araag2/TREC_Precision-Medicine huggingface dataset:
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Clinical and Histopathologic Characteristics of BAP1 Mutations

Summary: The goal of this protocol is to determine the prevalence of somatic and germline mutations in BAP1 (BRCA associated protein-1) among patients with mesothelioma , choroidal nevus, primary uveal melanoma (UM), or metastatic UM seen at our institution.


Eligibility: Inclusion Criteria:
  All consents:
  -  > or = to 18 years of age
  -  Ability to provide informed consent
  Consent 1:
  Mesothelioma
  -  Histologically proven diagnosis of Mesothelioma OR Choroidal nevus
  -  Diagnosis of choroidal nevus by direct examination and/or ultrasound/optical coherence tomography and possibly fluorescein angiography OR Primary uveal melanoma
  -  Diagnosis of uveal melanoma by direct examination and/or ultrasound/optical coherence tomography and possibly fluorescein angiography
  Consent 2:
  Mesothelioma
  -  Histologically proven diagnosis of Mesothelioma AND
  -  BAP1 mutation or loss of expression identified in tumor sample
  OR one of the following:
  -  Age<50 at diagnosis
  -  No history of asbestos exposure
  -  Personal history of choroidal nevus, uveal melanoma, melanoma, renal cell carcinoma, or cholangiocarcinoma
  -  Family history of choroidal nevus, uveal melanoma, mesothelioma, renal cell carcinoma, or cholangiocarcinoma
  -  History of malignancy in more than two first-degree relatives OR Choroidal nevus
  -  Diagnosis of choroidal nevus by direct examination and/or ultrasound/optical coherence tomography and possibly fluorescein angiography AND one of the following:
  -  More than one clinical risk factor, which may include: orange pigment, thickness > 1 < 2.5mm
  -  Personal history of uveal melanoma, skin melanoma, mesothelioma renal cell carcinoma, or cholangiocarcinoma
  -  Family history of choroidal nevus, uveal melanoma, mesothelioma renal cell carcinoma, or cholangiocarcinoma OR Primary uveal melanoma
  -  Diagnosis of uveal melanoma by direct examination and/or ultrasound/optical coherence tomography and possibly fluorescein angiography
  AND one of the following:
  -  Personal history of uveal melanoma, skin melanoma, mesothelioma, renal cell carcinoma, or cholangiocarcinoma
  -  Family history of choroidal nevus, uveal melanoma, mesothelioma, renal cell carcinoma, or cholangiocarcinoma
  -  History of malignancy in more than two first-degree relatives OR Metastatic uveal melanoma
  -  Histologically proven diagnosis of metastatic uveal melanoma AND
  -  BAP1 mutation or loss of expression identified in tumor sample
  OR one of the following:
  -  Personal history of uveal melanoma, skin melanoma, mesothelioma renal cell carcinoma, or cholangiocarcinoma
  -  Family history of choroidal nevus, uveal melanoma, mesothelioma renal cell carcinoma, or cholangiocarcinoma
  -  History of malignancy in more than two first-degree relatives
  Consent 3:
  -  Relative of patient with germline BAP1 mutation identified through identified testing
  Exclusion Criteria:
  -  none

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Efficacy and Safety of the Mammalian Target of Rapamycin (mTor Rapamycin) Inhibitor in Vascular Malformations

Summary: The phosphatidylinositol 3-kinase (PI3Kinase)/Protein Kinase B (AKT)/mammalian target of rapamycin (mTor) pathway plays a role on the development and the lymphatic-vascular organisations.
  The investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.

Detailed description: The complex vascular malformations induce chronical pains and organic dysfunctions causing significant morbidity and mortality. Therefore, the investigators need to establish guidelines in order to treat these pathologies. Standard treatments such as surgery or interventional radiology are of limited efficacy and related to a high level of recurrences as well as complications. Recent preclinical studies have shown the important role of the PI3Kinase/AKT/mTor pathway on the development and the lymphatic-vascular organisations suggesting an appealing therapeutic target to treat patients with complex vascular malformations.
  The aim of this clinical study is to prospectively evaluate the efficacy and the safety of the Rapamycin, an mTOR inhibitor, to treat children and adults with microcystic lymphatic malformations, general lymphatics abnormalities (GLA) or complex vascular malformations for which conventional therapies as surgery or sclerotherapy are ineffective or associated with high risk of important complications.

Eligibility: Inclusion Criteria:
  -  Patients with complex vascular abnormalities to be threat by a systemic therapy
  -  Patients must have adequate liver function (LDL-cholesterol, triglycerides,…)
  -  Patients must have adequate organ function: neutrophils >1500/mm³, Hb > 8,0 g et platelets> 50.000/mm³ (no platelets limits for the Kasabach Merritt syndrome)
  -  Patients must have adequate renal function(normal creatinin depending on the age), clearance > 70 ml/min/1.73m² and Urin Protein Creatinine ratio <0.3 g.
  -  Karnofsky or Landry > 50
  Exclusion Criteria:
  -  Dental equipments or prosthesis interfering onto a radiological examen
  -  Other uncontrolled medical condition (uncontrolled diabetes, hypertension…)
  -  Concomitant drugs such as inhibitors/inducers of cytochrome P450 3A4 (CYP3A4)
  -  Immunocompromised patients, including known seropositivity for HIV
  -  Digestive problems modifying the absorption of Rapamycin (gastric tube feeding accepted)
  -  Pregnant or nursing (lactating) women
  -  Prior treatment with phosphatidylinositol 3-kinase (PI3K) and/or mTOR inhibitors

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: BKM120 for Patients With PI3K-activated Tumors

Summary: The purpose of this signal seeking study was is to determine whether treatment with BKM120 demonstrates sufficient efficacy in select pathway-activated solid tumors and/or hematologic malignancies to warrant further study.


Eligibility: Inclusion Criteria:
  -  Patient had a confirmed diagnosis of a solid tumor or hematological malignancy with the exception of endometrial cancer, glioblastoma, nonsmall cell lung cancer, prostate cancer or breast cancer.
  -  Patient's tumor was evaluated and pre-identified to have activation of the PI3K pathway, at a CLIA certified laboratory
  -  Patient must have received at least one prior treatment for recurrent metastatic and /or locally advanced disease and for whom no standard therapy options was anticipated to result in a durable remission.
  -  Patient must have had progressive and measurable disease as per RECIST 1.1. or other appropriate hematological guidelines
  -  Patient had an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 1
  Exclusion Criteria:
  Patient had received previous treatment with BKM120 Patient had symptomatic CNS metastases Patient had mood disorder as outlined in Section 5 Patient had received chemotherapy or other anticancer therapy ≤ 4 weeks (6 weeks for nitrosourea, antibodies or mitomycin-C) prior to starting study drug.

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Role of Bile Duct Resection in Major Hepatectomy Due to Intrahepatic Cholangiocarcinoma

Summary: To investigate the prognosis due to presence or absence of bile duct resection in intrahepatic cholangiocarcinoma that require hepatic lobectomy.

Detailed description: The bile duct resection in hepatic lobectomy due to cholangiocarcinoma, mass forming type, is still controversial. We retrospectively evaluated the effect of bile duct resection in this surgery.

Eligibility: Inclusion Criteria:
  -  Cases of major hepatectomy due to intrahepatic cholangiocarcinoma
  Exclusion Criteria:
  -

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Study of INCB040093 in Subjects With Previously Treated B-Cell Malignancies

Summary: The study will be conducted in three parts. Part 1 is a dose escalation phase to determine the maximum tolerated dose (MTD) of INCB040093, a PI3Kδ inhibitor, or a tolerated, pharmacologically active dose; Part 2 will evaluate the combination of INCB040093 and itacitinib (INCB039110), a JAK1 inhibitor, to determine the MTD of the combination or a tolerated dose that produces substantial pharmacologic inhibition of both targets; Part 3 will further evaluate the chosen doses of INCB040093 alone and in combination with itacitinib (INCB039110) in subjects with relapsed/refractory B-cell malignancies.


Eligibility: Inclusion Criteria:
  • Aged 18 years or older, with lymphoid malignancies of B-cell origin as follows:
  *Indolent / aggressive B-cell (NHL) Non- Hodgkin's Lymphoma:
  EXCLUDING: Burkitt lymphoma and precursor B-lymphoblastic leukemia/lymphoma
  INCLUDING: any non-Hodgkin's B-cell malignancy such as CLL and rare non-Hodgkin's B-cell subtypes such as Hairy Cell Leukemia, Waldenstrom macroglobulinemia, Mantle cell lymphoma, transformed NHL histologies, etc.
  *Hodgkin's lymphoma
  -  Life expectancy of 12 weeks or longer.
  -  Subject must have received ≥ 1 prior treatment regimen.
  -  The subject must not be a candidate for potentially curative therapy, including stem cell transplant.
  Exclusion Criteria:
  -  Received an investigational study drug within 28 days or 5 half-lives (whichever is longer) prior to receiving the first dose of study drug.
  -  Received any approved anticancer medications within 21 days or 5 half-lives (whichever is longer) prior to receiving their first dose of study drug (42 days for nitrosoureas) EXCEPT steroids at ≤ 10 mg prednisone daily (or equivalent).
  -  Has any unresolved toxicity ≥ Grade 2 from previous anticancer therapy.
  -  Has history of brain metastases or spinal cord compression, or lymphoma involving the central nervous system.
  -  Has an Eastern Cooperative Oncology Group (ECOG) performance status of ≥ 3.
  -  Received allogeneic hematopoietic stem cell transplant within the last 6 months, or has active graft versus host disease (GVHD) following allogeneic transplant, or is currently receiving immunosuppressive therapy following allogeneic transplant.
  -  Received autologous hematopoietic stem cell transplant within the last 3 months.
  -  Laboratory parameters not within the protocol-defined range.
  -  Current or recent history (<30 days prior to screening and/or <45 days prior to dosing) of a clinically meaningful bacterial, fungal, parasitic or mycobacterial infection.
  -  Current clinically active viral infection.
  -  Known history of infection with the human immunodeficiency virus (HIV).
  -  History of active hepatitis or positive serology for hepatitis.

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Study of Letrozole With or Without BYL719 or Buparlisib, for the Neoadjuvant Treatment of Postmenopausal Women

Summary: The purpose of the study was to determine whether treatment with a PI3K inhibitor plus letrozole led to an increase in pathologic clinical response and Objective Response Rate compared to treatment with placebo plus letrozole in patients with Breast cancer.


Eligibility: Inclusion Criteria:
  1. Patient is an adult, female ≥ 18 years old at the time of informed consent
  2. Patient has a histologically and/or cytologically confirmed diagnosis of breast cancer
  3. Patient is postmenopausal.
  4. Patient has T1c-T3, any N, M0, operable breast cancer
  5. Patients must have measurable disease
  6. Patient has diagnostic biopsy available for the analysis of PIK3CA mutation and Ki67 level.
  7. Patient has estrogen-receptor and/or progesterone positive breast cancer as per local laboratory testing
  8. Patient has HER2 negative breast cancer defined as a negative in situ hybridization test or an IHC status of 0 or 1+ as per local laboratory testing
  Exclusion Criteria:
  1. Patient has locally recurrent or metastatic disease
  2. Patient has received any systemic therapy (e.g. chemotherapy, targeted therapy, immunotherapy) or radiotherapy for current breast cancer disease before randomization.
  3. Patient with type 1 diabetes mellitus or not adequately controlled type 2 diabetes mellitus
  4. History of acute pancreatitis within 1 year of study entry
  5. Uncontrolled hypertension

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Phase 1b Trial of BGJ398/BYL719 in Solid Tumors

Summary: To study the safety and efficacy of the combination of BGJ398 with BYL719 in patients whose tumors express mutations to PIK3CA with or without alterations to FGFR 1-3.

Detailed description: This dose escalation/dose expansion study will evaluate the combination of orally administered BGJ398 in combination with orally administered BYL719. During the dose escalation part, the MTD of the combination will be determined in patients whose advanced or metastatic tumors express mutations to PIK3CA. Once the MTD has been determined, the expansion part will begin. Patients will be addd to one of three arms based on the disease type and genetic changes. Patients with metastatic colorectal cancer are not eligible for participation in the expansion part.

Eligibility: Inclusion Criteria:
  -  Histologically/cytologically confirmed advanced or metastatic solid tumors who have failed standard therapy or for whom no effective standard anti-cancer therapy exists
  -  Documented PIK3CA mutations in all patients in dose escalation and expansion with or without documented genetic alterations in FGFR depending upon dose expansion cohort (either local or central determination)
  -  Measurable disease defined by RECIST v1.1
  -  ECOG performance status of ≤2
  Exclusion Criteria:
  -  Prior PI3Ki or selective FGFR inhibitor treatment (for patients enrolled to expansion part)
  -  Colorectal cancer (for patients enrolled to expansion part)
  -  Patients with diabetes mellitus requiring insulin treatment and/or with clinical signs or with fasting glucose ≥ 140 mg/dL / 7.8 mmol/L, history of clinically significant gestational diabetes mellitus or documented steroid-induced diabetes mellitus
  -  Use of medications that increase serum levels of phosphorus and/or calcium
  -  Inorganic phosphorus outside of normal limits
  -  Total and ionized serum calcium outside of normal limits

Relevance to patient case: possibly relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: S-1 in Combination With Abraxane in Treating Cholangiocarcinoma

Summary: This phase II study was designed to evaluate the efficacy and safety of S-1 plus Abraxane as second-line treatment for cholangiocarcinoma.

Detailed description: There are no standard second-line treatment for cholangiocarcinoma, new therapies are needed. The efficacy of S-1 as first-line treatment for cholangiocarcinoma was confirmed in several studies. The IMPACT study showed that Abraxane plus gemcitabine provide better survival than gemcitabine alone. Since cholangiocarcinoma displayed similar sensitivity to chemotherapy which was used in pancreatic carcinoma, we assume that S-1 plus Abraxane is effective and safe in treating cholangiocarcinoma.

Eligibility: Inclusion Criteria:
  -  Histologic or cytologic diagnosis of cholangiocarcinoma
  -  Not eligible for curative surgery
  -  Progressed after first-line gemcitabine-based chemotherapy
  -  Eastern Cooperative Oncology Group (ECOG) performance status of 2 or better
  -  No serious or uncontrolled concomitant medical illness
  -  Adequate bone marrow function (absolute neutrophil count ≧1,500 ul, platelet count ≧90,000/ul), hemoglobin: > or equal to 8 mg/dL, total bilirubin: < or equal to 1.5 X institutional upper limit of normal, calculated creatinine clearance greater than or equal to 60 mL/min (calculated by the Cockcroft and Gault method).
  Exclusion Criteria:
  -  Uncontrolled infection or severe active comorbid disease
  -  Previous malignancy in the past five years, excluding nonmelanoma skin cancers and in situ cervical, bladder or uterine cancer
  -  Pregnancy or lactating women

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Efficacy and Safety Study of PDT Using Photofrin in Unresectable Advanced Perihilar Cholangiocarcinoma (OPUS)

Summary: Photodynamic therapy (PDT) is a combination of a drug, porfimer sodium (Photofrin), which is activated by a light from a laser that emits no heat. This technique works to allow the medical doctor to specifically target and destroy abnormal or cancer cells while limiting damage to surrounding healthy tissue. The activation of the drug is done by lighting the abnormal areas using a fiber optic device (very fine fiber like a fishing line that permits light transmission) inserted into a flexible tube with a light called cholangioscope for the bile duct. The light will activate the porfimer sodium concentrated in the abnormal tissue, leading to its destruction.
  This research study will evaluate the efficacy and safety of PDT with porfimer sodium administered with Standard Medical Care (SMC) compared to SMC alone on the overall survival time of patients with non-operable advanced cholangiocarcinoma, a rare cancer of the bile ducts. It will involve 200 patients across North America and Europe. Other countries may participate if needed. Participation will last at least 18 months.


Eligibility: Inclusion Criteria:
  -  Males or females aged 18 or older
  -  Diagnosed with radiologically and biopsy or cytology confirmed inoperable perihilar cholangiocarcinoma Bismuth Tumor Stage III/IV
  -  Non-menopausal or non-sterile female subjects of childbearing potential must have a negative serum beta-HCG and use a medically acceptable form of birth control
  -  Able to sign an informed consent
  Exclusion Criteria:
  -  Diagnostic of cholangiocarcinoma made more than 45 days prior to randomization
  -  Cholangiocarcinoma with extra-hepatic metastasis or concurrent non-solid malignancy
  -  Presence or history of other neoplasms (treated during the last five years prior to study entry) other than carcinoma in situ of the cervix or basal carcinoma of the skin
  -  Previously received photodynamic therapy for cholangiocarcinoma
  -  Previously undergone surgical resection of the cholangiocarcinoma
  -  Previously undergone chemotherapy, brachytherapy, or radiotherapy prior to entering the study
  -  Previously undergone metal stent insertion
  -  Porphyria or hypersensitivity to porphyrins (constituents of porfimer sodium), gemcitabine, cisplatin or other platinum-containing compounds
  -  Presence of infection other than the infection of the bile duct (cholangitis)
  -  Acute or chronic medical or psychological illnesses that prevent endoscopy procedures
  -  Abnormal blood test results
  -  Severe impairment of your kidney or liver function
  -  Decompensated cirrhosis
  -  Pregnant or intend to become pregnant, breastfeeding or intend to breast-feed during this study
  -  Participated in another drug study within 90 days before this one
  -  Unable or unwilling to complete the follow-up evaluations required for the study

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: VEGF Signaling Promotes Cell Growth and Metastasis in Extrahepatic Cholangiocarcinoma in a VEGF Receptor Mediated Pathway (ECC)

Summary: The investigators study the VEGF signaling in ECC cell lines,patients and its mechanism in ECC growth, proliferation and apoptosis.

Detailed description: Several studies have documented the elevated expression of VEGF, VEGFR1 and VEGFR2 in cancer cell lines. Another study observed a sequential elevation of VEGF in low-grade dysplasia, high-grade dysplasia and early stage liver cancer. Clinical analyses have discovered the high expression of VEGF is correlated with tumor progression, vascular invasion, distal metastasis and poor prognosis. However, few studies have been conducted to investigate the VEGF signaling in ECC cells and its possible mechanism in regulating ECC growth.Therefore,we try to clarify the mechanism of VEGF signal in ECC growth,proliferation and apoptosis.

Eligibility: Inclusion Criteria:
  1. Extrahepatic cholangiocarcinoma patients diagnosed through biopsy; two or more dynamic imagine with a diagnosis of Extrahepatic cholangiocarcinoma
  2. Child-Pugh A or B
  3. well preserved renal and hematopoietic Function
  4. receive surgical therapy
  5. achieve complete section accessed by contrast-enhanced CT
  Exclusion Criteria:
  1. incomplete section
  2. remote metastasis
  3. Child-Pugh C
  4. combination with other hepatobiliary disease
  5. suffer from other tumors concurrently or in last five years

Relevance to patient case: not relevant
Patient case: 62-year-old male with cholangiocarcinoma; genetic feature: PIK3CA (1047H)

Clinical trial: Study of Sildenafil as a Therapy for Fatigue in Pancreatic Cancer

Summary: Investigation of the effects of daily sildenafil on patients with pancreatic or cholangiocarcinoma cancer undergoing treatment.

Detailed description: This is a double-blinded, placebo-controlled trial in men and women, 40-75 years old, diagnosed with pancreatic or cholangiocarcinoma cancer. Patients will receive placebo or sildenafil daily for 4 weeks during their initial chemotherapy cycle.

Eligibility: Inclusion Criteria:
  1. Men and women, aged 40-75 years.
  2. Diagnosed with Stage III or IV pancreatic or cholangiocarcinoma receiving the following therapy:
  -  Neoadjuvant chemotherapy
  -  Surgical resection followed by chemotherapy.
  3. Able to comprehend risks and sign a consent form.
  4. Performance Status of 0-2.
  Exclusion Criteria:
  1. Significant renal or heart disease or any acute metabolic disease.
  2. Evidence of hepatitis as indicated by a 3-fold increase in of 2 out of 3 liver enzymes.
  3. Diabetes mellitus or other untreated endocrine disease.
  4. Recent (within 3 months) treatment with anabolic steroids.
  5. Ongoing anticoagulant therapy.
  6. Androgen secreting tumors of the ovary and adrenal or any ovarian tumor (e.g., Sertoli-Leydig).
  7. Polycystic ovary syndrome (PCOS) and/or hyperthecosis.
  8. Non-classical adrenal hyperplasia.
  9. Cushing's syndrome.
  10. Glucocorticoid resistance.
  11. Pregnancy.
  12. Hyperprolactinoma, hypothyroidism.
  13. Use of nitrates.
  14. Use of alpha blockers.
  15. Use of protease inhibitors.
  16. Use of cytochrome p450 inhibitors.
  17. Systolic blood pressure <100 or >150, diastolic blood pressure <60 or >90. This range is smaller than the acceptable range stated in the prescribing information for sildenafil (>90/50 and <170/110).
  18. Peripheral vascular disease.
  19. Use of a phosphodiesterase 5 inhibitor.
  20. Any other circumstance deemed exclusionary by the PI or study physician.

Relevance to patient case: not relevant