RecruitingPhase 1/2

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)

United States50 participantsStarted 2023-03-06

Plain-language summary

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. ...

Who can participate

Age range18 Years – 120 Years

SexALL

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Inclusion criteria

✓. Moderate or severe cGVHD (after allogeneic hematopoietic stem cell transplantation) diagnosed and staged per NIH criteria
✓. cGVHD that did not respond to \>=2 lines of prior systemic therapy.
✓. Karnofsky performance score \>=60%
✓. Age \>=18 years.
✓. If participant is taking systemic therapy for cGVHD at the time of enrollment, they must be on a stable or tapering dose in the preceding 4 weeks.
✓. Participants must have adequate organ and marrow function as defined below:
✓. Primary malignancy for which the participant received transplant has been in complete clinical remission and stable for 3 months prior to enrollment on study.
✓. Individuals of child-bearing potential (IOCBP) and individuals able to father a child with a partner able to become pregnant who are sexually active must agree to use one (1) highly effective (e.g., intrauterine device \[IUD\], surgical) or two (2) effective forms of contraception (e.g., barrier method) at study entry, for the duration of study treatment, and for at least 90 days after last study drug exposure.

Exclusion criteria

What they're measuring

Phase I: Safety of pacritinib in refractory cGVHD.

Timeframe: 60 days

Phase II: Overall response rate (ORR)

Timeframe: 6 months

Trial details

NCT IDNCT05531786

SponsorNational Cancer Institute (NCI)

Sponsor typeNIH

Study typeINTERVENTIONAL

Primary completion2027-07-22

Contact for this trial

Rania S Hishmeh, R.N.

(240) 858-3681 rania.hishmeh@nih.gov

View on ClinicalTrials.gov More Graft vs Host Disease trials