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Patient-derived Xenograft (PDX) Modeling to Test Drug Response for High-grade Osteosarcoma

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03358628
Recruitment Status : Not yet recruiting
First Posted : November 30, 2017
Last Update Posted : November 30, 2017
Information provided by (Responsible Party):
GUO WEI, Peking University People's Hospital

Brief Summary:
By obtaining clinical specimens from participants with high-grade bone and soft tissue sarcomas to establish and profile as freshly implanted tumors in mice, the aim of this study is to identify agents with predicted activity in the host patient while also potentially providing them with individualized cancer treatment options

Condition or disease Intervention/treatment
Osteosarcoma Other: Molecular Profiling & In Vivo drug testing in PDX

Detailed Description:
Patient-derived xenografts (PDX) are increasingly used as tools for drug development in pre-clinical settings, and have been shown to recapitulate the histology and behavior of the cancers from which they are derived. Although, they have been commonly used productively as pre-clinical disease models to study disease biology and drug response, they have not been used prospectively to inform clinical management. PDX have been employed to inform clinical decision-making in small studies, which have shown high concordance between individual PDX and patient responses to therapy. While encouraging, the role of this approach in bone and soft tissue sarcomas and in the context of genomic drug matching strategies remains undefined. This has created an opportunity to evaluate the utility of PDX as clinical predictors to direct the use of chemo- and targeted therapies in combination with comprehensive genomic and epigenetic analysis for patients with bone and soft tissue sarcomas.

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Study Type : Observational
Estimated Enrollment : 20 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Patient-derived Xenograft (PDX) Modeling to Test Drug Response for High-grade Osterosarcoma
Estimated Study Start Date : February 1, 2018
Estimated Primary Completion Date : January 31, 2020
Estimated Study Completion Date : January 31, 2025

Resource links provided by the National Library of Medicine

Group/Cohort Intervention/treatment
Osteosarcoma patients with metastatic relapsed or unresectable progressive disease (total n= up to 20) following resection of the primary lesion and adjuvant chemotherapy.
Other: Molecular Profiling & In Vivo drug testing in PDX
Molecular profiling of host tumour sample and PDX will be performed and analyzed by an expert panel. In vitro drug testing using organoid culture generation may also be performed if sufficient fresh tissue is available. Matched treatment recommendation based on profiling and in vivo PDX drug testing results will be made, if available.

Primary Outcome Measures :
  1. Measure of drug sensitive PDX to a panel of drugs as a predictor of clinical response in matched host [ Time Frame: up to 2 years ]
    Sensitivity measured by tumor growth inhibition (>80%) or objective tumor response (regression) as per Response Evaluation Criteria In Solid Tumors (RECIST) criteria.

Biospecimen Retention:   Samples With DNA
Whole Blood, formalin fixed paraffin embedded blocks, or fresh tumor tissue

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with high-grade osteosarcoma referred to, or being treated at Peking University People's Hostpital.

Inclusion Criteria:

  1. Age >18 years;
  2. Diagnosis confirmed histologically and reviewed centrally;
  3. Prior treatment (completed >4 weeks before trial entry) consisted of standard high-grade osteosarcoma chemotherapy agents including doxorubicin, cisplatin, high-dose methotrexate, and ifosfamide; metastatic relapsed and unresectable progressive disease (PD);
  4. Eastern Cooperative Oncology Group performance status 0-1 with a life expectancy >3 months;
  5. Adequate renal, hepatic, and hemopoietic function;
  6. Normal or controlled blood pressure;
  7. Surgery and/or radiotherapy completion at least 1 month before enrollment.

Exclusion Criteria:

  1. Central nervous system metastasis;
  2. Have had other kinds of malignant tumors at the same time;
  3. Cardiac insufficiency or arrhythmia;
  4. Uncontrolled complications, such as diabetes mellitus and so on;
  5. Coagulation disorders;
  6. Urine protein≥ ++;
  7. Pleural or peritoneal effusion that needs to be handled by surgical treatment;
  8. Combined with other infections or wounds.

Information from the National Library of Medicine

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 identifier (NCT number): NCT03358628

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Contact: Tingting Ren, PhD 86-10-88324470
Contact: Yidan Zhang, MD 86-13810330739

Sponsors and Collaborators
Peking University People's Hospital
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Principal Investigator: Wei Guo, MD, PhD Peking University People's Hospital

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Responsible Party: GUO WEI, Chief of Musculoskeltal Tumor Center, Peking University People's Hospital Identifier: NCT03358628     History of Changes
Other Study ID Numbers: 2017PHB278-01
First Posted: November 30, 2017    Key Record Dates
Last Update Posted: November 30, 2017
Last Verified: November 2017

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by GUO WEI, Peking University People's Hospital:
Patient-derived xenograft

Additional relevant MeSH terms:
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Neoplasms, Bone Tissue
Neoplasms, Connective Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type