cyrilzakka/clinical-trials huggingface dataset:
Treatment of a Single Patient With CRD-TMH-001

The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.

The objective of the study is to assess the safety and preliminary efficacy of CRD-TMH-001 after intravenous administration for a period of 1 year with long-term follow-up out to 15 years.

Eligibility Criteria:
Inclusion Criteria:

* Completion of informed consent
* Confirmation of genetic mutation
* Confirmation of absence of elevated AAV9 NAbs

Exclusion Criteria:

- Any significant medical issue(s) (past or current) that would, in the opinion of the Principal Investigator (PI), prevent this patient from being dosed.
The Role of Functional Magnetic Resonance Imaging in Preoperative Assessment of Primary Penile Carcinoma

The proposed project is intended as a prospective study that includes 60 patients with newly diagnosed penile squamous cell carcinoma (SCC) referred to Oslo University Hospital (OUH), Radiumhospitalet, for surgery, primarily organ-sparing surgery (OSS).

OSS may improve not only quality of life, but also quality of sexual function. However, there is a potential for increased risk of local recurrence after OSS compared to the amputation of the penis. Appropriate preoperative staging, including multi-parametric magnetic resonance imaging (mpMRI), can substantially improve selection of patients and decrease the recurrence rate after surgery.

MpMRI without artificial erection is promising diagnostic tool that is poised to be all-in-one solution for staging and preoperative assessment of primary penile cancer, especially prior to OSS. The method is non-invasive and thus comfortable to perform for most of the patients. Novel MRI techniques are not incorporated into current clinical recommendations, and the potential of new, functional sequences has not been evaluated before. The accuracy of functional, non-erectile mpMRI for detecting and staging of primary penile cancer is not known. Thus, the main purpose of this study is to assess the diagnostic value of this method for preoperative assessment of penile cancer.


Eligibility Criteria:
Inclusion Criteria:

* newly diagnosed penile squamous cell carcinoma (SCC)
* the patient will benefit from organ-sparing surgery (OSS)
* signed broad consent for cancer research

Exclusion Criteria:

* N/A
Impact of Orthodontic Treatment on the Temporomandibular Joints (TMJ) Assessed by Modjaw

The aim of the study is to assess the correlation between orthodontic treatment and temporomandibular joint (TMJ). For that, an evaluation with a virtual articulator (Modjaw) is carried out before and after the treatment. The condylar course are compared. Furthermore, the eventual TMJ symptoms are also compared before and after.


Eligibility Criteria:
Inclusion Criteria:

* Major patient (≥18 years)
* First dentofacial orthopedic consultation at the dental clinic of Montpellier
* Patient requiring multi-attachment orthodontic treatment (vestibular or lingual) for a duration ≤2 years
* Signed informed consent

Exclusion Criteria:

* Syndromic patient
* Orthodontic treatment with aligners
* General joint or bone pathology
* Patients with oral hygiene incompatible with orthodontic treatment
The Cytoadherence in Pediatric Malaria (CPM) Study

The purpose of this study is to determine the importance of key blood group molecules in the clinical outcome of Plasmodium falciparum malaria infection in children.

Every year, nearly 2 million children die from infection with Plasmodium falciparum malaria. When red blood cells (RBC) become infected with malaria, a sticky parasite-derived knob protein, termed PfEMP-1, erupts on the RBC surfaces. PfEMP-1 attaches to several blood group molecules, including those found on other RBC, on blood vessels, and on the cells that normally help to stop bleeding (platelets). The cellular sticking results in a dangerous interruption in blood flow to vital organs, causing brain injury (cerebral malaria), systemic shock (lactic acidosis), and death. Depending on an individual's inherited blood groups of relevance, adhesion may be extensive or limited. In the laboratory, PfEMP-1 adheres to RBCs via the A or B (but not the O) antigens of the ABO blood group system, and to platelets and blood vessels via platelet glycoprotein IV (CD36) and ICAM-1. Consistent with the expected evolutionary advantage of being deficient in these binding targets, blood type O and low-expression of CD36 are found more frequently among Africans. The "Cytoadherence in Pediatric Malaria" (CPM) project is determining the distribution of adhesive blood group molecules in a cohort of 2000 Ugandan children according to the extent of malaria severity and death, and thus their ultimate clinical and evolutionary significance in malarial survival. This knowledge may serve as the grounds for developing targeted cytoadhesion-interruption therapies in our fight against malaria.

Eligibility Criteria:
Inclusion Criteria:

* Clinical diagnosis of Plasmodium falciparum malaria infection

Exclusion Criteria:

* HIV or significant malnutrition
Safety and Efficacy of SOF/VEL/VOX FDC for 8 Weeks and SOF/VEL for 12 Weeks in Adults Chronic Genotype 3 HCV Infection and Cirrhosis

The primary objective of this study is to evaluate the efficacy, safety, and tolerability of treatment with sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose combination (FDC) for 8 weeks and of treatment with sofosbuvir/velpatasvir (SOF/VEL) FDC for 12 weeks in participants naive to direct-acting antivirals (DAA) with chronic genotype 3 hepatitis C virus (HCV) infection and cirrhosis.


Eligibility Criteria:
Key Inclusion Criteria:

* Willing and able to provide written informed consent
* HCV RNA ≥ 10\^4 IU/mL at screening
* Chronic genotype 3 HCV infection (≥ 6 months)
* Presence of cirrhosis
* HCV treatment naive or treatment experienced with an interferon (IFN)-based regimen
* Use of protocol specified methods of contraception

Key Exclusion Criteria:

* Current or prior history of clinically significant illness that may interfere with participation in the study
* Screening ECG with clinically significant abnormalities
* Laboratory parameters outside the acceptable range at screening
* Pregnant or nursing female
* Chronic liver disease not caused by HCV
* Infection with hepatitis B virus (HBV) or human immunodeficiency virus (HIV)

Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)

Current diagnostic criteria for Immune ThrombocytoPenia (ITP) are mainly based on the presence of low numbers of platelets, excluding other multiple causes of thrombocytopenia, including immunodeficiencies, constitutional or acquired thrombocytopenia, hypersplenism and clonal hematological disorders such as MDS, disorders lymphoproliferative and acute myeloid leukemia (AML), among others. The analysis complementary tests for the diagnosis of ITP include studies basic systematic hematology, together with autoimmune assays and microbiological tests, while the evaluation of bone marrow is limited to elderly patients and/or patients resistant to treatment. Previous research has described the development of Myelodysplastic Syndrome (MDS) in patients with a previous diagnosis of ITP, and even the presence of MDS associated with genetic background. Therefore, it is conceivable fact that a percentage of cases with clinical signs of ITP in the moment of appearance may actually correspond to the first stages of MDS development in which bone marrow cells are not systematically evaluated in the initial presentation.

The anomalous immunophenotypic patterns between multiple compartments of bone marrow cells and peripherally blood (PB) platelets have been characterized through flow cytometry. The flow cytometry currently represents an important complementary tool for diagnosis of MDS that has shown great effectiveness and applicability in the differential diagnosis of non-clonal cytopenias against early MDS and for the detection of stages prior to MDS. Besides, the flow cytometry has made it possible to detect the presence of coexisting features related to MDS in patients with other malignancies hematologic conditions such as multiple myeloma, AML, and lymphocytic leukemia chronic. Therefore, the immunophenotypic analysis of the cells of the bone marrow of patients with ITP at the time of appearance would help to identify the cases that underlie clonal hematopoiesis MDS type. In the present study it is planned a broad characterization immunophenotyping of multiple compartments of bone marrow cells and PB platelets from patients with recently diagnosed ITP and investigate their morphological antecedents, in order to identify those patients who show compatible clonal hematopoietic patterns with MDS evident (or at risk of development), as candidates to receive most appropriate therapeutic methods.


Eligibility Criteria:
Inclusion Criteria:

* Patients aged ≥ 18 years old at diagnosis
* Informed consent in writing
* Newly diagnosed primary ITP patients, or
* Newly diagnosed MDS patients

Exclusion Criteria:

* Patients who participated in a interventional thrombopoietin receptor agonists (TPO-RA) clinical trial since TPO-RA treatment initiation
* Patients with secondary immune thrombopenia
Radiation Therapy in Treating Patients With Cervical Cancer

RATIONALE: Radiation therapy uses high-energy x-rays to kill tumor cells. Internal radiation uses radioactive material placed directly into or near a tumor to kill tumor cells. Giving radiation therapy in different ways may kill more tumor cells.

PURPOSE: This phase II trial is studying how well giving external-beam radiation together with internal radiation works in treating patients with cervical cancer.

OBJECTIVES:

Primary

* Determine the feasibility of magnetic resonance-guided, endocavitary, high-dose rate brachytherapy implants in patients with stage IB-IVB cervical cancer.
* Provide more reliable dose-volume estimations based on three-dimensional imaging-based treatment planning in patients treated with this regimen.

Secondary

* Determine the toxic effects and treatment tolerance in patients treated with this regimen.
* Correlate three-dimensional dose-volume histograms of organs at risk with treatment toxicity in patients treated with this regimen.
* Determine the disease status, time and patterns of relapse, and survival of patients treated with this regimen.

OUTLINE: Patients undergo external beam radiotherapy 4-5 days a week for up to 7 weeks. Patients also undergo MRI-guided high-dose brachytherapy comprising one brachytherapy implant a week for 3-6 implants. Brachytherapy may be administered during or after external beam radiotherapy.

After completion of study treatment, patients are followed periodically for 5 years.

PROJECTED ACCRUAL: A total of 20 patients will be accrued for this study.

Eligibility Criteria:
DISEASE CHARACTERISTICS:

* Histologically confirmed carcinoma of the cervix of 1 of the following cellular subtypes:

  * Squamous cell
  * Adenocarcinoma
  * Adenosquamous cell
* Stages IB-IVA disease

  * Stage IVB disease allowed provided the impact on the quality of life is tremendous (e.g., "frozen pelvis" or massive pelvic disease and fistula formation, severe pain, or bleeding)
* Measurable and/or evaluable disease on MRI

PATIENT CHARACTERISTICS:

* ECOG performance status 0-2
* No physical or physiological capacity that would preclude study treatment
* No cognitively impaired patients who cannot provide informed consent
* Not pregnant or nursing
* Negative pregnancy test
* No contraindication to MRI, including any of the following:

  * Weight \> 136 kg
  * Allergy to MR contrast agent
  * Pacemakers, cerebral aneurysm clips, shrapnel injury, or implantable electronic devices
* No significant unrelated systemic illness
* No serious infections
* No significant cardiac, pulmonary, hepatic, or other organ dysfunction that would preclude study treatment
* Must be medically fit to receive anesthesia

PRIOR CONCURRENT THERAPY:

* No prior definitive brachytherapy procedures

  * Ring implants or intravaginal cones for the relief of excessive bleeding allowed
* No prior definitive surgical oncologic procedures (e.g., radical hysterectomy)
* Concurrent chemotherapy, immunotherapy, or hormonal therapy allowed
Outcome Factors of Pneumonia Patients in Critical Care Unit

To determine the risk factor that affect pneumonic patients at the critical care unit regarding the history investigation and clinical data

Pneumonia remains a serious illness with important clinical impact . It is defined as acute respiratory tract infection that affect lower respiratory tract ,it is classified as community acquired pneumonia (CAP), hospital acquired pneumonia (HAP) and ventilator associated pneumonia .

In this work we assess the factors that affect the prognosis of critically ill patients with pneumonia regarding the age ,sex ,smoking, other co morbidity as diabetes mellitus, hypertension , liver diseases , kidney diseases, heart diseases and chest diseases, clinical date and investigation .

Eligibility Criteria:
Inclusion Criteria:

* 1- Age above 18 yrs.

  2- Critically ill patients admitted to ICU with clinical manifestations of pneumonia in assuit university hospital

Exclusion Criteria:

* 1- Age below 18 yrs. 2- Patients don't have critical illness 3- Patient who refused to contribute in this study
the Pharmacokinetic Profiles of Linagliptin With DW1029M

Phase I clinical study to evaluate DW1029M influence the pharmacokinetic profiles of Linagliptin after oral administration in healthy male volunteer.

A Randomized, open-label, multiple-dose, crossover phase I clinical study to evaluate DW1029M influence the pharmacokinetic profiles of Linagliptin after oral administration in healthy male volunteer.

Eligibility Criteria:
Inclusion Criteria:

* A healthy adult within the range of 19 to 55 years old at the time of screening
* Body Mass Index(BMI)=17.5\~30.5kg/m2, one with weight of more than 55kg (BMI= kg/(m)2)
* Congenital or chronic diseases within the last three years, there is no medical examination results of popular characters with no psychotic symptoms or findings
* Doctor checks conducted hematology, blood chemistry, urinalysis and laboratory tests, including an electrocardiogram(EKG) performed during screening tests such as a suitable test subjects who judged
* The purpose of the test participants prior to testing, information, and to hear about the free will fully explain to participate in this study, according to the Institutional Review Board(IRB)-approved consent form signed by the parties in writing

Exclusion Criteria:

* one with clinically significant blood, kidneys, endocrine, respiratory, gastrointestinal, urinary, cardiovascular, hepatic, psychiatric, neurological or allergic disease (but ,except for untreated seasonal allergies of asymptomatic at the time of administration) with medical history or evidence
* one with gastrointestinal disease(Such as esophageal stricture or achalasia of the esophagus disease, Crohn disease) to affect drug absorption or surgery(but, excluding simple appendectomy or hernia surgery) with medical history
* Alanine Aminotransferase(ALT) or Aspartate Aminotransferase(AST) \> 2 times the upper limit of the normal range
* Within 6 months, 210 g / week of alcohol in excess of a history of regular characters (beer (5%) 1 cup (250 mL) = 10 g, soju(20%) 1 cup (50 mL) = 8 g, Wine (12%) 1 cup (125 mL) = 12 g)
* Take part in other clinical trials within two months
* Systolic Blood Pressure(SBP) ≥ 140 mmHg or Diastolic Blood Pressure (DBP) ≥ 90 mmHg
* The great history of alcohol or drug abuse within 1 year
* Taking medication of drug-metabolizing enzymes that are known to significantly induce or inhibit within 30 days
* More than 20 cigarettes a day smoker
* Taking medication of a prescription drug or nonprescription within 10 days,
* Within two months the whole blood donation have, within one month of the apheresis donation have
* Participate in clinical trials to test drug administration and may be at increased risk due to interpretation of test results, or may interfere with severe supply / chronic medical or mental condition or abnormal laboratory test values in character
* Patient with hypersensitivity to investigational products or ingredients
* Patient with Type 1 Diabetes or Diabetic ketoacidosis
* Described lifestyle in this protocol can comply with or can not
* One with other investigator judge to unsuitable
The Relationship Between Arsenic Methylation Capability and Blood Metals in Children With Developmental Delays

The purpose of this study is to explore the relationship between arsenic methylation and blood metals in children with developmental delays and its correlation with health related quality of life.

The investigators recruited children with developmental delays and children with normal development from Shin-Kong Ho-Su Memorial Hospital as controls. Information obtained from the interview included socioeconomic and basic demographic characteristics as well as children function, health condition, and quality of life. On the other hand, blood and urine samples of the participants were collected for the analysis of arsenic methylation related enzymes using polymerase chain reaction and restriction fragment length polymorphism. 2 ml blood were digested for the determination of mercury, lead and arsenic. The relationship between arsenic methylation and blood metals in children with developmental delays and its correlation with health related quality of were analyzed.

Eligibility Criteria:
Study group:

Inclusion Criteria:

* children with developmental delays obtained written informed consent from parents

Exclusion Criteria:

* parents refused for participation of their children to the intervention failed to obtain the written informed consent from parents

Control group:

Inclusion Criteria:

* children with normal development obtained written informed consent from parents

Exclusion Criteria:

* parents refused for participation of their children to the intervention failed to obtain the written informed consent from parents
Predicting Response of Metastatic Triple Negative Breast Cancer to Immunotherapy Based on Patients Cytokine Profile

Recent progress in immunotherapy (IT) has shifted treatment paradigms for multiple malignancies including breast cancer. It has been shown that levels of certain cytokines were correlated with increased response to immunotherapy in patients with metastatic breast cancer. In this study investigators will perform cytokine profiling among patients diagnosed with metastatic triple-negative breast cancer at different time points during the treatment.

This is a prospective study that has two groups of patients as determined by their prescribed treatment plan. Group A (control group) are patients with metastatic triple-negative breast cancer (TNBC) who receive chemotherapy only (current standard of care). Group B (experimental group) are patients with mTNBC who receive a combination of chemotherapy and IT immunotherapy (Atezolizumab) which is a standard of care for patients whose tumor express \>1% of PD-L1 molecules. T-cell cytokines will be measures prior to treatment and at multiple time points during the treatment.

Eligibility Criteria:
Inclusion Criteria:

* Diagnosis of metastatic triple negative breast cancer
* 18 years of age or older

Exclusion Criteria:

* Younger than 18
* Unwilling to participate or unable to give informed consent
Oropharyngeal Administration of Mother's Milk in Preterm Infants and Gastrointestinal Motility

Mother's milk does not come in contact with the oropharyngeal pouch of preterm infants during gavage feeding. We hypothesized that stimulation of the oropharyngeal pouch using small amount of the mother's milk 5 minutes before initiation of regular gavage feeding will increase the level of GIT hormones.

Feeding preterm infants continues to challenge health care providers because of difficulty to provide adequate volume of milk that maintains optimum nutrition without increasing the risk of feeding intolerance. Preterm, VLBW, infants are at increased risk of feeding intolerance as they have shorter GIT with lower digestive, absorptive, and motility capabilities than those of full-term infants. Intolerance to enteral feeding has been associated with abdominal distention, initiation of inflammatory cascade, edema of the bowel, and subsequent development of necrotizing enterocolitis.

Oral feeding is the best physiologic method for enteral nutrition of preterm infants. However, because of immaturity of suckling reflex and poor coordination between suckling and swallowing, gavage feeding (oro-gastric or naso-gastric tube feeding) has been used an alternative method of enteral nutrition in preterm infants.

During breastfeeding, mother's milk comes in contact with mouth and oro-pharyngeal pouch which, theoretically, stimulates both oro-pharyngeal receptors that improves the motility, secretory, and absorptive ability of the GIT. Furthermore, anti-inflammatory and pro-inflammatory cytokines, which are present abundantly in mother's colostrum and milk, may exert an immuno-protective effect when they come in contact with oro-pharyngeal as well as GIT mucosa.

Preterm, VLBW, infant in the NICU receives enteral feedings by a naso-gastric or oro-gastric gavage tube. Thus, mother's milk does not typically come into contact with oro-pharyngeal pouch which delays the maturation of oral suckling and swallowing skills in preterm infants.

Oral stimulation has been shown to improve oral feeding performance, attain early oral feeding, improve weight gain and shorten the length of hospital stay. Investigators aimed to study the effect of Oro-pharyngeal administration of mother's milk before regular gavage feeding on gastrointestinal movement in preterm infants.

Eligibility Criteria:
Inclusion Criteria:

* Preterm infants delivered at less than 32 weeks gestation and less than 1500 grams birth weight will be included in the study

Exclusion Criteria:

1. Preterm infants \> 32 weeks gestation unable to be fed on own mother's colostrum or milk.
2. Preterm infants with major congenital anomalies or chromosomal abnormalities.
3. Preterm infants delivered to mothers with confirmed chorioamnionitis.
4. Preterm infants with confirmed early onset sepsis.
Efficacy and Safety of Xylitol Nasal Irrigation After Functional Endoscopic Sinus Surgery

Investigators tried to evaluate the efficacy and safety of xylitol nasal irrigation as an adjuvant therapy after FESS, particularly the influence of nasal irrigation on Eustachian tube function.

In this study, patients with chronic rhinosinusitis who received FESS were recruited and randomly assigned to 2 groups at one month post-surgery. Patients in the xylitol group received 400ml of 5% xylitol nasal irrigation daily for 2 months, and those in the normal saline (NS) group received 400ml of NS nasal irrigation daily for 2 months. Before FESS as well as before and after nasal irrigation, sino-nasal symptoms were assessed by a 22-item Sino-Nasal Outcome Test questionnaire and patients received endoscopic examination, nasal function tests, cytokine measurement of nasal irrigant, and bacterial culture from the middle meatus. The safety of nasal irrigation was assessed by self-reported adverse events, blood test, Eustachian Tube Dysfunction Patient Questionnaire and Eustachian tube function test. This study tried to evaluate the efficacy and safety of xylitol nasal irrigation as an adjuvant therapy after FESS, particularly the influence of nasal irrigation on Eustachian tube function.

Eligibility Criteria:
Inclusion Criteria:

1. Patients with chronic rhinosinusitis who failed medical treatment
2. Patients underwent bilateral primary functional endoscopic sinus surgery.

Exclusion Criteria:

1. Patients with a history of immunodeficiency
2. Patients with a history of sinus surgery
3. Patients who receiving antibiotic treatment within a week before functional endoscopic sinus surgery
4. Patients with a pathological diagnosis of fungal sinusitis
5. Patients with a pathological diagnosis of sinonasal tumor