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Osteosarcoma MetastaticPhase 2NCT04183062

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

A Phase 2 study testing Chemotherapy (gemcitabine & docetaxel) plus BIO-11006 in people with Osteosarcoma Metastatic and Ewing's Sarcoma Metastatic, run by Nicklaus Children's Hospital f/k/a Miami Children's Hospital at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 5 to 21 years
What is being tested?
Chemotherapy (gemcitabine & docetaxel) plus BIO-11006
Could I receive a placebo?
No, everyone receives an active treatment
How many people
0 participants
Where
1 siteUnited States
Study dates
4 Oct 2019to 12 Dec 2021
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 5 to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Histologic verification of Ewing's sarcoma or osteosarcoma at original diagnosis or at relapse
  • Recurrence within one year of end of previous therapy
  • Presence of lung metastases
  • Osteosarcoma patients must be ineligible for curative surgery
  • Ability to take inhaled medication by nebulizer and be willing to adhere to the BIO-11006 regimen
  • 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.
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  • Males of reproductive potential must agree to the use of condoms or other methods to ensure effective contraception with partner
  • Stated willingness to comply with all study procedures and availability for the duration of the study
  • Provision of signed and dated informed consent form

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Nicklaus Children's Hospital

    Miami, Florida, 33155

Other trials in United States

About this study

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

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.

Read the 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.

Being tested
Chemotherapy (gemcitabine & docetaxel) plus BIO-11006
Keywords
bone cancer, sarcoma, lung metastases, pediatric cancers
Medical subject headings
Bone Neoplasms, Sarcoma

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Chemotherapy (gemcitabine & docetaxel) plus BIO-11006Drug

    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.

Study groups in NCT04183062
Study groupRoleWhat they receive
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.
Receives: Drug: Chemotherapy (gemcitabine & docetaxel) plus BIO-11006

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety - Treatment-emergent adverse events (TEAEs)

    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.

    Measured: Up to 16 months

Secondary outcomes (1)

  1. Efficacy - Progression Free Survival (PFS)

    PFS will be measured from Cycle 1 Day 1 until occurrence of event (such as death, disease progression or end of study).

    Measured: Up to 16 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Dec 2019
  2. Study start4 Oct 2019
  3. Primary completion4 Oct 2019
  4. Study completion12 Dec 2021

Why it stopped: The drug for this study was not available by the manufacturer. No patients enrolled.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
0 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04183062
Sponsor's protocol ID
2019023
Status last verified
Nov 2024
First submitted
11 Nov 2019
Last updated
8 Nov 2024
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Blackshear PJ. The MARCKS family of cellular protein kinase C substrates. J Biol Chem. 1993 Jan 25;268(3):1501-4. No abstract available.

    PubMed 8420923

  • Brown HK, Schiavone K, Gouin F, Heymann MF, Heymann D. Biology of Bone Sarcomas and New Therapeutic Developments. Calcif Tissue Int. 2018 Feb;102(2):174-195. doi: 10.1007/s00223-017-0372-2. Epub 2017 Dec 13.

    PubMed 29238848

  • Green TD, Park J, Yin Q, Fang S, Crews AL, Jones SL, Adler KB. Directed migration of mouse macrophages in vitro involves myristoylated alanine-rich C-kinase substrate (MARCKS) protein. J Leukoc Biol. 2012 Sep;92(3):633-9. doi: 10.1189/jlb.1211604. Epub 2012 May 23.

    PubMed 22623357

  • Grohar PJ, Janeway KA, Mase LD, Schiffman JD. Advances in the Treatment of Pediatric Bone Sarcomas. Am Soc Clin Oncol Educ Book. 2017;37:725-735. doi: 10.1200/EDBK_175378.

    PubMed 28561686

  • Hartwig JH, Thelen M, Rosen A, Janmey PA, Nairn AC, Aderem A. MARCKS is an actin filament crosslinking protein regulated by protein kinase C and calcium-calmodulin. Nature. 1992 Apr 16;356(6370):618-22. doi: 10.1038/356618a0.

    PubMed 1560845

  • Singer M, Martin LD, Vargaftig BB, Park J, Gruber AD, Li Y, Adler KB. A MARCKS-related peptide blocks mucus hypersecretion in a mouse model of asthma. Nat Med. 2004 Feb;10(2):193-6. doi: 10.1038/nm983. Epub 2004 Jan 11.

    PubMed 14716307

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Chemotherapy (gemcitabine & docetaxel) plus BIO-11006 in people with Osteosarcoma Metastatic and Ewing's Sarcoma Metastatic, run by Nicklaus Children's Hospital f/k/a Miami Children's Hospital at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 9 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: withdrawn, as of the sponsor's last update on 8 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was withdrawn before enrolling participants, so there are no results.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety - Treatment-emergent adverse events (TEAEs) (time frame: Up to 16 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04183062, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2024.

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