- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT00391560
Phase II Study of Perifosine in Patients With Refractory and Relapsed Leukemia
Study Overview
Detailed Description
This is a Phase II study of perifosine in patients with refractory and relapsed leukemia. After a one time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed.
Patients will be assessed for efficacy at the end of each 28 day cycle of therapy, +/- 7 days. Complete remissions, partial remissions and hematological improvements of any kind will be counted towards an objective response for all diseases.
A maximum total of 74 patients will be enrolled on the study, all of them assigned to the same experimental treatment scheme (arm) described above.
A maximum total of 37 evaluable patients will be entered in each of two diagnostic groups, which are being distinguished due to different anticipated rates of accrual. Group 1: AML, MDS, CML-BP non-lymphoid, CMML, Agnogenic Myeloid Metaplasia (AMM); Group 2: CLL, ALL, CML-BP lymphoid.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- Patients must have relapsed/refractory leukemias for which no standard therapies are anticipated to result in a durable remission. Patients with poor-risk myelodysplasia (MDS) [i.e. refractory anemia with excess blasts (RAEB-1 or RAEB-2) by WHO classification] and chronic myelomonocytic leukemia (CMML) are also candidates for this protocol. Relapsed/refractory leukemias include acute non-lymphocytic leukemia (AML) by WHO classification, acute lymphocytic leukemia (ALL), chronic lymphocytic leukemia (CLL), or chronic myelogenous leukemia (CML) in blast crisis. Patients with agnogenic myeloid metaplasia (AMM) are also eligible.
- ECOG performance status of 0-2
- Sexually active men and women who are not surgically sterile or post menopausal must use acceptable contraceptive methods (physician will discuss acceptable methods) during the time on study and for 4 weeks following the completion of treatment. Women of child-bearing potential (i.e., women who are pre-menopausal or not surgically sterile) must have a negative serum or urine pregnancy test within 2 weeks prior to beginning treatment on this trial.
- In the absence of rapidly progressing disease, the interval from prior treatment to time of study drug administration should be at least 2 weeks for cytotoxic agents, or at least five half-lives for noncytotoxic agents. Persistent chronic toxicities from prior chemotherapy must not be greater than grade 1.
Patients must have the following clinical laboratory values:
- Serum creatinine: <= 2.0 mg/dl
- Total bilirubin: <=1.5x the upper limit of normal unless considered due to Gilbert's syndrome
- Alanine aminotransferase (ALT), or aspartate aminotransferase (AST): <= 3x the upper limit of normal unless considered due to organ leukemic involvement
- Must be able and willing to give written informed consent
- Age equal to or greater than 18 years
Exclusion Criteria:
- Uncontrolled intercurrent illness including, but not limited to uncontrolled infection, symptomatic congestive heart failure, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
- Active heart disease including myocardial infarction within previous 3 months, symptomatic coronary artery disease, arrhythmias not controlled by medication, or uncontrolled congestive heart failure
- Patients with a history of severe hyper-reactive airway system (e.g. active asthma, COPD)
- Patients receiving any other standard or investigational treatment for their hematologic malignancy
- Pregnant and nursing patients are excluded because the effects of perifosine on a fetus or nursing child are unknown.
Study Plan
How is the study designed?
Design Details
- Primary Purpose: TREATMENT
- Allocation: NON_RANDOMIZED
- Interventional Model: PARALLEL
- Masking: NONE
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
EXPERIMENTAL: Group 1 on Perifosine
Patients with AML, MDS, CML-BP non-lymphoid, CMML, or Agnogenic Myeloid Metaplasia (AMM). After a one-time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed. |
Identical intervention in both arms.
Other Names:
|
EXPERIMENTAL: Group 2 on Perifosine
Patients with CLL, ALL, or CML-BP lymphoid. After a one-time loading dose of 600 mg (150 mg x 4 at least 4 hours apart) during the first cycle, perifosine will be given orally at 100 mg once a day continuously. Cycles are 28 days in length. Intra-patient dose escalation for the maintenance dose to 150 mg daily will be done in the second cycle if no non-hematological toxicities beyond grade 0-1 occurred during the first cycle are observed. |
Identical intervention in both arms.
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Overall Response Rate (CR + PR)
Time Frame: Every 4 weeks
|
All patients included in the study must be assessed for response to treatment, unless there are major protocol treatment deviations or they are ineligible. Even a 5% response rate would be of interest for this agent, given the different nature and mechanism of action of this compound. |
Every 4 weeks
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Time to Tumor Progression
Time Frame: Every 4 weeks
|
"Time to Progression" is defined as the period of time from the date of first study drug administration to the date that the patient is withdrawn because of clinical or radiographic progressive disease or death from any cause.
|
Every 4 weeks
|
Hematologic Improvement
Time Frame: Every 4 weeks
|
Hematological improvements of any kind will be counted towards an objective response for all diseases.
|
Every 4 weeks
|
Collaborators and Investigators
Sponsor
Investigators
- Study Chair: Frank Giles, MD, M.D. Anderson Cancer Center
Study record dates
Study Major Dates
Study Start
Primary Completion (ACTUAL)
Study Completion (ACTUAL)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (ESTIMATE)
Study Record Updates
Last Update Posted (ACTUAL)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- Perifosine 217
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Leukemia
-
Stanford UniversityTerminatedLeukemia | Leukemia, Lymphocytic, Acute | Leukemia Acute Promyelocytic Leukemia (APL) | Leukemia Acute Lymphoid Leukemia (ALL) | Leukemia Chronic Myelogenous Leukemia (CML) | Leukemia Acute Myeloid Leukemia (AML) | Leukemia Chronic Lymphocytic Leukemia (CLL)United States
-
Massachusetts General HospitalCelgene CorporationTerminatedAcute Myelogenous Leukemia | Acute Myeloid Leukemia (AML) | Acute Myelocytic Leukemia | Acute Granulocytic Leukemia | Acute Non-Lymphocytic LeukemiaUnited States
-
Institute of Hematology & Blood Diseases HospitalBejing Institute for Stem Cell and Regenerative Medicine; Institute for Stem...RecruitingRefractory Leukemia | Relapsed Leukemia | Acute Myeloid Leukemia, ChildhoodChina
-
Betta Pharmaceuticals Co., Ltd.Not yet recruitingAcute Myeloid Leukemia LeukemiaChina
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedChildhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia Without Maturation (M1) | Childhood Acute Myelomonocytic Leukemia (M4) | Childhood Acute Myeloid Leukemia/Other Myeloid MalignanciesUnited States
-
Hybrigenics CorporationUnknownAcute Myelogenous LeukemiaUnited States, France
-
Center for International Blood and Marrow Transplant...National Marrow Donor Program; St. Baldrick's FoundationActive, not recruitingAcute Myelogenous LeukemiaUnited States
-
Massachusetts General HospitalCompleted
-
Beijing Boren HospitalRecruitingAcute Myeloid Leukemia | Refractory Acute Myeloid Leukemia | Relapse LeukemiaChina
-
Kinex Pharmaceuticals Inc.CompletedAcute Myelogenous LeukemiaUnited States
Clinical Trials on perifosine
-
AEterna ZentarisCompletedNon Small Cell Lung CancerUnited States
-
NCIC Clinical Trials GroupNational Cancer Institute (NCI)CompletedSarcoma | Endometrial CancerCanada
-
University of Wisconsin, MadisonNational Cancer Institute (NCI)CompletedUnspecified Adult Solid Tumor, Protocol SpecificUnited States
-
University Health Network, TorontoNational Cancer Institute (NCI)Completed
-
National Cancer Institute (NCI)TerminatedHead and Neck CancerUnited States
-
NCIC Clinical Trials GroupNational Cancer Institute (NCI)CompletedMelanoma (Skin)Canada
-
Memorial Sloan Kettering Cancer CenterUniversity of Wisconsin, Madison; Duke University; AEterna ZentarisCompleted
-
National Cancer Institute (NCI)CompletedLymphoma | Myelodysplastic Syndromes | Leukemia | Unspecified Adult Solid Tumor, Protocol Specific | Myelodysplastic/Myeloproliferative NeoplasmsUnited States
-
Dana-Farber Cancer InstituteCompletedWaldenstrom's MacroglobulinemiaUnited States
-
Daphne FriedmanKeryx Biopharmaceuticals; Keryx / AOI Pharmaceuticals, Inc.CompletedChronic Lymphocytic Leukemia | Small Lymphocytic LymphomaUnited States