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Study Summary
No. 2011-0016:.......Myeloproliferative Diseases......Srdan Verstovsek......Leukemia
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Study Summary Title
Study Summary
Number:
2011-0016
Study Title:A Phase 2 Study to Evaluate the Efficacy and Safety of GS-6624 in Adult Patients with Primary, Post Polycythemia Vera or Post Essential Thrombocythemia Myelofibrosis
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Physician New Patient Referral
Name:Srdan VerstovsekPatients Call:800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Dept:LeukemiaReferring MD
Call:
800-392-1611 (in U.S.A.) 713-792-6161 (outside U.S.A.)
Phone:713-792-7305
Contact us about clinical trials
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General Information
Disease Group:Myeloproliferative DiseasesSupported By:Gilead Sciences, Inc.
Phase of Study:Phase IIReturn
Visit:
Treatment
Agents:
AB0024Home Care:
Treatment Loc:Both at MDACC & and Other Sites
Estimated
Length of Stay
in Houston:
Description/
Intervention:
The goal of this clinical research study is to compare the effectiveness and
safety of GS-6624 at different dose levels with ruxolitinib in patients with
myelofibrosis.

GS-6624 is a drug that is designed to bind to certain proteins. This may
prevent or slow the growth of cancer cells.

Ruxolitinib is designed to block a gene mutation that may be important in
cancer cell growth and survival. Ruxolitinib is the standard of care drug.
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Study Objectives / Outcomes
Primary Objective:

· To evaluate the effects of GS-6624 on bone marrow fibrosis alone and in combination with ruxolitinib in subjects with Primary Myelofibrosis (PMF) and Post-Polycythemia Vera or Post-Essential Thrombocythemia Myelofibrosis (post-PV MF or post-ET MF).

Secondary Objective(s):

· To evaluate the effects of AB0024 on Myelofibrosis Symptoms Assessment Score;
· To evaluate the effects of AB0024 on cytokines;
· To evaluate the formation of anti-AB0024 antibodies.

  • To evaluate the effects of GS-6624 on hematologic parameters alone and in combination with ruxolitinib in subjects with PMF and post-PV MF or post-ET MF
  • To evaluate the safety of GS-6624
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Study Status Information
Study Activation / Registration Date:07/12/2011
IRB Review and Approval Date:07/12/2011
Study Type:Phase Ii Or Phase I/Ii
Recruitment Status:Open
Projected Accrual:54
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Enrollment Eligibility
If you do not meet the enrollment eligibility, there may be other treatment options for you. Please Contact the Referral Office for more information.

Inclusion Criteria:1) Must be equal to or more than 18 years of age.

2) Must be diagnosed with PMF or post ET/PV MF with intermediate-1, intermediate-2 or high risk disease according to the IWG prognostic scoring system, or if with low risk disease then with symptomatic splenomegaly that is equal to or more than 10 cm below left costal margin by physical exam.

3) Must have adequate organ function as demonstrated by the following: a. ALT (SGOT) and/or AST (SGPT) equal to or less than 2.5x upper limit of normal (ULN), or equal to or less than 4x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to MF); b. Direct bilirubin equal to or less than 1.5 x ULN; or equal to or less than 2x ULN (if upon judgment of the treating physician, it is believed to be due to extramedullary hematopoiesis [EMH] related to MF); c. Serum creatinine equal to or less than 2.5 mg/dL.

4) ECOG performance status (PS) equal to or less than 2.

5) Treatment-related toxicities from prior therapies must have resolved to Grade equal to or less than 1.

6) In Stage 2, subjects must be on ruxolitinib for at least 8 weeks and on a stable dose for at least 4 weeks.

7) Women of childbearing potential and men must agree to using one medically approved (i.e., mechanical or pharmacological) contraceptive measure and have their partners agree to an additional barrier method of contraception for the duration of the study and for 90 days after the last administration of study drug. A female is considered to be of childbearing potential unless she has had a hysterectomy, is at least one year post-menopausal, or has undergone tubal ligation.

Exclusion Criteria:1) Any serious medical condition or psychiatric illness that would prevent, (as judged by the treating physician) the subject from signing the informed consent form or any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.

2) Pregnant or lactating,

3) Known history of human immunodeficiency virus (HIV), hepatitis C, or hepatitis B.

4) History or presence of any form of cancer within the 3 years prior to enrollment, with the exception of excised basal cell or squamous cell carcinoma of the skin, or cervical carcinoma in situ or breast carcinoma in situ that has been excised or resected completely and is without evidence of local recurrence or metastasis.

5) Participation in an investigational drug or device trial within 2 weeks prior to study Day 1 or within 5 times the half-life of the investigational agent in the other clinical study, if known.

6) Use of any growth factors, cytotoxic chemotherapeutic agents (e.g., hydroxyurea), corticosteroids (prednisone equal to or less than 10 mg/day or corticosteroid equivalent is allowed), or immunodulators (e.g., thalidomide) within 2 weeks and interferon use within 4 weeks prior to study Day 1.

7) Symptomatic congestive heart failure, unstable angina, or unstable cardiac arrhythmia requiring medication.

8) History of surgery within 2 weeks prior to enrollment or anticipated surgery during the study period.

9) Any other condition that might reduce the chance of obtaining data required by the protocol or that might compromise the ability to give truly informed consent.

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Links
Registration Number: NCT01369498
Study Information on Clinical Trials Registry (clinicaltrials.gov)

Other Links:
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Results


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