A Study of Romiplostim to Prevent Low Platelet Counts in Children and Young Adults Receiving Chemotherapy for Solid Tumors
A Study of Romiplostim to Prevent Low Platelet Counts in Children and Young Adults Receiving Chemotherapy for Solid Tumors
The purpose of this study is to find out whether romiplostim can help prevent low platelet
 counts caused by N8 or EFT chemotherapy, reduce the number of platelet transfusions required
 during chemotherapy, and prevent treatment delays due to low platelet counts.
Inclusion Criteria:

 -  Documented diagnosis of a primary solid tumor. Patients must have histological
 verification of malignancy at MSKCC.

 -  Male and female patients aged 1-21 years with a primary solid tumor undergoing
 treatment with the pre-defined chemotherapy regimens of N8 or EFT. Prior to enrollment
 patient could have been undergoing induction therapy with a similarly myelosuppressive
 regimen as long as they will be continuing with N8 or EFT at the time of study
 enrollment.

 Note: Patients who received 3 prior cycles of induction chemotherapy, prior to enrollment
 will be eligible for this study.

 -  Total Bilirubin (sum of conjugated + unconjugated) ≤ 3 times institutional upper limit
 of normal (ULN) for age and ALT/AST ≤ 3 times institutional ULN for age.

 -  Normal cardiac function:

 -  Shortening fraction greater than or equal to 28% by echocardiogram OR Left
 ventricular ejection fraction (LVEF) greater than or equal to 50% on technetium-
 99m pertechnetate radionuclide cineangiography (MUGA) or echocardiogram.

 -  Screening ECG with corrected QT (QTc) interval of < 470 msec.

 -  Timing of cardiac assessment: We will utilize the most recent EKG/ECHO when
 assessing cardiac function. See section 9.0 for additional details.

 -  Adequate renal function, defined as an estimated Creatinine Clearance or GFR >40ml/min
 or an normal creatine for age (see below)

 Serum Creatinine by age:

 Age (years) <6: Maximum Serum Creatinine (mg/dL), Male 0.8, Female 0.8 Age (years) 6 to
 <10: Maximum Serum Creatinine (mg/dL), Male 1, Female 1 Age (years) 10 to <13: Maximum
 Serum Creatinine (mg/dL), Male 1.2, Female 1.2 Age (years) 13 to <16: Maximum Serum
 Creatinine (mg/dL), Male 1.5, Female 1.4 Age (years) >16: Maximum Serum Creatinine (mg/dL),
 Male 1.7, Female 1.4

 These threshold creatine values were derived from the Scwartz formula estimating GFR,
 utilizing child length and statured published by the CDC.

 Exclusion Criteria:

 -  Patients with history of hematologic malignancies or allogenic/autogenic stem cell
 transplant.

 -  Patients with a currently known predisposition to a myeloid stem cell disorder,
 myeloid leukemia, and/or bone marrow failure syndrome including, but not limited to:

 -  Aplastic anemia

 -  Ataxia telangiectasia

 -  Bloom syndrome

 -  Congenital amegakaryocytic thrombocytopenia

 -  Cyclic neutropenia

 -  Diamond Blackfan anemia

 -  Dyskeratosis congenita

 -  Familial AML/MDS syndromes (including ANKRD26, CEBPA, DDX41, ETV6, GATA2, RUNX1,
 SRP72)

 -  Fanconi anemia

 -  Kostmann disease

 -  Li-Fraumeni syndrome

 -  Neurofibromatosis

 -  Nijmegen breakage syndrome

 -  Noonan syndrome

 -  Paroxysmal nocturnal hemoglobinuria

 -  Pearson syndrome

 -  Poland syndrome

 -  Rothmund-Thomson syndrome

 -  Severe congenital neutropenia

 -  Thrombocytopenia absent radii syndrome

 -  Trisomy 8

 -  Trisomy 21

 -  WHIM syndrome

 -  Wiskott Aldrich syndrome

 -  Xeroderma pigmentosa

 -  Secondary malignancy in the past 5 years.

 -  Patients who have previously undergone up-front chemotherapy and have relapsed or
 progressed through therapy.

 -  Patients who have received 4 or more cycles of induction chemotherapy for their
 current malignancy prior to time of enrollment.

 -  Previous use of romiplostim, eltrombopag, recombinant human TPO, or any other TPO
 receptor agonist, or any investigational platelet producing agent.

 -  Patients receiving other investigational agents are not eligible for study entry.

 History of uncontrolled arrhythmias, clinically significant electrocardiogram (ECG)
 abnormalities, active heart failure or pericardial disease.

 -  Patients with current or prior venous thrombotic event or arterial thrombotic event at
 time of enrollment will be ineligible for this study.

 -  Pregnant women/lactating mothers.

 -  Patients unwilling to use effective contraception method, which includes abstinence.

 -  Patients with an inability to return for follow-up visits or obtain follow-up studies
 required to assess toxicity to therapy.