Kornimate/medical-research-clean huggingface dataset:
effect of anti-cd303 antibodies in autoimmune diseases

Conditions: Immune Disease

Summary: the pathogenic role of type i interferons (ifns) in the development of different autoimmune diseases has been extensively described in the literature. since plasmacytoid dendritic cells (pdcs) are the main source of type i ifns, there is evidence of the involvement of pdcs in autoimmune diseases. the cd303 surface protein (also called bdca-2) is specifically expressed by the pdcs. the hypothesis leading to the realization of this study is to observe, in vitro, an inhibition of the secretion of the type i ifns by pdcs in the peripheral blood in patients with autoimmune disease, thanks to the action of the anti-cd303 antibody developed by the lfb group, which could reduce the inflammatory response and improve patients with autoimmune disease


Eligibility criteria:
inclusion criteria: * have the ability to understand the requirements of the study, provide written informed consent, and comply with the study data collection procedures * patient followed in the department of internal medicine of chu lille * patient with one of the following autoimmune disease, defined according to international criteria: systemic lupus erythematosus, systemic sclerosis, gougerot-sjögren syndrome and idiopathic thrombocytopenic purpura * being socially insured exclusion criteria: * overlapping syndrome with another autoimmune disease * age ≤18 years * pregnant or nursing women * people in emergencies * person incapable of consent * persons deprived of liberty * persons without social security cover

Keywords: autoimmune diseases plasmacytoid dendritic cells type i interferons antibodies anti-cd303
a phase ii, open label, multiple arm study of auy922, byl719, inc280, ldk378 and mek162 in chinese patients with advanced non-small cell lung cancer

Conditions: Adenocarcinoma Lung Cancer; Squamous Cell Lung Carcinoma

Summary: the purpose of this study was to evaluate the anti-tumor activity of single agent byl719, inc280, ldk378 and mek162 in advanced nsclc patients carrying specific molecular alterations. there is a great unmet medical need in nsclc patients with advanced or metastatic disease. novel approaches using targeted therapeutic agents for these patient populations with molecular characterization could potentially identify subsets of advanced nsclc patients who would benefit from targeted kinase inhibition. study treatments, byl719, inc280, ldk378 and mek162, which target pik3ca, c-met, alk/ros1 and mek respectively, have shown promising data in either preclinical or clinical lung cancer settings.

to enter the screening phase of the study, the subjects' molecular alterations were determined using locally validated methodologies from a newly obtained tumor sample (preferred) or the most recent archival tumor sample available. based on the molecular alterations of the tumor, subjects were assigned to one of the treatment arms. subjects with multiple molecular alterations in epidermal growth factor receptor (egfr) and the relevant pathways were excluded, except under the conditions described in inclusion criteria. the treatment period began on cycle 1 day 1 and continued in 28-day cycles until disease progression, unacceptable toxicity, withdrawal of informed consent, death or the subject transferred to another novartis study that could continue to provide the study drug. all subjects were required to be followed up for 30 days for safety after receiving the last dose of study treatment. subjects who discontinued study treatment for any reason other than disease progression were followed up for progression of disease. all subjects were required to be followed for survival. for subjects transferred to another novartis study, an end of treatment visit (eot) was required to be performed and the subject would not enter the follow-up period.

Eligibility criteria:
inclusion criteria: * advanced (stage iiib or stage iv) nsclc * must have specific molecular alterations exclusion criteria: * symptomatic central nervous system (cns) metastases which are neurologically unstable or requiring increasing doses of steroids within the 4 weeks prior to study entry to control their cns disease * radiation therapy within ≤ 4 weeks prior to study entry, with the exception of limited field palliative radiotherapy for bone pain relief. * any other malignancies within the last 5 years before study entry * major surgery ≤ 2 weeks prior to study entry or who have not recovered from side effects of such therapy

Keywords: adenocarcinoma lung cancer, squamous cell lung carcinoma, nsclc
a study of et-743 (trabectedin) in patients with advanced breast cancer

Conditions: Breast Neoplasms; Neoplasms; Neoplasms by Site; Breast Diseases; Skin Diseases

Summary: the purpose of this study is to test the safety and effectiveness of an investigational chemotherapy agent in patients with advanced breast cancer.

patients will be enrolled in the study after all study specific-entry criteria are met and informed consent is obtained. patients will be required to attend regular clinic visits to receive study medication and have their status monitored. patients will also be required to have radiologic tumor assessments performed at multiple times throughout the study. a detailed explanation can be provided by the study physician (investigator) conducting this study. trabectedin 580 mcg/m2 once weekly for 3 consecutive weeks (treatment a) or 1,300 mcg/m2 once every 21 days (treatment b) will be given to patients as a 3-hour intravenous (i.v). infusion via a central venous catheter which is a tube placed into a large vein that is used to administer medications. all patients will be given dexamethasone 10 mg i.v. 30 minutes before each trabectedin infusion. patients may receive multiple cycles of trabectedin in the absence of disease progression.

Eligibility criteria:
inclusion criteria: * diagnosis of advanced breast cancer * prior therapy with anthracycline and taxane (2 types of chemotherapy drugs) * at least one measureable tumor lesion * adequate bone marrow, hepatic and renal function * eastern cooperative oncology group (ecog) performance status of 0 or 1 exclusion criteria: * known hypersensitivity to any components of the i.v. formulation of et-743 or dexamethasone * pregnant or lactating women * known metastases (spread) of cancer to the central nervous system * history of another neoplastic disease unless in remission for five years or more.

Keywords: trabectedin yondelis et743 advanced breast cancer breast cancer antineoplastic agents, alkylating agents molecular mechanisms of pharmacological action
residual dental pulp tissue and cord blood stem cells

Conditions: Irreversible Pulpitis

Summary: the experiments outlined in this proposal are designed to test the hypothesis that the in vivo injection of cord blood stem cells (invitrx®) into the root canal system will facilitate teeth initially diagnosed with irreversible pulpitis, to form normal healthy pulpal tissue.

patients will be recruited from the various dental clinics at ucsf (student clinic, advanced education in general dentistry clinic, post-graduate endodontics clinic, etc) that meet the inclusion and exclusion criteria. patients in the control group will have root canal therapy initiated; instrumentation and cleaning up to 4mm from the radiographic apex. bleeding will be induced and blood clot formed 3mm from the cej. mta followed by glass ionomer and composite will be placed over the clot. patients in the experimental group will have root canal therapy initiatied; instrumentation and cleaning up to 4mm from the radiographic apex. bleeding will be induced and blood clot formed 3mm from the cej. 1 cc (30 million cord blood stem cells) will then be injected into the clot formation. mta followed by glass ionomer and composite will be placed over the clot.

Eligibility criteria:
inclusion criteria: a. patients must be systemically healthy, class i dental category. b. involved teeth must have no periodontal disease detectible by periodontal examination. c. patients must present without radiographic signs of endodontic disease (i.e. no periapical radiolucencies). exclusion criteria: a. presence of any disease or medication that alters the immune system or interferes with healing ability b. smokers (more than 10 cigarettes per day) c. external or internal tooth resorption d. tooth perforation from the pulp cavity through the tip of the root exposing the tissue to material in the oral cavity. e. pregnant or nursing mothers because hormonal factors may influence the condition. f. allergies or adverse reactions to local anesthetic medications g. patients under the age of 18. h. patients with an asa classification ii or higher

Keywords: pulpal regeneration stem cells residual pulpal tissue irreversible pulpitis
aquatic treadmill therapy for improving gait, balance, fitness and quality of life in stroke patients

Conditions: Ischemic or Hemorrhagic Stroke; Hemiparesis

Summary: this study aims to determine if aquatic treadmill therapy is effective for improving economy of gait, gait speed, balance, and cardiovascular fitness in people with chronic stroke.


Eligibility criteria:
inclusion criteria: * individuals with ischemic or hemorrhagic stroke with hemiparesis who have chronic deficits in gait, falls and/or poor fitness exclusion criteria: * subjects with medical co-morbidities that may affect gait and endurance.
a study of mrna-3745 in adult and pediatric participants with glycogen storage disease type 1a (gsd1a)

Conditions: Glycogen Storage Disease

Summary: the main goal of this trial is to evaluate the safety and tolerability of mrna-3745 via intravenous (iv) administration in adult and pediatric participants with gsd1a.

the study includes a single ascending dose (sad) stage and a multiple ascending dose (mad) stage. participants enrolled in the mad stage have the option to continue treatment in an open-label extension (ole) period that will assess long-term safety and clinical activity of mrna-3745.

Eligibility criteria:
inclusion criteria: * documented gsd1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (g6pc) mutations by genetic testing. * absence of hospitalization for hypoglycemia in the 4 weeks prior to screening exclusion criteria: * solid organ transplant * received gene therapy for gsd1a * presence of liver adenoma \>5 centimeters (cm) in size * diagnosis of type 1 or type 2 diabetes mellitus * presence of liver adenoma with growth of \>2 cm or \>5 newly diagnosed liver adenomas, in the previous 2 years note: additional inclusion/exclusion criteria may apply, per protocol.

Keywords: glycogen storage disease type 1a gsd1a von gierke disease glucose metabolism disorder genetic disorder autosomal recessive disorder messenger rna mrna pediatric
real-world ar101 market-supporting experience study in peanut-allergic children (ramses)

Conditions: Peanut Allergy

Summary: this is a multicenter, randomized, double-blind, placebo-controlled safety study of ar101 using the characterized oral desensitization immunotherapy (codit™) regimen in peanut-allergic children.

the primary objective is to assess the safety and tolerability of ar101 when used in a codit™ regimen for approximately 6 months in peanut-allergic children children aged 4 to 17 years, inclusive.

Eligibility criteria:
key inclusion criteria: * ages 4 to 17 years, inclusive * history of physician-diagnosed peanut allergy that includes allergic signs and symptoms within two hours of known oral exposure to peanut * mean peanut wheal diameter on spt of at least 8mm and elevated psige of at least 14 kua/l at screening * written informed consent from the subject's parent/guardian * written assent from the subject as appropriate (per local regulatory requirements) * use of effective birth control by sexually active female subjects of childbearing potential key exclusion criteria: * subjects in whom the clinical diagnosis of peanut allergy is uncertain * severe or uncontrolled asthma * history of cardiovascular disease, including uncontrolled or inadequately controlled hypertension * history of severe or life-threatening episode of anaphylaxis or anaphylactic shock within 60 days of screening * history of eosinophilic esophagitis (eoe), other eosinophilic gastrointestinal disease, chronic, recurrent, or severe gastroesophageal reflux disease (gerd), symptoms of dysphagia or recurrent gastrointestinal symptoms of undiagnosed etiology * history of a mast cell disorder, including mastocytosis, urticaria pigmentosa, chronic idiopathic or chronic physical urticaria beyond simple dermatographism (e.g., cold urticaria, cholinergic urticaria), and hereditary or idiopathic angioedema * any other condition that, in the opinion of the investigator, precludes participation for reasons of safety

Keywords: ar101 characterized peanut allergen cpna (characterized peanut allergen) oit (oral immunotherapy) peanut allergy peanut-allergic children desensitization arc007 characterized oral desensitization immunotherapy (codit™)
the effect of reading a pictorial storybook on children's fear, anxiety and emotional symptoms before dental treatment

Conditions: Dental Fear

Summary: the aim of this study was to determine the effect of reading a picture storybook on children's fear, anxiety and emotional symptoms before dental treatment.

in children with receiving dental treatment will be randomly divided into two groups: picture storybook group and control. interventions will be applied for only 1 times. while the illustrated storybook is read to the experimental group, no intervention will be applied to the control group. the children will be assessed before, during and after. fear, anxiety and emotional symptoms due to dental treatment procedure will be evaluated using the children's emotional indicator scale, child anxiety scale-stateness and children's fear scale.

Eligibility criteria:
inclusion criteria: * no audiovisual impairment, * being open to communication and * must be accompanied by a parent. exclusion criteria: * having special needs, * having an audio-visual impairment, * learning difficulties or mental retardation and * children whose parents do not agree to participate in the study.

Keywords: dental fear children dental anxiety emotional indicators pictorial storybook
open-label hepatic impairment study

Conditions: Hepatitis C

Summary: this study will be conducted in hepatitis c positive patients to determine whether the pharmacodynamic effects of psi-7977 or psi-352938 are similar to hcv-infected patients with normal hepatic function, which may allow inclusion of patients with cirrhosis and varying degrees of hepatic dysfunction in future clinical studies.

this study is designed per the food and drug administration (fda) guidance for patients with impaired hepatic function to assess the influence of hepatic impairment on the pk and pharmacodynamics (pd) of psi-7977 and psi-352938 this study will be conducted in hepatitis c positive patients to ascertain whether the pd effects of psi-7977 or psi-352938 are similar to hcv-infected patients with normal hepatic function, which may allow inclusion of patients with cirrhosis and varying degrees of hepatic dysfunction in future clinical studies. data from subjects who participated in the p2938-0212 study (psi-352938 mad) will be used as the control group. these subjects were documented non-cirrhotic subjects with normal hepatic function. hepatitis c virus (hcv) genotypes 1-6 will be enrolled in this study.

Eligibility criteria:
inclusion criteria: * hepatic impaired males or females of non-childbearing potential aged \> 18 years with chronic hcv-infection * naïve to all direct acting anti-viral (daa) treatments for chronic hcv infection. * documented cirrhosis exclusion criteria: * prior peg/rbv null responders. * unstable cardiac disease, recent myocardial infarction, or family history of qtc prolongation or unexplained cardiac arrest. * positive test at screening for anti-hav igm ab, hbsag, anti-hbc igm ab, or anti-human immunodeficiency virus (hiv) ab. * history of clinically significant medical condition associated with other chronic liver disease * any current signs or symptoms of severe hepatic encephalopathy * history of gastric or esophageal variceal bleeding in which varices have not been adequately treated with medication and surgical procedures * prior placement of a portosystemic shunt * history of hepatorenal, or hepatopulmonary syndrome. * active spontaneous bacterial peritonitis. * use of medications associated with qt prolongation within 28 days prior to dosing. * current hypotension * history of torsades de pointes, evidence of an active or suspected cancer, or a history of malignancy, abnormal hematological and biochemical parameters

Keywords: hepatitis c, chronic hcv hepatic impairment
ozurdex for macular edema post membrane peeling

Conditions: Epiretinal Membrane; Cellophane Maculopathy; Macular Edema; Retinal Edema

Summary: an epiretinal membrane is scar tissue on the retina that can cause blurring and distortion of vision and lead to swelling, or macular edema. despite surgery to remove the scar tissue(membrane peeling), residual swelling of the retina may continue to interfere with vision. in this study the investigators will inject an implantable steroid device into the back, fluid-filled portion of the eye. steroids have been found to decrease the swelling in the retina. ozurdex™ is an implantable steroid. once implanted, ozurdex™ is slowly dissolved by the vitreous gel that fills the eye, releasing the steroid. the steroid drug delivery system in this study, known as ozurdex™ has been fda-approved by the us food and drug administration (fda) for decreasing swelling due to another condition in the eye. this study will help to find out whether or not this device ozurdex™ is also effective for reducing the swelling of the retina in patients who have already had surgery to remove scar tissue on the retina.


Eligibility criteria:
inclusion criteria * patients with macular edema who have had previous epi-retinal membrane peeling surgery for macular edema associated with epi-retinal membranes. * all patients must have clear ocular media/lenses determined visually by the investigator in order to permit good quality stereoscopic fundus photography, fluorescein angiography and ocular coherence tomography. exclusion criteria: * best corrected visual acuity 20/50 or better in the study eye * sub-macular hemorrhage in the study eye * sub-retinal fibrosis in the study eye * macular hole in the study eye * active inflammatory disease of the study eye * choroidal neovascularization in the study eye * history of other ophthalmic disorders with the exception of cataract or previous cataract extraction in the study eye * active ocular infection in the study eye * previous subfoveal laser treatment in the study eye * previous verteporfin photodynamic therapy in the study eye * any systemic medical condition that would preclude them from undergoing surgery with monitored sedation.

Keywords: epiretinal membrane macular pucker cellophane maculopathy macular edema retinal edema ozurdex dexamethasone intravitreal implant
validation of a simplified severity score (investigator global assessment: iga) in bullous pemphigoid

Conditions: Bullous Pemphigoid

Summary: the aim of the study is to validate a global and simple score : iga (investigator global assessment) score for the evaluation of the extent and severity of the disease in patients with bullous pemphigoid


Eligibility criteria:
inclusion criteria: * consecutive adult patients aged ≥ 18 years * newly diagnosed or relapsing bp * clinical features suggestive of classic bp and suggestive histological features and deposition of igg and/ or c3 deposits on the dermal epiderma junction, and detection of circulating anti-epidermal antibodies labelling the epidermal side of salt-split skin * patient having read and understood the information letter and not opposed to participation * must be willing and able to adhere to all specified requirements, including but not limited to adherence to the follow-up visits exclusion criteria: * predominant or exclusive mucosal involvement leading to suspect the diagnosis of mucous membrane pemphigoid * pemphigoid gestationis * linear iga dermatosis (predominant or exclusive iga deposits on the dej) * skin lesions suggesting the diagnosis of epidermolysis bullosa acquisita (skin fragility, atrophy, milia)
trial of vitamin d in hiv progression, birth outcomes, and child health

Conditions: HIV

Summary: the purpose of this study to assess the effect of maternal vitamin d3 (cholecalciferol) supplementation on maternal hiv progression, small-for-gestational age infants, and infant stunting at 12 months postpartum for hiv-infected tanzanian pregnant women who are receiving highly active antiretroviral therapy (haart).

the study is a randomized, double-blind, placebo-controlled trial of maternal vitamin d3 (cholecalciferol) supplementation starting in the second trimester (12-27 weeks gestation) and continuing until 12 months postpartum among hiv-infected tanzanian pregnant women who are receiving haart. eligible individuals are randomized to receive a) a vitamin d3 regimen consisting 3,000 iu of vitamin d3 taken orally once daily starting in the second trimester and continuing until 12 months postpartum or b) a placebo regimen taken orally once daily starting in the second trimester and continuing until 12 months postpartum. mothers will be followed at study visits during pregnancy and mother/infant pairs will attend study visits from birth until trial discharge at 12 months postpartum.

Eligibility criteria:
inclusion criteria: * women aged ≥18 years old * hiv-positive * receiving haart * pregnant and of 12-27 weeks gestation (second trimester) * calcium levels in the normal physiologic range (≤2.6 mmol/l) exclusion criteria: * enrolled in any other clinical trial

Keywords: vitamin d pregnant women
juvenile recurrent respiratory papillomatosis: establishment of a french national cohort (prr : national cohort " repa ")

Conditions: Juvenile Recurrent Respiratory Papillomatosis

Summary: recurrent respiratory papillomatosis (rrp) is a rare disease. however, it is the most common benign laryngeal tumor in children. to date, no epidemiological data are available in france. the aim of this study is to establish the epidemiology of juvenile ppr.

recurrent respiratory papillomatosis (rrp) is characterized by the recurrent proliferation of multiple polypoid squamous tumors in the respiratory tract, linked to hpv infection. rrp is a rare disease. however, it is the most common benign laryngeal tumor in children. there are currently no french national epidemiological data on this disease. the data collected by the reference centers will enable us to gain a better understanding of this pathology in terms of epidemiology, risk factors, genetic susceptibility and clinical manifestations, as well as to significantly study the efficacy of the various existing therapies and preventive measures, in order to establish a clear, standardized and optimal guideline.

Eligibility criteria:
inclusion criteria: * patients with juvenile prr (disease onset before age 18) * followed in the : * the malo crmr * one of the malo network's ccmrs * one of the main correspondent centers exclusion criteria: * patients objecting to the collection of their personal data * parents of minor patients objecting to the collection of their child's personal data