Saracatinib will be given orally at a dose of 125 milligrams once daily for 9 months. Saracatinib is provided as a pink tablet.

Pulmonary Lymphangioleiomyomatosis

Subjects will receive enough tablets for 90 days +/- 14 days at each visit. Subject will have visits every 90 days for drug accountability as well as safety and efficacy testing to include pulmonary function testing, laboratory testing to include liver and kidney profile, urine pregnancy testing at each visit, vital signs, physical examination - any medically significant changes from baseline visit will be recorded, all adverse events will be monitored until resolution.

saracatinib

Stanford University

Loyola Medical Center

Laboratory of Translational Research NHLBI

University of Cincinnati

Baylor College of Medicine - Ben Taub General Hospital

Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

Tony Eissa

Principal Investigator

Baylor College of Medicine

Brigham and Women's Hospital

Loyola University

University of South Florida

National Institutes of Health (NIH)

Sirolimus and MEC (Mitoxantrone, Etoposide, and Cytarabine)

Sirolimus and MEC (Mitoxantrone, Etoposide, and Cytarabine)
Sirolimus: Sirolimus, by mouth, will be given as a 12mg loading dose followed by 8 daily doses of 4mg/day.
MEC (Mitoxantrone, Etoposide, and Cytarabine): MEC (Mitoxantrone 8mg/m2/day IV, Etoposide 100mg/m2/day IV and Cytarabine 1000mg/ m2/day IV every 24 hours for 5 days) will be administered after sirolimus loading dose and 3 daily doses.

Sirolimus and MEC

Sex: Female, Male

Region of Enrollment

Percent change compared between response groups (responder vs nonresponder).
This outcome measure only includes patients who survived to outcome assessment.

Association Between the Magnitude of mTOR Target Inhibition Post-treatment in Leukemic Blasts and Clinical Response in Patients With High Risk AML Treated With Sirolimus MEC

Complete response is defined as:
Peripheral Blood Counts -Neutrophil count >1 x 109/L.
Platelet count ≥ 100 x 109/L.
Reduced hemoglobin concentration or hematocrit has no bearing on remission status.
Leukemic blasts must not be present in the peripheral blood.
Cellularity of bone marrow biopsy must be > 20% with maturation of all cell lines with < 5% blasts and no Auer rods.
Extramedullary leukemia, such as CNS or soft tissue involvement, must not be present

Complete Response

Complete response in the absence of platelet recovery is defined as:
- Bone marrow (<5% blasts) with adequate bone marrow cellularity, no evidence of circulating blasts or extramedullary disease and normalization of peripheral blood counts except for platelets (neutrophil count =1,000/µL)

Complete Response in the Absence of Platelet Recovery

Partial response is defined as:
Requires that all of the criteria for complete remission be satisfied except that the bone marrow may contain ≥ 5% blasts but < 25% blasts.
A marrow with <5% blasts that contain Auer rods will also be considered a PR

Partial Response

Overall Study

Margaret Kasner, MD

Sirolimus, by mouth, will be given as a 12mg loading dose followed by 8 daily doses of 4mg/day.

Sirolimus

Mitoxantrone

Etoposide

1000mg/m2/day IV every 24 hours for 5 days

Cytarabine

Kimmel Cancer Center at Thomas Jefferson University, an NCI-Designated Cancer Center

University of Pennsylvania

Thomas Jefferson University

Pilot Trial of Sirolimus/MEC in High Risk Acute Myelogenous Leukemia (AML)

A Pilot, Pharmacodynamic Correlate, Multi-Institutional Trial of Sirolimus in Combination With Chemotherapy (Mitoxantrone, Etoposide, Cytarabine) for the Treatment of High Risk, Acute Myelogenous Leukemia

tuberous sclerosis/tyrosine

Safety and Efficacy of Saracatinib In Subjects With Lymphangioleiomyomatosis

Pilot Trial of Sirolimus/MEC in High Risk Acute Myelogenous Leukemia (AML)

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