A Phase 1b Study of Safety, Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis (RopeRux in Myelofibrosis)

University of Utah1 个研究点分布在 1 个国家目标入组 15 人开始时间: 2026年8月1日最近更新: 2026年7月8日

适应症

干预措施

试验速览

阶段: 1 期
状态: 尚未招募
发起方: University of Utah
入组人数: 15
试验地点: 1
主要终点: The frequency of adverse events (AEs) and serious adverse events (SAEs) characterized by type

研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

The purpose of this clinical trial is to learn if the study drug ropeginterferon alfa- 2b added to, standard of care, ruxolitinib is safe and effective in treating patients with Myelofibrosis.

研究设计

研究类型: Interventional
分配方式: Na
干预模型: Single Group
主要目的: Treatment
盲法: None

入排标准

年龄范围: 18 Years 至 —（Adult, Older Adult）
性别: All
接受健康志愿者: 否

入选标准

•Male or female subject aged ≥ 18 years.
•Diagnosed with PMF, post-PV MF, or post-ET MF per WHO 2016 or 2022 criteria, bearing one of these MPN phenotype defining mutations (JAK2, CALR, and MPL), and with a DIPSS score of low, intermediate-1 or intermediate-
•Subjects must be already on standard of care ruxolitinib per the treating physician for at least 3 months or more, and on a stable dose for at least 6 weeks prior to screening.
•Subjects must have spleen volume of > 450ml by either MRI or CT scan
•Subject must have a JAK2, CALR, or MPL allelic burden of ≥20% at screening
•-Prior treatment for PV or ET with hydroxyurea or ruxolitinib is allowed. If the patient was on pegylated interferon in the past, the progression from PV/ ET to post-PV/ET MF must not have occurred while on pegylated interferon therapy.
•ECOG Performance Status ≤
•Adequate organ function as defined as:
•Hematologic:
•WBC count ≥ 4 x 109/L
•Absolute neutrophil count (ANC) ≥1500/mm3
•Platelet count ≥ 75,000/mm3
•Hemoglobin ≥ 8 g/dL
•--Total Bilirubin ≤ 1.5x institutional upper limit of normal (ULN)
•--AST(SGOT)/ALT(SGPT) ≤ 3 × institutional ULN
•Estimated creatinine clearance ≥ 50 mL/min by Cockcroft-Gault formula
•Recovery to baseline or ≤ Grade 1 CTCAE v 6.0 from toxicities related to any prior treatments, unless AE(s) are clinically nonsignificant and/or stable on supportive therapy per the treating investigator.
•Participants must adhere to the following sex and contraceptive/barrier requirements:
•If participant is of childbearing potential, they must have a negative pregnancy test
•For participants of non-childbearing potential: The post-menopausal status will be defined as having been amenorrheic for 12 months without an alternative medical cause. The following age-specific requirements apply:
•< 50 years of age:
•--Amenorrheic for ≥ 12 months following cessation of exogenous hormonal treatments; and
•--Luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution
•-≥ 50 years of age:
•--Amenorrheic for 12 months or more following cessation of all exogenous hormonal treatments; or
•--Had radiation-induced menopause with last menses >1 year ago; or
•--Had chemotherapy-induced menopause with last menses >1 year ago
•--Luteinizing hormone and follicle-stimulating hormone levels in the post-menopausal range for the institution
•Participants of childbearing potential and participants with a sexual partner of childbearing potential must agree to use a highly effective method of contraception and lactation requirements as described in Sections 6.4.1 and 6.4.
•Able to provide informed consent and willing to sign an approved consent form that conforms to federal and institutional guidelines.

排除标准

•PV or ET patients who progressed while on pegylated interferon or ropeginterferon therapy.
•Receiving other investigational agents.
•Existence of, or history of severe psychiatric disorders, particularly severe depression, suicidal ideation, or suicide attempt (Patients with pre-existing depression who are well-controlled and on stable doses of antidepressants are eligible).
•Evidence of severe retinopathy or clinically significant eye disease.
•History or presence of active serious or untreated autoimmune disease.
•History of solid organ transplant.
•Liver cirrhosis Child-Pugh score B or C. -≥ 5% blasts in peripheral blood or bone marrow.
•Prior systemic anti-cancer therapy or any investigational therapy ≤ 14 days or within five half-lives prior to starting study treatment, whichever is shorter.
•Major surgery 4 weeks prior to starting study drug or who have not fully recovered from major surgery.
•The diagnosis of another malignancy which, in the investigator's opinion, is likely to significantly impact study participation.
•Current evidence of uncontrolled, significant intercurrent illness including, but not limited to, the following conditions:
•Cardiovascular disorders:
•Uncontrolled hypertension, in the opinion of the investigator
•Congestive heart failure New York Heart Association Class II or greater, unstable angina pectoris, serious cardiac arrhythmias.
•Stroke or myocardial infarction within the past 3 months
•Significant coronary stenosis, in the opinion of the investigator
•QTc prolongation defined as a QTcF > 500 ms.
•Known congenital long QT.
•Any other condition that would, in the Investigator's judgment, contraindicate the subject's participation in the clinical study due to safety concerns or compliance with clinical study procedures (e.g., infection/inflammation, intestinal obstruction, unable to swallow medication, [subjects may not receive the drug through a feeding tube], social/ psychological issues, etc.)
•Known HIV infection with a detectable viral load within 6 months of the anticipated start of treatment.
•Note: Subjects on effective antiretroviral therapy with an undetectable viral load within 6 months of the anticipated start of treatment are eligible for this trial.
•Active infection requiring systemic therapy, including, but not limited to: tuberculosis (clinical evaluation that includes clinical history, physical examination, radiographic findings, and TB testing in line with local practice), hepatitis B (known positive HBV surface antigen (HBsAg) result), or hepatitis C.
•Note: Subjects with a past or resolved HBV infection (defined as the presence of hepatitis B core antibody [anti-HBc] and absence of HBsAg) are eligible. Subjects positive for hepatitis C (HCV) antibody are eligible only if polymerase chain reaction is negative for HCV RNA.
•Medical, psychiatric, cognitive, or other conditions that may compromise the subject's ability to understand the subject information, give informed consent, comply with the study protocol or complete the study.
•Known prior severe hypersensitivity to investigational product (IP) or any component in its formulations (NCI CTCAE v 6.0 Grade ≥ 3).
•Subjects taking prohibited medications as described in Section 7.
•A washout period of prohibited medications for a period of at least five half-lives or as clinically indicated should occur before the start of treatment.

研究组 & 干预措施

Treatment: All Patients

Experimental

This study will investigate the safety and tolerability of ropeginterferon alfa- 2b added on to standard of care ruxolitinib.

干预措施: ropeginterferon alfa- 2b (Drug)

Treatment: All Patients

Experimental

This study will investigate the safety and tolerability of ropeginterferon alfa- 2b added on to standard of care ruxolitinib.

干预措施: Ruxolitinib (Drug)