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Primary MyelofibrosisPhase 2NCT04884191

Phase 2 Study: An Open-Label, Randomized, Phase 2 Dose-Finding Study of Pacritinib in Patients With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis, or Post- Essential Thrombocythemia Myelofibrosis Previously Treated With Ruxolitinib

A Phase 2 study testing Pacritinib in people with Primary Myelofibrosis and Post-Polycythemia Vera Myelofibrosis, run by CTI BioPharma at 62 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial summary

This was an open-label, randomized, dose-finding study in patients with primary or secondary MF (Dynamic International Prognostic Scoring System [DIPSS] risk score of Intermediate-1 to High-Risk) who were previously treated with ruxolitinib. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
Pacritinib
Could I receive a placebo?
No, everyone receives an active treatment
How many people
165 participants
Where
62 sitesin 8 countries
Study dates
31 Jul 2017to 4 Sep 2019
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

  • PMF, PPV-MF, or PET-MF (as defined by Tefferi and Vardiman 2008)
  • DIPSS Intermediate-1, Intermediate -2, or High-risk (Passamonti et al 2010)
  • Prior ruxolitinib treatment with failure to benefit or intolerance as defined by at least one of the following:
  • Treatment for ≥3 months with inadequate efficacy response defined as <10% SVR by MRI or <30% decrease from baseline in spleen length by physical examination or regrowth to these parameters following an initial response; and/or
  • Treatment for ≥28 days complicated by either
  • Development of a red blood cell (RBC) transfusion requirement (at least 2 units/month for 2 months) ii. National Cancer Institute (NCI) CTCAE grade ≥3 AEs of thrombocytopenia, anemia, hematoma, and/or hemorrhage while being treated with a dosage of <20 mg BID
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  • Palpable splenomegaly ≥5 cm below the lower costal margin (LCM) in the midclavicular line as assessed by physical examination
  • TSS of ≥10 on the MPN-SAF TSS 2.0 or patients with a single symptom score of ≥5 or 2 symptoms of ≥3, including only the symptoms of left upper quadrant pain, bone pain, itching, or night sweats
  • Age ≥18 years old
  • Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
  • Peripheral blast count of <10% throughout the Screening period
  • Absolute neutrophil count of >500/μL
  • Adequate liver and renal function, defined by liver transaminases (aspartate aminotransferase [AST]/serum glutamic-oxaloacetic transaminase [SGOT] and alanine aminotransferase [ALT]/serum glutamic-pyruvic transaminase [SGPT]), ≤3 × the upper limit of normal (ULN) (AST/ALT ≤5 × ULN, if transaminase elevation is related to MF), direct bilirubin ≤4× ULN, and creatinine ≤2.5 mg/dL
  • Adequate coagulation function, defined by prothrombin time (PT)/international normalized ratio (INR), partial thromboplastin time (PTT), or thrombin time (TT) of ≤1.5 × ULN
  • Left ventricular cardiac ejection fraction of ≥45% by echocardiogram or multigated acquisition (MUGA) scan
  • If fertile, willing to use effective birth control methods during the study
  • Willing to undergo and able to tolerate frequent MRI or CT scan assessments during the study
  • Able to understand and willing to complete symptom assessments using a PRO instrument
  • Provision of informed consent

Who cannot join

  • Life expectancy <6 months
  • Completed allogeneic stem cell transplant (allo-SCT) or are eligible for and willing to complete allo-SCT
  • History of splenectomy or planning to undergo splenectomy
  • Splenic irradiation within the last 6 months
  • Previously treated with pacritinib
  • Patients receiving high-dose ruxolitinib (more than 10 mg BID or 20 mg QD) who cannot tolerate tapering down ruxolitinib to 10 mg BID or less prior to the first dose of pacritinib
Show all 23Show fewer
  • Treatment with anticoagulation or antiplatelet agents, except for aspirin dosages of ≤100 mg per day, within the last 2 weeks
  • Treatment with a strong CYP3A4 inhibitor or a strong cytochrome P450 inducer within the last 2 weeks
  • Treatment with medications that can prolong the QTc interval within the last 2 weeks
  • Treatment with an experimental therapy within the last 28 days
  • Significant recent bleeding history defined as NCI CTCAE grade ≥2 within the last 3 months, unless precipitated by an inciting event (eg, surgery, trauma, or injury)
  • Any history of CTCAE grade ≥2 non-dysrhythmia cardiac conditions within the last 6 months. Patients with asymptomatic grade 2 non-dysrhythmia cardiac conditions may be considered for inclusion, with the approval of the medical monitor, if stable and unlikely to affect patient safety.
  • New York Heart Association Class II, III, or IV congestive heart failure
  • Any history of CTCAE grade ≥2 cardiac dysrhythmias within the last 6 months. Patients with non-QTc CTCAE grade 2 cardiac dysrhythmias may be considered for inclusion, with the approval of the medical monitor, if the dysrhythmias are stable, asymptomatic, and unlikely to affect patient safety.
  • QTc prolongation >450 ms based on the mean of triplicate ECGs or other factors that increase the risk for QT interval prolongation (eg, heart failure, hypokalemia [defined as serum potassium <3.0 mEq/L that is persistent and refractory to correction], family history of long QT interval syndrome, or concomitant use of medications that may prolong QT interval)
  • Any active gastrointestinal or metabolic condition that could interfere with absorption of oral medication
  • Active or uncontrolled inflammatory or chronic functional bowel disorder such as Crohn's Disease, inflammatory bowel disease, chronic diarrhea, or constipation
  • Other malignancy within the last 3 years, other than curatively treated basal cell or squamous cell skin cancer, carcinoma in situ of the cervix, organ-confined or treated nonmetastatic prostate cancer with negative prostate-specific antigen, in situ breast carcinoma after complete surgical resection, or superficial transitional cell bladder carcinoma
  • Uncontrolled intercurrent illness, including, but not limited to, ongoing active infection or psychiatric illness or social situation that, in the judgment of the treating physician, would limit compliance with study requirements
  • Known seropositivity for human immunodeficiency virus
  • Known active hepatitis A, B, or C virus infection
  • Women who are pregnant or lactating
  • Concurrent enrollment in another interventional trial

Trial locations and contacts

62 sites in 8 countries.

62 sites in 8 countries

United States33 sites

  • University of Michigan Comprehensive Cancer Center

    Ann Arbor, Michigan, 48109

  • Saint Agnes Hospital

    Baltimore, Maryland, 21229

  • Dana-Farber Cancer Institute

    Boston, Massachusetts, 02215

  • Northwestern University Feinberg School of Medicine

    Chicago, Illinois, 60611

  • The University of Chicago Medical Center

    Chicago, Illinois, 60637

  • Cleveland Clinic

    Cleveland, Ohio, 44195

  • The Ohio State University Comprehensive Cancer Center

    Columbus, Ohio, 43210

  • City of Hope

    Duarte, California, 91010

  • Duke University Hospital

    Durham, North Carolina, 27710

  • Florida Cancer Specialists & Research Institute

    Fort Myers, Florida, 33901

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601

  • The University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

  • USC Norris Comprehensive Cancer Center

    Los Angeles, California, 90033

  • UCLA Jonsson Comprehensive Cancer Center

    Los Angeles, California, 90095

  • Tennessee Oncology

    Nashville, Tennessee, 37203

  • Vanderbilt-Ingram Cancer Center

    Nashville, Tennessee, 37232

  • Yale School of Medicine

    New Haven, Connecticut, 06520

  • Ochsner Medical Center

    New Orleans, Louisiana, 70121

  • Weill Cornell Medical College

    New York, New York, 10021

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029

  • Columbia University

    New York, New York, 10032

  • Memorial Sloan-Kettering Cancer Center

    New York, New York, 10065

  • Mayo Clinic Hospital

    Phoenix, Arizona, 85054

  • University of Rochester

    Rochester, New York, 14642

  • University of Utah School of Medicine

    Salt Lake City, Utah, 84112

  • University of Texas Health Science Center at San Antonio School of Medicine

    San Antonio, Texas, 78229

  • Fred Hutchinson Cancer Research Center

    Seattle, Washington, 98109

  • Washington University School of Medicine in St. Louis

    St Louis, Missouri, 63110

  • Florida Cancer Specialists & Research Institute

    St. Petersburg, Florida, 33705

  • Stanford Cancer Institute

    Stanford, California, 94305

  • Medical Faculty Associates, Inc.

    Washington D.C., District of Columbia, 20037

  • Florida Cancer Specialists & Research Institute

    West Palm Beach, Florida, 33401

  • University of Kansas Cancer Center

    Westwood, Kansas, 66205

Other trials in United States

France7 sites

  • CHU Hopital Sud

    Amiens, 80054

  • Centre Hospitalier Regional Universitaire de Lille - Hopital Claude Huriez

    Lille, 59037

  • CHU de Nimes - Hopital Universitaire Caremeau

    Nîmes, 30029

  • Hôpital Saint-Louis

    Paris, 75010

  • CHU Hopitaux de Bordeaux - Hôpital Haut-Lévêque

    Pessac, 33604

  • Centre Hospitalier Lyon-Sud

    Pierre-Bénite, 69495

  • Centre Hospitalier Universitaire de Toulouse- Hôpital Purpan

    Toulouse, 31059

Other trials in France

United Kingdom6 sites

  • Beatson West of Scotland Cancer Center

    Glasgow, G12 0YN

  • Barts Health NHS Trust - The Royal London Hospital

    London, E1 2ES

  • Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital

    London, SE1 9RT

  • Imperial College Healthcare NHS Trust - Hammersmith Hospital

    London, W12 0HS

  • The Christie NHS Foundation Trust

    Manchester, M20 4BX

  • Oxford University Hospitals NHS Trust - Churchill Hospital

    Oxford, OX3 7LE

Other trials in United Kingdom

South Korea4 sites

  • Yeungnam University Medical Center

    Daegu, 42415

  • Severance Hospital

    Seoul, 03722

  • Samsung Medical Center

    Seoul, 06351

  • The Catholic University of Korea, Seoul St. Mary's Hospital

    Seoul, 06591

Other trials in South Korea

Spain4 sites

  • Hospital del Mar

    Barcelona, 08003

  • Hospital Clínic de Barcelona

    Barcelona, 08036

  • Hospital Universitario Ramón y Cajal

    Madrid, 28034

  • Clínica Universidad de Navarra

    Pamplona, 31008

Other trials in Spain

Hungary3 sites

  • SE AOK I. sx. Belgyogyaszati Klinika

    Budapest, 1083

  • Debreceni Egyetem Klinikai Központ

    Debrecen, 4032

  • Somogy Megyei Kaposi Mór Oktató Kórház

    Kaposvár, 7400

Other trials in Hungary

Italy3 sites

  • Azienda Ospedaliero-Universitaria Careggi

    Florence, 50134

  • Istituto Scientifico Romagnolo per lo Studio e la Cura Dei Tumori

    Meldola, 47014

  • ASST Monza - Ospedale San Gerardo

    Monza, 20900

Other trials in Italy

Sweden2 sites

  • Skane University Hospital Lund

    Lund, 22185

  • Orebro University Hospital

    Örebro, 70185

Other trials in Sweden

About this study

This was an open-label, randomized, dose-finding study in patients with primary or secondary MF (Dynamic International Prognostic Scoring System [DIPSS] risk score of Intermediate-1 to High-Risk) who were previously treated with ruxolitinib. The study was designed to support a pacritinib dosage selection decision with evaluation of 3 dosages.

Being tested
Pacritinib
Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • PacritinibDrug

    Pacritinib

Study groups in NCT04884191
Study groupRoleWhat they receive
Pacritinib 100 mg QDExperimentalNot described
Receives: Drug: Pacritinib
Pacritinib 100 mg BIDExperimentalNot described
Receives: Drug: Pacritinib
Pacritinib 200 mg BIDExperimentalNot described
Receives: Drug: Pacritinib

What is being measured

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

  1. Spleen Volume Reduction Response (≥ 35%)

    Number of patients achieving a ≥ 35% spleen volume reduction (SVR) as measured by magnetic resonance imaging (MRI, preferred) or computed tomography (CT) scans

    Measured: From Baseline to Weeks 12 and 24

  2. Percent Change in Spleen Volume

    Percent change from baseline

    Measured: From Baseline to Weeks 12 and 24

  3. Total Symptom Score Analysis

    Proportion of patients with ≥ 50% reduction in Total Symptom Score from baseline as assessed by the validated PRO instrument MPN-SAF TSS 2.0

    Measured: From Baseline to Weeks 12 and 24

  4. Patient Global Impression Assessment

    Number of patients with improvement in PGIA. The Patient Global Impression Assessment questionnaire was completed at the end of Week 12 and end of Week 24. The scores were summarized by treatment group at each visit.

    Measured: From Baseline to Weeks 12 and 24

Secondary outcomes (4)

  1. Spleen Length Reduction

    Rate of reduction in spleen length from baseline

    Measured: From Baseline to Weeks 24

  2. Frequency of RBC's or Platelet Transfusions

    Number of patients

    Measured: At week 24

  3. Eastern Cooperative Oncology Group Performance Status

    0 = Fully active, able to carry on all pre-disease performance without restriction

    1. = Restricted in physically strenuous activity but ambulatory and able to carry out work of a light or sedentary nature, e.g., light house work, office work
    2. = Ambulatory and capable of all selfcare but unable to carry out any work activities; up and about more than 50% of waking hours
    3. = Capable of only limited selfcare; confined to bed or chair more than 50% of waking hours
    4. = Completely disabled; cannot carry on any selfcare; totally confined to bed or chair
    5. = Dead

    Measured: At weeks 4, 12, 24, and 30 days post End-of-Treatment visit

  4. Number of Participants With Adverse Events

    Measured: Randomization through 30 days post End-of-Treatment visit

Timeline

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

  1. First posted12 May 2021
  2. Study start31 Jul 2017
  3. Primary completion4 Sep 2019
  4. Study completion4 Sep 2019

Resources

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
Randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
165 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04884191
Sponsor's protocol ID
PAC203
Status last verified
May 2022
First submitted
10 May 2021
Last updated
1 Jun 2022
FDA-regulated drug
Yes
FDA-regulated device
No

Publications

  • Verstovsek S, Mesa R, Talpaz M, Kiladjian JJ, Harrison CN, Oh ST, Vannucchi AM, Rampal R, Scott BL, Buckley SA, Craig AR, Roman-Torres K, Mascarenhas JO. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia. Haematologica. 2022 Jul 1;107(7):1599-1607. doi: 10.3324/haematol.2021.279415.

    PubMed 34551507

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pacritinib in people with Primary Myelofibrosis and Post-Polycythemia Vera Myelofibrosis, run by CTI BioPharma at 62 sites in 8 countries.

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 19 inclusion and 23 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: finished, as of the sponsor's last update on 1 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 62 sites in United States, France, United Kingdom, South Korea, Spain and 3 more countries. 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 1 Jun 2022). 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?

Spleen Volume Reduction Response (≥ 35%) (time frame: From Baseline to Weeks 12 and 24). The study lists 4 primary outcomes in all. 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 NCT04884191, which also lists 1 linked publication.

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 1 Jun 2022.

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