Actively Recruiting
A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia
Led by Shayna Sarosiek, MD · Updated on 2025-12-05
30
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
S
Shayna Sarosiek, MD
Lead Sponsor
S
Sobi, Inc.
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying pacritinib as a possible treatment for people with Waldenström macroglobulinemia (WM), a type of blood cancer. This Phase II trial aims to evaluate how safe and effective pacritinib is for participants whose disease has returned or not responded to previous treatments. Pacritinib works by blocking proteins, JAK2 and IRAK1, that help WM cells survive and grow. The U.S. Food and Drug Administration has approved pacritinib for another condition called Myelofibrosis, but not yet for WM. In this open-label study, about 30 participants will receive pacritinib capsules taken orally twice daily in 28-day cycles. The treatment period can last up to 4 years, including cycles 1 through 48 with specific visits for assessments at cycles 3, 6, 9, 12, and every 3 cycles thereafter. Bone marrow biopsies and aspirations will be performed at cycles 6 and 12 and then yearly. After treatment, participants will be followed every 12 weeks for 2 years or until they start a new therapy. Participants will undergo various tests and procedures including blood and urine tests, CT scans, X-rays, heart monitoring (ECGs), and bone marrow biopsies. Questionnaires will also be completed. Researchers will assess response rates to treatment, time to response and survival outcomes, and monitor any treatment-related side effects. The total participation may last several years, combining treatment and follow-up visits to track safety and effectiveness.
CONDITIONS
Brief Title
A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- ECOG performance status 2 or less
- Clinicopathological diagnosis of Waldenström Macroglobulinemia
- Symptomatic disease meeting treatment criteria, including at least one of: constitutional symptoms, progressive lymphadenopathy or splenomegaly, low hemoglobin (≤10 g/dL), low platelet count (≤100 k/uL), hyperviscosity syndrome, symptomatic peripheral neuropathy, systemic amyloidosis, renal insufficiency, symptomatic cryoglobulinemia
- Serum IgM level at least twice the upper limit of normal
- Adequate organ and marrow function as defined by blood counts and liver/kidney tests
- Ability to understand and willing to sign informed consent
- At least two prior treatments for Waldenström Macroglobulinemia; must be BTK inhibitor exposed or not a candidate for BTK therapy
- Women of childbearing potential must use two forms of contraception or remain abstinent during and for 3 months after the study
You will not qualify if you...
- Current uncontrolled HIV infection
- Known HIV with detectable viral load or unstable antiretroviral treatment
- Active hepatitis B or C infection based on specific testing
- Chronic liver disease with severe impairment (Child-Pugh class B or C)
- Pregnant, breastfeeding, or planning pregnancy during or shortly after the study
- Central nervous system involvement by Waldenström Macroglobulinemia
- Active alcohol or drug abuse
- Use of medications strongly affecting CYP3A within 14 days before treatment
- Participation in another therapeutic clinical trial
- History of other cancers unless adequately treated and in remission
- Significant illnesses affecting safety or study assessments
- Inability to swallow pills
- Significant cardiovascular disease including unstable angina, recent heart attack, poor heart function, severe heart failure, symptomatic arrhythmias
- Prolonged QT interval (>480 msec)
- Active infections or bleeding requiring medical intervention
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 4 years
Participants receive Pacritinib treatment in 28-day cycles with assessments including ECGs, blood and urine tests, CT scans, X-rays, and bone marrow biopsies at prescribed intervals.
Baseline visit, then treatment cycles with visits on Day 1 of Cycles 1-2, Cycles 3, 6, 9, 12, and every 3 cycles up to 48 cycles total; bone marrow biopsies at Cycles 6, 12, and yearly
Duration - Up to 2 years
Participants are monitored after treatment ends for safety and efficacy assessments.
Visits every 12 weeks
Trial Site Locations
Total: 1 location
1
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
Actively Recruiting
Research Team
S
Shayna Sarosiek, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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