Ruxolitinib in Thrombocythemia and Polycythemia Vera

NCT04644211 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 20-364
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 4

Brief Summary

This research is being done to see if the drug ruxolitinib is effective in reducing the symptoms caused by low-risk essential thrombocythemia (ET) and polycythemia vera (PV). \- This research study involves the study drug Ruxolitinib.

Conditions

Essential Thrombocythemia; Polycythemia Vera

Keywords

Essential Thrombocythemia; Polycythemia Vera; Sympton Burden

Outcome Measures

Primary Outcomes (1)

• Percentage of patients who achieve >50% reduction from baseline to Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score

  Percentage of patients with \>50% change in Myeloproliferative Neoplasm Symptom Assessment Total Symptom Score (MPN-SAF TSS), which has use in major clinical trials and as symptom assessment in all patients in clinical practice. Modeling has established baseline MPN-SAF TSS cut-off scores (total MPN-SAF TSS \>20 or an individual component score \>5) at which symptomatic treatment would be significantly beneficial

  baseline to 12 weeks

Secondary Outcomes (6)

• Proportion of patients achieving complete hematologic rate at week 12

  Week 12

• Proportion of patients achieving complete hematologic rate at 24 Weeks

  Week 24

• Best MPN-SAF TSS score

  12 Weeks

• Best MPN-SAF TSS score

  24 Weeks

• Percentage of change in Spleen Volume

  baseline to 12 weeks

Study Arms (2)

Ruxolitinib Stage 1

EXPERIMENTAL

In stage 1, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ETpatients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.

Drug: Ruxolitinib

Ruxolitinib Stage 2

EXPERIMENTAL

Stage 2 will commence based on 3 or more participants in Stage 1 showing a predetermined positive response to Ruxolitinib. In stage 2, participants will be divided into two cohorts: * Very low, Low, and Intermediate-risk ET patients with significant symptom burden and Low-risk PV patients with significant symptom burden * Study cycles are 28 days long, participants in both cohorts will receive: * Ruxolitinib 2x daily for 6 study cycles.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Pill taken by mouth.

Also known as: Jakafi

Ruxolitinib Stage 1; Ruxolitinib Stage 2

Eligibility Criteria

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

You may qualify if:

• Patients who have been diagnosed with essential thrombocythemia or polycythemia vera by World Health Organization 2016 diagnostic criteria.
• Patients with essential thrombocythemia must be very low (no history of thrombosis, age \<60, and no JAK2 mutation), low (no history of thrombosis, age \<60, presence of JAK2 mutation), or intermediate risk (no history of thrombosis, age \>60, no JAK2 mutation) by IPSET criteria. Patients with polycythemia vera must be low risk (no history of thrombosis and age \<60) by NCCN guidelines.
• Patients with an MPN-SAF TSS (MPN-10) score \>10 AND at least one individual feature \>5 documented on a separate visit within 3 months prior to study registration, as documented in the clinical record or obtained by clinician. If not previously documented in the electronic medical record, participants must be blinded to purpose of MPN SAF TSS scoring for eligibility determination. Average daily MPN-SAF TSS (MPN-10) score must remain \>10 with any individual feature \>5 for the week-long baseline assessment prior to ruxolitinib initiation.
• Patients who have previously received or are receiving cytoreductive therapy (i.e. hydroxyurea, anagrelide, interferon) are eligible for the study if therapy was used for the indication of symptom control, or if therapy was used for pre-operative control of blood counts. If a subject is still receiving cytoreductive therapy at the time of screening and enrollment, there will be a wash-out period from prior cytoreductive therapy at least 7 days prior to ruxolitinib initiation.
• Age ≥18 years.
• ECOG performance status ≤2 (Karnofsky ≥60%)
• Participants must have adequate organ and marrow function as defined below:
• leukocytes ≥3,000/mcL
• absolute neutrophil count ≥1,500/mcL
• platelets ≥100,000/mcL
• total bilirubin ≤ institutional upper limit of normal (ULN)
• AST(SGOT)/ALT(SGPT) ≤3 × institutional ULN
• creatinine ≤ institutional ULN OR glomerular filtration rate (GFR) ≥60 mL/min/1.73 m2 unless data exists supporting safe use at lower kidney function values, no lower than 30 mL/min/1.73 m2
• Participants with a prior or concurrent malignancy not receiving treatment for concurrent cancer diagnosis and/or prior concurrent malignancy within 5 years except for basal cell carcinoma or squamous cell carcinoma of the skin.
• For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.

You may not qualify if:

• Essential thrombocythemia patients who are high risk by IPSET-R criteria (age \> 60 with JAK2 V617F mutation and/or history of thrombosis).1 Polycythemia vera patients who are high risk by NCCN guidelines (age \> 60 and/or history of thrombosis).
• Patients with \>5% blasts on baseline marrow exam or at any other time in peripheral blood
• Participants who are receiving any other investigational agents.
• Participants with a history of splenectomy. Participants may still be eligible after discussion with and approval by the Overall PI, however.
• History of allergic reactions attributed to compounds of similar chemical or biologic composition to ruxolitinib or excipients of ruxolitinib.
• Participants requiring any medications or substances that are strong inhibitors or 3A4 isozyme are ineligible. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product.
• Participants with uncontrolled intercurrent illness.
• Participants with inadequate liver or renal function at screening as evidenced by lab values not meeting criteria
• Participants with psychiatric illness/social situations that would limit compliance with study requirements.
• Pregnant women are excluded from this study because ruxolitinib is a Class C agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with ruxolitinib, breastfeeding should be discontinued if the mother is treated with ruxolitinib.
• The effects of ruxolitinib on the developing human fetus are unknown. Pregnant women and subjects of childbearing potential who are unwilling to take appropriate precautions to avoid becoming pregnant or fathering a child are ineligible. Women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of ruxolitinib administration.

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Incyte Corporation: collaborator

Study Sites (4)

Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

RECRUITING

Beth-Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

NOT YET RECRUITING

Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States

RECRUITING

Massachusetts General North Shore Cancer Center

Danvers, Massachusetts, 01923, United States

RECRUITING

MeSH Terms

Conditions

Thrombocythemia, Essential; Polycythemia Vera

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombocytosis; Blood Platelet Disorders; Myeloproliferative Disorders; Bone Marrow Diseases; Hemorrhagic Disorders; Bone Marrow Neoplasms; Hematologic Neoplasms; Neoplasms by Site; Neoplasms

Study Officials

• Gabriela Hobbs, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gabriela Hobbs, MD

CONTACT

(617) 726-8748; ghobbs@partners.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: November 10, 2020
• First Posted: November 25, 2020
• Study Start: March 21, 2022
• Primary Completion (Estimated): July 1, 2027
• Study Completion (Estimated): July 1, 2034
• Last Updated: January 8, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Data can be shared no earlier than 1 year following the date of publication
• Access Criteria: Contact the Partners Innovations team at http://www.partners.org/innovation

Locations

US (4)