Trial Outcomes & Findings for A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression (NCT NCT02891603)

Last Updated: 2026-02-06

Results Overview

STAT3 activity in circulating CD4+ T-cells. This is equivalent to 5.5 tablespoons of blood for each assessment. Peripheral blood mononuclear cells (PBMC) will be isolated by Ficoll density gradient. PBMCs will be stimulated with IL-6 for 20 minutes to activate STAT3. Phosphoproteins will be analyzed within T-cells by flow cytometry. Result reported is %pSTAT3+CD4+T cells at day +21.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

40 participants

Primary outcome timeframe

up to 21 days

Results posted on

2026-02-06

Participant Flow

Participant milestones

Participant milestones
Measure	Phase 1, Level 1: Pacritinib With Sirolimus and Tacrolimus After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth the day of the participant's transplant (Day 0) and will continue until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 1, Level 2: Pacritinib With Sirolimus and Tacrolimus After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 2: Pacritinib With Sirolimus and Tacrolimus After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). Patients will take Pacritinib at the MTD: 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.
Overall Study STARTED	10	8	22
Overall Study COMPLETED	10	8	22
Overall Study NOT COMPLETED	0	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Phase 1, Level 1: Pacritinib With Sirolimus and Tacrolimus n=10 Participants After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth the day of the participant's transplant (Day 0) and will continue until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 1, Level 2: Pacritinib With Sirolimus and Tacrolimus n=8 Participants After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 2: Pacritinib With Sirolimus and Tacrolimus n=22 Participants After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). Patients will take Pacritinib at the MTD: 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Total n=40 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=192 Participants	4 Participants n=170 Participants	10 Participants n=185 Participants	22 Participants n=177 Participants
Age, Categorical >=65 years	2 Participants n=192 Participants	4 Participants n=170 Participants	12 Participants n=185 Participants	18 Participants n=177 Participants
Sex: Female, Male Female	5 Participants n=192 Participants	3 Participants n=170 Participants	9 Participants n=185 Participants	17 Participants n=177 Participants
Sex: Female, Male Male	5 Participants n=192 Participants	5 Participants n=170 Participants	13 Participants n=185 Participants	23 Participants n=177 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	1 Participants n=192 Participants	0 Participants n=170 Participants	1 Participants n=185 Participants	2 Participants n=177 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	9 Participants n=192 Participants	7 Participants n=170 Participants	21 Participants n=185 Participants	37 Participants n=177 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=192 Participants	1 Participants n=170 Participants	0 Participants n=185 Participants	1 Participants n=177 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Race (NIH/OMB) Asian	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Race (NIH/OMB) Black or African American	1 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	1 Participants n=177 Participants
Race (NIH/OMB) White	9 Participants n=192 Participants	8 Participants n=170 Participants	22 Participants n=185 Participants	39 Participants n=177 Participants
Race (NIH/OMB) More than one race	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=192 Participants	0 Participants n=170 Participants	0 Participants n=185 Participants	0 Participants n=177 Participants
Region of Enrollment United States	10 participants n=192 Participants	8 participants n=170 Participants	22 participants n=185 Participants	40 participants n=177 Participants

PRIMARY outcome

Timeframe: up to 21 days

Population: Evaluable participants treated at MTD (Phase 1 level 2 \& Phase 2). Phase 1 level 1 participants were not analyzed for efficacy, therefore there were not included in the analysis population.

Outcome measures

Outcome measures
Measure	Pacritinib With Sirolimus and Tacrolimus n=28 Participants Patients treated with Pacritinib combined with Sirolimus and Tacolimus at Phase 1 Level 2 dose/Phase 2 dose (MTD)
STAT Activity	9.623 %pSTAT3+CD4+T cells at day +21 Standard Deviation 10.01

SECONDARY outcome

Timeframe: up to 100 days

Cumulative incidence of acute GVHD . Participants will be monitored for clinical signs of acute GVHD. Acute GVHD will be graded per the 1995 consensus guidelines.

Outcome measures

Outcome measures
Measure	Pacritinib With Sirolimus and Tacrolimus n=28 Participants Patients treated with Pacritinib combined with Sirolimus and Tacolimus at Phase 1 Level 2 dose/Phase 2 dose (MTD)
Incidence of Acute GVHD	46.4 percentage of participants Interval 27.1 to 63.7

Adverse Events

Phase 1, Level 1: Pacritinib With Sirolimus and Tacrolimus

Serious events: 5 serious events

Other events: 10 other events

Deaths: 0 deaths

Phase 1, Level 2: Pacritinib With Sirolimus and Tacrolimus

Serious events: 3 serious events

Other events: 8 other events

Deaths: 1 deaths

Phase 2: Pacritinib With Sirolimus and Tacrolimus

Serious events: 8 serious events

Other events: 18 other events

Deaths: 5 deaths

Serious adverse events

Other adverse events

Additional Information

Joseph Pidala, MD, PhD

Moffitt Cancer Center

Phone: 813-745-2556

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: GT60

Measure	Phase 1, Level 1: Pacritinib With Sirolimus and Tacrolimus n=10 participants at risk After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth the day of the participant's transplant (Day 0) and will continue until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 1, Level 2: Pacritinib With Sirolimus and Tacrolimus n=8 participants at risk After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.	Phase 2: Pacritinib With Sirolimus and Tacrolimus n=22 participants at risk After standard of care allogenic hematopoietic cell transplantation, Pacritinib will be added to standard treatment with Sirolimus and Tacrolimus (PAC/SIR/TAC). Patients will take Pacritinib at the MTD: 100 mg Pacritinib will begin taken by mouth twice daily starting the day of the participant's transplant (Day 0) and continuing until 70 days after the transplant.. Sirolimus will be given the day before transplant and continued daily for at least one year. Tacrolimus will begin 3 days before transplant and will be given for at least 50 days.
General disorders Chills	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
General disorders Fever	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Vascular disorders Hypotension	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Cardiac disorders LVEF decrease	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Cardiac disorders Increase in cardiac enzymes	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Respiratory, thoracic and mediastinal disorders Adult respiratory distress syndrome	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Gastrointestinal disorders Abdominal Pain	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Gastrointestinal disorders Colitis -Clostridium difficile colitis	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Metabolism and nutrition disorders Dehydration	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Gastrointestinal disorders Gastrointestinal Disorders - Other	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Investigations Alanine aminotransferase increased - VOD	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Investigations Alanine aminotransferase increased - suspected VOD	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Renal and urinary disorders Acute kidney injury	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	9.1% 2/22 • Number of events 2 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Infections and infestations Sepsis	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Infections and infestations Lung infection -Pneumonia	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Renal and urinary disorders Urinary tract pain	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Nervous system disorders Encephalopathy	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Skin and subcutaneous tissue disorders Erythroderma -acute GVHD	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Cardiac disorders Myocardial infarction	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
General disorders Mulit-organ failure	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Skin and subcutaneous tissue disorders Acute GVHD of skin	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Skin and subcutaneous tissue disorders Pain of skin	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
General disorders Edema trunk	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Skin and subcutaneous tissue disorders Periorbital edema	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Immune system disorders Anaphylaxis	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Infections and infestations Sinusitis	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Nervous system disorders Headache -migraine	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Injury, poisoning and procedural complications Fracture	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 2 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Injury, poisoning and procedural complications Fall	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	4.5% 1/22 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Metabolism and nutrition disorders Metabolism and Nutrition disorders - Other	10.0% 1/10 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/8 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.
Gastrointestinal disorders Gastrointestinal disorders -Other	0.00% 0/10 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	12.5% 1/8 • Number of events 1 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.	0.00% 0/22 • Adverse events collected from first dose of study drug until 30 days after continuation of study drug, an average of 105 days.