Symptoms and General Pathology

All Conditions

Behaviors and Mental Disorders

Disease Progression

Quality of Life

Antineoplastic Agents

All Drugs and Chemicals

Apatinib

Lenvatinib

Angiogenesis Inhibitors

Immunomodulating Agents

Sorafenib

Immunologic Factors

2000 patients with advanced hepatobiliary tumors will be enrolled and the enroll patients should be treat with any type of the following three treatment program:

1. Monotherapy or combination therapy with the targeted drug related to genetic variation of the subject;
2. Treatment with pan-target anti-angiogenic drugs, such as sorafenib, regorafenib, lenvatinib, apatinib, etc;
3. Immunotherapy or immunotherapy combined with targeted therapy or (and) chemotherapy.

Therapeutic regimens are based on the clinicopathological features, genomic alterations, advices from multidisciplinary tumor board and willings from patients.

Precision oncology including targeted therapy and immunotherapy

Xiaobo Yang

Haitao Zhao

The investigators design a large sample size study in the real-world to explore whether targeted therapy and immunotherapy can improve the survival, quality of life and drug safety of patients with advanced hepatobiliary tumors, and analyze the correlation between genetic variation and the effectiveness of therapy.

This trial is a multicenter, non-random, open and observational real-world study. It is estimated that 2000 patients with advanced hepatobiliary tumors will be enrolled in about 20 research centers. And it is planned to complete the enrollment within 2 years and it is expected that all enrolled subjects will reach the observation end point in 5 years.

After primary screening of all the patients, the investigators will further collect next-generation sequence(NGS) data and immunohistochemical data from the subjects, and enroll patients with any type of the following three treatment program: 1. Monotherapy or combination therapy with the targeted drug related to genetic variation of the subject; 2. Treatment with pan-target anti-angiogenic drugs, such as sorafenib, regorafenib, lenvatinib, apatinib, etc; 3. Immunotherapy or immunotherapy combined with targeted therapy.

The investigators will collect subjects' data from assessment centers each month and the efficacy, quality of life, and safety of treatment will be evaluated.

This trial plans to conduct an interim analysis and final analysis after collecting 500 cases, 1000 cases, and 2000 subjects, 3000 subjects,and explore the clinical application value of targeted therapy and immunotherapy in advanced hepatobiliary tumors in real world.

Real-world Study for Patients With Advanced Hepatobiliary Tumors

Real-world Study for Targeted Therapy and Immunotherapy in Patients With Advanced Hepatobiliary Tumors: a Multi-centers, Open-assess Observational Study.

Time from the date of initial treatment to death for any cause.

A duration from the date of initial treatment with apatinib to disease progression (as defined by RECIST) or death.

Percentage of subjects with complete response and partial response.

Percentage of subjects with complete remission, partial remission, or stable disease during treatment or within 30 days of termination of test drug.

Percentage of subjects who develop any adverse events (CTCAE 4.0).

The life quality of every subject will be assessed every 3 months according to the 45-item FACT-Hep questionnaire, which assesses generic HRQL concerns and disease-specific issues.

OrigiMed

Peking Union Medical College Hospital

Neoplasms

Skin and Connective Tissue Diseases

Rare Diseases

Osteosarcoma

Sarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Bone Tissue

Neoplasms, Connective Tissue

Soft Tissue Sarcoma

Amino Acids

Tyrosine

Treatment will be divided in 28 days cycles, including a 21-day period of treatment by regorafenib and best supportive care followed by a 7-day period of rest.

In case of toxicity, dose can be reduced or treatment interrupted according to Specific Product Characteristics (SPC).

Patients can receive up to a maximum of 13 cycles (maximum treatment period : 12 Months).

Treatment will be divided in 28 days cycles, including a 21-day period of treatment by placebo and best supportive care followed by a 7-day period of rest.

In case of toxicity, dose can be reduced or treatment interrupted according to Specific Product Characteristics (SPC).

Patients can receive up to a maximum of 13 cycles (maximum treatment period : 12 Months).

Treatment for 13 cycles (12 months) maximum.

During each cycle :

* patients ≥ 16 years old and patients \<16 old with Body Surface Area (BSA) ≥ 1,70 m2 will take 3 tablets, once a day, corresponding to a total of 120 mg Regorafenib, during 21 days, followed by 7 days without treatment.
* patients \< 16 years old with a 1,30 m2 ≤ BSA ≥ 1,70 m2 will take 2 tablets, once a day, corresponding to a total of 80 mg Regorafenib, during 21 days, followed by 7 days without treatment

Treatment by regorafenib and best supportive care

Treatment for 13 cycles (12 months) maximum.

During each cycle :

* patients ≥ 16 years old and patients \<16 old with Body Surface Area (BSA) ≥ 1,70 m2 will take 3 tablets, once a day, corresponding to a total of 120 mg Placebo, during 21 days, followed by 7 days without treatment.
* patients \< 16 years old with a 1,30 m2 ≤ BSA ≥ 1,70 m2 will take 2 tablets, once a day, corresponding to a total of 80 mg Placebo, during 21 days, followed by 7 days without treatment

Treatment by placebo and best supportive care

Julien GAUTIER

Loïc CHAIGNEAU

Maud TOULMONDE

Alice HERVIEUX

Nicolas PENEL

Mehdi BRAHMI

Nelly FIRMIN

Morgane CLEIREC

Sophie PIPERNO-NEUMANN

Pascaline BOUDOU-ROUQUETTE

Emmanuelle BOMPAS

Claire BERGER

Natacha ENTZ WERLE

Justine GANTZER

Thibaud VALENTIN

Nathalie GASPAR

This is a randomized, double-blinded, 2 arms study concerning patients with high-grade bone sarcoma (HGBS) without complete remission after standard treatment at diagnosis or first relapse.

In the first arm, patients will be treated with regorafenib + best supportive care (BSC) for a maximum of 12 months as maintenance therapy after standard line therapy completion, whereas in the second arm, patients will be treated with placebo + BSC (standard of care).

The comparison between this two arms will allow to determine whether or not regorafenib and BSC is efficient for disease control, in terms of Progression-Free Survival improvement.

This is a randomized, placebo-controlled, double-blinded, prospective, comparative, multicentre phase II study.

Patients with measurable unresectable residual disease will be accrued after they completed standard of care, consisting of:

* At diagnosis: multimodal treatment with neoadjuvant chemotherapy, surgery and adjuvant chemotherapy
* At relapse: chemotherapy

Patients who meet the eligibility criteria will be randomly assigned (1:1) into one of the following treatment groups:

* Experimental arm: regorafenib + best supportive care (BSC) maintenance (12 months maximum)
* Standard arm: placebo + BSC (12 months maximum)

A randomization procedure (centralized implementation of concealed random permuted blocks) will be used to obtain a balanced distribution of the setting of the disease (stratification factor): residual disease at diagnosis or at relapse after standard multimodal treatment.

After their eligibility has been confirmed, patients will receive regorafenib or its matching placebo until disease progression, or for a maximum of 12 months, or unacceptable toxicity or willingness to stop, whichever occurs first.

In case of radiological disease progression during treatment period, patients from the standard arm (placebo) who have unresectable disease will be allow to switch to experimental arm (regorafenib). After the switch, patients will be treated with regorafenib until disease progression or unacceptable toxicity or willingness to stop which occurs first.

After the completion of the maintenance therapy (12 months) patients will be followed-up until the first radiological disease progression, unless a premature disease progression occurred. All patients will be followed-up until the data cut-off (12 months after the last randomization).

The vital status will be updated once for all patients at the end of the study, based on patient's medical file.

The end of the study will be 24 months after the last randomisation or at the end of treatment of the last patient under treatment (either blinded or open label) whichever occurs last.

Efficacy of Regorafenib Combined With Best Supportive Care as Maintenance Treatment in High Grade Bone Sarcomas Patients

A Randomized, Placebo-controlled, Double-blinded, Phase II Study of Efficacy of Regorafenib as Maintenance Treatment in Patients With High Grade Bone Sarcomas at Diagnosis or Relapse and Without Complete Remission After Standard Treatment

The Progression-Free Survival (PFS) will be defined as the time from the date of randomization to the date of first documented disease progression (according to the RECIST V1.1) or the date of death due to any cause.

Patients alive and progression-free at the time of data cut-off will be censored at the time of the latest tumour evaluation.

In patients from the placebo arm who switched into the active treatment group, PFS will be studied from regorafenib initiation: PFS from switch will be defined as the time from the date of switch to the date of first documented disease progression (according to the RECIST V1.1) post-switch or the date of death due to any cause.

The Objective Response Rate (ORR) will be defined as the proportion of patients with a best overall response of Complete Response (CR) or Partial Response (PR) (RECIST V1.1)

In patients from the placebo arm who switched into the active treatment group, the ORR will be studied from regorafenib initiation:

The ORR from switch will be defined as the proportion of patients in the placebo arm with a best overall response of Complete Response (CR) or Partial Response (PR) from regorafenib initiation.

The Disease Control Rate (DCR) will be defined as the proportion of patients with a best overall response of CR, PR or Stable Disease (SD) (RECIST V1.1) 2 months after randomization

The Disease Control Rate (DCR) will be defined as the proportion of patients with a best overall response of CR, PR or Stable Disease (SD) (RECIST V1.1) 4 months after randomization

In patients from the placebo arm who switched into the active treatment group, the DCR will be studied from regorafenib initiation:

The DCR from switch will be defined as the proportion of patients in the placebo arm with a best overall response of CR, PR or Stable Disease (SD) 2 months after regorafenib initiation.

In patients from the placebo arm who switched into the active treatment group, the DCR will be studied from regorafenib initiation:

The DCR from switch will be defined as the proportion of patients in the placebo arm with a best overall response of CR, PR or Stable Disease (SD) 4 months after regorafenib initiation.

The Time to Treatment Failure (TTF) will be defined as the time from the date of randomization to the date of permanent discontinuation of the study treatment, whichever is the cause. Patient not known to have withdrawn treatment before 12 months (study treatment duration) will be censored at the time of treatment stop.

The Overall Survival (OS) will be defined as the time from date of randomization to the date of death, from any cause. Patient not known to have died at the time of analysis will be censored based on the last recorded date on which the patient was known to be alive.

In patients from the placebo arm who switched into the active treatment group, the OS will be studied from regorafenib initiation:

The Overall Survival (OS) from switch will be defined as the time from date of switch to the date of death, from any cause. Patient not known to have died at the time of analysis will be censored based on the last recorded date on which the patient was known to be alive.

The patient's Quality of Life (QoL) will be assessed using the EORTC Quality Of Life Questionnaire (QLQ)-C30 file. Scores will be calculated at each time point according to the scoring manuals. Descriptive statistics will be used to evaluate baseline scores and evolution of scores from baseline to each time point. Data will be compared between arms using the Student's t-test. The QoL data will also be presented graphically if deemed relevant.

The safety will be described mainly on the frequency of adverse events (AE) coded using the common toxicity criteria (NCI-CTCAE v5.0) grade. Descriptive statistics will be provided for characterizing and assessing patient tolerance to treatment. AE will be coded according to the MedDRA®.

Centre Leon Berard

Respiratory Tract (Lung and Bronchial) Diseases

Lung Neoplasms

Carcinoma, Non-Small-Cell Lung

Carcinoma

Respiratory Tract Neoplasms

Thoracic Neoplasms

Lung Diseases

Respiratory Tract Diseases

Carcinoma, Bronchogenic

Bronchial Neoplasms

Pemetrexed

Docetaxel

Cisplatin

Endostatins

Gefitinib

Tubulin Modulators

Antimitotic Agents

Growth Inhibitors

Jianhua Chang, MD,PhD

The purpose of this study is to examine the efficacy and safety of endostatin combined with Docetaxel in advanced Non-Small-Cell Lung Carcinoma (NSCLC) patients failure to first-line or second-line chemotherapy.

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Real-world Study for Patients With Advanced Hepatobiliary Tumors	NCT03892577	Objective Respo... Progression-fre... Overall Surviva... Disease Control... Duration of Res... Five-year Survi... Quality of Life	Precision oncol...	18 Years -	Peking Union Medical College Hospital
Efficacy of Regorafenib Combined With Best Supportive Care as Maintenance Treatment in High Grade Bone Sarcomas Patients	NCT04698785	Bone Sarcoma Osteosarcoma	Treatment by re... Treatment by pl...	12 Years -	Centre Leon Berard
Efficacy of Regorafenib Combined With Best Supportive Care as Maintenance Treatment in High Grade Bone Sarcomas Patients	NCT04698785	Bone Sarcoma Osteosarcoma	Treatment by re... Treatment by pl...	12 Years -	Centre Leon Berard
Study of Endostatin Combined With Docetaxel in the Treatment of Advanced Non-small Cell Lung Cancer Patients	NCT01192230	Carcinoma, Non-...	Endostatins Docetaxel	18 Years - 80 Years	Fudan University

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