araag2/TREC_Precision-Medicine huggingface dataset:
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Safety and Efficacy Study of Intramyocardial Stem Cell Therapy in Patients With Dilated Cardiomyopathy

Summary: BACKGROUND. In patients with non-ischemic dilated cardiomyopathy, intracoronary stem cell transplantation has been shown to improve exercise capacity, reduce ventricular remodelling and improve 1-year survival. Pre-clinical data demonstrate that stem cell effects on the diseased heart can be further enhanced by direct intramyocardial delivery route.
  AIMS.
  1. To evaluate safety and efficacy of intramyocardial stem cell therapy in patients with non-ischemic dilated cardiomyopathy.
  2. To directly compare clinical effects of intracoronary and intramyocardial stem cell delivery.
  METHODS. Of 60 patients with dilated cardiomyopathy, 30 will be randomized to intramyocardial transplantation of CD34+ cells (Study Group), and 30 will receive intracoronary stem cell therapy (Control Group). In both groups peripheral blood stem cells will be mobilised by daily subcutaneous injections of filgrastim; CD34+ cells will be collected via apheresis and labelled with technetium. In the Study Group electromechanical mapping will be used to identify viable myocardium and intramyocardial injections in the target areas will be performed with NOGA catheter. In the Control group patients will undergo myocardial perfusion scintigraphy and CD34+ cells will be injected intracoronary in the artery supplying segments of reduced viability. Patients will be followed for 1 year. Primary endpoints will include changes in left ventricular ejection fraction and left ventricular dimensions (measured by echocardiography). Secondary endpoints will include changes in exercise capacity and changes in NT-proBNP values.
  HYPOTHESES.
  1. At 1 year, intramyocardial stem cell therapy will be associated with improved left ventricular ejection fraction, reduced left ventricular dimensions, improved exercise capacity and reduced levels of NT-proBNP.
  2. Beneficial effects of intramyocardial stem cell therapy will be superior to those observed with intracoronary stem cell delivery.


Eligibility: Inclusion Criteria:
  -  Established dg. of dilated CMP (defined according to ESC position statement - absence of any stenotic lesions on coronary angiography, no congenital heart disease, no primary valve disease on echocardiography, and no history of hypertension or alcohol abuse1)
  -  left ventricular ejection fraction < 30%
  -  NYHA functional class III or IV for at least 3 months before referral
  -  Optimal medical management for at least 6 months
  Exclusion Criteria:
  -  Left ventricular aneurysm or thrombus
  -  Hematologic disease
  -  Multiorgan failure
  -  Active malignancy

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: A Phase I Study of Oral LGX818 in Adult Patients With Advanced or Metastatic BRAF Mutant Melanoma

Summary: CLGX818X2101 is a first-time in-human, phase I study to establish the maximum tolerated dose (MTD) and/or recommended phase 2 dose (RP2D) of daily administered LGX818 (daily, twice daily and/or every-other-day), a RAF kinase inhibitor. Patients with locally advanced or metastatic melanoma harboring the BRAF V600 mutation (during dose escalation phase and expansion phase) and patients with metastatic colorectal cancer harboring the BRAF V600 mutation (during the expansion phase) will be enrolled. The study consists of a dose escalation part were cohorts of patients will receive escalating oral doses of LGX818, followed by a safety dose expansion part were patients will be treated with oral dose of LGX818 given at the MTD or RP2D.


Eligibility: Inclusion Criteria:
  For the dose escalation phase:
  1. Histologically confirmed diagnosis of locally advanced or metastatic melanoma (stage IIIB to IV per American Joint Committee on Cancer [AJCC]). For the dose expansion phase: (i) Histologically confirmed diagnosis of locally advanced or metastatic melanoma (stage IIIB to IV per American Joint Committee on Cancer [AJCC]), or (ii) confirmed diagnosis and non-resectable advanced metastatic colorectal cancer (mCRC) for which no further effective standard therapy exists.
  2. Written documentation of BRAF V600E mutation, or any other BRAF V600 mutation.
  3. Evidence of measurable disease
  Exclusion Criteria:
  1. Previous therapy with a MEK inhibitor.
  2. Symptomatic or untreated leptomeningeal disease.
  3. Symptomatic or untreated brain metastasis.Patients previously treated for these conditions that are asymptomatic in the absence of corticosteroid therapy are allowed to enroll. Brain metastasis must be stable with verification by imaging.
  4. Known acute or chronic pancreatitis.
  5. Clinically significant cardiac disease
  6. Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of oral LGX818
  7. Previous or concurrent malignancy. Exceptions to this exclusion criteria include: adequately treated basal cell or squamous cell skin cancer; in situ carcinoma of the cervix, treated curatively and without evidence of recurrence for at least 3 years prior to study entry; or other solid tumor treated curatively, and without evidence of recurrence for at least 3 years prior to study entry.
  8. Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test (> 5 mIU/mL).
  9. History of thromboembolic or cerebrovascular events within the last 6 months
  Other protocol-defined inclusion/exclusion criteria may apply

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Value of Cardiac Magnetic Resonance (CMR) Derived Parameters for Diagnosing Left Ventricular Non-compaction Cardiomyopathy

Summary: Left ventricular non-compaction (LVNC) is a rare cardiomyopathy characterized by numerous excessively prominent left ventricular (LV) trabeculation and deep intertrabecular recesses communicating with the ventricular cavity and severely altering myocardial structure. Although most authors assume a developmental arrest in embryogenesis as the underlying pathology, the mechanisms of LVNC are not fully understood yet. Several gene mutations have been identified to be linked with LVNC and an autosomal dominant inheritance pattern is frequent To date the most commonly used imaging tool for diagnosing LVNC is echocardiography applying the criteria established by Jenni and coauthors However, qualitative parameters to differentiate normal compaction of the myocardium in healthy subjects from LVNC or from other cardiomyopathies like dilative cardiomyopathy (DCM) or hypertrophic cardiomyopathy (HCM) may fail due to highly variable LV trabeculation. Therefore, absolute quantification should be performed. Cardiac magnetic resonance (CMR) has been reported as a promising imaging modality to characterize patients with LVNC as it provides both a high spatial resolution and a good contrast between trabeculation and blood pool Jacquier et al. recently described a value of trabeculated LV myocardial mass above 20% of the global mass of the LV to be highly sensitive and specific for LVNC However, in their approach, a substantial degree of the LV cavity was included into calculated trabecular LV mass and led to systemic overestimation of the latter. Furthermore, the role and prognostic value of myocardial scarring as assessed by delayed enhancement (DE) CMR was not evaluated.
  The aim of the retrospective study was to establish revised and extended CMR criteria to distinguish LVNC from DCM, HCM and a group of healthy controls and to improve the assessment of trabeculated mass by excluding intertrabecular blood pool.


Eligibility: Inclusion Criteria:
  -  left ventricular non-compaction cardiomyopathy
  -  dilatative cardiomyopathy
  -  hypertrophic cardiomyopathy or healty controls
  Exclusion Criteria:
  -  contraindications for magnetic resonance imaging like pacemakers or other metallic implants

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Electrical Interactions in Heart Disease

Summary: This study will evaluate the relationship of markers of cardiac activation and their determinants. The hypothesis is that these markers will be interrelated.


Eligibility: Inclusion Criteria:
  -  ventricular dysfunction
  Exclusion Criteria:
  -  normal ventricular function

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Study of Mitomycin-C Application in Laryngotracheal Stenosis

Summary: This is a randomized, prospective, double-blind, placebo-controlled clinical trial of the use of mitomycin-C topical application as an adjunctive treatment in the endoscopic surgical treatment of patients with laryngotracheal stenosis. We hypothesize that the use of mitomycin-C improves patient outcome in the endoscopic surgical treatment of laryngotracheal stenosis.

Detailed description: Obstruction of the upper airway caused by laryngotracheal stenosis (LTS) often results in severe morbidity and even mortality. Treatment of LTS continues to present a challenge and a wide array of surgical techniques have been employed. Despite multiple endoscopic and/or open reconstructive procedures, patients often experience restenosis as a result of the abnormal wound-healing process that initially instigated the airway obstruction. The high rate of stenosis relapse has therefore motivated researchers to find new methods to modulate and control the wound-healing process of the airway. Although other adjuvant treatments such as steroids and antibiotics have been investigated in LTS, much attention in recent years has turned to the use of topical mitomycin-C (MMC). As a topical application, MMC has been shown to inhibit fibroblast proliferation in wound-healing processes. The use of MMC in the treatment of airway stenosis was first reported in 1998 and is now routinely used in the endoscopic management of LTS. However, despite numerous animal and human studies, the benefit of MMC in LTS patients remains questionable. While previously published retrospective data suggest that the addition of MMC improves outcome, there have been no prospective studies to directly address the efficacy of MMC in endoscopic LTS surgery. This study will be the first randomized, prospective, double-blind, placebo controlled clinical trial designed to investigate the efficacy of MMC as an adjunctive therapy to endoscopic surgical treatment in patients with LTS. In addition, the study will investigate the relationship between patient symptoms and objective pulmonary function measurements. Ultimately, the results of this study may influence the treatment and evaluation of patients with laryngotracheal stenosis.

Eligibility: Inclusion Criteria:
  -  Patients with laryngotracheal stenosis, including subglottic stenosis or tracheal stenosis
  -  Patients with disease amenable to treatment with endoscopic CO2 laser radial incisions and balloon dilation
  -  Age greater than or equal to 18 years
  Exclusion Criteria:
  -  Age less than 18 years
  -  Pregnancy
  -  Patients with glottic and supraglottic stenosis
  -  Patients with disease not amenable to treatment with endoscopic CO2 laser radial incisions and balloon dilation
  -  Patients with cartilaginous subglottic or tracheal stenosis

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Genetic Test in Detecting Minimal Residual Disease in Samples From Younger Patients Registered on the COG-AALL08B1 Trial

Summary: RATIONALE: Testing for minimal residual disease in blood samples from patients with acute lymphoblastic leukemia may help doctors plan better treatment.
  PURPOSE: This research trial studies a genetic test in detecting minimal residual disease in samples from younger patients registered on COG-AALL08B1 trial.

Detailed description: OBJECTIVES:
  -  Assess the feasibility of minimal residual disease (MRD) determination in pediatric B-lineage acute lymphoblastic leukemia (ALL) using deep sequencing of the immunoglobulin heavy chain locus.
  OUTLINE: Archived blood and tumor tissue samples are analyzed for MDR using deep sequencing of immunoglobulin heavy chain locus. MDR quantification results are then compared with the flow cytometry reference methods used in COG studies.

Eligibility: DISEASE CHARACTERISTICS:
  -  Samples from patients registered on the Children Oncology Group (COG)-AALL08B1 protocol and stored in the Hematopathology Laboratory at the University of Washington
  -  Pretreatment and after induction therapy samples
  PATIENT CHARACTERISTICS:
  -  Not specified
  PRIOR CONCURRENT THERAPY:
  -  Not specified

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Vitamin D3 Supplementation for Heart Failure Patients

Summary: The purpose of this study is to determine if treatment with vitamin D3 of 10,000 International Units (IU) daily in vitamin D deficient patients for 6 months will improve B type natriuretic peptide (BNP), a marker of heart function, compared to placebo. The investigators also aim to determine if vitamin D helps cardiopulmonary function as evaluated by cardiopulmonary exercise test (CPX), laboratory values, strength, and quality of life in patients with stable congestive heart failure (Class II or III).


Eligibility: Inclusion Criteria:
  -  NYHA Heart Failure Class II or II, stable
  -  Vitamin D deficiency (32 ng/ml or less)
  -  No recent medication changes for 3 months
  -  Females of childbearing age must use effective contraceptive if they are sexually active
  Exclusion Criteria:
  -  Hypercalcemia
  -  Nephrolithiases
  -  Sarcoidosis
  -  Acute cardiac insufficiency
  -  Pregnancy
  -  Breastfeeding
  -  Any clinically unstable medical condition
  -  Supplements of greater than or equal to 1000 units of vitamin D per day

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Study of LGX818 and Cetuximab or LGX818, BYL719, and Cetuximab in BRAF Mutant Metastatic Colorectal Cancer

Summary: This study will assess the safety and efficacy of LGX818 when combined with cetuximab or combined with cetuximab and BYL719 in patients with BRAF mutant metastatic colorectal cancer


Eligibility: Inclusion Criteria:
  -  Metastatic colorectal cancer
  -  Progression after at least one prior standard of care regimen or be intolerant to irinotecan-based regimens
  -  Life expectancy ≥ 3 months
  -  ECOG performance status ≤ 2
  Exclusion Criteria:
  -  Symptomatic or untreated leptomeningeal disease
  -  Symptomatic brain metastasis
  -  Patients with clinically manifested diabetes
  -  Acute or chronic pancreatitis
  -  Clinically significant cardiac disease
  Other protocol-defined inclusion/exclusion criteria may apply.

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Carvedilol Effect in Preventing Chemotherapy - Induced Cardiotoxicity

Summary: The purpose of this study is to evaluate if carvedilol can prevent the cardiotoxicity after chemotherapy in breast cancer.

Detailed description: Dilated cardiomyopathy secondary to chemotherapy accounts for approximately 1% of all dilated cardiomyopathies.
  Initial studies showed beneficial effect of the use of carvedilol for the prevention of chemotherapy-induced cardiomyopathy. This study has the objective to evaluate the effectiveness of carvedilol for the prevention of chemotherapy-induced cardiomyopathy. Will be selected 200 patients referred for chemotherapy that includes anthracyclines for breast cancer.These patients will be randomized to carvedilol or placebo and will have periodic assessment of cardiac function with echocardiography and biomarkers until complete chemotherapy and 24 months later.

Eligibility: Inclusion Criteria:
  Patients diagnosed with breast cancer, with an indication of chemotherapy that includes anthracycline.
  Exclusion Criteria:
  Failure analysis of ventricular function; History of chemotherapy or radiotherapy; Previous symptoms of heart failure; Presence of cardiomyopathy; Presence of Coronary Artery Disease; Aortic valve disease or moderate to severe mitral regurgitation; Contraindication to the use of β-blocker; Use of inhibitors of angiotensin converting enzyme, angiotensin receptor blockers or β-blockers.
  Patients with HER 2 expression

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Long-Term Study of IGIV, 10% in Alzheimer´s Disease

Summary: The purpose of this long-term study is to provide additional evidence of safety and efficacy of IGIV, 10% treatment in participants with Alzheimer´s Disease who have completed the Phase 3 Baxter precursor study 160701. All participants will receive IGIV, 10% at either 0.2 g/kg or 0.4 g/kg body weight depending on their treatment assignment in Baxter study 160701. Participants and investigators will be blinded to dose unless otherwise notified by the sponsor.


Eligibility: Main Inclusion Criteria:
  -  Completed 18 months of study treatment and assessments in Baxter precursor study 160701
  -  Diagnosis of probable Alzheimer´s Disease (AD)
  -  Able to comply with testing and infusion regimen (including adequate corrected visual acuity and hearing ability)
  -  Has a caregiver (study partner) who is willing and able to participate
  Main Exclusion Criteria:
  -  Significant neurological disease other than AD
  -  Clinically significant cardiac/cardiovascular problems (e.g. uncontrolled blood pressure, atrial fibrillation, heart disease, clotting disorders, strokes, or recent heart attack)
  -  Contraindication to undergoing MRI (e.g. pacemaker [with the exception of an MRI-compatible pacemaker], severe claustrophobia, ferromagnetic implants such as a metal plate)
  -  Specific findings on brain MRI (microhemorrhages, superficial siderosis, vasogenic edema, a macrohemorrhage, major stroke, or multiple lacunae)
  -  Active malignancy or history of malignancy within 5 years prior to screening with the exception of the following: adequately treated basal cell or squamous cell carcinoma of the skin, carcinoma in situ of the cervix, and stable prostate cancer not requiring treatment
  -  Uncontrolled major depression, psychosis, or other major psychiatric disorder(s)
  -  Poorly controlled diabetes
  -  Serious problems with liver or kidneys
  -  Known history of hypersensitivity following infusions of human blood or blood components (e.g. human immunoglobulins or human albumin)
  -  Current or recent treatment with immunomodulatory therapies (with the exception of immunoglobulin and non-systemic and low-dose systemic corticosteroids)
  -  Recent use of investigational drugs or biologics, including those aimed at altering AD progression (with the exception of immunoglobulin)
  -  Active immunization for the treatment of AD at any time
  There are reasons why it might not be appropriate to participate in this trial. Please contact Medical Information at medinfo@baxter.com for details.

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Brain Function and Perfusion in Patients With Heart Failure

Summary: The investigators will evaluate the determinants of cerebral impairment in patients with non-ischemic heart failure compared to controls, and its relation to cognitive function. They hypothesize that patients with heart failure have impaired brain perfusion and hemodynamic factors are associated with cognitive dysfunction.


Eligibility: Inclusion Criteria Control Group:
  -  Healthy subject, as assessed by clinician
  -  Age matched with patient group
  Exclusion Criteria Control Group:
  -  Age < 18 years
  -  Carotid artery or intracranial disease, as assessed by echo-doppler (significant stenosis > 50%)
  -  Unable or unwilling to undergo MRI
  -  The presence of ferromagnetic material in or on the body, for example tattoos with ferrous ink or pacemakers or cardiac defibrillators. Also the presence of pregnancy and claustrophobia.
  -  History of cognitive impairment
  -  Unable to understand procedures
  -  Unable or unwilling to provide informed consent
  Inclusion Criteria DCM-group:
  -  Heart failure due to DCM
  -  15 patients with NYHA class II
  -  15 patients with NYHA class III/IV
  Exclusion Criteria DCM-Group:
  -  Age < 18 years
  -  History of myocardial infarction
  -  History of peripheral artery disease
  -  History of cerebrovascular disease or
  -  Carotid artery or intracranial disease, as assessed by echo-doppler (significant stenosis > 50%)
  -  History of cognitive impairment
  -  Unable to undergo MRI (eg pacemaker)
  -  Unable to understand procedures
  -  Unable or unwilling to provide informed consent

Relevance to patient case: not relevant
Patient case: 32-year-old male with dilated cardiomyopathy; genetic feature: LMNA

Clinical trial: Dexamethasone in Prevention of Respiratory Morbidity in Elective Caesarean Section in Term Fetus

Summary: Introduction: Babies born at term by elective caesarean section (CS) and before onset of labor are more likely to develop respiratory complications than babies born vaginally. In developing countries resources are scarce and it is difficult to provide expensive treatments as neonatal care.
  Aim of the Work: To assess the effect of prophylactic dexamethasone administration before elective cesarean section at term in reducing neonatal respiratory complications.
  Patients and methods: 600 women were included in the study and were planned to have elective caesarean section. 300 received dexamethasone 12 mg twice, 12 hours apart 48 hours before delivery. 300 patients were the control group.
  The outcomes were: incidence of admission to neonatal intensive care unit (NICU), incidence of transient tachypnea of newborn (TTN), the incidence of respiratory distress syndrome (RDS) and the need for mechanical ventilation.

Detailed description: Dexamethasone which is a long acting corticosteroid can be used prior to elective cesarean section to decrease neonatal respiratory morbidity by enhacing fetal lung maturity.

Eligibility: Inclusion Criteria:
  -  Previous caesarean section
  -  Complete 37 week calculated from the first day of the last menstrual period.
  Exclusion Criteria:
  -  Obstetric complications such as pre-eclampsia, antepartum hemorrhage or known fetal anomaly.
  -  Hypertensive patients.
  -  Chronic disease e.g. diabetes mellitus.
  -  Known renal disease.
  -  Pre-operative infection.

Relevance to patient case: not relevant