A Phase 1 Study of XL019 in Adults With Polycythemia Vera

Study Overview
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Brief Summary
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The purpose of this study is to evaluate the safety and tolerability of the JAK2 inhibitor XL019 administered orally in adults with Polycythemia Vera.

Official Title
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A Phase 1 Dose-Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of XL019 Administered to Subjects With Polycythemia Vera

Conditions
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Polycythemia Vera

Intervention / Treatment
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* Drug: XL019


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: The subject has a diagnosis of polycythemia vera (PV), and has failed, or is intolerant of, standard therapies or refuses to take standard medications. The subject is ≥18 years old. The subject has an Eastern Cooperative Oncology Group (ECOG) performance status of ≤2. The subject has adequate organ function. Subjects who have received phlebotomy due to PV must have documented phlebotomy history for 12 weeks prior to enrollment. The subject has the capability of understanding the informed consent document and has signed the informed consent document. Sexually active subjects (male and female) must use medically acceptable methods of contraception during the course of the study. Female subjects of childbearing potential must have a negative pregnancy test at screening. The subject has had no other diagnosis of malignancy or evidence of other malignancey for 2 years prior to screening for this study (except non-melanoma skin cancer or in situ carcinoma of the cervix). Exclusion Criteria: The subject has received treatment for PV within 14 days prior to first dose of XL019 The subject has uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, hypertension, symptomatic congestive heart failure, unstable angina pectoris, myocardial infarction within 3 months, or cardiac arrhythmias. The subject is pregnant or breastfeeding. The subject is known to be positive for the human immunodeficiency virus (HIV). The subject is unable or unwilling to abide by the study protocol or cooperate fully with the investigator or designee.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: XL019<br>* XL019 capsules administered orally<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability of XL019 as a single agent when orally administered in adults with Polycythemia Vera (PV) |  | Assessed at each visit | 
| Determine the maximum tolerated dose (MTD) and dose-limiting toxicities (DLTs) for XL019 |  | Assessed at periodic visits | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluate pharmacokinetic and pharmacodynamic parameters of XL019 in adults with PV |  | Assessed during periodic visits | 
| Evaluate preliminary efficacy of XL019 |  | Assessed weekly or bi-weekly | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Polycythemia Vera, PV