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Prospective national multicenter randomized open label phase IIb RUXBETA trial.
Full description
A randomized, open label, multicenter phase IIb study to evaluate the efficacy and safety of Ruxolitinib versus best available therapy in patients with high risk essential thrombocythemia, who are resistant or intolerant to hydroxyurea.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
Target Population
Men and women, age more than or equal18 years and less than 75 years.
Confirmed diagnosis of Essential Thrombocythemia for at least 6 months, according to the 2008 WHO criteria, with a high-risk status.
Patients must have a treatment history for ET that meet the definition of resistance or intolerance to hydroxyurea therapy according to the ELN criteria as follow:
ECOG Performance Status (ECOG PS) less than or equal 2 at screening and at baseline.
Adequate Organ Function:
Direct bilirubin less than 2.0 times the institutional Upper Limit of Normal (ULN).
Hepatic enzymes (AST, ALT) less than or equal 2.5 times the institutional ULN.
Adequate renal function at screening as demonstrated by MDRD-eGFR more than 30 mL/min/1.73m2.
Women of childbearing potential (WOCBP) must be using an adequate method of contraception to avoid pregnancy during and after the study.
Women of childbearing potential must have a negative serum (beta-human chorionic gonadotropin hCG pregnancy test at Screening.
Signed Written Informed Consent.
Health insurance coverage.
Exclusion criteria
Medical history and concurrent diseases:
Clinically significant cardiac disease (NYHA Class III or IV).
Chronic hepatocellular disease.
Subjects with impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of Ruxolitinib
Subjects with clinically significant bacterial, fungal, parasitic or viral infection which requires therapy:
History of progressive multifocal leucoencephalopathy (PML).
Other malignant disease during the last 5 years prior to the inclusion except treated cervical intraepithelial neoplasia, basal cell carcinoma of the skin, or squamous cell carcinoma of the skin, with no evidence for recurrence in the past 3 years.
History of significant bleeding disorder not related to the ET.
Subjects with an uncontrolled undercurrent illness or any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the subject or compliance with the protocol.
Subjects being treated concurrently with a potent systemic inhibitor of CYP3A4 at the time of screening.
Subjects being treated concurrently with any prohibited medications.
Women who are pregnant or breastfeeding are not eligible for this study.
Inability to freely provide consent through judiciary or administrative condition.
Ongoing participation to another clinical investigational study.
Primary purpose
Allocation
Interventional model
Masking
13 participants in 2 patient groups
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Data sourced from clinicaltrials.gov
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