Expanded Treatment Protocol of ruxolitinib in Polycythemia Vera patients who are HU resistant or intolerant

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 500

Inclusion Criteria

1. Male or female aged 18 or over
2. Confirmed diagnosis of PV according to the revised World Health Organization (WHO) criteria with resistance or intolerance to
hydroxyurea
3. HU resistant or intolerant
4. Peripheral blood blast count of 0% at screening
5. Palpable spleen (unless the patient is entering the protocol after he/she has successfully completed another clinical trial with ruxolitinib in PV and the study team has agreed that the patients can be included in this protocol)
6. ECOG performance status of 0, 1, or 2 at Baseline
7. Must have recovered or stabilized sufficiently from adverse drug reactions associated with any prior treatments before beginning treatment with ruxolitinib
8. Does not have access to a comparable or satisfactory alternative treatment (i.e., comparable or satisfactory treatment is not available or does not exist)
9. Is not eligible for participation in any of the ruxolitinib ongoing clinical trials or has recently completed a clinical trial that has been terminated and, after considering other options (e.g., trial extensions, amendments, etc.), the clinical team has determined that treatment is necessary and there are no other feasible alternatives for the patient)
10. Is not being transferred from an ongoing clinical trial for which he/she is still eligible
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 200
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 300

Exclusion Criteria

1. Inadequate liver or renal function
2. Platelet counts < 50 ×109/L or an Absolute Neutrophil Count (ANC) < 1 × 109/L at screening
3. Presence of active bacterial, fungal, parasitic or viral infection which require therapy
4. History of progressive multifocal leukoencephalopathy (PML)
5. Significant concurrent, uncontrolled medical condition that, in the Investigator's opinion would jeopardize the safety of the patient or compliance with the protocol

Other exclusion criteria as per protocol section 5.3 may apply.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: to evaluate the safety of ruxolitinib;Secondary Objective: Efficacy<br>- to evaluate change in hematrocrit levels<br>- to evaluate change in speen length<br><br>Patient Reported Outcomes:<br>- to evaluate change in MPN-SAF TSS score;Primary end point(s): 1. AEs, Grade 3&4 AEs & SAEs, events of special interest, AEs leading to<br>discontinuation, and deaths.<br>2. Evaluate changes in hematology;Timepoint(s) of evaluation of this end point: refer to protocol section 10.4

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Efficacy:<br>- Change in Hct levels from Baseline to each visit where measured<br>- Change in spleen length from Baseline to each visit where measured<br><br>Patient Reported Outcome:<br>Change in MPN-SAF TSS score from baseline to each visit where measured;Timepoint(s) of evaluation of this end point: Efficacy:<br>- Week 4, 8, 12, 16, 20, 24, 36, 48, 60, 72, 84, 96, 108<br>- Week 12, 24, 36, 48, 60, 72, 84, 96, 108<br><br>Patient Reported Outcome:<br>Week 12, 24, 36, 48, 60, 72, 84, 96, 108

Expanded Treatment Protocol of ruxolitinib in Polycythemia Vera patients who are HU resistant or intolerant

Recruitment & Eligibility

Study & Design

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