Back to resultsA Safety Study of XL019 in Adults With Myelofibrosis
Primary Purpose
Myeloproliferative Disorders, Myelofibrosis, Polycythemia Vera
Status
Terminated
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
XL019
Sponsored by
About this trial
This is an interventional treatment trial for Myeloproliferative Disorders focused on measuring Myeloproliferative Disorders
Eligibility Criteria
Inclusion Criteria:
- The subject has primary myelofibrosis (PMF), post-polycythemia vera MF, or post-essential thrombocythemia MF and requires therapy, including subjects who have received prior MF-directed therapy and relapsed or subjects with refractory disease; or if newly diagnosed, then with intermediate or high risk according to the Lille scoring system.
- The subject is unwilling to undergo or is not a candidate for peripheral stem cell/bone marrow transplant.
- 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.
- 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 diagnosis of malignancy or evidence of other malignancy 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 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.
Sites / Locations
- UCSF - Division of Hematology/Oncology
- H. Lee Moffitt Cancer Center & Research Institute
- Dana Farber Cancer Institute
- Mt. Sinai School of Medicine
- MD Anderson Cancer Center
Outcomes
Primary Outcome Measures
To evaluate the safety, tolerability, maximum tolerated dose, and dose-limiting toxicities of XL019 as a single agent when orally administered in subjects with PMF, post-PV MF, or post-ET MF.
Secondary Outcome Measures
Determine plasma pharmacokinetics, evaluate pharmacodynamic correlates, and estimate renal elimination of XL019
Evaluate preliminary efficacy of XL019 as a single agent when administered orally
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00522574
Brief Title
A Safety Study of XL019 in Adults With Myelofibrosis
Official Title
A Phase 1 Dose-Escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of XL019 Administered to Subjects With Myelofibrosis
Study Type
Interventional
2. Study Status
Record Verification Date
April 2011
Overall Recruitment Status
Terminated
Why Stopped
Due to emerging safety data
Study Start Date
August 2007 (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Name of the Sponsor
Exelixis
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of this study is to evaluate the safety and tolerability of XL019 in adults with myelofibrosis. XL019 is a selective inhibitor of the cytoplasmic tyrosine kinase JAK2. JAK2 is activated by cytokine and growth factor receptors and phosphorylates members of the STAT family of inducible transcription factors. Activation of the JAK/STAT pathway promotes cell growth and survival, and is a common feature of human tumors. JAK2 is activated by mutation in the majority of patients with myelofibrosis, polycythemia vera and essential thrombocytosis and appears to drive the inappropriate growth of blood cells in these conditions.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myeloproliferative Disorders, Myelofibrosis, Polycythemia Vera, Thrombocythemia, Essential
Keywords
Myeloproliferative Disorders
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
XL019
Intervention Description
gelatin capsules supplied in 5-mg, 25-mg, and 100-mg strengths
Primary Outcome Measure Information:
Title
To evaluate the safety, tolerability, maximum tolerated dose, and dose-limiting toxicities of XL019 as a single agent when orally administered in subjects with PMF, post-PV MF, or post-ET MF.
Time Frame
Assessed at periodic visits
Secondary Outcome Measure Information:
Title
Determine plasma pharmacokinetics, evaluate pharmacodynamic correlates, and estimate renal elimination of XL019
Time Frame
Assessed at periodic visits
Title
Evaluate preliminary efficacy of XL019 as a single agent when administered orally
Time Frame
Assessed at periodic visits
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
The subject has primary myelofibrosis (PMF), post-polycythemia vera MF, or post-essential thrombocythemia MF and requires therapy, including subjects who have received prior MF-directed therapy and relapsed or subjects with refractory disease; or if newly diagnosed, then with intermediate or high risk according to the Lille scoring system.
The subject is unwilling to undergo or is not a candidate for peripheral stem cell/bone marrow transplant.
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.
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 diagnosis of malignancy or evidence of other malignancy 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 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.
Facility Information:
Facility Name
UCSF - Division of Hematology/Oncology
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
H. Lee Moffitt Cancer Center & Research Institute
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Dana Farber Cancer Institute
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Mt. Sinai School of Medicine
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
12. IPD Sharing Statement
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A Safety Study of XL019 in Adults With Myelofibrosis
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