Study to Find a Safe Dose and Show Early Clinical Activity of Weekly Nab-paclitaxel in Pediatric Patients With Recurrent/ Refractory Solid Tumors

Inclusion Criteria

• Patients must meet all of the following criteria to be enrolled in the study:
• Patient has a confirmed solid tumor diagnosis according to the

following:

• Phase 1: patient has a recurrent or refractory solid tumor that has

progressed or did not respond to standard therapy, or for which no

standard anticancer therapy exists

• Phase 2: patient has radiologically documented measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) 1.1 (for neuroblastoma, evaluable disease by 123^I-metaiodobenzylguanidine [MIBG]/Curie score is also acceptable) in 1 of the following tumor types and has failed up to 3 lines of treatment: Group 1: neuroblastoma, Group 2: rhabdomyosarcoma; Group 3: Ewing's sarcoma.
• The patient has a Lansky/Karnofsky performance status score of ≥ 70%.
• The patient has adequate serum chemistry levels, evidenced by the

following laboratory values

• aspartate aminotransferase (AST)/serum glutamic-oxaloacetric

transaminase (SGOT), alanine aminotransferase (ALT)/serum glutamic

pyruvate transaminase (SGPT) ≤ 2.5 × upper limit of normal range (ULN)

• Total bilirubin ≤ 1.5 × ULN
• Creatinine ≤ 1.5 × ULN
• The patient has adequate bone marrow function, evidenced by the

following:

• Absolute neutrophil count ≥ 1.0 × 10^9 cells/L
• Platelets ≥ 80 × 10^9 cells/L (transfusion independent, defined as not

receiving platelet transfusions within 7 days prior to laboratory sample). In the phase 2 portion, for patients with known bone marrow involvement, platelets ≥ 50 × 10^9 cells/L

• Hemoglobin ≥ 8 g/dL (transfusion is permitted to fulfill this criterion).
• The patient (when applicable) or patient's parent(s) or legal guardian(s)

understand(s) and voluntarily signed an informed consent document prior

to any study-related assessments/procedures being conducted. Where

locally applicable, the patient also understands and voluntarily provides

his/her assent prior to any study-related assessments/procedures being

conducted.

• Male patients of childbearing potential must use a condom during

sexual intercourse and shall not father a child during the study and for 6 months after the last dose of study medication.

• Female patients of childbearing potential [defined as all female

patients ≥ 12 years old or who have reached menarche, whichever occurs

first] must have both of the following:

a. Agree to the use of two physician-approved contraceptive methods

simultaneously or practice complete abstinence while on study medication or for a longer period if required by regulations.

i. True abstinence: When this is in line with the preferred and usual

lifestyle of the patient. Periodic abstinence (e.g., calendar, ovulation,

symptothermal, postovulation methods) and withdrawal are not

acceptable methods of contraception.

ii. Acceptable contraceptive methods include: oral, injectable, or

implantable hormonal contraceptive; tubal ligation; intra-uterine device;

barrier contraceptive with spermicide; or vasectomized partner) including

at least one barrier method.

b. Have negative serum pregnancy test result at screening confirmed by

negative urine pregnancy dipstick within 72 hours prior to first dose of

investigational product (if serum test occurred > 72 hours from first

dose); pregnancy test with sensitivity of at least 25 mIU/mL.

Exclusion Criteria

• The presence of any of the following will exclude a patient from enrollment:
• The patient has a primary brain tumor(s) or brain metastasis (unless metastasis is treated and stable for > 28 days). In patients who are symptomatic, a brain scan is required to exclude metastasis.
• The patient has received therapeutic dose chemotherapy or radiotherapy ≤ 21 days prior to start of investigational product.
• The patient has received maintenance dose chemotherapy (e.g., low dose cyclophosphamide) ≤ 7 days from the first dose of investigational product.
• The patient has received any investigational therapy ≤ 28