ClinicalTrials.gov record NCT06986174
A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia
A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This is a Phase 2, single-arm, open-label study testing the oral drug pacritinib in adults with symptomatic relapsed or refractory Waldenström macroglobulinemia. Participants receive pacritinib in one treatment group (no placebo) for up to 4 years and are followed for up to 2 years after stopping. About 30 people are expected to join.
What it is studying. The study is testing how safe pacritinib is and how patients' disease responds when treated with pacritinib for symptomatic Waldenström macroglobulinemia, including measuring tumor and blood responses and tracking side effects.
What the researchers hope to learn. To collect data on pacritinib's safety profile and on response rates (objective, complete, very good partial, partial, minimal, and stable disease) in this population; to measure time-to-response, progression-free and overall survival metrics; to assess bone marrow changes and response differences by specific gene mutations; and to record treatment-related adverse events.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
This study is being done to examine the safety and effectiveness of pacritinib as a possible treatment for participants with Waldenström macroglobulinemia (WM). The name of the study drug involved in this study is: -Pacritinib (a type of kinase inhibitor)
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Drug
- Pacritinib
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- Age ≥18 years
- ECOG performance status ≤2
- Clinicopathological diagnosis of Waldenström Macroglobulinemia
- Symptomatic disease meeting criteria for treatment using consensus panel criteria from the Second International Workshop on Waldenström macroglobulinemia. At least one of the following:
- constitutional symptoms: recurrent fever, night sweats, fatigue or weight loss
- progressive or symptomatic lymphadenopathy or splenomegaly
- hemoglobin ≤10 g/dL
- platelet count ≤100 k/uL
- hyperviscosity syndrome
- symptomatic peripheral neuropathy
- systemic amyloidosis
- renal insufficiency
- symptomatic cryoglobulinemia
- Serum IgM level ≥ 2 times the upper limit of normal
- Participants must meet the following organ and marrow functions as defined below:
- absolute neutrophil count ≥0.5 k/uL without growth factor within 7 days
- platelet count ≥50 k/uL without platelet transfusion within 7 days
- total bilirubin ≤1.5 times the upper limit of normal or ≤3 times the upper limit of normal with documented liver involvement, hemolysis or Gilbert's disease
- AST (SGOT) and ALT (SGPT) ≤2.5 times the upper limit of normal or ≤5 times the upper limit of normal with documented liver involvement
- Creatinine clearance ≥30 ml/min using Cockcroft/Gault equation
- Ability to understand and the willingness to sign a written informed consent document. (Providing consents in as many languages as possible is encouraged)
- At least 2 prior lines of treatment for Waldenström Macroglobulinemia. Participants must either be BTK inhibitor exposed or not be a candidate for BTK therapy.
- Women of childbearing potential: Females of childbearing potential (FCBP) will be required to use two highly effective forms of contraception simultaneously or will remain abstinent from heterosexual intercourse during the following periods related to this study:
- while participating in the study; and 2) for at least three months (90 days) after discontinuation from the study. FCBP must be referred to a qualified provider of contraceptive methods if needed.
Exclusion criteria
- Current history of uncontrolled HIV
- Patients with a known history of HIV must have a viral load assessed for eligibility and must be on a stable antiretroviral regimen that can be administered concurrent with pacritinib.
- Known active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection based on criteria below
- Hepatitis B virus (HBV): Patients with positive hepatitis B surface antigen (HBsAg) are excluded. Patients with positive hepatitis B core antibody (antiHBc) and negative HBsAg require hepatitis B polymerase chain reaction (PCR) evaluation before enrollment. Patients who are hepatitis B PCR positive will be excluded.
- Hepatitis C virus (HCV): positive hepatitis C antibody. If positive hepatitis C antibody result, patient will need to have a negative result for hepatitis C ribonucleic acid (RNA) before enrollment. Patients who are hepatitis C RNA positive will be excluded.
- Participants with chronic liver disease and hepatic impairment meeting Child-Pugh class B or C (Appendix B)
- Participants who are pregnant, breast feeding, or planning to become pregnant while enrolled in this study or within 3 month after last study dose (2 weeks for breastfeeding)
- Current CNS involvement by WM
- Active alcohol or drug abuse
- Concurrent administration of medications that are moderate or strong inhibitors or inducers of CYP3A within 14 days or 5 half-lives, whichever is shorter, prior to first dose of study drug.
- Concurrent participation in another therapeutic clinical trial
- History of another malignancy, except adequately treated local basal cell or squamous cell carcinoma of the skin, cervical carcinoma in situ, superficial bladder cancer, localized prostate cancer, or other adequately treated cancer currently in complete remission
- Prior or ongoing clinically significant illness, including active infections requiring antibiotics, of medical condition that, in the investigator's opinion, could affect the safety of the patient; alter the absorption, distribution, metabolism or excretion of the study drug; or impair the assessment of study results
- Inability to swallow pills
- Significant cardiovascular disease defined as:
- Unstable angina, or
- History of myocardial infarction within 6 months prior to planned start
- Previously documented left ventricular ejection fraction (LVEF) by any method of ≤ 45% in the 12 months prior to planned start; assessment of LVEF via echocardiogram or multigated acquisition (MUGA) scan during screening should be performed in selected patients as medically indicated, or
- Any Class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification, or
- Uncontrolled or symptomatic arrhythmias
- Prolonged QT Interval with baseline QTc >480 msec using the Bazette formula
- Ongoing, active infection.
- Active bleeding requiring blood transfusion or other medical intervention. Participants requiring anticoagulation therapy are not excluded.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
This is a single-arm, open-label, Phase II study to evaluate the safety and efficacy of pacritinib in participants with symptomatic Waldenström macroglobulinemia (WM). Pacritinib is a targeted therapy that blocks a protein called JAK2 that helps cells live and grow. It also inhibits IRAK1, which is important for the survival of WM cells. By blocking JAK2 \& IRAK1, pacritinib may kill abnormal cells or stop them from growing. The U.S. Food and Drug Administration (FDA) has not approved pacritinib for WM but it has been approved for Myelofibrosis. The research study procedures include screening for eligibility, in-clinic visits, questionnaires, blood tests, urine tests, Computerized Tomography (CT) scans, X-rays, echocardiograms (ECGs), bone marrow biopsies and aspirations Participants will receive study treatment for up to 4 years and will be followed for 2 years, or until there is start of a new treatment. It is expected that about 30 people will take part in this research study. Sobi AG, Inc. is supporting this research study by providing study drug and funding.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants will take oral pacritinib capsules twice daily and attend regular clinic visits for treatment (up to 4 years) and safety monitoring, then be followed every 12 weeks for up to 2 years or until a new therapy starts. Before and during the study they will have screening and on‑treatment assessments including questionnaires, blood and urine tests, CT scans, electrocardiograms and cardiac assessments as needed. Bone marrow biopsies/aspirations are required at cycles 6 and 12 and then yearly, with an end‑of‑treatment CT scan and bone marrow biopsy. Women of childbearing potential must follow the study contraception requirements during treatment and for 90 days after stopping the drug.
How the study is structured. Interventional Phase 2 trial with a single treatment group. All participants receive oral pacritinib (no randomization or blinding). Treatment is given in repeated 28‑day cycles for up to 4 years, with scheduled clinic visits, imaging, blood and urine tests, periodic bone marrow biopsies, and follow-up for up to 2 years after treatment ends. Planned enrollment is about 30 participants.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on Waldenström macroglobulinemia
Important dates
- Study start
- Nov 21, 2025
- Primary completion
- Oct 1, 2028 (estimated)
- Study completion
- Oct 1, 2032 (estimated)
- Target enrollment
- 30 participants (estimated)
- Last updated on registry
- Dec 5, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Shayna Sarosiek, MD
- Collaborators
- Sobi, Inc.
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Shayna Sarosiek, MD 📞 617-632-4218 ✉ Shayna_Sarosiek@dfci.harvard.edu
Participating sites (1)
- Dana Farber Cancer InstituteRecruiting
Boston, Massachusetts, 02115 · United States
- Shayna Sarosiek, MD · 617-632-4218 · Shayna_sarosiek@dfci.harvard.edu
- Shayna Sarosiek, MD
Terms used on this page · explanations by MedicalUpdates.ai
- study drug
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- ECOG performance status
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- Creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- written informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- prior lines
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- Women of childbearing potential
- Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
- enrollment
- The number of participants a study plans to include, or has included. 'Estimated' means the planned number; 'actual' means the number who joined.
- 5 half-lives
- The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
- complete remission
- No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
- echocardiogram
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- during screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- QTc
- A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- CT
- An X-ray scan that builds detailed cross-section images of the body. 'High-resolution' CT (HRCT) is used to look closely at the lungs.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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