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

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ClinicalTrials.gov Identifier: NCT04183062
Recruitment Status : Recruiting
First Posted : December 3, 2019
Last Update Posted : April 14, 2021
Sponsor:
Information provided by (Responsible Party):
Nicklaus Children's Hospital f/k/a Miami Children's Hospital

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Osteosarcoma Metastatic Ewing's Sarcoma Metastatic Drug: Chemotherapy (gemcitabine & docetaxel) plus BIO-11006 Phase 2

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : October 4, 2019
Estimated Primary Completion Date : November 4, 2022
Estimated Study Completion Date : November 4, 2023

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Arms and Interventions
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Arm Intervention/treatment
Experimental: Chemotherapy plus BIO-11006
Patients 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.
 Drug: Chemotherapy (gemcitabine & docetaxel) plus BIO-11006

BIO-11006 will be administered at a dose of 75 mg twice daily for patients over 5 years of age and under 18 years of age or 125 mg twice daily for patients 18 years of age or older. The study drug will be administered twice daily by nebulizer over 20 minutes.

GemTax will be administered in 21-day cycles. Gemcitabine will be administered at a dose of 900 mg/m2 IV over 90 minutes on days 1 and 8. Docetaxel will be administered at a dose of 75 mg/m2 IV over 60 minutes on day 8.


Outcome Measures
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Primary Outcome Measures :
  1. Safety - Treatment-emergent adverse events (TEAEs) [ Time Frame: Up to 16 months ]
    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 :
  1. Efficacy - Progression Free Survival (PFS) [ Time Frame: Up to 16 months ]
    PFS will be measured from Cycle 1 Day 1 until occurrence of event (such as death, disease progression or end of study).


Eligibility Criteria
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Ages Eligible for Study:   5 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04183062


Contacts
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Contact: Guillermo De Angulo, MD 786-624-3513 guillermo.deangulo@nicklaushealth.org
Contact: Guido Elias, CRC 786-624-3513 guido.elias@nicklaushealth.org

Locations
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United States, Florida
Nicklaus Children's Hospital Recruiting
Miami, Florida, United States, 33155
Contact: Guillermo De Angulo, MD    786-624-3513    guillermo.deangulo@nicklaushealth.org   
Contact: Guido Elias, CRC    786-624-3513    guido.elias@nicklaushealth.org   
Principal Investigator: Guillermo De Angulo, MD         
Sponsors and Collaborators
Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Investigators
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Principal Investigator: Guillermo De Angulo, MD Nicklaus Children's Hospital
More Information
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Publications:

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Responsible Party: Nicklaus Children's Hospital f/k/a Miami Children's Hospital
ClinicalTrials.gov Identifier: NCT04183062    
Other Study ID Numbers: 2019023
First Posted: December 3, 2019    Key Record Dates
Last Update Posted: April 14, 2021
Last Verified: April 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Nicklaus Children's Hospital f/k/a Miami Children's Hospital:
bone cancer
sarcoma
lung metastases
pediatric cancers
Additional relevant MeSH terms:
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Neoplasm Metastasis
Sarcoma
Osteosarcoma
Sarcoma, Ewing
Neoplastic Processes
Neoplasms
Pathologic Processes
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Gemcitabine
Docetaxel
 Antimetabolites, Antineoplastic
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Enzyme Inhibitors
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Tubulin Modulators
Antimitotic Agents
Mitosis Modulators