Clinical Trials /

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

NCT04183062

Description:

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.

Related Conditions:
  • Ewing Sarcoma
  • Osteosarcoma
Recruiting Status:

Recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: BIO-11006 for Osteosarcoma and Ewing's Sarcoma Lung Metastases
  • Official Title: A Phase 2 Study of the Safety and Efficacy of BIO-11006 in the Treatment of Recurrent Osteosarcoma and Ewing's Sarcoma in Patients With Lung Metastases

Clinical Trial IDs

  • ORG STUDY ID: 2019023
  • NCT ID: NCT04183062

Conditions

  • Osteosarcoma Metastatic
  • Ewing's Sarcoma Metastatic

Interventions

DrugSynonymsArms
Chemotherapy (gemcitabine & docetaxel) plus BIO-11006Chemotherapy plus BIO-11006

Purpose

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.

Detailed Description

      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.
    

Trial Arms

NameTypeDescriptionInterventions
Chemotherapy plus BIO-11006ExperimentalPatients will receive BIO-11006 in addition to GemTax chemotherapy. The BIO-11006 inhalation solution will be given by mouth inhalation twice daily. BIO-11006 will be given during the first three cycles of GemTax and then will be stopped. Subjects will continue with GemTax treatment for three additional cycles. 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.
  • Chemotherapy (gemcitabine & docetaxel) plus BIO-11006

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
      
Maximum Eligible Age:21 Years
Minimum Eligible Age:5 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Safety - Treatment-emergent adverse events (TEAEs)
Time Frame:Up to 16 months
Safety Issue:
Description:Frequency, type, severity, and duration of TEAEs. Sources of TEAEs may include spontaneous patient reports, results of physical examinations, and clinical laboratory measurements. Simple descriptive statistics of frequencies, centrality and variability will be utilized to tabulate and summarize TEAEs. If sufficient number of TEAEs will be captured, frequencies, duration and severity will be compared.

Secondary Outcome Measures

Measure:Efficacy - Progression Free Survival (PFS)
Time Frame:Up to 16 months
Safety Issue:
Description:PFS will be measured from Cycle 1 Day 1 until occurrence of event (such as death, disease progression or end of study).

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Trial Keywords

  • bone cancer
  • sarcoma
  • lung metastases
  • pediatric cancers

Last Updated

November 27, 2019