Randomized Polycythemia Vera Symptom Study comparing the effectiveness and patient assessed effects of Ruxolitinib with Hydroxyurea in a Phase 3 trial

Conditions: Polycythemia Vera
Therapeutic area: Diseases [C] - Cancer [C04]

Registration Number: EUCTR2012-002318-37-IE

Lead Sponsor: Incyte Corporation

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 100

Inclusion Criteria

Men or women aged = 18 years with a confirmed diagnosis of PV according to the revised World Health Organization criteria.
• Subjects must currently be reporting symptoms on a stable dose of HU monotherapy and be eligible to continue HU on study after randomization. Before screening, the subject must have been receiving HU for at least 12 weeks and on the same dose for the last 4 weeks.
• Subjects must have completed the modified MPN-SAF screening symptom form and have a screening total symptom score (TSS) of = 8 for the cytokine-related symptoms, or symptom cluster C (TSS-C): tiredness, itching, muscle aches, night sweats, and sweats while awake.
• Subjects should meet at least one of the following criteria with respect to phlebotomy and splenomegaly:
- No more than 2 phlebotomies within the 6 months before screening OR
- No palpable splenomegaly
• Subjects must have a hematocrit that can be controlled within 35% to 48% (inclusive) before randomization. (A phlebotomy may be performed during the screening phase to achieve this target range.)
• Subjects must have recovered from all side effects associated with phlebotomy, and at least 1 week must have elapsed between the phlebotomy and the beginning of the baseline phase.
• Subjects with Eastern Cooperative Oncology Group performance status of 0, 1, or 2 at screening.
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 50
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 50

Exclusion Criteria

• Subjects with inadequate liver or renal function at screening as demonstrated by:
- Encephalopathy = Grade 2 as per Child-Pugh System
- Hepatocellular disease
- Total bilirubin > 2 × upper limit of normal (ULN), unless direct bilirubin is < 2 × ULN
- Alanine aminotransferase > 2.5 × ULN
- Modification of Diet in Renal Disease estimated glomerular filtration rate < 30 mL/min/1.73 m2 or on dialysis
• Subjects with platelet count < 100 × 10^9/L or an absolute neutrophil count of < 1 × 10^9/L
• Subjects with peripheral blood blast count of > 0% at screening.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: To compare the efficacy of ruxolitinib and HU for reducing the cluster of symptoms defined by tiredness, itching, muscle aches, night sweats, and sweats while awake in subjects with PV.;Secondary Objective: To compare the efficacy of ruxolitinib and HU for reducing the following individual symptoms:<br>- Tiredness<br>- Itching<br>- Muscle aches<br>- Night sweats<br>- Sweats while awake<br>• To evaluate the durability of symptomatic improvement in subjects experiencing relief from the cluster of symptoms that includes tiredness, itching, muscle aches, night sweats, and sweats while awake, and for each of these symptoms individually.<br>• To evaluate the safety of ruxolitinib and HU.;Primary end point(s): Proportion of subjects with = 50% reduction in the TSS-C (tiredness, itching, muscle aches, night sweats, and sweats while awake) at Week 16 compared to baseline, as measured by the modified MPN-SAF diary.;Timepoint(s) of evaluation of this end point: Week 16

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Proportion of subjects with = 50% reduction in individual symptoms at Week 16 compared to baseline, as measured by the modified MPN-SAF diary for the following items:<br>- Tiredness<br>- Itching<br>- Muscle aches<br>- Night sweats<br>- Sweats while awake<br>Noncomparative Secondary Endpoints<br>• Proportion of subjects in Arm A who achieve a durable response, defined as a = 50% reduction in TSS-C at Week 16 that is maintained at Week 48.<br>• Proportions of subjects in Arm A who achieve a durable response, defined as a = 50% reduction in the individual symptoms in TSS-C (tiredness, itching, muscle aches, night sweats, and sweats while awake) at Week 16 that is maintained at Week 48.<br>• Safety of ruxolitinib and HU.;Timepoint(s) of evaluation of this end point: Week 16 and week 48

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