MPN CLINICAL TRIALS

MPN CLINICAL TRIALS

Clinical trials are an essential step in the process of discovering new treatments for MPNs. It takes a long time from initial studies in a lab for a therapy to gain FDA approval, to be prescribed by a doctor and available at the pharmacy. You can learn more about that process here.

When drugs enter the clinical trial phase, researchers rely on volunteers (both healthy and those with the target disease) to participate in trials to determine if the drug is safe and efficacious. Without the participation of those volunteers, new drugs cannot be approved.

The MPN Research Foundation has compiled a list of MPN trials that are currently recruiting and we update this list on a quarterly basis. There are links to the ClinicalTrials.gov website for each trial in the document for each trial listed. You may find additional trials not on this list on that website. Our goal is to capture as many trials as possible and to keep the status of each trial up-to-date.

What can you do?

  1. Review the list – MPN CT 2021_06_30 FINAL
  2. Share the list (or the link to this site) with other patients you know
  3. Consider whether you might be a good candidate for a clinical trial? If the answer is yes..
  4. Print the list and bring it to your next conversation with your doctor.
 

This list is intended for reference purposes only – it is NOT medical advice.  Patients should consult their doctors on whether any of these trials may be appropriate for them.

Additionally, MPN Research Foundation has a searchable catalog of trials below, some of which are currently recruiting patients.

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.

  • All Essential Thrombocythemia (ET)Myelofibrosis (MF)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).

    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.

    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.

    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.

    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

    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.

    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.

    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.

    Status: Recruiting

    et pv

    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.

    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.

    Status: Recruiting

    et mf pv

    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).

    Status: Active, Not Yet Recruiting

    et mf pv

    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.

    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.

    Status: https://www.clinicaltrials.gov/ct2/show/NCT03165734?term=PAC203&rank=1

    mf

    AVID200

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

    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.

    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

    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.

    Status: Recruiting

    et mf

    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.

    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.

    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.

    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.

    Status: Completed

    et mf pv

    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.

    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.

    Status: Recruiting

    mf

    Selumetinib + Azacitidine

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

    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

    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.

    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.

    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).

    Status: Recruiting

    et mf

    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.

    Status: https://clinicaltrials.gov/ct2/show/NCT03123588?term=NCT03123588&cond=RESET-272&rank=1

    mf

    Ruxolitinib + INCB050465

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

    Status: Recruiting

    mf

    Ruxolitinib + Thalidomide

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

    Status: Recruiting

    et pv

    Ruxolitinib – Ruxo-BEAT trial

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

    Status: Active, Not Recruiting

    mf pv

    Ruxolitinib + TGR-1202

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

    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.

    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.

    Status: Recruiting

    et mf pv

    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

    Status: Not Yet Recruiting

    et mf pv

    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.

     

    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.

    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.

    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.

    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.

    Status: Active, Not Recruiting

  • 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.