Trial Outcomes & Findings for Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS) (NCT NCT02469415)

Last Updated: 2018-10-16

Results Overview

The primary efficacy outcome of both parts is the overall response rate (ORR) based mainly on hematologic improvement defined by (International Working Group) IWG-2006 criteria, and which also includes complete remission (CR), partial remission (PR) and marrow complete remission.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

3 participants

Primary outcome timeframe

28 days

Results posted on

2018-10-16

Participant Flow

Recruitment Period: October 2015 through February 2016. This study was closed by the FDA due to the Pacritinib hold. The study was closed to new patients February 10, 2016. All active patients were required to come off treatment at that time.

Three participants were registered on this study. One participant withdrew consent before receiving the study medication. Two participants were taken off study when the investigational agent was placed on full clinical hold by the Food and Drug Administration (FDA).

Participant milestones

Participant milestones
Measure	Pacritinib + Azacitidine or Decitabine Pacritinib: Part 1: Pacritinib 200 mg taken by mouth twice daily. Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined t
Overall Study STARTED	3
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Pacritinib + Azacitidine or Decitabine Pacritinib: Part 1: Pacritinib 200 mg taken by mouth twice daily. Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined t
Overall Study Withdrawal by Subject	1
Overall Study Investigational agent placed on hold	2

Baseline Characteristics

Pacritinib for Patients With Lower-Risk Myelodysplastic Syndromes (MDS)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Pacritinib + Azacitidine or Decitabine n=3 Participants Pacritinib: Part 1: Pacritinib 200 mg taken by mouth twice daily. Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined therapy. 5-azacitidine: Part 2 Starting Dose of 5-azacitidine: 75 mg/m2 by vein on Days 1 - 5 of Cycles 5 and beyond. Decitabine: Part 2 Starting Dose of Decitabine: 20 mg/m2 by vein on on Days 1 - 7 of Cycles 5 and beyond.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	52 years n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 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
Region of Enrollment United States	3 Participants n=5 Participants

PRIMARY outcome

Timeframe: 28 days

Population: Two participants were taken off study when the investigational agent was placed on full clinical hold by the Food and Drug Administration (FDA). The two participants who received the study medication were not on study long enough to make a formal response assessment.

Outcome measures

Outcome data not reported

Adverse Events

Pacritinib + Azacitidine or Decitabine

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Pacritinib + Azacitidine or Decitabine n=3 participants at risk Pacritinib: Part 1: Pacritinib 200 mg taken by mouth twice daily. Part 2: Pacritinib dose decreased to 200 mg in the morning and 100 mg in the evening for the first cycle of combined therapy. If no toxicity is observed in first cycle of combined therapy, Pacritinib dose may be increased to 200 mg twice a day on subsequent cycles of combined t
Infections and infestations Pneumonia	33.3% 1/3 • Number of events 1 • Participants will be assessed for adverse events from the first dose of study medication to the completion of study treatment, for up to 12 months.

Other adverse events

Adverse event data not reported

Additional Information

Courtney DiNardo, MD/Assistant Professor

The University of Texas MD Anderson Cancer Center

Phone: 713-794-1141

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place