A Phase 2 Study to Evaluate the Safety and Efficacy of Pacritinib in Relapsed or Refractory Waldenström Macroglobulinemia

NCT06986174 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 24-660
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

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)

Conditions

B-Cell Lymphoproliferative Disorder; Lymphoplasmacytic Lymphoma; Indolent Lymphoma; Waldenström Macroglobulinemia

Keywords

Waldenström Macroglobulinemia; Lymphoplasmacytic Lymphoma; Lymphoma; B-Cell Lymphoproliferative Disorder; Indolent Lymphoma

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR)

  ORR was defined as the percentage of participants achieving complete response (CR), very good partial response (VGPR), partial response (PR) and minimal response (MR) on treatment based on IWWM-11 criteria.

  Up to 48 months

Secondary Outcomes (20)

• Complete Response Rate

  Up to 48 months

• Very Good Partial Response Rate

  Up to 48 months

• Partial Response Rate

  Up to 48 months

• Minimal Response Rate

  Up to 48 months

• Stable Disease Rate

  Up to 48 months

Study Arms (1)

Pacritinib

EXPERIMENTAL

30 participants will complete: * Baseline visit with assessments and ECGs * Cycles 1 through 2 (28 days per cycle): --Day 1: Predetermined dose of Pacritinib 2x daily * Cycle 3, 6, 9, 12, and every 3 cycles for 48 cycles total (28 days per cycle): --Day 1: Predetermined dose of Pacritinib 2x daily * Bone marrow biopsies/aspirations at Cycles 6 and 12 and then yearly * End of Treatment visit with CT scan and bone marrow biopsy/aspiration * Follow up: every 12 weeks for 2 years

Drug: Pacritinib

Interventions

Pacritinib DRUG

Kinase inhibitor, capsule, taken orally per protocol.

Also known as: SB1518, VONJO, Pacritinib Citrate

Pacritinib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:

You may not qualify if:

• 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:

Sponsors & Collaborators

• Shayna Sarosiek, MD: lead
• Sobi, Inc.: collaborator

Study Sites (1)

Dana Farber Cancer Institute

Boston, Massachusetts, 02115, United States

RECRUITING

MeSH Terms

Conditions

Waldenstrom Macroglobulinemia; Lymphoma, B-Cell, Marginal Zone; Lymphoma

Interventions

11-(2-pyrrolidin-1-ylethoxy)-14,19-dioxa-5,7,26-triazatetracyclo(19.3.1.1(2,6).1(8,12))heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Lymphoma, B-Cell; Lymphoma, Non-Hodgkin

Study Officials

• Shayna Sarosiek, MD

  Dana-Farber Cancer Institute

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Shayna Sarosiek, MD

CONTACT

617-632-4218; Shayna_Sarosiek@dfci.harvard.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Sponsor-Investigator

Study Record Dates

• First Submitted: May 15, 2025
• First Posted: May 22, 2025
• Study Start: November 21, 2025
• Primary Completion (Estimated): October 1, 2028
• Study Completion (Estimated): October 1, 2032
• Last Updated: December 5, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Belfer Office for Dana-Farber Innovations (BODFI) at innovation@dfci.harvard.edu

Locations

US (1)