- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02584777
A Phase II Non-Controlled, Open-Label, Efficacy, Safety, Pharmacokinetic, and Pharmacodynamic Study of Pacritinib in Myelofibrosis
May 3, 2021 updated by: Baxalta now part of Shire
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).
Study Overview
Study Type
Interventional
Phase
- Phase 2
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (ADULT, OLDER_ADULT)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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%
- 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
Exclusion Criteria:
- 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
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NA
- Interventional Model: SINGLE_GROUP
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Pacritinib
Oral administration
|
QD (Once a day)
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of participants achieving a ≥35% reduction in spleen volume
Time Frame: Baseline to Week 24
|
Measured by MRI or CT scan
|
Baseline to Week 24
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of participants with ≥50% reduction in total symptom score (TSS)
Time Frame: Baseline to Week 24
|
Measured by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) TSS 2.0
|
Baseline to Week 24
|
Proportion of participants with baseline platelet count <100,000/μL achieving ≥35% reduction in spleen volume
Time Frame: Baseline to Week 24
|
Measured by MRI or CT scan
|
Baseline to Week 24
|
Proportion of participants with baseline platelet count <100,000/μL achieving ≥50% reduction in total symptom score (TSS)
Time Frame: Baseline to Week 24
|
Baseline to Week 24
|
|
Proportion of participants with baseline platelet count <50,000/μL achieving ≥35% reduction in spleen volume
Time Frame: Baseline to Week 24
|
Measured by MRI or CT scan
|
Baseline to Week 24
|
Proportion of participants with baseline platelet count <50,000/μL achieving ≥50% reduction in total symptom score (TSS)
Time Frame: Baseline to Week 24
|
Baseline to Week 24
|
|
Clinically significant adverse events (AEs)
Time Frame: Throughout the study period of approximately 5 years
|
Throughout the study period of approximately 5 years
|
|
Clinically significant changes in laboratory results
Time Frame: Throughout the study period of approximately 5 years
|
Throughout the study period of approximately 5 years
|
|
Clinically significant changes in vital signs
Time Frame: Throughout the study period of approximately 5 years
|
Throughout the study period of approximately 5 years
|
|
Clinically significant changes in electrocardiograms (ECGs)
Time Frame: Throughout the study period of approximately 5 years
|
Throughout the study period of approximately 5 years
|
|
Pacritinib pharmacokinetic (PK) parameter: maximum observed concentration (Cmax)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pacritinib pharmacokinetic (PK) parameter: Time of maximum observed concentration (Tmax)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pacritinib pharmacokinetic (PK) parameter: minimum observed concentration (Cmin)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pacritinib pharmacokinetic (PK) parameter: area under the concentration curve (AUC)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pacritinib pharmacokinetic (PK) parameter: apparent volume of distribution (V/F)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pharmacodynamic parameter: Maximum observed effect (Emax)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pharmacodynamic parameter: time of maximum observed effect (tEmax)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
|
Pharmacodynamic parameter: area under the effect curve (AUEC)
Time Frame: Baseline; weeks 3, 12 & 24
|
Baseline; weeks 3, 12 & 24
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (ACTUAL)
November 30, 2015
Primary Completion (ACTUAL)
September 30, 2017
Study Completion (ACTUAL)
August 31, 2020
Study Registration Dates
First Submitted
October 21, 2015
First Submitted That Met QC Criteria
October 21, 2015
First Posted (ESTIMATE)
October 23, 2015
Study Record Updates
Last Update Posted (ACTUAL)
May 5, 2021
Last Update Submitted That Met QC Criteria
May 3, 2021
Last Verified
May 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- 381401
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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