2024-515619-23-00招募中2 期
Ruxolitinib versus Best Available Therapy in patients with high-risk polycythemia vera or high-risk essential thrombocythemia – The Ruxo-BEAT Trial
Universitaetsklinikum Aachen AöR17 个研究点 分布在 1 个国家目标入组 223 人开始时间: 2024年8月9日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 223
- 试验地点
- 17
- 主要终点
- The rate of complete clinicohematologic responses (CHR) at month 6, compared to baseline, as defined by (Barosi, et al., 2009).
研究概览
简要总结
The objective of the Ruxo-BEAT trial is to assess the feasibility, safety, and efficacy of administration of ruxolitinib vs. best available therapy in patients with high-risk PV or high-risk ET.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •Subjects must provide written informed consent prior to study-specific procedures or assessments which are not routinely performed for diagnosis or monitoring of PV or ET, and the subjects must be willing to comply with treatment and to follow up assessments and procedures
- •Patient must be 18 years of age or older
- •Patient´s ECOG performance status must be 0-2
- •Patient must fulfill WHO 2008 diagnostic criteria for either polycythemia vera (PV) or essential thrombocythemia (ET). Moreover, PV- and ET-patients have to be classified as high risk according to defined criteria as outlined below. - For patients with high risk PV OR PV with indication for cytoreductive therapy due to progressive myeloproliferation, AT LEAST ONE of the following must be fulfilled (according to DGHO onkopedia) (Barbui, et al., 2011), (Passamonti, 2009): Age > 60 years; Previous documented thrombosis or thromboembolism; Platelet count > 1500 x 10^9/l; Poor tolerance of phlebotomy or frequent phlebotomy requirement; Symptomatic or progressive splenomegaly; Severe disease-related symptoms (according to the investigators definition); Progressive leukocytosis with leukocyte count > 20 x 10^9/l. - For patients with high risk ET, AT LEAST ONE of the following must be fulfilled (according to DGHO guidelines): Age > 60 years; Platelet count > 1500 x 10^9/l; Previous thrombosis or thromboembolism; Previous severe hemorrhage related to ET (defined as decrease of Hgb of at least 2 g/dl)
- •Patients must fulfill the following criteria regarding prior therapy: PV patients: Never treated with cytoreductive drugs except hydroxyurea, anagrelide, or interferon for up to 6 weeks maximum (phlebotomy and/or aspirin are allowed). ET patients: Naïve and pretreated patients may be entered in this trial.
- •Patient must have adequate liver function as indicated by a total bilirubin, AST, and ALT ≤ 2 of the institutional upper limit of normal (ULN) value, unless directly attributable to the patient’s MPN.
- •Patient must have a creatinine clearance >40ml/min calculated according to the modified formula of Cockcroft and Gault, eGFR, or directly measured after 24h-urine collection.
- •Patient must be able to swallow and retain oral medication
排除标准
- •Patients who meet criteria for post PV-MF or post ET-MF (IWG-MRT).
- •Any circumstance at the time of study entry that would preclude completion of the study or the required follow-up prohibits inclusion into this study.
- •Subjects who have had an active malignancy during the previous 3 years except for treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, each with no evidence for recurrence in the past 3 years.
- •Patients who have uncontrolled bacterial, viral, or fungal infection.
- •Patients who have any medical condition requiring prolonged use of oral corticosteroids with a dose of more than 20 mg per day (> 1 month).
- •Patients who have severe cerebral dysfunction and/or legal incapacity.
- •Patients who have had active splanchnic vein thrombosis within the last 3 months (includes Budd-Chiari, portal vein, splenic and mesenteric thrombosis).
- •Patients who have thyroid dysfunction which is not adequately controlled.
- •Fertile men or women of childbearing potential cannot be included unless they are: -surgically sterile or > 2 years after the onset of menopause and/or -willing to use a highly effective contraceptive method (Pearl Index <1) such as oral contraceptives, intrauterine device, sexual abstinence, or barrier method of contraception (i.e. condoms) in conjunction with spermicidal jelly during study treatment.
- •Patients who are taking any of the following prohibited medication: -clarithromycin, telithromycin, troleandomycin (antibiotics) -ritonavir, indinavir, saquinavir, nelfinavir, amprenavir, lopinavir (HIV protease inhibitors) -itraconazole, ketoconazole, voriconazole, fluconazole (antifungals).
- •Patients with a diagnosis of galactose or lactose intolerance or a glucosegalactose- malabsorption.
- •Patients who have received previous ruxolitinib treatment
- •Patients who have a history of anaphylaxis following exposure to the BAT drug of choice.
- •Patients who have an inadequate bone marrow reserve as demonstrated by ANC ≤ 1 x 10^9/l OR platelet count <50 x 10^9/l.
- •Patients who have known hepatitis B or C or HIV infection.
- •Patients who suffer from other severe, concurrent diseases, including tuberculosis, serious cardiac functional dysfunction (class III or IV as defined by the New York Heart Association Classification), uncontrolled diabetes, uncontrolled hypertension, severe pulmonary disease (i.e. COPD with hypoxemia), or major organ malfunction that could interfere with the patient’s ability to participate in the study.
- •Patients who have history of active substance or alcohol abuse within the last year.
- •Female patients who are pregnant or nursing.
- •Patients who have participated in another interventional trial and/or used investigational agents or concurrent anticancer treatment for concomitant disease within the last 4 weeks of registration.
结局指标
主要结局
The rate of complete clinicohematologic responses (CHR) at month 6, compared to baseline, as defined by (Barosi, et al., 2009).
The rate of complete clinicohematologic responses (CHR) at month 6, compared to baseline, as defined by (Barosi, et al., 2009).
次要结局
- Safety of both regimens.
- The rate of complete clinicohematologic responses (CHR) at month 12 and month 24 as defined by Barosi et al. Blood 2009 (with the clinical modification that splenomegaly assessment may be done by ultrasound or palpation).
- To determine the complete response (CR) rate at month 6 as defined by Barosi et al. Blood 2013 (revised ELN response criteria, see section 15.6) (Barosi, et al., 2013).
- The efficacy as assessed by both the absence of phlebotomy (Hct <45%) and reduction in spleen size (palpable spleen that is reduced by > 50% from baseline measured by palpation or ultrasound) OR platelet count < 600 x 10^9/l (ET) at month 6 and month 24
- The proportion of subjects achieving both durable absence of phlebotomy eligibility and durable spleen size reduction measured by palpation (PV) OR durable platelet count < 600 x 10^9/l (ET) (durable defined as > 3 months) (Barosi et al. 2009/2013) at month 6, month 12 and month 24.
- The rate of overall clinicohematologic responses (CR + PR) according to both guidelines (Barosi et al. 2009 and 2013) at month 6 and month 24.
研究者
Univ.-Prof. Dr. med. Steffen Koschmieder
Scientific
Universitaetsklinikum Aachen AöR
研究点 (17)
Loading locations...
相似试验
招募中
1 期
Ruxolitinib in Combination With CHOP Chemotherapy for the Treatment of Untreated Nodal T-Follicular Helper Cell LymphomasNCT07278856City of Hope Medical Center20
招募中
4 期
Zanubrutinib Combined With Rituximab in the Treatment of Secondary HLH in B-cell LymphomaHemophagocytic LymphohistiocytosisNCT07270835The First Affiliated Hospital of Soochow University40
招募中
2 期
Multicenter, open-label, phase II study in patients with immunoglobulin M monoclonal gammopathy of unknown significance and Myelin Associated Glycoprotein antibodies related polyneuropathy and Zanubrutinib Treatment2023-505933-29-00University Medical Center Utrecht42
招募中
2 期
Evaluation of the efficacy and safety of Rituximab in patients with Amyotrophic Lateral Sclerosis (ALS)2022-502743-35-00Deutsches Zentrum Fuer Neurodegenerative Erkrankungen e.V.52
尚未招募
3 期
Innovating Shorter, All- Oral, Precised Treatment Regimen for Rifampicin Resistant Tuberculosis：BLMZ Chinese CohortPulmonary TuberculosisTuberculosisDrug-resistant TuberculosisNCT07268664Beijing Chest Hospital120