Trial Outcomes & Findings for Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN) (NCT NCT02410551)

Last Updated: 2018-10-30

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

4 participants

Primary outcome timeframe

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.

Results posted on

2018-10-30

Participant Flow

Patients with Idiopathic Myelofibrosis or Myelofibrosis secondary to Ploycythemia Vera or Essential Thrombocythemia that are 18 to 70 years old that want to pursue a transplant. They start the Pacritinib per-transplant and can proceed to transplant 60 days after starting Pacritinib but no more than 180 days.

Participant milestones

Participant milestones
Measure	Pacritinib Pre- Transplant Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than 180 days
Overall Study STARTED	4
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Reasons for withdrawal
Measure	Pacritinib Pre- Transplant Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than 180 days
Overall Study Adverse Event	1
Overall Study Withdrawal by Subject	1
Overall Study FDA Clinical hold-patients taken off	2

Baseline Characteristics

Pacritinib Before Transplant for Myeloproliferative Neoplasms (MPN)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pacritinib Pre- Transplant n=4 Participants Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	3 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	3 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants
Race (NIH/OMB) White	4 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	4 Participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Population: Four participants were not evaluable and no analysis was done.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Start of Pacritinib to one year post transplant

Population: Four participants was invaluable and no analysis was done.

Evaluate safety and efficacy of this therapy determined by Neutrophil and platelet engraftment, Non-relapse mortality at one year post transplant, Overall survival at one year post transplant, Liver and spleen response to Pacritinib, Immune recovery, quality of life and symptom score, Primary and secondary graft failure,Complete remission, Relapse.

Outcome measures

Outcome data not reported

Adverse Events

Pacritinib Pre- Transplant

Serious events: 0 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Pacritinib Pre- Transplant n=4 participants at risk Patients will start pacritinib 200 mg po bid and can proceed to transplant after 60 days of starting pacritinib but not more than
Skin and subcutaneous tissue disorders Rash maculo-papular	25.0% 1/4 • 07/31/2015 to 02/25/2016 The severity of the adverse events (AEs) will be graded according to the Common Terminology Criteria v4.0 (CTCAE). We will not capture expected Grade 1 and Grade 2 AEs. Grade 3 and 4 AEs will be collected and recorded in the medical record. Only Pacritinib-related adverse events and protocol specific data will be entered into PDMS/CORe. PDMS/CORE will be used as the electronic case report form for this protocol.

Additional Information

Popat,Uday,M.D. / Stem Cell Transplantation

UT MD Anderson Cancer Center

Phone: 713-792-8750

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place