Clinical trial NCT02715089: Precise Treatment in Hepatobiliary Cancers (PTHBC)
Official title: Single Center, Single Arm, Open Study, to Explore and Evaluate the Precise Treatment in Hepatobiliary Cancers(PTHBC)
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Liver Neoplasms
Biliary Tract Neoplasms
Hepatobiliary Neoplasms
Keywords: NGS
Liver Neoplasms
Biliary Tract Neoplasms
Hepatobiliary Neoplasms
Lead sponsor: Peking Union Medical College Hospital
Sponsor class: OTHER
Collaborators: name: 3D Medicines
First posted: 2016-03-22
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The subjects : Hepatobiliary Cancer patients.

Brief summary: The purpose of this study is to explore the precise treatment in hepatobiliary cancer patients and evaluate drug safety, progression free and overall survival. This trial study is based on genetic tests, then therapeutic target drugs are administered according to the genetic test reports. Patients with genetic abnormalities (such as mutations, amplifications, or translocations) may benefit from precise treatment which targets particular genetic abnormality. The identifications of these genetic abnormalities may help treat hepatobiliary cancer patients better.

Detailed description: The genetic tests are performed for the eligible subjects in this study, then therapeutic target drugs are administered according to the genetic test reports. While the precise treatments, follow-ups are conducted to evaluate the efficacy and safety of the target drugs for the subjects, until the overall survival.

Study Type: Non-Interventional. Masking: Open Label.

Eligibility criteria: Inclusion Criteria:

* Age from 18 to 65, male or female.
* Radiologically and pathologically confirmed as hepatobiliary cancers with stage IV.
* Palliative care as the preferred.
* The result of next-generation sequencing (NGS) test show the patient has gene mutation and also can be treated by the right commercial products that have been approved by the China Food and Drug Administration (CFDA) or the Food and Drug Administration (FDA).
* ECOG performance status 0-2.
* Life expectancy ≥3 months.
* Voluntarily participate in the study and agree to sign informed consent form.

Exclusion Criteria:

* Hepatobiliary cancer patient with stageI-III, or with any of the following items will not be eligible for screening.Such as, suitable for the treatment of radical resection, radical resection but evaluation unmeasurable.
* The result of NGS test show the patient has no gene mutation, or has gene mutation but no medicine.
* ECOG performance status ≥ 3.
* Female patients who are pregnant or not using a contraceptive method of birth control.
* History or presence of serious cardiovascular or cerebrovascular abnormalities.
* Abnormalities of the hepatic or renal functions, such as jaundice, ascites, bilirubin ≥ 1.5×ULN, alkaline phosphatase ≥ 3×ULN, persistent protein urine≥ grade 3 (according to National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTC-AE\] v4.0), creatinine ratio \> 3.5g/24 hours, renal failure.
* Persistent infection \> grade 2 (according to National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTC-AE\] v4.0).
* Patient has underwent a major operation 4 weeks prior to screening or has not yet recovered from the operation.
* Patient with epilepsy, known or untreated brain metastases.
* The presence of wounds, ulcers or fractures that can not be healed, or with a past history of transplantation.
* The presence of bleeding events ≥grade 3 (according to National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTC-AE\] v4.0) , present evidence or past history of coagulation dysfunction disorders.
* Known human immunodeficiency virus (HIV) infection history.
* Patient with drug abuse or unstable compliance.
* The presence of unresolved toxicity caused by any previous treatment/operation \> grade 1 (according to National Cancer Institute \[NCI\] Common Terminology Criteria for Adverse Events \[CTC-AE\] v4.0), except alopecia, anemia or hypothyroidism).
* Investigator consider that the patient should not be enrolled in this study by careful assessment.
* The subjects participate in any other clinical trial in the meantime.

Primary outcomes:
measure: Objective response rates (ORR)
timeFrame: 6 weeks
measure: Progression-free survival (PFS)
timeFrame: 8 months
Secondary outcomes:
measure: Overall survival (OS)
timeFrame: Through study completion, an average of 1 year.
measure: Duration of Response (DOR)
timeFrame: 6 months
measure: Disease control rate (DCR)
timeFrame: 6 weeks
measure: Adverse Drug Reaction (ADR)/Adverse Event (AE)
timeFrame: Through study completion, an average of 2 years.
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
Study officials:
affiliation: Peking Union Medical College Hospital
name: Hai-Tao Zhao, Doctor
role: PRINCIPAL_INVESTIGATOR
References:
citation: Lin J, Shi J, Guo H, Yang X, Jiang Y, Long J, Bai Y, Wang D, Yang X, Wan X, Zhang L, Pan J, Hu K, Guan M, Huo L, Sang X, Wang K, Zhao H. Alterations in DNA Damage Repair Genes in Primary Liver Cancer. Clin Cancer Res. 2019 Aug 1;25(15):4701-4711. doi: 10.1158/1078-0432.CCR-19-0127. Epub 2019 May 8.