BIO-11006 for Osteosarcoma and Ewing's Sarcoma Lung Metastases

This is a Phase 2 study of an investigational drug, BIO-11006, for the treatment of lung metastases in pediatric patients with advanced osteosarcoma or Ewing's sarcoma. This study will enroll up to 10 patients aged between 5 and 21 at Nicklaus Children's Hospital in Miami, FL. Patients will receive BIO-11006 in addition to chemotherapy consisting of gemcitabine and docetaxel. This study will test the hypothesis that BIO-11006 will enhance the effect of the gemcitabine and docetaxel chemotherapy to treat lung metastases in osteosarcoma and Ewing's sarcoma.

This is a Phase 2, controlled study of BIO-11006 peptide given in addition to Gemcitabine and Docetaxel (GemTax) in the treatment of patients with advanced osteosarcoma or Ewing's sarcoma with lung metastases. Patients will receive BIO-11006 in addition to GemTax chemotherapy, which is a current institutional treatment option for recurrent Osteosarcoma and Ewings's Sarcoma. The BIO-11006 inhalation solution will be given by mouth inhalation twice daily. BIO-11006 will be given during the first three cycles and then will be stopped. Patients will continue on GemTax for up to 6 additional cycles and will be clinically assessed before every cycle and have standard-of-care imaging assessments after cycle 3, 6 and 9. If the patient shows lung progression (either clinical or on imaging) at any point after cycle 4 has been given, but had shown at least a partial response during the tumor assessment after cycle 3, BIO-11006 may be re-started at the discretion of the investigator and continued for the duration of the GemTax treatment. Subjects will be followed for disease status during routine clinical follow-ups for 9 months post the end of study treatment. The trial will be conducted at a single site.

Eligibility Criteria:
Inclusion Criteria:

1. Histologic verification of Ewing's sarcoma or osteosarcoma at original diagnosis or at relapse
2. Recurrence within one year of end of previous therapy
3. Presence of lung metastases
4. Osteosarcoma patients must be ineligible for curative surgery
5. Ability to take inhaled medication by nebulizer and be willing to adhere to the BIO-11006 regimen
6. Females of reproductive potential must have a negative pregnancy test at screening and must agree to use a method of highly effective contraception during study participation.
7. Males of reproductive potential must agree to the use of condoms or other methods to ensure effective contraception with partner
8. Stated willingness to comply with all study procedures and availability for the duration of the study
9. Provision of signed and dated informed consent form

Exclusion Criteria:

1. Previously treated and progressed on gemcitabine and docetaxel
2. Central Nervous System (CNS) metastases
3. Contraindication to gemcitabine or docetaxel
4. Known allergic reactions to components of BIO-11006
5. Use of medications that are primarily metabolized by cytochrome P450 isoenzymes
6. Treatment with another investigational drug within 8 weeks of enrollment.
7. Current pneumonia or idiopathic pulmonary fibrosis
8. Absolute Neutrophil Count (ANC) \< 750 cells/mm3
9. Platelet count \< 75,000 cells/mm3
10. Hemoglobin \<8.0 g/dL
11. Creatinine clearance (CrCL) \< 45 mL/min
12. Bilirubin \> upper limit of normal (ULN) for age
13. AST and/or ALT \>1.5 x ULN concomitant with alkaline phosphatase \>2.5 x ULN
14. Organ transplant or bone marrow transplant recipient
15. Pregnancy or lactation
16. Current smoker or tobacco use within 3 months prior to enrollment
17. Any febrile illness or other severe illness within 2 weeks of enrollment
18. Known history of human immunodeficiency virus, hepatitis B, hepatitis C or tuberculosis