Can a single drug stop JAK2 mutations from turning into cancer?
NCT ID NCT07249840
First seen Jun 25, 2026 · Last updated Jul 29, 2026 · Updated 3 times
Summary
This pilot study tests whether the drug ropeginterferon is safe and feasible for 12 people with a JAK2 mutation and high-risk features who do not yet have a blood cancer. Participants receive an injection every 4 weeks and are monitored closely. The goal is to see if this approach can prevent progression to myeloproliferative neoplasms.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- ropeginterferon alfa-2b
- What this could lead to
- If successful, this could point toward a way to reduce the risk of blood cancers in people with JAK2 mutations.
- What could go wrong
- This is a very early, small pilot study focused on safety and feasibility, not effectiveness. The drug may cause side effects and may not work as hoped.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Started
-
Jul 2026
- Expected to finish
-
Aug 2030
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Adults age 18 years or older * Evidence of JAK2 V617F clonal hematopoiesis of indeterminate potential, as defined by a JAK2 V617F mutation detected on quantitative PCR. By definition, these patients do NOT have a diagnosis of an MPN, and must have at least one additional high-risk feature. * Have high-risk clinical/laboratory features, as defined as at least ONE of the following criteria: * Patients with a venous or arterial thrombotic event within 1 year prior to or any time after diagnosis of JAK2 clonal hematopoiesis * Patients with elevated laboratory parameters above normal limits at screening, but not meeting criteria for an MPN by WHO 2016 criteria. This would include patients with elevated laboratory parameters but an otherwise normal bone marrow biopsy. * White blood cell count \> 10 K/uL OR * Hemoglobin \> 16 g/dL in women and \> 16.5 g/dL in men; OR hematocrit \>48% for women and \>49% for men OR * Platelets \>400 K/uL * Patients with JAK2 VAF \>20% * Willing to have a bone marrow biopsy at study entry to exclude an MPN diagnosis. Screening bone marrow biopsy must not be diagnostic of any overt hematologic malignancy by morphologic assessment and must be consistent with a diagnosis of clonal hematopoiesis as determined by multi-institutional hematopathology review. A historical bone marrow biopsy is allowed if within 3 months of C1D1 and records and pathology can be obtained. In cases where the bone marrow biopsy results are uncertain, study eligibility should be discussed with the PI. All patients will have an erythropoietin level drawn to rule out a PV diagnosis by WHO 2016 criteria. * Must have adequate organ function as demonstrated by the following: * ALT (SGPT) and AST (SGOT) ≤ 2.5x upper limit of normal (ULN), * Direct bilirubin ≤ 1.5 x ULN * eGFR \>60 mL/min * leukocytes ≥3,000/mcL * absolute neutrophil count ≥1,500/mcL * Platelets ≥100,000/mcL * ECOG performance status (PS) ≤ 3 * Human immunodeficiency virus (HIV)-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months of C1D1 are eligible for this trial. * For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated. * Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load. * Participants with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial. * Participants with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, participants should be class 2B or better. * The effects of ropeginterferon on the developing human fetus are unknown. For this reason, 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 and women of childbearing potential 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 ropeginterferon administration. * Ability to understand and the willingness to sign a written informed consent document. Exclusion Criteria: * Meeting WHO 2016 criteria for a MPN. * Pregnant or lactating. * Any active malignancy in the past 2 years prior to C1D1, with the exception of non-melanoma skin cancer, or cervical carcinoma in situ or breast carcinoma in situ that has been excised or resected completely and is without evidence of local recurrence or metastasis. Any malignancy treated with curative intent and no evidence for active disease in the last 2 years are eligible. * Evidence of severe retinopathy or clinically relevant ophthalmologic disorder. * Participation in an investigational drug or device trial within 2 weeks prior to C1D1 * Documented autoimmune disease at screening or in the medical history which is active and serious * History of significant and clinically relevant psychiatric illnesses, including prior suicide attempts or risk of suicide on screening * History of thyroid dysfunction not adequately controlled * History of major organ transplantation * History of uncontrolled severe seizure disorder * Participants who are receiving any other investigational agents. * History of allergic reactions attributed to compounds of similar chemical or biologic composition to ropeginterferon.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for JAK2 mutation are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
-
Mass General Brigham
RECRUITINGBoston, Massachusetts, 02115, United States