cyrilzakka/clinical-trials huggingface dataset:
Orlistat Treatment of Crigler-Najjar Disease

The purpose of this study was to determine whether orlistat is effective in decreasing plasma unconjugated bilirubin levels in patients with Crigler-Najjar disease.

Unconjugated hyperbilirubinemia in Crigler-Najjar (CN) disease is conventionally treated with phototherapy and/or phenobarbital. Life-long daily phototherapy has considerable disadvantages. Main problems are a decreasing efficacy with age and a profound impact of the intensive phototherapy regimen on the quality of (social) life. An alternative treatment option for unconjugated hyperbilirubinemia is based on intestinal capture of UCB by oral treatment. Particularly when plasma UCB concentrations are high as in CN disease, UCB can diffuse from the blood into the intestinal lumen across the mucosa. Intestinal capture of UCB followed by fecal excretion reduces the enterohepatic circulation of UCB and subsequently decreases plasma UCB concentration. We demonstrated in Gunn rats, the animal model for CN disease, that orlistat treatment decreases plasma UCB concentrations parallel with increased fecal fat excretion, and induces net transmucosal excretion of UCB from the blood into the intestinal lumen. In human adults, orlistat has been widely applied for treatment of obesity, without serious side effects. Recent studies in obese adolescents and prepubertal children indicate that short-term orlistat treatment is well-tolerated by children and generally has only mild side effects. In the present randomized, placebo-controlled trial we determined in patients with CN disease the effects of orlistat treatment on plasma UCB concentrations, and on fecal excretion of fat and UCB.

Eligibility Criteria:
Inclusion Criteria:

* patients with Crigler-Najjar disease above the age of 7 years

Exclusion Criteria:

* cholestasis, chronic malabsorption syndrome, pregnancy
Curcumin Pharmacokinetics

Each year, there are about 150,000 new colorectal cancer (CRC) cases and 50,000 CRC deaths in the United States. A safe and effective preventive agent could reduce the burden of colorectal cancer as well as reduce the costs associated with screening patients who are at high risk of developing CRC. Preclinical studies strongly suggest that curcumin is active against multiple pathways implicated in tumor initiation, progression, and metastasis. However, little is know how curcumin performs in humans. The investigators propose to enroll 12 healthy volunteers who will undergo blood draw and rectal biopsy after 7 days of curcumin administration. The investigators will look at curcumin preparations with low and high oral bioavailability and calculate the blood and rectal tissue concentrations associated with these two formulations to determine which preparation produces the highest tissue concentrations.


Eligibility Criteria:
Inclusion Criteria:

1. 18 - 65 years of age
2. BMI 18 - 30 kg/m\^2
3. Ability/willing to provide informed consent
4. Good general health

Exclusion Criteria:

1. History of any pancreatic or biliary disease (eg. Familial colorectal cancer syndromes, Ulcerative colitis or Crohn's disease)
2. History of any acute or chronic illness that requires current medical therapy, including active gastrointestinal conditions, that might interfere with drug absorption
3. History of large bowel resection for any reason
4. Diagnosed narcotic or alcohol dependence
5. Women with childbearing potential who do not agree to practice effective birth control.
6. Use of curcumin within the last 14 days
7. Allergy to study agent
8. Individuals with creatinine, AST or ALT above 1.5 times the upper limit of normal at baseline.
9. Personal or inherited bleeding disorders or therapeutic anti-coagulation with warfarin.
10. Women who are pregnant or nursing.
Study on the Correlation Between TCM Syndrome, Inflammatory Phenotype and Biomarker of Bronchial Asthma

This study aims to determine the TCM syndrome pattern and the distribution of inflammation phenotype in different stages of bronchial asthma; to explore the correlation between TCM syndrome and inflammation phenotype. Secondly screening biomarkers that can be recognized by TCM syndromes and inflammatory phenotypes of bronchial asthma, and provide a basis for individualized diagnosis and treatment of diseases.

The clinical heterogeneity of bronchial asthma was strong.The TCM syndrome was complicated and mostly treated on stage, type, excess and deficiency.The phenotype was numerous and more classified from inflammation type, clinical classification, internal phenotype, etc.The flammatory phenotype was the more common type.The pathological basis of asthma was closely related to the infiltration of inflammatory mediators such as Airway eosinophil cells, so it was important to find the specific biomarker. The correlation between TCM syndrome, inflammatory phenotype and its biomarker wsa not clear. So we put forward theoretical hypothesis:There was of correlation between TCM syndrome and inflammatory phenotype of asthma.The biological target was contributes to the confirmation for TCM syndrome and inflammatory phenotype.The following research was planned to be carried out: relying on the registration system of National TCM Clinical Research Base to enroll in 900 cases patients in the acute and remission period of asthma and healthy physical examination crowd, filling in the patient's general demographic data, disease status, lung function, TCM syndrome and score and collecting the sputum, exhaled gas, urine, peripheral venous serum specimens and biomarker to be tested on the same day. Based on the clinical research database, confirming syndrome and in phenotype, clarifying the correlation between syndrome and phenotype and selecting identifiable biomarker by using correlation analysis, principal component analysis, multiple regression analysis, neural network analysis and other methods.The research provided evidence for the study of the essence of syndrome and phenotype and provided evidence for individualized diagnosis and treatment of diseases.

Eligibility Criteria:
Inclusion Criteria:

1. Patients with definite dignosis of bronchial asthma or healthy volunteer;
2. Ages range from 18 years to 80 years old;
3. Not anticipated in other clinical studies within 1 month before enrollment;
4. Signed informed consent.

Exclusion Criteria:

1. Acute asthma exacerbation patients with respiratory failure, mechanical ventilation;
2. Patients with unconsciousness, dementia, various mental disorders;
3. Complicated with hematological malignancies , heart failure(Acute myocardial infarction, cardiac function grade 3 or above), severe liver or kidney diseases;
4. Complicated with neuromuscular disorders affecting respiratory and motor function, unable to complete the six-minute walk test;
5. People who are participating in other clinical trials.
SBRT Compared With IMRT Concurrently With Chemotherapy in Treating Patients With LS-SCLC

As stereotactic radiotherapy (SBRT) has been widely used in clinical practice at present, the aim of this study is to evaluate the efficacy and safety of stereotactic body radiation therapy compared with intensity modulated radiation therapy (IMRT) concurrently with EP regimen(cisplatin plus etoposide) in treating patients with limited-stage small cell lung cancer


Eligibility Criteria:
Inclusion Criteria:

* Histologic or cytologic diagnosis of SCLC
* limited-stage disease is defined as disease confined to the ipsilateral hemithorax, which can be safely encompassed within a radiation field
* No prior chemotherapy, or radiotherapy
* Performance status of 0, 1, 2 on the ECOG criteria.
* At least one unidimensional measurable lesion meeting Response Evaluation Criteria in Solid Tumors (RECIST. 2000).
* Adequate hematologic (neutrophil count \>= 1,500/uL, platelets \>= 100,000/uL), hepatic (transaminase =\< upper normal limit(UNL)x2.5, bilirubin level =\< UNLx1.5), and renal (creatinine =\< UNL) function
* If female: childbearing potential either terminated by surgery, radiation, or menopause, or attenuated by use of an approved contraceptive method (intrauterine device \[IUD\], birth control pills, or barrier device) during and for 3 months after trial. If male, use of an approved contraceptive method during the study and 3 months afterwards. Females with childbearing potential must have a urine negative HCG test within 7 days prior to the study enrollment.

Exclusion Criteria:

* Non small cell lung cancer and carcinoid
* Supraclavicular lymphadenopathy
* Inability to comply with protocol or study procedures.
* Moderate and severe ventilation dysfunction
* Medically uncontrolled serious heart, lung, neurological, psychological, metabolic disease
* Second primary malignancy that is clinically detectable at the time of consideration for study enrollment.
* Pregnant or breast-feeding.
* Enrollment in other study within 30 days
Training Health Care Professional in Detecting BCC on OCT Using E-learning and CUSUM-analysis

In this study, various health care professionals will follow an E-learning module in which BCC detection on OCT is explained. Thereafter, the participants will test their skill by assessing OCT-scans. Their performance will be monitored using cumulative-sum analysis. After completion, newly trained OCT assessors will test their diagnostic accuracy for BCC detection on OCT in a exploratory study. The trainability, amount of required training and diagnostic accuracy will be compared between dermatologist and non-dermatologists.

Accurate detection of BCC on OCT requires substantial training and for future implementation of OCT, many OCT assessors need to be trained. Cumulative-sum analysis (CUSUM-analysis) can be used to objectify the competence of OCT assessors and has been previously used to monitor the competence of OCT assessors. Hence CUSUM-analysis may be valuable in training new assessors. A consensus statement from 2021 states that OCT scans should be acquired and interpreted by dermatologists. But this consensus is challenged by various studies in which OCT assessors were non-dermatologists. All non-dermatologist OCT assessors achieved high diagnostic accuracy. In addition, as diagnosis, treatment and follow-up of BCC patients constitute a substantial proportion of the workload of dermatologists, the question arises whether the acquisition of OCT scans and the subsequent assessment thereof could be outsourced to non-dermatologists. This could reduce the workload of the dermatologist, shorten waiting lists and potentially reduce costs.

In an E-learning dermatologists and non-dermatologists will learn about BCC features on OCT as described by Hussain et al. In this E-learning they will learn how to systematically examine an OCT-scan and detect BCC features. They will also be informed on common pitfalls in BCC detection. Thereafter they will start a CUSUM-module containing 400 OCT-scans (50% BCC vs. 50% non-BCC) to monitor cumulative successes and failures in diagnostic assessments. The competence of OCT assessors can be objectified using preset acceptable (16%) and unacceptable error rates (25%). The error rate is defined as the sum of false negative and false positive diagnoses divided by the total number of assessed OCT scans. For all OCT scans, the histologically verified diagnosis based on punch or excision biopsy is available. The OCT assessor will practice until an acceptable performance rate is achieved and maintained (over 50 scans). A secondary objective will be to explore the diagnostic accuracy in terms of sensitivity and specificity. Once an acceptable performance rate is achieved and maintained, assessors will assess a new cohort of 100 OCT scans (50% BCC and 50% non-BCC). Their level of suspicion for BCC will be expressed on a 5-point Likert-Scale. Diagnostic parameters will be compared between dermatologists and non-dermatologists.

Eligibility Criteria:
Inclusion Criteria:

* Health care provider or medical student

Exclusion Criteria:

* Previous experience with OCT
Effectiveness of Embrace Scar Therapy Device After Cutaneous Wound Closure

The purpose of this study is to determine whether the use of the embrace device after repair of linear cutaneous surgery wounds improves scar cosmesis.

The purpose of this study is to determine whether the use of the embrace device after repair of linear cutaneous surgery wounds improves scar cosmesis. We will use a split wound model, half of the wound is treated with the embrace device and the other half is not treated. Three-months post-surgery, the scar will be measured via the physician observer scar assessment scale, a validated scar instrument. The scar width and adverse events will also be recorded.

Following the surgical repair of cutaneous wounds, scar formation is inevitable and results in varying degrees of aesthetic and/or functional impairment. Numerous treatment modalities have been employed to treat scars. Carbon dioxide and pulse dye lasers, as well as dermabrasion can reduce erythema and irregular topology of the scar surface1,2. Silicone-based products have also been used to treat post-surgical scars, including gels, sheets, and tape3-5. Intralesional steroids are often injected into to induce flattening of a scar6. More recent research has highlighted the impact of mechanical forces and tension on scar formation. In one report, incisions in both pigs and humans were treated with a tension-shielding device and showed a reduction in scarring7. More recently two clinical trials have been published in the plastic surgery literature showing that the use of the embrace device, a silicone-based dressing designed to minimize wound tension, is effective in improving the aesthetic outcome following scar revision surgery8,9. While these initial studies of the embrace device have promising findings, there are significant drawbacks to both studies including small study population, inclusion of patients seeking scar revision (a select group likely predisposed to poor scar cosmesis and not representative of first-time surgical patients), investigator conflict of interest, and the use of a digital software-based scar assessment tool using patients photos that were not standardized with respect to lighting or distance. Therefore, larger studies in first-time surgical patients with standardized photos for scar assessment are required to validate this potentially promising device for improved scar cosmesis.

Eligibility Criteria:
Inclusion Criteria:

* 18 years of age or older
* Able to give informed consent themselves
* Patient scheduled for cutaneous surgical procedure along a flat surface suitable to application of the embrace with predicted primary closure.
* Able to apply dressings themselves.
* Willing to return for follow up visits.

Exclusion Criteria:

* Mentally handicapped
* Unable to understand written and oral English
* Incarceration
* Under 18 years of age
* Pregnant Women
* Wounds with predicted closure length less than 3 cm
* Patients with known adverse reactions to adhesives
* Patients with history of collagen vascular disease
EUS-guided Celiac Plexus Block vs. Sham in Chronic Pancreatitis

The aim of this randomized trial is to assess the efficacy of EUS-CPB in the alleviation of abdominal pain in patients with chronic pancreatitis.

Chronic pancreatitis is a complex fibroinflammatory disease arising from numerous etiological factors and a variety of clinical manifestations. Abdominal pain is the most debilitating complication and can result in significant morbidity and impact on quality of life. Endoscopic ultrasonography (EUS) has been increasingly utilized for celiac plexus block (EUS-CPB), whereby a steroid in combination with a local anesthetic agent are injected into the celiac plexus within the abdominal cavity. The aim of this randomized trial is to assess the efficacy of EUS-CPB in the alleviation of abdominal pain in patients with chronic pancreatitis.

Eligibility Criteria:
Inclusion Criteria:

* Age ≥ 18 years
* Abdominal pain of at least 3 months duration and a VAS score ≥ 3, with or without the use of narcotic analgesics.
* Diagnosis of chronic pancreatitis on EUS examination, with ≥ 5 features on EUS.
* No other cause of abdominal pain

Exclusion Criteria:

* Age \< 18 years.
* History of prior EUS-CPB.
* Use of anticoagulants that cannot be discontinued for the procedure.
* Clinically significant allergy to bupivacaine or triamcinolone.
* Unable to obtain consent for the procedure from either the patient or LAR.
* Intrauterine pregnancy.
Comparison of Dialysis Strategies in Critically Ill Patients With Acute Renal Failure

The goal of the present study is the comparison of different dialysis strategies in critically ill patients with acute renal failure on the intensive care unit. Patients are treated with either continuous dialysis or hemofiltration. Outcome measures are death, restitution of renal function, days on ICU, hemodynamic stability, dialysis efficiency.

The goal of the present study is the comparison of different renal replacement strategies in critically ill patients with acute renal failure on the intensive care unit. In a prospectively randomized clinical study patients are treated with either continuous dialysis (SLED) or continuous hemofiltration (CVVHD). 200 patients should be included in the study (n=100 each). The primary outcome is death at ICU, secondary outcome measures are restitution of renal function, days on ICU, hemodynamic stability, dialysis efficiency.

Eligibility Criteria:
Inclusion Criteria:

* Patients 18 years or older
* Written informed consent
* No pre-existing dialysis dependency

Exclusion Criteria:

* Minors / children
* No consent
* Severe electrolyte disturbances which necessitates special dialysis therapy
Binding of Lu AG06466 in the Brain in Healthy Men

A study to learn how Lu AG06466 and one of its break down products binds in the brain after single and repeated dosing

The study will be conducted in two Parts. Part A: cross over, fed/fasted determined by randomized sequence Part B: sequential group

PET scans will be used to quantify MAGL occupancy

Eligibility Criteria:
Inclusion Criteria:

* body weight ≥60 kg.
* body mass index ≥18.5 and ≤27 kg/m2.
* waist circumference ≤94 cm.
* The subject has a normal sMRI performed during the screening period.
* The subject is suitable for radial artery blood sampling and cannulation as demonstrated by the Allen test.
* Subject must be healthy as assessed using detailed medical history, laboratory tests, and physical examination.
* The subject must make use of contraception.

Exclusion Criteria:

* The subject is left handed.

Other in- and exclusion criteria may apply
Observational Study to Assess Adherence Oral Anticancer Therapies

Oral anticancer treatments are increasingly numerous. They represent an additional alternative in the therapeutic arsenal of the clinician, and appear to satisfy patients who prefer this route of administration over intravenous treatment. The objective of oral therapies is twofold: to remove the constraints and risks associated with infusions and to allow the patient to follow his treatment at home.

However, they have significant adverse effects that may affect patients, who are sometimes at a disadvantage compared to how they are treated, and potentially lead to non-compliance with the consequences.

This study will identify the factors associated with non-adherence and determine the impact of this non-adherence in terms of treatment efficacy and tolerance.

The aim of this routine care study is to evaluate the adherence to oral anticancer therapies during 3 months.

Primary objective

- To evaluate adherence to oral anti-cancer therapies at 3 months

Secondary objectives

* To evaluate the adherence to 3 months according to the age of the patients (less than 75 years versus 75 years and more)
* Evaluate compliance at 1 and 2 months
* Evaluate toxicity at 1, 2 and 3 months according to compliance according to criteria CTCAE 4.03.
* Evaluate response to treatment at 3 months based on adherence
* Evaluate the factors associated with adherence and describe the reasons for non-adherence.

Eligibility Criteria:
Inclusion Criteria:

* Age\> 18 years
* Cancer proved histologically.
* Patient receiving an initial prescription for oral anticancer therapy, excluding hormone therapy
* Illness measurable or assessable by imaging
* Patient affiliated to a social security scheme
* Patient having been informed of the study
* Non-opposition of the patient

Exclusion Criteria:

* Contraindication to oral treatment
* Patient's refusal
* Patient under tutelage, curatorship or safeguard of justice
* Psychiatric illness and / or condition of the patient compromising understanding of information or conduct of the study
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France

Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.


Eligibility Criteria:
Inclusion Criteria:

* Have a diagnosis of haemophilia B and been treated previously with factor IX Product
* Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
* Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations

Exclusion Criteria:

* Participation in an investigational medicinal product trial at enrolment visit
Evaluation of a New Strategy for the Diagnosis of Peroxisomal Diseases

The principal aim of the study is to avoid the diagnostic wanderings of patients suffering from a peroxisomal disorder. For this purpose, a new diagnostic strategy is proposed. It rests on functional metabolic explorations and gene studies directly connected to a first-line enlarged physico-chemical detection of metabolites from peroxisomal origin in clinically suspect patients.


Eligibility Criteria:
Inclusion Criteria:

I - CLINICAL/BIOLOGICAL CRITERIA For an efficient screening of the patients of the 4 contributing University Hospitals (Amiens, Caen, Lille and Rouen), various inclusion criteria were selected: In general, the inclusion criterion is the existence of a positive biology or in turn the persistence of a clinical suspicion in spite of a negative biology.

I A - Children from 0 to 17 years:

The clinical inclusion criterion is a positive biology or the persistence of a clinical suspicion in spite of a negative biology. This persistent clinical suspicion is left to the discretion of the clinician. It is essentially based on the existence of a family history of peroxisomal (or suspected) pathology and / or the combination of several clinical signs of craniofacial dysmorphism, skeletal abnormalities, encephalopathy (seizures, ataxia, Hypotonia), demyelinating peripheral neuropathy, ophthalmopathy (retinopathy, cataract), hepatic impairment (hyperbilirubinemia, hepatomegaly, cholestasis) and growth retardation OR I B - Adults from 18 years

The neurological symptoms of peroxisomal diseases in adulthood are numerous and non-specific. The three inclusion criteria selected for patient selection are as follows:

* Cognitive impairment (delayed acquisition or regression) and / or leukodystrophy AND At least one of the following signs: Cerebellar ataxia, Spastic paraparesis, Peripheral neuropathy, Neurosensory deafness, Retinitis pigmentosa, Epilepsy, Unexplained unexplained vigilance, Peripheral corticotropic insufficiency +/- gonadotropic AND
* no evidence for an extraperoxisomal origin of the patient disease stated after the usual paraclinic explorations

II - NON CLINICAL CRITERIA

* Social insurances
* Having understood the information note and having signed the informed consent form.
* Patients under guardianship or curatorship may be included, since peroxisomal diseases as a cause of neurological impairment may potentially lead to guardianship.

Exclusion Criteria:

* Pregnant or nursing women
* Person deprived of liberty or in emergency situations