Actively Recruiting
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Led by National Cancer Institute (NCI) · Updated on 2026-03-27
50
Participants Needed
2
Research Sites
228 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Background: Chronic graft-versus-host disease (cGVHD) is an immune system disorder that can occur in people who have had a stem cell transplant. cGVHD can affect multiple organs and increase risk of disability and death. New treatments are needed to treat cGVHD after stem cell transplant. Objective: To test a drug (pacritinib) in people with moderate or severe cGVHD that has not responded to previous treatment. Eligibility: People aged 18 years and older with moderate or severe cGVHD that has not responded to 2 or more lines of previous treatment. Design: Participants will be screened. They will have blood and urine tests. They will have tests of their heart and lung function. They may also have a CT scan. Some may have other specialized tests. Participants will take the study drug at home every day. Pacritinib is a capsule taken by mouth. The study doctor will determine the dosage and schedule. Participants will keep a medication diary. They will record the date and time of each drug dose and any missed doses. Participants will visit the clinic every 2 weeks for the first 4 months. Then they will visit the clinic once every 4 weeks. They will have blood and urine tests. During some visits, other screening tests will be repeated, and participants will fill out questionnaires about their quality of life. Photographs may be taken of skin rashes and joints affected by cGVHD. Participants will give saliva samples. Optional biopsies may be taken of the skin and mouth. Participants will take pacritinib for 6 to 12 months if no side effects develop. Follow-up visits will continue for up to 2 years. ...
CONDITIONS
Official Title
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Moderate or severe chronic graft-versus-host disease (cGVHD) diagnosed and staged per NIH criteria
- cGVHD not responding to two or more prior systemic treatments
- Disease progression or persistence despite corticosteroids or other systemic therapies as defined
- Karnofsky performance score of 60% or higher
- Age 18 years or older
- Stable or tapering dose of systemic therapy for cGVHD in the last 4 weeks if on treatment
- Adequate organ and marrow function including neutrophil count ≥1,000/mcL, platelets ≥50,000/mcL, and acceptable liver and kidney function
- Primary malignancy in complete remission and stable for at least 3 months
- Agreement to use effective contraception during and after treatment if of child-bearing potential
- Ability to understand and sign informed consent
You will not qualify if you...
- Active acute GVHD without chronic GVHD (overlapping acute and chronic GVHD is allowed)
- Treatment with ruxolitinib or ibrutinib within 14 days before starting study drug
- Active HIV-1, Hepatitis B or C infection
- Symptomatic congestive heart failure, unstable angina, uncontrolled arrhythmias, or high risk for QT prolongation
- Left ventricular ejection fraction ≤50%
- Poor lung function (FEV1 ≤39%)
- Active bleeding or history of severe bleeding in past year
- Use of other investigational agents
- Concurrent use of strong CYP3A4 inducers or inhibitors without stopping 2 weeks prior
- Known allergy to JAK inhibitors
- Refusal of blood transfusions
- Pregnancy or breastfeeding
- Active uncontrolled infections
- Other active cancers except certain skin or cervical/breast cancers needing no treatment
- Any uncontrolled illness limiting study compliance
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
University of Miami
Miami, Florida, United States, 33101
Actively Recruiting
2
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
R
Rania S Hishmeh, R.N.
CONTACT
N
Najla El Jurdi, M.D.
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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