Kornimate/medical-research-clean huggingface dataset:
study of ataluren (ptc124) in hemophilia a and b

Conditions: Hemophilia A; Hemophilia B

Summary: hemophilia a (ha) and hemophilia b (hb) are inherited bleeding disorders caused by mutations in the gene for factor viii (fviii) and factor ix (fix), respectively. these proteins are essential for blood clotting. the lack of fviii/fix can produce bleeding episodes that cause damage of the bone, muscles, joints, and tissues. a specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of the disease in approximately 10-30% of participants with hemophilia and results in severe manifestations. ataluren (ptc124) is an orally delivered, investigational drug that acts to overcome the effects of the premature stop codon, potentially enabling the production of functional fviii/fix. this study is a phase 2a trial evaluating the safety and efficacy of ataluren in participants with ha or hb due to a nonsense mutation. the main purpose of this study is to understand whether ataluren can safely increase fviii/fix activity levels.

in this study, participants with hemophilia a or hemophilia b due to a nonsense mutation were treated with an investigational drug called ataluren (ptc124). evaluation procedures to determine if a participant qualifies for the study was performed within 14 days prior to the start of treatment. eligible participants who elected to enroll in the study then participated in a 28-day treatment period. within the 28-day period, ataluren (ptc124) treatment was to be taken for 2 cycles of 14 days each 3 times per day with meals at a dose level of 5, 5, 10 milligrams/kilograms (mg/kg) in the first cycle and a dose level of 20, 20, 40 mg/kg in the second cycle. after the first 14-day cycle, study doses were changed to 10 mg/kg (morning), 10 mg/kg (midday), and 20 mg/kg (evening) and the doses were administered for 1 cycle only. then, there was an interval of approximately 14 days without treatment. during the study, ataluren (ptc124) efficacy, safety, and pharmacokinetics were evaluated periodically with measurement of fviii/fix activity and inhibitor levels, other blood tests, and urinalysis.

Eligibility criteria:
inclusion criteria: * ability to provide written informed consent * age ≥18 years * presence of a nonsense mutation as the sole disease-causing mutation in the fviii or fix gene * at least 20 prior treatments with fviii or fix concentrates * willingness and ability to comply with scheduled visits, drug administration plan, study restrictions, and study procedures exclusion criteria: * known hypersensitivity to any of the ingredients or excipients of the study drug * any history of prior anti-fviii/fix inhibitors * unable or unwilling to forego prophylactic fviii/fix concentrate use during the screening and on-study periods (note: participants were allowed use of fviii/fix concentrates for treatment of bleeding episodes while on study)

Keywords: hemophilia a hemophilia b factor viii factor ix fviii fix nonsense mutation premature stop codon ha hb ptc124 ataluren
the effect of yoga on cardiac sympathetic innervation evaluated by i-123 mibg

Conditions: Heart Failure, Systolic

Summary: the purpose of this study is to evaluate if yoga practice will reduce cardiac sympathetic activity and subsequently cardiac arrhythmias.

subjects with reduced ejection fraction will be randomized to 8 weeks yoga training (1 in class session and home practice) vs. no yoga. they will undergo holter monitoring, cardiac device interrogation, and i-123 mibg imaging at the beginning and end of the study.

Eligibility criteria:
inclusion criteria: * negative pregnancy test in females * ejection fraction \</= 40% assessed by echocardiogram within the last 12 months * stable dose of heart failure medications including afterload reducing medication such as ace-i, arb and hydralazine; beta blockers; digoxin and aldosterone antagonist for at least 4 months and no anticipated changes for 8 weeks. (i.e. no greater than a 50% dose change within the past month) * has an implantable pacemaker or icd * nyha ii-iv exclusion criteria: * pregnant or lactating female * females without a pregnancy test * co-administration of a positive inotrope (i.e. milrinone or dobutamine) * history of significant medical non-compliance * unwilling to adhere to the protocol * orthopedic limitation making yoga participation difficult * underlying cardiac rhythm other than sinus rhythm * recent history within 6 months prior to enrollment of unstable coronary artery disease (unstable angina, recent heart attack, recent revascularization, or decompensated heart failure) * implantation of a cardiac resynchronization therapy device in the past 3 months. * tia, cva, or major surgery in the past 3 months * iodine or adreview (123-mibg) allergy

Keywords: heart failure, systolic defibrillators, implantable pacemaker, artificial
tackling dry socket via laser therapy in smokers

Conditions: Alveolar Osteitis

Summary: this study examines the efficiency of diode laser therapy to manage dry sockets after tooth extraction.

the study compares the efficiency of diode laser therapy and using only alveogyl to accelerate the healing of dry sockets after tooth extraction in smokers. smoker patients with dry sockets were enrolled randomly into two different groups. group 1 received dual wavelength laser diode therapy, three times per week. while group 2 received only a topical application of alveogl. the treatment lasted for two consecutive weeks. the treatment efficiency was evaluated by measuring the volume of the socket using the syringe method. the pain level was measured by the visual analog scale.

Eligibility criteria:
inclusion criteria: * smokers patient exclusion criteria: * females on contraceptives. * diabetic patients * hepatitis c virus patients * patients on corticosteroids * patients with renal diseases * anemic patients

Keywords: diode laser dry socket
modified sling and conventional suture techniques in free gingival graft operations

Conditions: Gingival Recession, Localized

Summary: the aim of this study is to investigate the effects of conventional suture (cs) and modified sling suture (mss) techniques, applied in free gingival graft (fgg) surgery using gingival unit graft (gug) and conventional graft (cg) techniques, on clinical parameters and graft dimensions.

background: the aim of this study is to investigate the effects of conventional suture (cs) and modified sling suture (mss) techniques, applied in free gingival graft (fgg) surgery using gingival unit graft (gug) and conventional graft (cg) techniques, on clinical parameters and graft dimensions. methods: 52 individuals having cairo type 2 (rt2) and type 3 (rt3) gingival recessions in mandibular anterior region were divided into four groups as a) gug+mss (n=13), b) gug+cs (n=13), c) cg+mss (n=13) and d) cg+cs (n=13). keratinized gingival width (kgw), keratinized tissue thickness (kgt), relative gingival recession height (rgrh), and relative vestibule depth (rvd) measurements were recorded using a digital caliper and periodontal probe unc 15. dimensional changes (δ) of the graft surface area (gsa) was determined by imagej software. all measurements were done at baseline and repeated 1st and 3rd months.

Eligibility criteria:
inclusion criteria: * systemically healthy individuals * \<1 mm of attached gingiva on mandibular incisors * pi and gi scores \<1 * patients who had not previously undergone soft tissue surgery in the relevant area exclusion criteria: * pregnant or lactating women * patients younger than 18 years old * mandibular incisors with having restoration, probing depth ≥ 3 mm, malposition and/or endodontic problem * maxillary premolars with gingival recession in the palatal area and/or prosthetic restoration

Keywords: gingival unit graft sling suture shrinkage
severe pid with lymphoproliferation and neutropenia

Conditions: Primary Immune-Deficiency (PID) Common Variable Immune Deficiency (CVID)

Summary: the purpose of this study is to analyse the phenotype in a sub-population of adults with severe primary immunodeficiency with lymphoproliferation and neutropenia and to decipher the possible pathways involved, especially under the hypothesis of a ctla4/lrba schema


Eligibility criteria:
inclusion criteria : * \>18 years old * cvid (common variable immunodeficiency) * neutropenia * lymphoproliferation exclusion criteria : - secondary immunodeficiency

Keywords: cvid neutropenia autoimmunity lymphoproliferation
nt-probnp and new biomarkers in patients with dyspnea and/or peripheral edema

Conditions: Dyspnea; Peripheral Edema

Summary: the objectives of this study are to: * evaluate the sensitivity and specificity of nt-probnp (n-terminal prohormone brain natriuretic peptide) and new biomarker measurements for the diagnosis of heart failure in patients with acute dyspnea and/or edema. * determine the exact biomarker cut-off point(s) for the differentiation of healthy persons, patients with heart failure and patients with dyspnea of other origin. * evaluate treatment costs in patients with dyspnea under special consideration of nt-probnp measurement.

primary endpoint: * sensitivity and specificity of biomarker measurements for discrimination of heart failure from dyspnea of other origin. secondary endpoints: * determination of cut-off(s) * discrimination of cardiac and non-cardiac diseases causing to dyspnea or peripheral edema * determination of treatment costs of the initial hospital stay * evaluation of resource savings when implementing nt-probnp for determination of diagnosis. * time to diagnosis. * hospitalization rate /intensive care treatment rate between day 1 and 28 * duration of initial hospital stay * 30-day re-hospitalization rate * 30 day mortality rate and adverse event rate * 1 and 5 year mortality rates * 1 and 5 year rehospitalization rates

Eligibility criteria:
inclusion criteria: * age 18-90 years * dyspnea or/and peripheral edema * informed consent exclusion criteria: * renal insufficiency with creatinine \> 2.8mg/dl * anemia (hemoglobin \< 8.0mg/dl) * pregnancy

Keywords: natruretic peptides nt-probnp new biomarkers congestive heart failure atrial fibrillation acute myocardial infarction lung embolism
comparison of one anastomisis gastric bypass and duodeno-jejunostomy for treating sma syndrome

Conditions: Superior Mesenteric Artery Syndrome; Duodenal Obstruction; Weight Loss; Gastrointestinal Disease

Summary: the goal of this clinical trial is to find out which surgery works better to treat people with a rare condition called superior mesenteric artery syndrome (smas). this condition causes the duodenum to be squeezed between two arteries, leading to severe nausea, vomiting, and weight loss. the researchers are comparing two types of surgery: * one anastomosis gastric bypass (oagb) * duodeno-jejunostomy (dj) the main questions this study will answer are: * which surgery improves symptoms and nutritional status better? * which surgery leads to fewer complications and better quality of life? participants will: * be randomly assigned to one of the two surgeries * be followed for 12 months after the operation * complete follow-up visits and nutritional assessments * answer questions about their symptoms and overall well-being

superior mesenteric artery syndrome (smas) is a rare but serious condition where part of the small intestine (the duodenum) gets compressed between major blood vessels. this can cause severe digestive symptoms and weight loss. surgery is often needed when other treatments do not work. this prospective randomized controlled trial compares two surgical options to relieve the compression: one anastomosis gastric bypass (oagb) and duodeno-jejunostomy (dj). both surgeries aim to improve food passage and relieve symptoms, but they work differently and have different effects on digestion and nutrition. participants will be randomly assigned to one of the two surgical procedures. the study will collect data before and after surgery on symptoms, nutritional status, complications, and quality of life. follow-up will continue for 12 months. the goal is to help surgeons and patients choose the most effective and safest surgical treatment for smas.

Eligibility criteria:
inclusion criteria: * clinical diagnosis of superior mesenteric artery syndrome (smas) based on symptoms and radiologic findings * no prior gastric or intestinal surgery * willingness and ability to participate in follow-up for 12 months exclusion criteria: * presence of other gastrointestinal pathologies mimicking smas * severe comorbidities contraindicating surgery (e.g., advanced cardiac or pulmonary disease) * history of previous bariatric or upper gi surgery * refusal or inability to provide informed consent

Keywords: superior mesenteric artery syndrome smas oagb one anastomisis gastric bypass duodenojejunostomy gastrc bypass intestinal obstruction malnutrition minimally invasive surgery quality of life randomized controlled trial
opioid free anesthesia and major spine surgery

Conditions: Opioid Use; Postoperative Pain; Spine Disease

Summary: this study compares the intraoperative opioid free anesthesia approach in multilevel spine posterior instrumentation surgery to a conventional opioid-based anesthesia. half of participants will receive opioid free anesthesia with dexmedetomidine, lidocaine and ketamine while the other half will receive opioid based anesthesia with fentanyl, remi-fentanyl and ketamine

opioid analgesics are commonly used in clinical practice for per and postoperative pain treatment. however their use is associated with a lot of undesirable effects. from this comes the idea of using opioid free anesthesia intra-operatively in an attempt to decrease opioids postoperative use and accordingly, their associated side effects. intravenous (iv) lidocaine has been found to have analgesic, antihyperalgesic, and anti-inflammatory properties. it was found to significantly improve postoperative pain, when its intraoperative use was compared with placebo . dexmedetomidine is a selective alpha-two adrenergic receptor agonist that has antinociceptive, analgesic, opioid sparing and sedative properties. similar to lidocaine, dexmedetomidine was shown to lower postoperative pain, opioid consumption and accordingly, opioid related side effects.

Eligibility criteria:
inclusion criteria: * age group: 18-80 years old * american society of anesthesiologists (asa )class i, ii and iii * elective spine posterior instrumentation surgery of at least two levels. exclusion criteria: * renal, hepatic or cardiac insufficiency. * alcohol or drug abuse. * psychiatric disease. * allergy or contraindication to any of the study drugs. * inability to comprehend pain assessment or inability to use a patient controlled analgesia (pca) device.

Keywords: lidocaine dexmedetomidine opioid-free
study of ngr-htnf in combination with cisplatin in solid tumor

Conditions: Solid Tumors

Summary: the main objective of the trial is to document the safety of the combination (escalation doses of ngr-htnf, from 0.2 mcg/sqm to 1.6 mcg/sqm , with a fixed dose of cisplatin, 80 mg/sqm). safety will be established by clinical and laboratory assessment according to national cancer institute common toxicity criteria (nci-ctc ).

this is a phase ib, open-label, non-randomized, dose-escalation study that will be conducted in sequential cohorts of patients. three patients per each cohort are planned. patients, with advanced or metastatic solid tumor not amenable of standard therapy will be enrolled.

Eligibility criteria:
inclusion criteria: * patients ≥18 years with advanced or metastatic solid tumor not amenable to any clinical improvement by current standard treatments and suitable for a treatment with cisplatin * life expectancy more than 3 months * ecog performance status 0-1 * absence of any conditions in which hypervolemia and its consequences (e.g. increased stroke volume, elevated blood pressure) may represent a risk for the patient * adequate baseline bone marrow, hepatic and renal function, defined as follows: * neutrophils \> 1.5 x 10\^9/l and platelets \> 100 x 10\^9/l * bilirubin \< 1.5 x uln * ast and/or alt \< 2.5 x uln in absence of liver metastasis * ast and/or alt \< 5 x uln in presence of liver metastasis * serum creatinine \< 1.5 x uln * creatinine clearance (estimated according to cockcroft-gault formula) ≥ 50 ml/min * patients may have had prior therapy providing the following conditions are met: * chemotherapy, radiation therapy, hormonal therapy or immunotherapy: wash-out period of 28 days * corticosteroid therapy wash out period of 14 days * surgery: wash-out period of 14 days * patients must give written informed consent to participate in the study exclusion criteria: * previous signs of severe toxicity platinum related * patients must not receive any other investigational agents while on study * new york heart association class iii or iv cardiac disease * unstable angina * patients with myocardial infarction within the last six (6) months * patient with significant peripheral vascular disease * clinical signs of cns involvement * patients with active or uncontrolled systemic disease/infections or with serious illness or medical conditions, which is incompatible with the protocol * known hypersensitivity/allergic reaction to human albumin preparations or to any of the excipients * any psychological, familial, sociological or geographical condition potentially hampering compliance with the study protocol * pregnancy or lactation. patients - both males and females - with reproductive potential (i.e. menopausal for less than 1-year and not surgically sterilized) must practice effective contraceptive measures throughout the study. women of childbearing potential must provide a negative pregnancy test (serum or urine) within 14 days prior to registration

Keywords: ngr-htnf cisplatin solid tumors
cpap or bipap for motion mitigation during radiotherapy

Conditions: Radiotherapy Side Effect; NSCLC; Esophageal Cancer; Malignant Lymphoma

Summary: when using highly conformal radiotherapy techniques, such as proton therapy, a controlled breathing pattern and a minimal breathing amplitude could greatly benefit the treatment of mobile tumors. this reduction in tumor motion may be achieved with the use of a ventilator that is able to regulate and modulate the breathing pattern. cpap provides a constant level of positive airway pressure. compared to spontaneous breathing, the use of cpap increased lung volume and can result in a significant decrease in tumor movement and a significant decrease in both mean lung and mean heart radiation dose. these results were found in patients treated for limited stage disease, it is not clear if this approach is feasible for patients with more advanced stage of disease that undergo radiotherapy with curative intent. with bilevel positive airway pressure (bipap), tidal volume excursions are determined by the pressure difference between the set inspiratory positive airway pressure (ipap) and the set expiratory positive airway pressure (epap). this mode of ventilation increases lung volume comparable to cpap, but also to control tidal volumes and breathing frequency. however, bipap has never been studied in the setting of motion mitigation during radiotherapy and bipap might be more difficult to adjust to for patients compared to cpap. therefore, the current study is proposed to evaluate whether or not cpap or bipap is of benefit in patients that undergo radiotherapy for larger intra-thoracic tumor volumes.

the study will follow a 2-step approach. first (phase 1, n=10), the feasibility of short-term use of cpap and bipap and its effects on (regional) lung volumes, tidal volumes and breathing frequency will be investigated in patients with intra-thoracic tumors who are planned for radiotherapy. this is necessary to select the best setting of cpap or bipap with which minimal tumor motion is expected (and that is feasible for the patient). in the second phase (n=21), the particular setting found in fase 1 is investigated: weekly repeated radiotherapy planning (4d) ct scans with and without cpap/bipap will be aquired to evaluate the influence on tumor motion and dose to the target and organs at risk. radiotherapy will be deliverd according to local protocol without the use of cpap/bipap.

Eligibility criteria:
inclusion criteria: * age ≥18 years * stage iii/iv (n)sclc, esophageal cancer or malignant lymphoma that will be treated with curative intent * who 0-2. * written informed consent exclusion criteria: * facial deformations so that facial mask is impossible to fit * noncompliance with any of the inclusion criteria. * planned for radiotherapy with fraction dose ≥3 gy. * severe heart failure (lvef\<30%)

Keywords: bipap cpap mechanical ventilation
glaucoma treatment using focused ultrasound in open angle glaucoma patients

Conditions: Glaucoma

Summary: the purpose of the this study is to collect safety and efficacy data on focused ultrasound treatment (ucp procedure) in open angle glaucoma patients without previous glaucoma surgery.


Eligibility criteria:
inclusion criteria: * open angle glaucoma patient (primary open angle glaucoma (poag) including pigmentary glaucoma (pg) and pseudoexfoliative glaucoma (pxf)) * any patients without previous conventional glaucoma surgery failure (trabeculectomy, deep sclerectomy, ahmed valve, drainage device implantation, cyclo cryotherapy, diode laser cyclo-destruction) * subjects where the iop is not adequately controlled with glaucoma medication, with iop ≥ 21 mm hg and \<30 mmhg * no previous intraocular surgery or laser treatment during the 90 days before hifu day * age \> 18 years and \< 90 years * patient able and willing to complete postoperative follow-up requirements exclusion criteria: * patient who has been diagnosed for normal tension glaucoma * ocular or retrobulbar tumor * ocular infection within 14 days prior to the hifu procedure * ocular disease other than glaucoma that may affect assessment of visual acuity and/or iop (choroidal hemorrhage or detachment, lens subluxation, thyroid ophthalmopathy, proliferative diabetic retinopathy, clinically significant macular edema...)

Keywords: glaucoma ultrasound ciliary body
prospective trial comparing oxybutynin and tamsulosin for stent pain in the pediatric population

Conditions: Disorder of Urinary Stent

Summary: the primary objective of this study is to evaluate the efficacy and safety tamsulosin in reducing stent discomfort in the pediatric population in the peri-operative period. the secondary objective is to compare the efficacy of tamsulosin with that of oxybutynin in reducing stent discomfort.


Eligibility criteria:
inclusion criteria: * patients aged 4-18 years will be enrolled in this study. we will include all patients who will have a ureteral stent placement after their procedure. exclusion criteria: * patients with developmental delay or unable to verbalize their pain level will be excluded.