NCT02410551

Brief Summary

The goal of this clinical research study is to learn if giving pacritinib before standard of care drugs followed by an allogeneic stem cell transplant can help to control myeloproliferative neoplasms. The safety of this therapy will also be studied.

Trial Health

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Monitor

Trial Health Score

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

Enrollment
4

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2015

Geographic Reach
1 country

1 active site

Status
terminated

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

First Submitted

Initial submission to the registry

April 2, 2015

Completed
5 days until next milestone

First Posted

Study publicly available on registry

April 7, 2015

Completed
2 months until next milestone

Study Start

First participant enrolled

June 15, 2015

Completed
1.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 20, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 20, 2017

Completed
1.7 years until next milestone

Results Posted

Study results publicly available

October 5, 2018

Completed
Last Updated

October 30, 2018

Status Verified

October 1, 2018

Enrollment Period

1.6 years

First QC Date

April 2, 2015

Results QC Date

February 28, 2018

Last Update Submit

October 4, 2018

Conditions

Keywords

Myeloproliferative DiseasesMyeloproliferative NeoplasmsMPNMyelofibrosisPolycythemia veraPVEssential thrombocythemiaAllogeneic stem cell transplantationAllo TPPacritinibBusulfanBusulfexMyleranQuestionnairesSurveysPhone calls

Outcome Measures

Primary Outcomes (1)

  • Progression-free Survival (PFS)

    The protocol was to enroll at least 21 evaluable participants, defined as patients who received Pacritinib for \>/=60 days but less than 180 days. We enrolled four participants, however all four were not evaluable since no one was able to complete 60 days of Pacritinib.

    participants who received Pacritinib for >/= 60 days but less than 180 days who undergo transplant with a matched related or at least 7/8 matched unrelated donor. The protocol was to evaluate progression free survival at one year.

Other Outcomes (1)

  • Evaluate Safety and Efficacy of Pacritinib.

    Start of Pacritinib to one year post transplant

Study Arms (1)

Pacritinib + Allogeneic Stem Cell Transplantation

EXPERIMENTAL

Participants start Pacritinib 200 mg by mouth twice a day. Participants proceed to transplant after 60 days of Pacritinib but not more than 180 days. Pacritinib stopped 21 days prior to starting preparative regimen for standard of care stem cell transplantation (SOC Allo TP). SOC transplant conditioning with Fludarabine and Busulfan AUC of 4000 microMol-min per day providing that pharmacokinetic can be done, otherwise Busulfan dose given as a fixed dose of 100 mg/m2 daily for four days. Questionnaires about symptoms and quality of life completed at baseline, 1, 3, 6, and 12 months after transplant. Phone calls made by study staff to participant on second and third week of each month.

Drug: PacritinibDrug: BusulfanBehavioral: QuestionnairesBehavioral: Phone CallsProcedure: Allogeneic Stem Cell TransplantationDrug: Fludarabine

Interventions

200 mg by mouth twice a day for 60 days.

Pacritinib + Allogeneic Stem Cell Transplantation

Busulfan AUC of 4000 microMol-min per day providing that pharmacokinetic can be done, otherwise Busulfan dose given as a fixed dose of 100 mg/m2 daily for four days.

Also known as: Busulfex, Myleran
Pacritinib + Allogeneic Stem Cell Transplantation
QuestionnairesBEHAVIORAL

Questionnaires completed at baseline, 1, 3, 6, and 12 months after transplant.

Also known as: Surveys
Pacritinib + Allogeneic Stem Cell Transplantation
Phone CallsBEHAVIORAL

Phone calls made by study staff to participant on second and third week of each month.

Pacritinib + Allogeneic Stem Cell Transplantation

Allogeneic stem cell transplantation (Allo TP) 60 days after starting Pacritinib but not more than 180 days.

Also known as: Stem cell transplant
Pacritinib + Allogeneic Stem Cell Transplantation

Fludarabine taken along with Busulfan as per standard of care as preparative regimen for allogeneic stem cell transplantation (Allo TP).

Also known as: Fludarabine phosphate, Fludara
Pacritinib + Allogeneic Stem Cell Transplantation

Eligibility Criteria

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

You may qualify if:

  • Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Polycythemia Vera or Essential Thrombocythemia.
  • Patients 18 years to less than or equal to 70 years.
  • Patients wanting to pursue transplant.
  • Patients must have a Zubrod PS equal or less than 2.
  • Calculated creatinine clearance greater than 50ml/min. using the Cockcroft-Gault equation.
  • Ejection fraction equal or above 40%.
  • Serum direct bilirubin less than 1 mg/dl (unless due to Gilbert's syndrome or hemolysis).
  • SGPT equal or less than 4 x normal values.
  • Corrected DLCO equal or above 50% of expected.
  • Negative Beta HCG test in a woman with childbearing potential defined as not post-menopausal for 12 months or no previous surgical sterilization) and if fertile, males and females must agree to use contraceptives.

You may not qualify if:

  • Patients with low risk myelofibrosis.
  • Uncontrolled life-threatening infections.
  • HIV positive.
  • Patients with active viral hepatitis.
  • Prior treatment with Pacritinib.
  • Prior stem cell transplant.
  • QTc greater than 450 ms.
  • CYP3A4 strong or moderate inhibitors/inducers in the past 7 days.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of Texas MD Anderson Cancer Center

Houston, Texas, 77030, United States

Location

Related Links

MeSH Terms

Conditions

Myeloproliferative DisordersPrimary MyelofibrosisPolycythemia VeraThrombocythemia, Essential

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-decaeneBusulfanSurveys and QuestionnairesStem Cell Transplantationfludarabinefludarabine phosphate

Condition Hierarchy (Ancestors)

Bone Marrow DiseasesHematologic DiseasesHemic and Lymphatic DiseasesBone Marrow NeoplasmsHematologic NeoplasmsNeoplasms by SiteNeoplasmsBlood Coagulation DisordersThrombocytosisBlood Platelet DisordersHemorrhagic Disorders

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsData CollectionEpidemiologic MethodsInvestigative TechniquesHealth Care Evaluation MechanismsQuality of Health CareHealth Care Quality, Access, and EvaluationPublic HealthEnvironment and Public HealthCell TransplantationCell- and Tissue-Based TherapyBiological TherapyTherapeuticsTransplantationSurgical Procedures, Operative

Results Point of Contact

Title
Popat,Uday,M.D. / Stem Cell Transplantation
Organization
UT MD Anderson Cancer Center

Study Officials

  • Uday Popat, MD

    M.D. Anderson Cancer Center

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

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

April 2, 2015

First Posted

April 7, 2015

Study Start

June 15, 2015

Primary Completion

January 20, 2017

Study Completion

January 20, 2017

Last Updated

October 30, 2018

Results First Posted

October 5, 2018

Record last verified: 2018-10

Locations