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)

Part of paid clinical trials in Miami, Florida.

Sponsor
National Cancer Institute (NCI)
Study ID
NCT05531786
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Graft vs Host Disease

Eligibility Criteria

Sex
ALL
Age
18 Years - 120 Years
Healthy Volunteers
Not accepted

Interventions

  • Pacritinib — DRUG
    Pacritinib will be given as 100 mg or 200 mg tablets to be taken orally twice daily (12 hours apart) on days 1-28 of a 28 day cycle. Morning and evening should be taken at approximately the same time of day.

Study Details

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. ...

Key Dates

Start date
Mar 6, 2023
Status verified
May 2026
Primary completion
Jul 22, 2027
Completion
Jul 22, 2027

Study Design

Enrollment
50 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Arm 2 - Low-dose
    Expansion dosing to evaluate the efficacy of pacritinib 100 mg PO BID
  • Experimental: Arm 3 - High-dose
    Expansion dosing to evaluate the efficacy of pacritinib 200 mg PO BID
  • Experimental: Escalating doses of treatment
    Escalating doses of pacritinib to confirm safety in cGVHD

Primary Outcome Measure

Phase I: Safety of pacritinib in refractory cGVHD. [ Time Frame: 60 days ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
University of MiamiMiamiFlorida33101-
National Institutes of Health Clinical CenterBethesdaMaryland20892
For more information at the NIH Clinical Center contact National Cancer Institute Referral Office
888-624-1937

Find similar trials in Miami, FL

By research site
University of Miami· Miami, FLNational Institutes of Health Clinical Center· Bethesda, MD

Related Studies