Kornimate/medical-research-clean huggingface dataset:
study to understand the genetic risk of developing an immune response after blood transfusions among individuals with sickle cell disease

Conditions: Sickle Cell Disease

Summary: the purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. this response is called an alloimmune response. we strongly believe that when someone has an alloimmune response, it is caused by changes in their genes. we plan to compare changes in the genes of individuals that develop red blood cell alloimmunization after blood transfusions with those that do not develop alloimmunization. this may help us to create more targeted therapeutic interventions, which may improve the health of alloimmune responders.

study description: this study seeks to fine-map risk variants associated with increased susceptibility to developing red blood cell alloantibodies in patients with sickle cell disease (scd), with the goal of characterizing the molecular basis of the alloimmunization response. this will allow for improved clinical management for individuals susceptible to alloimmunization responses. objectives: primary objective: elucidate the role of previously identified risk loci in the development of alloantibodies among individuals with scd. secondary objective: validate and characterize additional, novel alloimmunization-related candidate loci. endpoints: primary endpoint: completion of analysis of previously identified risk loci to determine the relationship between genome structure and expression. secondary endpoint: no additional candidate loci from concurrent discovery studies to evaluate.

Eligibility criteria:
* inclusion criteria to be eligible to participate in this study, an individual must meet all of the following criteria: 1. individual (\> 2 years of age) with confirmed scd diagnosis who meets at least one of the following conditions: 1. history of greater than ten administered transfusions or 20 transfusion units (where known) 2. history of one or more antibody screens 3. known candidate variant genotype exclusion criteria an individual who meets any the following criteria will be excluded from participation in this study: 1. impaired decision-making capability, with or without a legally authorized representative 2. history of transplant (e.g., organ, bone marrow, stem cell) 3. taking immunosuppressive medications at time of enrollment 4. confirmed pregnancy

Keywords: sickle cell disease transfusion alloimmunization
use of rfxiii in treatment of congenital fxiii deficiency, a prospective multi-centre observational study

Conditions: Congenital Bleeding Disorder; Congenital FXIII Deficiency

Summary: this study is conducted globally. the aim of this observational study is to investigate the incidence of specific adverse drug reactions associated with the use of recombinant factor xiii (novothirteen®) in patients with congenital fxiii a-subunit deficiency (congenital fxiii deficiency), comprising fxiii antibodies, allergic reactions, embolic and thrombotic events and lack of therapeutic effect. the study will aim at observing all patients exposed to novothirteen® in the eu, and additional patients from selected non-eu countries. recombinant fxiii (rfxiii) is registered in eu and switzerland as novothirteen® and in canada as tretten®.


Eligibility criteria:
inclusion criteria: * informed consent obtained before any study-related activities. (study-related activities are any procedure related to recording of data according to the protocol) * able and willing to provide signed informed consent (or patient's legally acceptable representative (lar) consent, if applicable), as required by local ethics committee, governmental or regulatory authorities * congenital fxiii a-subunit deficiency * actual or planned exposure to rfxiii
aav gene therapy study for subjects with pku

Conditions: Phenylketonuria (PKU)

Summary: this is a phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of bmn 307 in adult pku subjects with pah deficiency. participants will receive a single administration of bmn 307 and will be followed for safety and efficacy.


Eligibility criteria:
inclusion criteria: * male and female subjects with diagnosis of pku which is a condition characterized by pah deficiency * ability and willingness to maintain dietary protein intake consistent with baseline intake * willingness to abstain from hepatotoxic substances post-bmn 307 administration * willingness and capable per investigator opinion to comply with study procedures and requirements * willingness to use effective methods of contraception * plasma phe levels \> 600 µmol/l exclusion criteria: * subjects with primary bh4 deficiency or other forms of bh4 metabolism deficiency * clinically significant liver dysfunction or disease * prior treatment with gene therapy * any condition that, in the opinion of the investigator or sponsor, would prevent the subject from fully complying with the requirements of the study * history of malignancy

Keywords: pku pah deficiency
safety, tolerability and effectiveness of natalizumab in adolescents with active crohn's disease

Conditions: Crohn's Disease

Summary: the purpose of this study is to determine the safety and tolerability of natalizumab in adolescents (ages 12-17) diagnosed with moderately to severely active crohn's disease (cd). it is thought that natalizumab may stop the movement of certain cells, known as white blood cells, into bowel tissue. these cells are thought to cause damage in the bowel leading to the symptoms of crohn's disease. patients who complete this study may be eligible for long-term natalizumab therapy via extension protocol eln100226-352.


Eligibility criteria:
male and female patients, 12-17 years of age, who have at least a six-month history of crohn's disease and who are currently experiencing moderately to severely active crohn's disease. females must not be breastfeeding or pregnant, and must not become pregnant during the study.
photodynamic therapy clinical trial for cutaneous aids-related kaposi's sarcoma study summary.

Conditions: Sarcoma, Kaposi; HIV Infections

Summary: to determine the objective tumor response and remission rate of aids-related kaposi's sarcomas (ks) following a single dose of tin ethyl etiopurpurin (snet2) followed by photodynamic therapy (pdt). to determine the systemic and local toxicity, and morbidity safety profile of snet2-pdt.

all patients receive a single dose of snet2 and are randomized to receive either pdt light treatment or no light treatment (control group). patients are assessed prior to and at 4, 12 and 24 weeks following treatment, and are followed for 7 months.

Eligibility criteria:
inclusion criteria patients must have: * documentation of at least one biopsy-confirmed ks lesion. * a minimum of 4 and no more than 36 ks lesions. * all eligible lesions must be bidimensionally measurable, treatable by surface (non-contact) light illumination, and \<= 40 mm in diameter of the longest bidimensional axis. * actg disease state t(0) l(0) or (1) s(0) or (1). * life expectancy greater than 6 months. exclusion criteria co-existing condition: patients with the following symptoms or conditions are excluded: * active opportunistic infection or condition except thrush or herpes simplex virus infections. * advanced ks tumor stages including the presence of tumor associated edema or ulceration, extensive oral ks, or ks in other non-nodal viscera. * hematopoietic dysfunction. * coagulation dysfunction. * hepatic dysfunction. * renal dysfunction. * cardiovascular dysfunction - presence of significant coronary artery disease requiring current treatment or myocardial infarction. * pulmonary dysfunction. * sepsis. * known disorder of lipoprotein metabolism or clearance. patients with the following prior conditions are excluded: * history of allergic or hypersensitivity reactions to light, egg proteins or egg yolks, history of porphyria, systemic lupus erythematosus or xeroderma pigmentosum. excluded within 7 days of therapy: * hematopoietic dysfunction. * coagulation dysfunction. * hepatic dysfunction. * renal dysfunction. excluded within 3 months of therapy: * pulmonary dysfunction. excluded within 6 months of therapy: * myocardial infarction. prior medication: excluded: * intralesional chemotherapy within the past 12 weeks. * systemic chemotherapy or investigational drugs within the past 4 weeks. prior treatment: excluded within 3 months prior to therapy: * local cryotherapy or surgery to study lesions. * systemic or topical photodynamic therapy agents.

Keywords: skin neoplasms sarcoma, kaposi acquired immunodeficiency syndrome photochemotherapy tin etiopurpurin radiation-sensitizing agents
does a soft drink mixture improve tolerance of activated charcoal in the adult poisoned patient without affecting efficacy

Conditions: Overdose

Summary: activated charcoal (ac) is an established, effective means of gastrointestinal decontamination. providers give it to patients who have ingested something that is thought to be potentially poisonous to prevent it from being absorbed. however, one limitation to its use is palatability of the ac for the patient, potentially limiting how much, if any, is taken. other studies have suggested that mixing ac with various substances improves the rating on various scales (taste, etc). an important question is whether mixing the ac with other substance effects the ability of the ac to bind to xenobiotic in the gut. this small study investigates whether mixed cola with charcoal affected its ability to prevent the absorption of acetaminophen. it also performs a survery to see if participants preferred the ac-cola mixture. the investigators hypothesize that the ac will be equally as effective with cola as without. the investigators also hypothesize that participants will prefer the ac-cola mixture.


Eligibility criteria:
inclusion criteria: * no self-reported history of any hepatic, gastrointestinal, or renal disease * no self-reported history of alcohol or substance use disorder * no daily prescribed medications * weight between 60-93 kg exclusion criteria: * pregnant, * imprisoned * allergy or intolerance to acetaminophen or gluten
the efficacy of butylphthalide soft capsules in patents with vcind

Conditions: Vascular Cognitive Impairment no Dementia

Summary: vascular cognitive impairment no dementia (vcind) is very common among the aged and tends to progress to dementia, but there have been no proper large-scale intervention trials dedicated to it. vcind caused by subcortical ischemic small vessel disease (hereinafter, subcortical vcind) represents a relatively homogeneous disease process and is a suitable target for therapeutic trials investigating vcind. preclinical trials showed that butylphthalide soft capsules is effective for cognitive impairment of vascular origin. in this randomized, double-blind, placebo-controlled trial, the investigators apply fmri study the effects of butylphthalide soft capsules in patients with vcind.


Eligibility criteria:
inclusion criteria: * literate han chinese aged from 40 to 65 years * mmse ≥24 * normal or slightly impaired activities of daily living * the mri entry criteria are as follows: * subcortical small infarcts (3-20 mm in diameter),or one or more strategically located subcortical small infarcts in the caudate nucleus globus pallidus, or thalamus * absence of cortical and watershed infarcts, hemorrhages, hydrocephalus, and wmls with specific causes (e.g., multiple sclerosis); and * no hippocampal or entorhinal cortex atrophy (scored 0 according to medial temporal lobe atrophy scale of scheltens) exclusion criteria: * patients with diabetes mellitus * disorders other than subcortical vcind that may affect cognition; the score of hamilton depression scale more than 17 or schizophrenia * clinically significant gastrointestinal, renal, hepatic, respiratory, infectious, endocrine, or cardiovascular system disease; cancer; alcoholism; drug addiction; use of medications that may affect cognitive functioning * known hypersensitivity to celery * inability to undergo a brain mri

Keywords: butylphthalide soft capsules vcind fmri
trivalent, live, cold adapted influenza vaccine (caiv-t) against inactivated influenza vaccine (tiv) in children 6-59 months of age

Conditions: Influenza

Summary: the primary objective of this study is to estimate the relative efficacy and assess the safety of caiv-t compared to tiv.


Eligibility criteria:
inclusion criteria: * age 6-59 months of age (not yet reached their 5th birthday) * parent or guardian available by telephone * available for illness visits at clinic or at home during the influenza surveillance period * written informed consent (and health insurance portability and accountability act \[hipaa\] authorization for u.s. participants) obtained from the participant's parent or legal guardian and * ability of the parent/guardian to understand and comply with the requirements of the protocol exclusion criteria: * history of hypersensitivity to any component of caiv-t or inactivated influenza vaccine, including egg or egg protein * history of hypersensitivity to gentamicin * any known immunosuppressive condition or immune deficiency disease (including hiv infection), or ongoing receipt of any immunosuppressive therapy * history of guillain-barre syndrome * medically-diagnosed wheezing, bronchodilator use, or steroid use (systemic or inhaled) within the previous 42 days by parent report or chart review (e.g., children with recent persistent asthma are excluded), or history of severe asthma * acute febrile (\>100.0 degrees f or \>37.8 degrees c oral or equivalent) illness or acute respiratory illness, including sore throat, within three days prior to enrollment * receipt of an investigational product within 30 days prior to enrollment or expected receipt during this study * use of aspirin or salicylate-containing products 30 days prior to enrollment or expected receipt during this study * use of anti-influenza medications (including amantadine, rimantadine, oseltamivir, and zanamivir) within 14 days prior to enrollment or expected receipt during this study * receipt of any blood product within 90 days prior to vaccination or expected receipt during this study * administration of any live virus vaccine within 30 days prior to enrollment, or if receipt of another live virus vaccine is expected within 30 days of any study vaccination * administration of any inactivated vaccine within 14 days prior to enrollment or if receipt of another inactivated vaccine is expected within 14 days prior to enrollment or if receipt of another inactivated vaccine is expected within 14 days of any study vaccination * close contact who is severely immunocompromised (e.g., transplant recipient) * family member or household contact who is an employee of the research center or otherwise involved with the conduct of the study and * any condition that, in the opinion of the investigator, might interfere with the interpretation or evaluation of the vaccines.
effect of dairy consumption on glycemic control, body weight and cardiovascular risk in patients with type 2 diabetes

Conditions: Type 2 Diabetes

Summary: this is a randomized prospective clinical study in patients with type 2 diabetes to evaluate the effect of dairy products with full or low fat on glycemic control and cardio-metabolic risk factors in comparison to a regular diet.

this is a randomized, controlled clinical study in which 108 subjects with type 2 diabetes will be randomized into 3 different groups: full fat dairy group (group a): will receive nutritional counseling aiming to maintain body weight from a registered dietician and will be instructed to include at least ≥3 servings of full fat dairy products into their diet low fat dairy group (group b): will receive nutritional counseling aiming to maintain body weight from a registered dietician and will be instructed to include at least ≥3 servings of low fat dairy products into their diet control group (group c): will receive nutritional counseling aiming to maintain body weight from a registered dietician and will not be asked to include a specified amount or type of dairy products subjects in groups a and b will be educated about different dairy products' serving sizes and fat content. subjects in all 3 groups will be asked to record their daily intake of dairy products in a dedicated log book. subjects randomized to groups a and b will be asked to only use only dairy products with either the full fat or and low (≤1%) fat dairy (≤1% fat content) content respectively. purchase of the dairy products will be the responsibility of study subjects for which a subsidy of $500 (about $20/week for 24 weeks) will be provided for each subject. all study participants will also be asked to record their food intake for 3 days before each study visit in a 3-day food log.

Eligibility criteria:
inclusion criteria: * having type 2 diabetes for at least three months prior to screening and treated with any antihyperglycemic medication * a1c ≥7 % * consuming \<3 servings of dairy products per day * on stable dose of diabetes, blood pressure or cholesterol medications for 3 months * body weight is within 10% of current weight over the last 6 months before starting the study exclusion criteria: * pregnancy or lactation * lactose intolerance * allergy to milk or any of its components * use of orlistat * history of pancreatitis * history of gastric bypass surgery or sleeve gastrectomy * active malignancy * history of recent cardiovascular event * having a heart pacemaker * enrollment in other studies that may affect study outcomes
the tisa trial - senegal

Conditions: Severe Acute Malnutrition

Summary: this study evaluates the addition of a simple, scalable "wash kit", including household water treatment products, a safe water storage container, and hygiene promotion, to the standard national protocol for outpatient treatment of uncomplicated severe acute malnutrition among children aged 6-59 months of age in northern senegal.

severe acute malnutrition (sam) is defined as a weight-for-height z-score (whz) below -3 of the world health organisation growth standards. it is a short-term acute condition with a high case-fatality rate that increases both the susceptibility of children to infection and the risk of mortality. the global prevalence of sam in children is estimated to be over 17 million with most of those affected living in sub-saharan africa and asia. community-based management of acute malnutrition (cmam) has significantly changed standard practice for the treatment of sam. uncomplicated sam cases are now predominantly managed on an outpatient basis and action contre la faim (acf) supports outpatient treatment programs (otp) across the globe, particularly in africa and asia. this innovation has increased coverage, and reduced the cost of treatment for both the health system and individuals. however, under cmam, the treatment context has moved from a more controlled health facility setting to the generally less controlled household setting where the risk of infection due to environmental hazards, such as contaminated water, may be higher. the tisa trial described here will evaluate the effectiveness of combining a simple, scalable "wash kit" with otp on the recovery rate among uncomplicated sam cases aged 6-59 compared to the national standard otp. the wash kit includes a plastic container, a supply of aquatabs® (effervescent chlorine tablets), and training in their use and associated hygiene practices. the study is a cluster randomized controlled trial (crct), with health centres constituting the clusters. clusters are randomly allocated to either a control group receiving the standard otp or an intervention group receiving otp plus the wash kit. the primary outcome of the trial is the recovery rate and the secondary outcomes are weight gain, referral rate, longitudinal prevalence of diarrhoea, prevalence of enteric pathogen detection and all-cause mortality.

Eligibility criteria:
inclusion criteria: * weight-for-height z-score \<-3; or * brachial perimeter (mid-upper arm circumference) \<115; or * bilateral oedema exclusion criteria: * not able and/or willing to participate for up to 8-weeks * clinical complications requiring referral and/or inpatient treatment
safety & tolerability of cinacalcet in pediatric patients with chronic kidney disease and secondary hyperparathyroidism

Conditions: Chronic Kidney Disease; Hyperparathyroidism, Secondary

Summary: the primary objective was to characterize corrected serum calcium levels on treatment with cinacalcet in pediatric patients with secondary hyperparathyroidism (hpt).

this is a multicenter, 26-week, single-arm, open-label, safety study. participants were to remain on study for 26 weeks or until time of kidney transplantation, whichever came first. the study and enrollment was placed on partial clinical hold in february 2013 which resulted in changes to the protocol. the study was restarted in april 2014 following these changes. participants who completed the 26-week study or were on study when the study was closed in june 2016 were eligible to participate in an open-label extension study (study 20140159; nct02341417).

Eligibility criteria:
inclusion criteria: * subjects between the ages of 28 days to \< 6 years of age at enrollment (czech republic minimum age is ≥ 2 years of age at enrollment) * screening plasma ipth level \> 300 pg/ml (31.8 pmol/l) from the central laboratory, and not have received any cinacalcet therapy for at least 30 days prior to start of dosing * screening corrected calcium from the central laboratory: * ≥ 9.4 mg/dl (2.35 mmol/l) if age 28 days to \< 2 years * ≥ 8.8 (2.2 mmol/l) if age ≥ 2 to \< 6 years * serum phosphorus from the central laboratory: * ≥ 5.0 mg/dl (1.25 mmol/l) if age 28 days to \< 1 year * ≥ 4.5 mg/dl (1.13 mmol/l) if age ≥ 1 to \< 6 years * shpt not due to vitamin d deficiency, per investigator assessment * dry weight ≥ 7 kg at the time of screening exclusion criterion: * history of congenital long qt syndrome, second or third degree heart block, ventricular tachyarrhythmias or other conditions associated with prolonged qt interval * corrected qt interval (qtc) \> 500 ms, using bazett's formula * qtc ≥ 450 to ≤ 500 ms, using bazett's formula, unless written permission to enroll is provided by the investigator after consultation with a pediatric cardiologist * use of grapefruit juice, herbal medications, or potent cyp 3a4 inhibitors (e.g., erythromycin, clarithromycin, ketoconazole, itraconazole) * use of concomitant medications that may prolong the qtc interval (e.g., ondansetron, albuterol)

Keywords: dialysis sensipar mimpara hemodialysis peritoneal dialysis renal parathyroid hormone pediatric