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 at 773-453-9917 or 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)
PV

KRT-232 vs Ruxolitinib

A Two-Part, Randomized, Open-label, Multicenter, Phase 2a/2b Study of the Efficacy, Safety, and Pharmacokinetics of KRT-232 Compared to Ruxolitinib in Patients With Phlebotomy-Dependent Polycythemia Vera.

Read the full description here.

Status: Recruiting
Posted to page: 9/18/18
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
Posted to page: 9/18/18
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: Recruiting
Posted to page: 8/29/18
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
Posted to page: 8/16/18
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: Recruiting
Posted to page: 6/25/18
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: Recruiting

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: Recruiting

MF

Ruxolitinib + Navitoclax

The purpose of this study is to evaluate the addition of navitoclax to ruxolitinib in patients who have been receiving ruxolitinib alone. Ruxolitinib treatment alone has not been fully controlling disease but preclinical data suggests that the combination of navitoclax with ruxolitinib may help to overcome disease resistance to ruxolitinib.

Click here for more information.

Status: Recruiting

MF

Oral Rigosertib

The goal of this clinical research study is to study the efficacy and safety of oral Rigosertib in patients with Myelofibrosis (MF) and Anemia. This trial is being conducted at MD Anderson. If you have questions please call Jorge Cortes, MD at 713-794-5783.

Read the full description here.

Status: 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: Not Yet Recruiting

MF

IMG-7289

This is a Phase 1 open label study of an orally administered LSD1 inhibitor, IMG-7289, in patients with myelofibrosis.

Read the full description here.

Status: Recruiting

PV

Idasanutlin

A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Idasanutlin Monotherapy in Participants With Hydroxyurea-Resistant/Intolerant Polycythemia Vera.

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
Posted to page: 9/5/17

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
Posted to page: 7/13/2017

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
Posted to page: 6/26/17
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: Recruiting
Posted to page: 6/15/2017

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
Posted to page: 6/15/2017
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: 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: 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: 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

CPI-0610

A Phase 1 Study Evaluating CPI-0610 in Patients With Acute Leukemia, Myelodysplastic Syndrome, Myelodysplastic/ Myeloproliferative Neoplasms, and Myelofibrosis.

Read the full description here.

Status: Recruiting

ETPV

Oral RG7388

Study of Single Agent Oral RG7388 in Patients With PV and ET (With Pilot Feasibility Study in Combination With Pegylated Interferon for Patients Who do Not Respond to the Single Agent at Each Dose Level). If you have question contact John Mascarenhas at john.mascarenhas@mssm.edu or Jill Kleczko at jill.kleczko@mssm.edu

Read the full description here.

Status: Not Yet Recruiting

ETPV

Pegasys

Randomized Trial of Pegylated Interferon Alfa-2a Versus Hydroxyurea in Polycythemia Vera (PV) and Essential Thrombocythemia (ET). If you have questions contact Dr. Ronald Hoffman at ronald.hoffman@mssm.edu

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
PV

AOP2014 and Hydroxyurea

A Randomized, Open-label, Multicenter, Controlled, Parallel Arm, Phase III Study Assessing the Efficacy and Safety of AOP2014 vs. Hydroxyurea in Patients With Polycythemia Vera.

Read the full description here.

Status: Completed

PV

Pegylated Interferon

An Open-label, Multicenter, Phase IIIb Study Assessing the Long-term Efficacy and Safety of AOP2014 and Standard First Line Treatment (BAT) in Patients With Polycythemia Vera Who Previously Participated in the PROUD-PV Study

Contact Michael Zoerer at michael.zoerer@aoporphan.com with any questions.

Read the full description here.

Status: Not Yet Recruiting

MF

SL-401

Phase I/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

Research Tissue Bank

This study that will allow for the preservation and/or storage of a small portion of certain tissue samples. This material will be used for the study of Myeloproliferative Disorders (MPD) by researchers.

Contact: Dr. Rona S. Weinberg at 212-570-3412 or rweinberg@nybloodcenter.org with any questions.

Read the full description here.

Status: Recruiting

MF

Correlative Bio-Marker Study

Correlative Biomarker Study in Patients with Myeloproliferative Disorders

Contact: Rona S. Weinberg, PhD at 212-570-3412 or rweinberg@nybloodcenter.org or Ronald Hoffman, MD at 212-241-2297 or ronald.hoffman@mssm.edu

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

TAK-901

A Phase 1 Dose Escalation Study of TAK-901 in Subjects with Advanced Hematological Malignancies

Read the full description here.

Status: Completed 

MF

Ruxolitinib & Pracinostat

Evaluation of Ruxolitinib & Pracinostat Combination as a therapy for Patients with Myelofibrosis

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

Read the full description here.

Status: Completed

MF

INC424

An Open-label, Multicenter, Expanded Access Study of INC424 for Patients With Primary Myelofibrosis (PMF) or Post Polycythemia MF or Post-essential Thrombocythemia MF.

Contact: Novartis Pharmaceuticals at +1-862-778-8300 with any questions.

Read the full description here.

Status: Completed

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

Oral Arsenic

A Phase I Study of Oral Arsenic Trioxide With or Without Ascorbic Acid in Adults with Myelofibrosis

Contact Roswell Park Cancer Center Institute at 877-275-7724 or askrpci@rosewellpark.org with any questions.

Read the full description here.

Status: Terminated (low accrual)

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: Recruiting

MF

Pomalidomide

A Phase-3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Compare Efficacy and Safety of Pomalidomide in Subjects With Myelofibrosis and Red Blood Cell Transfusion Dependence

Contact Mary Bartlett at 913-266-0513 or mbartlett@celgene.com with any questions.

Read the full description here.

Status: Not Yet Recruiting

MF

Ruxolitinib & Panobinostst

Panobinostat and Ruxolitinib In MyElofibrosis (PRIME STUDY) - Phase I/II Study of Combination Oral JAK2 Tyrosine Kinase Inhibitor (JAK2-TKI) and Histone Deacetylase Inhibitor (HDACI) Therapy in Patients With Myelofibrosis

Contact Jill Kleczko at jill.kleczko@mssm.edu or John Mascarenhas at john.mascarenhas@mssm.edu.

Read the full description here.

Status: Not Yet Recruiting

MF

Ruxolitinib & LDE225

A Phase Ib/II, Open-label, Multi-center, Dose-finding Study to Assess the Safety and Efficacy of the Oral Combination of LDE225 and INC424 (Ruxolitinib) in Patients With Myelofibrosis

Read the full description here.

Status: Completed

MF

Ruxolitinib & SCT

Exploring the Potential of Dual Kinase JAK 1/2 Inhibitor Ruxolitinib (INC424) With Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients With Myelofibrosis

Contact: Jill Kleczko at jill.kleczko@mssm.edu, or Vikas Gupta at 416-946-4521.

Read the full description here.

Status: Not Yet Recruiting

MF

PRM-151

A Phase 2, Prospective Study of PRM-151 In Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (post-PV MF), Or Post-Essential Thrombocythemia MF (post-ET MF)

Read the full description here.

Status: Not Yet Recruiting

MF

Ruxolitinib | Allogeneic Hematopoeitic Cell Transplant

A study of Dual Kinase JAK 1/2 Inhibitor Ruxolitinib (INC424) with Reduced Intensity Allogeneic Hematopoietic Cell Transplantation in Patients with MF.

Contact: Robert H. Lurie at 312.695.1102 or cancertrials@northwestern.edu

Read the full description here.

Status: Withdrawn (The study design was revised so a new protocol will be opened.)

MF

Alisertib (MLN8237)

A Multicenter, Open-Label, Pilot Study of Alisertib (MLN8237), A Novel Inhibitor of Aurora Kinases, in Adult Patients with Relapsed/Refractory Acute Megakaryoblastic Leukemia or Myelofibrosis (Including Primary and Post-Essential/Post-Polycythemic MF)

For more information about this study please contact: 312.472.123 or nmdti@northwestern.edu

Read the full description here.

Status: Not Yet Recruiting

MF

PF-04449913

A Phase 2, Double-blind, Randomized Safety And Efficacy Study Of Glasdegib (Pf-04449913) Versus Placebo In Patients With Myelofibrosis Previously Treated With Ruxolitinib

Contact: Pfizer Clinical Trial Call Center: 1-800-718-1021

Read the full description here.

Status: Terminatedg

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