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Primary MyelofibrosisPhase 2NCT02584777

A Phase II Non-Controlled, Open-Label, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Pacritinib in Myelofibrosis

A Phase 2 study testing Pacritinib in people with Primary Myelofibrosis, run by Baxalta now part of Shire.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial summary

To evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of pacritinib in Asian subjects with myelofibrosis (MF), which includes primary MF (PMF), post-polycythemia vera MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Primary Myelofibrosis
What is being tested?
Pacritinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
No sites listed yet
Study dates
30 Nov 2015to 31 Aug 2020
Phase
Phase 2
Are there results?
None posted in the registry

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

  • Intermediate-1, intermediate-2, or high-risk PMF, PPV-MF or PET-MF as based on The Dynamic International Prognostic Scoring System (DIPSS) criteria
  • Palpable splenomegaly ≥5 cm below the LCM in midclavicular line by physical examination
  • TSS ≥13 on the MPN-SAF TSS 2.0, not including the inactivity question, based on a single assessment during screening visit
  • Age ≥18 years old at the time of screening (or minimum age of legal consent consistent with local regulations, if minimum is >18 years of age)
  • ECOG performance status 0 to 3
  • Peripheral blast count <10%
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  • Absolute neutrophil count >500/μL
  • Participants who are platelet or RBC transfusion dependent are eligible
  • Adequate liver and renal function, defined by liver transaminases (AST/serum glutamic oxaloacetic transaminase [SOOT] and alanine aminotransferase [ALT]/serum glutamic pyruvic transaminase [SGPT]) ≤3 × 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
  • At least 6 months from prior splenic irradiation
  • At least 12 months from prior 32P therapy
  • At least 1 week since prior treatment (most recent dose) with a potent CYP3A4 inhibitor or inducer
  • At least 4 weeks since any experimental treatment for PMF, PPV-MF, or PET-MF
  • At least 2 weeks since any treatment for PMF, PPV-MF, or PET-MF
  • If fertile, both males and females must agree to use effective birth control.
  • Able to understand and willing to complete symptom assessments using a patient-reported outcomes instrument and comply with treatment and study procedures of the protocol
  • Able to understand and willing to sign the informed consent form (ICF)
  • Participant is willing and able to comply with the requirements of the protocol

Who cannot join

  • Any GI or metabolic condition that could interfere with absorption of oral medication
  • Life expectancy <6 months
  • Prior treatment with a JAK2 inhibitor
  • Completed ASCT, or are eligible for and willing to complete ASCT
  • History of splenectomy or planning to undergo splenectomy
  • 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
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  • 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 non-metastatic prostate cancer with negative prostate specific antigen, in situ breast carcinoma after complete surgical resection, or superficial transitional cell bladder carcinoma
  • Inflammatory or chronic functional bowel disorder, such as Crohn's disease, inflammatory bowel disease, chronic diarrhea, or constipation
  • Clinically symptomatic and uncontrolled cardiovascular disease
  • History of any of the following within 6 months prior to first dose of pacritinib: myocardial infarction, severe/unstable angina, or symptomatic congestive heart failure
  • New York Heart Association Class II, III, or IV congestive heart failure
  • Participants with NCI CTCAE (version 4.03) Grade 2 cardiac arrhythmias may be considered for inclusion, with the approval of the medical monitor, if the arrhythmias are stable, asymptomatic and unlikely to affect participant safety. Participants will be excluded if they have ongoing cardiac dysrhythmias of NCI CTCAE Grade ≥3, QTc prolongation >450 ms, or other conditions 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], or family history of long QT interval syndrome)
  • Erythropoietic agent within 28 days prior to first dose of pacritinib
  • Thrombopoietic agent within 14 days prior to first dose of pacritinib
  • Known seropositivity for human immunodeficiency virus or syphilis, or known active hepatitis A, B or C virus infection
  • Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study
  • Participant is a family member or employee of the investigator
  • If female, participant is pregnant or breastfeeding at the time of enrollment. Even if breastfeeding can be discontinued, the participant should not be enrolled in the study

About this study

Official title: A Phase II, Prospective, Non-Controlled, Open-Label, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Pacritinib in Asian Subjects With Primary Myelofibrosis, Post-Polycythemia Vera Myelofibrosis or Post- Essential Thrombocythemia Myelofibrosis

To evaluate the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of pacritinib in Asian subjects with myelofibrosis (MF), which includes primary MF (PMF), post-polycythemia vera MF (PPV-MF) or post-essential thrombocythemia MF (PET-MF).

Being tested
Pacritinib
Keywords
Post-essential thrombocythemia myelofibrosis, Post-Polycythemia Vera Myelofibrosis
Medical subject headings
Primary Myelofibrosis

What participants receive

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

  • PacritinibBiological

    QD (Once a day)

Study groups in NCT02584777
Study groupRoleWhat they receive
PacritinibExperimentalOral administration
Receives: Biological: Pacritinib

What is being measured

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

  1. Proportion of participants achieving a ≥35% reduction in spleen volume

    Measured by MRI or CT scan

    Measured: Baseline to Week 24

Secondary outcomes (17)

  1. Proportion of participants with ≥50% reduction in total symptom score (TSS)

    Measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) TSS 2.0

    Measured: Baseline to Week 24

  2. Proportion of participants with baseline platelet count <100,000/μL achieving ≥35% reduction in spleen volume

    Measured by MRI or CT scan

    Measured: Baseline to Week 24

  3. Proportion of participants with baseline platelet count <100,000/μL achieving ≥50% reduction in total symptom score (TSS)

    Measured: Baseline to Week 24

  4. Proportion of participants with baseline platelet count <50,000/μL achieving ≥35% reduction in spleen volume

    Measured by MRI or CT scan

    Measured: Baseline to Week 24

  5. Proportion of participants with baseline platelet count <50,000/μL achieving ≥50% reduction in total symptom score (TSS)

    Measured: Baseline to Week 24

  6. Clinically significant adverse events (AEs)

    Measured: Throughout the study period of approximately 5 years

  7. Clinically significant changes in laboratory results

    Measured: Throughout the study period of approximately 5 years

  8. Clinically significant changes in vital signs

    Measured: Throughout the study period of approximately 5 years

  9. Clinically significant changes in electrocardiograms (ECGs)

    Measured: Throughout the study period of approximately 5 years

  10. Pacritinib pharmacokinetic (PK) parameter: maximum observed concentration (Cmax)

    Measured: Baseline; weeks 3, 12 & 24

  11. Pacritinib pharmacokinetic (PK) parameter: Time of maximum observed concentration (Tmax)

    Measured: Baseline; weeks 3, 12 & 24

  12. Pacritinib pharmacokinetic (PK) parameter: minimum observed concentration (Cmin)

    Measured: Baseline; weeks 3, 12 & 24

  13. Pacritinib pharmacokinetic (PK) parameter: area under the concentration curve (AUC)

    Measured: Baseline; weeks 3, 12 & 24

  14. Pacritinib pharmacokinetic (PK) parameter: apparent volume of distribution (V/F)

    Measured: Baseline; weeks 3, 12 & 24

  15. Pharmacodynamic parameter: Maximum observed effect (Emax)

    Measured: Baseline; weeks 3, 12 & 24

  16. Pharmacodynamic parameter: time of maximum observed effect (tEmax)

    Measured: Baseline; weeks 3, 12 & 24

  17. Pharmacodynamic parameter: area under the effect curve (AUEC)

    Measured: Baseline; weeks 3, 12 & 24

Timeline

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

  1. First posted23 Oct 2015 (estimated)
  2. Study start30 Nov 2015
  3. Primary completion30 Sep 2017
  4. Study completion31 Aug 2020

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02584777
Sponsor's protocol ID
381401
Status last verified
May 2021
First submitted
21 Oct 2015
Last updated
5 May 2021
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Pacritinib in people with Primary Myelofibrosis, run by Baxalta now part of Shire.

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 18 inclusion and 18 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: withdrawn, as of the sponsor's last update on 5 May 2021. Status can change between updates, so confirm with the study contact before making plans.

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?

The study was withdrawn before enrolling participants, so there are no results.

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?

Proportion of participants achieving a ≥35% reduction in spleen volume (time frame: Baseline to Week 24). 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 NCT02584777.

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 5 May 2021.

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