NCT03645824

Brief Summary

The only curative treatment for patients with myelofibrosis (MF) is allogeneic stem cell transplantation (SCT). Treatment with JAK2 inhibitors like pacritinib improves condition of MF patients, decreases spleen size and might diminish graft-versus-host disease (GvHD), thereby improving the outcome of SCT.

Trial Health

78
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
61

participants targeted

Target at P50-P75 for phase_2

Timeline
7mo left

Started Jun 2018

Longer than P75 for phase_2

Geographic Reach
2 countries

12 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress93%
Jun 2018Feb 2027

First Submitted

Initial submission to the registry

March 26, 2018

Completed
2 months until next milestone

Study Start

First participant enrolled

June 4, 2018

Completed
3 months until next milestone

First Posted

Study publicly available on registry

August 24, 2018

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2023

Completed
3.6 years until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2027

Expected
Last Updated

August 22, 2022

Status Verified

August 1, 2022

Enrollment Period

5.1 years

First QC Date

March 26, 2018

Last Update Submit

August 19, 2022

Conditions

Myelofibrosis

Keywords

Hemato-OncologyAllogeneic Stem Cell TransplantationPacritinibJAK2 inhibitorMyelofibrosis

Outcome Measures

Primary Outcomes (1)

  • Proportion of patients receiving allo-SCT, with failure within or at day 180 post-transplant.

    Failure can be defined by one of the following parameters: * Primary graft failure * Acute graft versus host disease grade 3-4 * Secondary graft failure * Death, from any cause

    2 years

Secondary Outcomes (6)

  • Adverse events

    5 years

  • Progression free survival

    5 years

  • Overall survival

    5 years

  • Relapse mortality

    5 years

  • Non-relapse mortality

    5 years

  • +1 more secondary outcomes

Study Arms (1)

Pacritinib treatment befor allo-SCT

EXPERIMENTAL

The effect of pacritinib treatment during 3 to 4 cycles before allo-SCT on engraftment 6 months (day +180) post allo-SCT in MF patients.

Drug: Pacritinib

Interventions

PacritinibDRUG

Patients receive up to 4 cycles of pacritinib before allo-SCT

Pacritinib treatment befor allo-SCT

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Patients with a confirmed diagnosis of post-ET, post-PV or primary myelofibrosis
  • Intermediate-2 or high-risk according to DIPSS plus (Appendix E)
  • Age 18-70 years inclusive
  • WHO performance status 0-2 (Appendix C)
  • All men and women of childbearing potential must agree to use adequate contraception during the study
  • Written informed consent
  • Patient is capable of giving informed consent

You may not qualify if:

  • Any GI or metabolic condition (e.g. inflammatory or chronic functional bowel disorder such as Crohn's Disease, Inflammatory Bowel Disease, chronic diarrhea or constipation) that could interfere with absorption of oral medication
  • Left ventricular cardiac ejection fraction of ≤ 45% by echocardiogram or multigated acquisition (MUGA) scan
  • Impaired 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 clearance ˂ 40 ml/min.
  • Impaired coagulation function, defined by prothrombin time (PT)/international normalized ratio (INR), partial thromboplastin time (APTT)\>1.5 x ULN.
  • Severe pulmonary dysfunction (CTCAE grade III-IV, see appendix D)
  • Treatment with a potent strong CYP3A4 inhibitor or a strong cytochrome P450 (CYP450) inducer within the last 2 weeks
  • Treatment with anticoagulation or antiplatelet agents, except for aspirin dosages of ≤100 mg per day, within the last 2 weeks
  • New York Heart Association Class II, III, or IV congestive heart failure
  • QTc prolongation \>450 ms as assessed by ECG and corrected by Federicia method or other factors that increase the risk for QT interval prolongation (e.g., 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)
  • Significant recent bleeding history defined as NCI CTCAE grade ≥2 within the last 3 months, unless precipitated by an inciting event (e.g., surgery, trauma, injury)
  • Patients with active, uncontrolled infections
  • Patients known to be HIV (human immunodeficiency virus)-positive
  • Active hepatitis A, B or C
  • History of active malignancy during the past 3 years, except basal carcinoma of the skin or stage 0 cervical carcinoma
  • Concurrent severe and/or uncontrolled medical condition (e.g. uncontrolled diabetes, infection, hypertension, cancer, etc.)
  • +2 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (12)

BE-Antwerpen-ZNASTUIVENBERG

Antwerp, Belgium

Location

BE-Gent-UZGENT

Ghent, Belgium

Location

BE-Leuven-UZLEUVEN

Leuven, Belgium

Location

BE-Roeselare-AZDELTA

Roeselare, Belgium

Location

NL-Amsterdam-AMC

Amsterdam, Netherlands

Location

NL-Amsterdam-VUMC

Amsterdam, Netherlands

Location

NL-Groningen-UMCG

Groningen, Netherlands

Location

NL-Maastricht-MUMC

Maastricht, Netherlands

Location

NL-Nijmegen-RADBOUDUMC

Nijmegen, Netherlands

Location

NL-Rotterdam-EMCDANIEL

Rotterdam, Netherlands

Location

NL-Rotterdam-ERASMUSMC

Rotterdam, Netherlands

Location

NL-Utrecht-UMCUTRECHT

Utrecht, Netherlands

Location

Related Links

MeSH Terms

Conditions

Primary Myelofibrosis

Interventions

11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Condition Hierarchy (Ancestors)

Myeloproliferative DisordersBone Marrow DiseasesHematologic DiseasesHemic and Lymphatic Diseases

Study Officials

  • Peter AW te Boekhorst, M.D. PhD

    Erasmus Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 26, 2018

First Posted

August 24, 2018

Study Start

June 4, 2018

Primary Completion

July 1, 2023

Study Completion (Estimated)

February 1, 2027

Last Updated

August 22, 2022

Record last verified: 2022-08

Data Sharing

IPD Sharing
Will not share

Locations

NL(8)BE(4)