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Sorafenib and Low Dose Cytarabine in Older Patients With Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome

Phase 1
Completed
Conditions
Leukemia
Myelodysplastic Syndromes
Interventions
Registration Number
NCT00516828
Lead Sponsor
NCIC Clinical Trials Group
Brief Summary

RATIONALE: Sorafenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth or by blocking blood flow to the cancer. Drugs used in chemotherapy, such as cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving sorafenib together with cytarabine may kill more cancer cells.

PURPOSE: This phase I/II trial is studying the side effects and best dose of giving sorafenib together with cytarabine and to see how well it works in treating older patients with acute myeloid leukemia or high-risk myelodysplastic syndrome.

Detailed Description

OBJECTIVES:

* To determine the recommended dose of sorafenib tosylate and cytarabine when given in combination to elderly patients with acute myeloid leukemia or high-risk myelodysplastic syndromes who are not suitable for intensive chemotherapy. (Phase I)

* To determine the safety, tolerability, toxicity profile, and dose-limiting toxicities in patients treated with this regimen. (Phase I)

* To estimate the efficacy (as measured by complete response rate) in patients treated with this regimen. (Phase II)

* To describe the toxic effects and overall response rate (complete and partial) in patients treated with this regimen. (Phase II)

* To evaluate potential correlates of response in translational research studies including FLT-3 internal tandem duplications and point mutations in blasts. (Phase II)

OUTLINE: This is a multicenter study.

* Phase I: Patients receive oral sorafenib tosylate twice daily on days 2-28 and cytarabine subcutaneously twice daily on days 1-10 at the dose level assigned at registration. Doses of both drugs will be escalated and the recommended doses for the combination will be determined. Treatment repeats every 28 days for up to 6 courses in the absence of disease progression or unacceptable toxicity. Patients who respond to treatment will receive 2 cycles after response criteria are met.

* Phase II: Patients receive sorafenib tosylate and cytarabine as in phase I at the recommended doses for the combination determined in phase I.

Bone marrow (or blood) samples are collected at baseline and at the end of each course of study treatment. Baseline samples are analyzed for mutational status of FLT-3 (i.e., internal tandem duplication \[ITD\] and point mutations).

After completion of study treatment, patients are followed at 4 weeks and then every 3 months thereafter until progression and toxicities resolve.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
21
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Sorafenib and CytarabinecytarabineCytarabine: subcutaneously twice daily from day 1 - 10. Sorafenib: Days 2-28; at the dose level assigned at registration. Sorafenib will be given orally twice daily.
Primary Outcome Measures
NameTimeMethod
Recommended phase II dose of sorafenib tosylate when given in combination with cytarabine (Phase I)29 months
Dose-limiting toxicity (Phase I)29 months
Complete remission (Phase II)29 months
Secondary Outcome Measures
NameTimeMethod
Overall response rate (complete and partial response) (Phase II)29 months
Time to progression (Phase II)29 months
Overall survival (Phase II)29 months
FLT-3 ITD endpoint mutation response correlation.29 months
Toxicity (Phase II)29 months

Trial Locations

Locations (4)

Juravinski Cancer Centre at Hamilton Health Sciences

🇨🇦

Hamilton, Ontario, Canada

Univ. Health Network-Princess Margaret Hospital

🇨🇦

Toronto, Ontario, Canada

McGill University - Dept. Oncology

🇨🇦

Montreal, Quebec, Canada

QEII Health Sciences Center

🇨🇦

Halifax, Nova Scotia, Canada

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