI-0610

This study is a Phase 3, randomized, blinded study comparing CPI-0610 and ruxolitinib with placebo and ruxolitinib in myelofibrosis (MF) patients that have not been exposed previously to Janus kinase inhibitors (JAKi).

Read the full description here.

Status: Recruiting
MF

Oral TP-3654

This study is a Phase 1, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of TP-3654 in patients with intermediate-2 and high-risk primary or secondary MF.

Read the full description here.

Status: Recruiting
PV

PTG-300

This is a Phase 2 study with an open-label dose escalation phase followed by a blinded withdrawal phase and an open label extension. The study is designed to monitor the PTG-300 safety profile and to obtain preliminary evidence of efficacy of PTG-300 for the treatment of phlebotomy-requiring polycythemia vera.

Read the full description here.

Status: Recruiting
MF

TRANSFORM-2

This is A Randomized, Open-Label, Phase 3 Study Evaluating Efficacy and Safety of Navitoclax in Combination With Ruxolitinib Versus Best Available Therapy in Subjects With Relapsed/Refractory Myelofibrosis.

Read the full description here.

Click here to see if you qualify.

Status: Recruiting
MF

9-ING-41

9-ING-41 has anti-cancer clinical activity while not causing myelosuppression, and has both pre-clinical anti-fibrotic activity and activity against myelofibrosis. This Phase 2 study will study its efficacy in patients with advanced myelofibrosis.

Read the full description here.

Status: Recruiting
MF

REFINE

This is a A Phase 2 Open-Label Study Evaluating Tolerability and Efficacy of Navitoclax Alone or in Combination With Ruxolitinib in Subjects With Myelofibrosis.

Read the full description here.

Click here to see if you qualify.

Status: Recruiting
MF

Momelotinib

This is a A Randomized, Double-Blind, Phase 3 Study of Momelotinib vs Danazol in Symptomatic, Anemic Subjects With Previously JAKi Treated Primary Myelofibrosis, Post-PV Myelofibrosis, or Post-ET Myelofibrosis.

Read the full description here.

Status: Recruiting
MF

TRANSFORM-1

This is a Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study Of Navitoclax In Combination With Ruxolitinib Versus Ruxolitinib In Subjects With Myelofibrosis.

Read the full description here.

Click here to see if you qualify.

Status: Recruiting
ETPV

CPAP Treatment

This early phase I trial studies how well the use of a continuous positive airway pressure (CPAP) machine works in treating obstructive sleep apnea in patients with polycythemia vera or essential thrombocythemia.

Read the full description here.

Status: Recruiting
MF

ABBV-744

This is a Phase 1 study to test the safety and Tolerability of Oral ABBV-744 Tablet Alone or in Combination With Oral Ruxolitinib Tablet or Oral Navitoclax Tablet in Adult Participants With Myelofibrosis.

Read the full description here.

Status: Recruiting
ETMFPV

Vactosertib

This is a 2-tiered, Phase 2, Rule-based, Intra-patient Dose Escalation Study to Investigate Safety and Feasibility of Vactosertib (TEW-7197) in the Treatment of Anemic Patients With Philadelphia Chromosome-negative MPNs (Ph-neg MPNs).

Read the full description here.

Status: Active, Not Yet Recruiting
ETMFPV

PRT543

This is a Phase 1 cohort, dose-escalation study of PRT543 in patients with advanced cancers who have exhausted available treatment options. The purpose of this study is to define a safe dose and schedule to be used in subsequent development of PRT543.

Read the full description here.

Status: Recruiting
MF

Pacritinib

CTI Biopharma is recruiting patients for the PACIFCA trial, a randomized phase 3 study evaluating PACRITINIB in patients with myelofibrosis and less than 50,0000 platelet count who have never received a JAK2 inhibitor or who have had no more than 90 days of prior JAK2 inhibitor exposure.

Read the full description here.

Status: Recruiting
MF

AVID200

This is an open-label, multicenter, Phase I Study of AVID200 in Patients With Myelofibrosis, post ET-Myelofibrosis, and post PV-Myelofibrosis.

Read the full description here.

Status: Recruiting
MF

Fedratinib

This is Single-Arm, Open-Label Efficacy and Safety Trial of Fedratinib in Subjects with DIPSS (Dynamic International Prognostic Scoring System)-Intermediate or High- Risk Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (post-PV MF), or Post-Essential Thrombocythemia Myelofibrosis (post-ET MF) and Previously Treated with Ruxolitinib.

Read the full description here.

Status: Recruiting
PV

KRT-232 vs Ruxolitinib

This study evaluates KRT-232, a novel oral small molecule inhibitor of MDM2, for the treatment of patients with phlebotomy-dependent polycythemia vera (PV). Inhibition of MDM2 in PV is a new mechanism of action in PV

Read the full description here

Status: Active, Not Yet Recruiting
MF

KRT-232

An Open-Label, Phase 2a/2b Study of KRT-232 in Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV-MF), Or Post-Essential Thrombocythemia MF (Post-ET-MF) Who Have Failed Ruxolitinib.

Read the full description here.

Status: Recruiting
ETMF

CALR Exon 9 Mutant Peptide Vaccine

A phase-I-first in man study in patients with calreticulin(CALR)-mutant MPN by vaccinating with exon 9 mutated peptide with the adjuvant Montanide ISA-51 to monitor safety and toxicity and the immunological response to vaccination.

Read the full description here.

Status: Active, Not Yet Recruiting
MF

Ruxolitinib + Pevonedistat

This phase I combination trial is studying the safety and tolerability of pevonedistat in combination with ruxolitinib in patients with myelofibrosis.

Read the full description here.

Status: Recruiting
MF

Ruxolitinib + Chemotherapy

This is a pilot, Open-Label Study of Safety and Efficacy of Ruxolitinib Given Peri-transplant During Reduced Intensity Allogeneic Hematopoietic Cell Transplantation (HCT) in Patients With Myelofibrosis.

Read the full description here.

Status: Active, Not Yet Recruiting
MF

Cyclophosphamide

The goal of this clinical research study is to learn about the safety of giving a stem cell transplant from a tissue-mismatched (haploidentical) donor, followed by cyclophosphamide, to patients with advanced myelofibrosis (MF). Melphalan, fludarabine, and total body irradiation (TBI) will also be given before the transplant as part of your standard care.

Read the full description here.

Status: Completed

ETMFPV

Givinostat

This is a multi-center, open label study testing the long-term safety, tolerability and efficacy of Givinostat in patients with Polycythemia Vera, Essential Thrombocythemia, primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis following core protocols in chronic myeloproliferative neoplasms and/or patient-named compassionate use program.

Read the full description here.

Status: Active, Not Yet Recruiting

MF

CPI-0610 +/- Ruxolitinib

A Phase 2 study of CPI-0610 alone or in combination with ruxolitinib in patients with myelofibrosis. This study is for MF patients who are currently taking ruxolitinib but have a sub-optimal response or for patients who are resistant, intolerant or have lost response to a JAK inhibitor.

Read the full description here.

Status: Recruiting

MF

Selumetinib + Azacitidine

Study of MEK Inhibitor Selumetinib in Combination With Azacitidine in Patients With Higher Risk Chronic Myeloid Neoplasia.

Read the full description here.

Status: Recruiting

MF

IMG-7289

A Multi-Center, Open-Label, phase 2 Study to Assess the Safety, Steady-State Pharmacokinetics and Pharmacodynamics of IMG-7289 in Patients With Myelofibrosis

Read the full description here.

Status: Recruiting

MF

Navitoclax

A Study Evaluating Tolerability and Efficacy of Navitoclax in Combination With Ruxolitinib in Subjects With Myelofibrosis. The trial is for patients who have been receiving ruxolitinib for 6 months.

Read the full description here.

Status: Recruiting
MF

Luspatercept

A Safety and Efficacy Study to Evaluate Luspatercept in Subjects With Myeloproliferative Neoplasm-associated Myelofibrosis Who Have Anemia With and Without Red Blood Cell-transfusion Dependence.

Read the full description here.

Status: Recruiting
MF

Pacritinib

CTI Biopharma is recruiting patients for the PAC203 study, a randomized phase 2 trial evaluating different doses of pacritinb in myelofibrosis patients regardless of platelet count and who have previously received ruxolitinib (Jakafi/Jakavi).

Read the full description here.

Status: Recruiting
ETMF

Ruxolitinib vs. Anagrelide

A Double-Blind, Double-Dummy Phase 2 Randomized Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Anagrelide in Subjects With Essential Thrombocythemia Who Are Resistant to or Intolerant of Hydroxyurea.

Read the full description here.

Status: Completed
MF

Ruxolitinib + INCB050465

A Phase 2 Study of the Safety, Tolerability, and Efficacy of INCB050465 in Combination With Ruxolitinib in Subjects With Myelofibrosis.

Read the full description here.

Status: Recruiting
MF

Ruxolitinib + Thalidomide

A Clinical Study to Test the Effects of Ruxolitinib And Thalidomide Combination for Patients With Myelofibrosis.

Read the full description here.

Status: Recruiting

ETPV

Ruxolitinib - Ruxo-BEAT trial

The Ruxo-BEAT Trial in Patients With High-risk Polycythemia Vera or High-risk Essential Thrombocythemia (Ruxo-BEAT)

Click here for more information.

Status: Active, Not Recruiting

MFPV

Ruxolitinib + TGR-1202

TGR-1202 + Ruxolitinib in Subjects With Myelofibrosis, MDS/MPN, or Polycythemia Vera Resistant to Hydroxyurea.

Click here for more information.

Status: Active, Not Recruiting

ET

Ruxolitinib vs BAT

A Randomized, Multicenter Phase IIb Study to Evaluate the Efficacy and Safety of Ruxolitinib Versus Best Available Therapy in Patients With High Risk Essential Thrombocythemia, Who Are Resistant or Intolerant to Hydroxyurea: A FIM Study.

Read the full description here.

Status: Not Yet Recruiting

MF

Ruxolitinib + Pomalidomide

A Phase-Ib/II Study to assess the efficacy of oral drug combination ruxolitinib and pomalidomide in primary and secondary myelofibrosis patients.

Read the full description here.

Status: Recruiting

ETMFPV

PU-H71

The First-in-human Phase I Trial of PU-H71 in Patients With Advanced Malignancies. If you have question please contact John Gerecitano, MD, PhD at 212-639-3748 OR Shanu Modi, MD at 646-888-5243

Read the full description here.

Status: Not Yet Recruiting

ETMFPV

Familial Study of MPDs

This study is designed to characterize the clinical picture and genetic pattern of Polycythemia and Thrombocytosis. The purpose of this project is to find a gene and its mutation that causes these disorders. Contact Dr. Josef Prchal at 801-581-4220 or Kim Hickman at 801-581-3707 if you have questions.

Read the full description here.

Status: Recruiting
MF

SL-401

Phase II trial of SL-401 in Patients With Advanced, High Risk Myeloproliferative Neoplasms (Systemic Mastocytosis, Advanced Symptomatic Hypereosinoophic Disorder, Myelofibrosis, Chronic Myelomonocytic Leukemia)

Contact: Dr. Srdan Verstovsek at 713-745-3429 with any questions.

Read the full description here.

Status: Recruiting

MF

Sotatercept (ACE-011)

A Phase-2, Prospective, Open-Label Study to Determine the Safety and Efficacy of Sotatercept (ACE-011) in Subjects With Myeloproliferative Neoplasm (MPN) -Associated Myelofibrosis and Anemia

Contact: Srdan Verstovsek, MD, PhD, at 713-745-3429 with questions.

Read the full description here.

Status: Recruiting

MF

NS-018

Dose-Escalation Multi-center Study to Assess the Safety, Tolerability, PK and PD of Orally Administered NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF, or Post-essential Thrombocythemia MF.

Read the full description here.

Status: Not Yet Recruiting

MF

LCL-161

Phase 2 Single Agent Study of LCL-161 in Patients With Primary Myelofibrosis, Post-Polycythemia Vera MF(Post-PV MF), or Post-Essential Thrombocytosis MF.

Contact Naveen Pemmaraju, MD at 713-792-4956 with any questions.

Read the full description here.

Status: Active, Not Recruiting

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