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Dose Escalation Phase I/II Study of Lovastatin With High-Dose Cytarabine for Refractory or Relapsed AML

Phase 1
Terminated
Conditions
Acute Myeloid Leukemia
Interventions
Registration Number
NCT00583102
Lead Sponsor
University of Iowa
Brief Summary

The purpose of this study is to test the safety and effectiveness of combining a drug known as Lovastatin to the chemotherapy drug cytarabine. Lovastatin is currently used to lower blood cholesterol levels and lab data suggests that it increases the anti-leukemia activity of cytarabine. This research is being done because high doses of cytarabine induce remissions in only about 25% of patients with acute myeloid leukemia.

Detailed Description

The rationale for combining lovastatin with cytosine arabinoside (HiDAC) in this trial is based on a study in press in Leukemia Research. This study demonstrated that there are synergistic interactions between cytosine arabinoside and lovastatin against human leukemia cell lines. In particular, this synergistic activity was observed in MTT assay. Given that there is such synergistic interaction in vitro it is reasonable to determine whether such interaction occurs in vivo. The proposed trial thus uses standard doses of HiDAC with incrementally increasing dose of lovastatin. This particular trial will follow an accelerated titration for lovastatin. The first dose level will be lovastatin at 0.5 mg/kg/day, divided into two daily PO doses given Q 12 hours on Days 1 -7 for a total of 14 doses. Doses should be rounded to the nearest 20 mg. After each subject reaches day 14, subsequent subjects will be treated at incrementally increasing doses that are 1 mg/kg/day, 2 mg/kg/day, 4 mg/kg/day, 8 mg/kg/day, 12 mg/kg/day, 18 mg/kg/day, and 24 mg/kg/day, with all doses divided into two daily PO doses given Q 12 hours on Days 1 - 7 for a total of 14. If MTD is not reached at the 24 mg/kg/day dose level, further dose escalations will occur with a 33% increase in dose at each level, rounded to the nearest 20 mg/kg/day.

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
23
Inclusion Criteria
  • Patients with primary refractory AML (that is no prior remission). Patients who have greater than 10% AML blasts in the bone marrow or blood upon recovery from two cycles of standard cytarabine- and anthracycline-based induction chemotherapy are eligible. Patients who have received etoposide and/or 6-thioguanine during remission induction will be eligible.
  • Patients with relapsed AML. Patients must have had a documented remission lasting > 30 days at some point during their prior therapy. Their current relapse must be untreated. Relapse is defined as the presence of greater than 10% AML blasts in the bone marrow or blood after having had a documented remission.
  • Patients who have received a high-dose cytarabine containing regimen (>2 g/m2/dose) within 3 months prior to registration on this protocol are not eligible.
  • No active CNS involvement. A lumbar puncture prior to treatment is not required and should not be performed in the absence of significant CNS symptoms or signs.
  • Non-pregnant and non-nursing. Treatment under this protocol would expose an unborn child to significant risks. Women and men of reproductive potential should agree to use an effective means of birth control.
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Exclusion Criteria

Although NOT considered formal Exclusion Criteria, study physicians are strongly encouraged as part of this decision-making process to recognize that the following may increase the risks to a subject entering this protocol:

  • Other serious illnesses which would limit survival to <2 years, or a psychiatric condition which would prevent compliance with treatment or informed consent.
  • Performance Status > 2.
  • Uncontrolled or severe cardiovascular disease, diabetes, pulmonary disease, or infection, which in the opinion of the treating physician, would make this protocol treatment unreasonably hazardous for the patient.
  • Patients with a "currently active" second malignancy other than non-melanoma skin cancers. Patients are not considered to have a "currently active" malignancy if they have completed therapy and considered by their physician to be at less than 30% risk of relapse within one year.
  • Patients who have received any investigational agent within the prior 4 weeks.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Lovastatin followed by CytarabineCytarabineThe subject will receive high dose cytarabine as well as lovastatin. The subject will take doses of lovastatin twice a day, about 12 hours apart. On the third day, the subject will begin high-dose cytarabine IV over 3 hours, twice a day, starting 1 hour after the lovastatin dose for 5 days.
Lovastatin followed by CytarabineLovastatinThe subject will receive high dose cytarabine as well as lovastatin. The subject will take doses of lovastatin twice a day, about 12 hours apart. On the third day, the subject will begin high-dose cytarabine IV over 3 hours, twice a day, starting 1 hour after the lovastatin dose for 5 days.
Primary Outcome Measures
NameTimeMethod
Complete Remission Rate5 weeks

The primary study end point will be complete remission rate.

Complete Remission (CR):

Complete remission is defined as the presence of all of the following:

Peripheral Blood Counts (sustained \> 30 days)

* Absolute neutrophil count ³1500/ml.

* Platelet count ³100,000/ml.

* No leukemic blasts in the peripheral blood.

* Transfusion independent for red cells and platelets. Bone Marrow

* Cellularity \>20% with maturation of all cell lines.

* No Auer rods.

* \<5% blast cells. No extramedullary leukemia (such as CNS or soft tissue involvement). OR Complete Response with Incomplete Platelet Recovery (CRp): CRp satisfies all CR criteria except platelets \< 100,000/µL.

Partial Remission (PR):

Must meet all criteria of a CR except that the bone marrow may contain 5-24% blasts.

Treatment Failure:

Failure to achieve a CR.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (1)

Holden Comprehensive Cancer Center

🇺🇸

Iowa City, Iowa, United States

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