Myelofibrosis (MF)PACERNCT07774455
Pacritinib Effectiveness in Real-world Settings
An observational study of Myelofibrosis (MF), run by Swedish Orphan Biovitrum at 5 sites in United States.
Trial summary
This study aims to evaluate real-world treatment patterns and effectiveness of pacritinib, including hematologic and clinical outcomes, and survival through a site-based retrospective chart review of medical records of patients with MF. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelofibrosis (MF)
- What is being tested?
- Not applicable- observational study
- How many people
- 60 participants
- Where
- 5 sitesUnited States
- Study dates
- 13 Aug 2026to 10 Mar 2027
- Phase
- Observational
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
Study population: Patients with platelet counts ≥50 x 10^9/L at pacritinib initiation from a total of 20-25 centers across the US over a 4-year time period (2022 to 2026).
Who may be able to join
- Adult patients must be ≥18 years of age at the index date
- Patients diagnosed with MF with platelet counts ≥50 x 109/L at the time of treatment initiation with pacritinib. If multiple values are available within 30 days prior to initiating treatment with pacritinib will use the value closest to index date
- Patients will be required to have ≥1 month of treatment with pacritinib and ≥6 months of observation from the start of pacritinib unless the patient died within 6 months of starting pacritinib
- If peripheral blasts were evaluated prior to index, they must be <10%. If not evaluated, patients will be included unless a healthcare provider has indicated in the medical record that there is a concern that the patient has transitioned to accelerated/blast phase disease at or prior to index
- According to local regulations, waivers of consent will be sought for study patients from the appropriate regulatory authorities and/or the independent ethics committee (IEC)/institutional review board (IRB). For patients not covered by waivers of consent, signed and dated informed consent provided by the patient, or the patient's legally authorized representative(s) for patients under the legal age (with patient assent, as applicable), should be obtained before any study-related activities are undertaken.
Who cannot join
- Diagnosis of acute myeloid leukemia prior to index
- Physician-concern that the patient has transitioned to accelerated/blast phase disease if peripheral blasts were not evaluated
- Treated with 2 or more JAK inhibitors prior to initiating treatment with pacritinib
- Treated with pacritinib in a clinical trial setting
Trial locations and contacts
5 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Clinical Operations Lead contact
5 sites in 1 country
United States5 sites, 5 recruiting
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Coltoff, coltoff@musc.edu
- Recruiting
The Ohio State University
Columbus, Ohio, 43210
Srisuwananukorn, andrew.srisuwananukorn@osumc.edu
- Recruiting
OneOncology Research Network
Nashville, Tennessee, 37219
Favaro, (704) 342-1900, jfavaro@oncologycharlotte.com
- Recruiting
Yale School of Medicine
New Haven, Connecticut, 06511
Podoltsev, nikolai.podoltsev@yale.edu
- Recruiting
Tampa General Hospital
Tampa, Florida, 33606
Gesiotto, (813) 844-9575, qgesiotto@tgh.org
About this study
Official title: Pacritinib Effectiveness in Real-world Settings (PACER)
This study aims to evaluate real-world treatment patterns and effectiveness of pacritinib, including hematologic and clinical outcomes, and survival through a site-based retrospective chart review of medical records of patients with MF.
Read the detailed description
This is a multicenter, observational, retrospective chart review study of patients with Myelofibrosis (MF) who received treatment with pacritinib in routine clinical settings with platelet count ≥50 x 109/L at the time of treatment initiation with pacritinib. This study will be conducted entirely through medical chart abstraction; all data will be taken from the patient's medical record, with no additional assessments.
- Conditions
- Myelofibrosis (MF)
- Being tested
- Not applicable- observational study
- Keywords
- Myelofibrosis, Pacritinib, Sobi, Sobi.PACRIT-RWE-101, PACER
- Medical subject headings
- Primary Myelofibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Not applicable- observational studyOther
Not Applicable - Observational Study
What is being measured
The primary outcome is the main question the study is designed to answer.
≥50% spleen length reduction
Measured: From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months.
≥20% spleen length reduction
Measured: From the Index Date to Week 24 or best response in spleen reduction, assessed up to 49 months.
Change in spleen length
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Improvement in spleen size category
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Among those with ≥1 MF-related symptom at index Symptom-specific resolution
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Among those with ≥1 MF-related symptom at index Decrease in total number of MF-symptoms
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Among those with ≥1 MF-related symptom at index Change in total number of MF-symptoms
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Change in blood counts (i.e., white blood cells [WBC], platelets, absolute neutrophil counts, peripheral blast percentage)
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index RBC-TI - absence of RBC transfusions over any 12-week period
Measured: Index to Week 24 and at other timepoints, assessed up to 49 months.
Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index ≥50% reduction in number of RBC units transfused
Measured: Index to Week 12 & Index to Week 24
Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index ≥50% reduction in monthly rate of RBC units transfused
Measured: Index to Week 12 & Index to Week 24
Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index change in the number of RBC units transfused
Measured: Index to Week 24
Among those who are non-transfusion independent (TI) (≥1 RBC transfusion in prior 12 weeks) at index change in the monthly rate of RBC units transfused
Measured: Index to Week 24
Among those with a platelet count <100 x 109/L at index date Absolute increase in platelet counts ≥30 x 109/L without a platelet transfusion in the prior 2 days
Measured: Index to best response, assessed up to 49 months.
Among patients with Hb <10 g/dL at index and who are RBC-transfusion dependent (TD) (≥3 RBC units transfused in prior 12 weeks) at index: achieved RBC-TI
Measured: 12-Week Period with no RBC transfusions administered during the period
Among patients with Hb <10 g/dL at index and who are not RBC-transfusion dependent (TD) (≥3 RBC units transfused in prior 12 weeks) at index: ≥1.5 g/dL increase in average Hb
Measured: 12-Week Period with no RBC transfusions administered during the period
Among patients who have IWG Hb Minor Response at index and are RBC-TD at index ≥50% reduction in RBC transfusion
Measured: 12-Week Period without being fully RBC-TI
Among patients who have IWG Hb Minor Response at index and are not RBC-TD at index ≥1 g/dL increase in average Hb
Measured: 12-Week Period without being fully RBC-TI
Physician-reported progression to leukemia (AML)
Measured: Index to Follow-up, assessed up to 49 months.
Overall survival
Measured: Index to Follow-up, assessed up to 49 months.
Leukemia-free Survival
Measured: Index to Follow-up, assessed up to 49 months.
Type, dose, and number of MF-directed therapies administered
Measured: At Index (Baseline)
Reasons for treatment switch from a prior MF-directed therapy to pacritinib
Measured: At Index (Baseline)
Method used to switch from prior JAKi to pacritinib
Measured: At Index (Baseline)
Physician reported (or Physician confirmed) JAKi withdrawal syndrome after switching to pacritinib from a different JAKi
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Pacritinib line of therapy, treatment dose and frequency, changes in dose/and or frequency, frequency of dose changes and reasons for change
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Duration of treatment with pacritinib
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Concomitant MF-related medications
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Reason for discontinuation of pacritinib
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Subsequent MF-therapy following discontinuation of pacritinib and time to next Treatment
Measured: Prior to (index date) and after initiation of pacritinib (up to 49 months).
Demographic characteristics include age, sex, race/ethnicity, geographic region, and insurance type
Measured: At Index (Baseline)
Body mass index (BMI) for all participants enrolled
Measured: At Index (Baseline)
Receipt of allo-HSCT among patients referred to allo-HSCT
Measured: Index to Week 24
Duration of treatment with pacritinib prior to allo-HSCT
Measured: Index to Week 24
Dose of pacritinib prior to allo-HSCT
Measured: Index to Week 24
Discontinuation of pacritinib prior to allo-HSCT
Measured: Index to Week 24
Treatment with pacritinib during conditioning
Measured: Index to Week 24
Dose of pacritinib during condition
Measured: Index to Week 24
Duration of treatment with pacritinib after allo-HSCT
Measured: Index to Week 24
Dose of pacritinib after allo-HSCT
Measured: Index to Week 24
Type of MF for all participants enrolled
Measured: At Index (Baseline)
Comorbidities for all participants enrolled
Measured: At Index (Baseline)
MF Risk Category for all participants enrolled
Measured: At Index (Baseline)
Bone Marrow Fibrosis Grade for all participants enrolled
Measured: At Index (Baseline)
Type of MF Mutations for all participants enrolled
Measured: At Index (Baseline)
Variant Allele Frequency (VAF) for all participants enrolled
Measured: At Index (Baseline)
Number of Driver Mutations for all participants enrolled
Measured: At Index (Baseline)
Number of High-Risk Mutations for all participants enrolled
Measured: At Index (Baseline)
Karyotype for all participants enrolled
Measured: At Index (Baseline)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2026
- Study start13 Aug 2026
- Primary completion10 Mar 2027 (estimated)
- Study completion10 Mar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelofibrosis (MF)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Retrospective
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- IQVIA RDS Inc.
- Responsible party
- Sponsor
- Investigators
- Raman Garcha, MD, Principal investigator, Swedish Orphan Biovitrum
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07774455
- Sponsor's protocol ID
- Sobi.PACRIT-RWE-101
- Acronym
- PACER
- Status last verified
- Oct 2026
- First submitted
- 22 Jul 2026
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No