Kornimate/medical-research-clean huggingface dataset:
efficacy of dalfampridine on upper extremity function in patients with ms

Conditions: Multiple Sclerosis

Summary: this study evaluates the effects of sustained-release oral dalfampridine in the treatment of upper limb deficits in people with multiple sclerosis (ms). in this double-blind randomized pilot study half of participants will dalfampridine, while the other half will receive a placebo.

dalfampridine (ampyra), a drug with a mechanism for symptomatic management of ms among blocks potassium channels on demyelinated neurons, allows normal electrical conduction. dalfampridine has recently been found to be associated with improvements in visual function, strength, ambulation, fatigue, and endurance in individuals with ms. although this medication has a widespread effect, its influence on upper extremity function has never been investigated in a double blind randomized case control study. following the fact that during the disease course, approximately 3 out of 4 multiple sclerosis patients encounter upper limb dysfunction the primary objective of this study will be to investigate the efficacy of sustained-release oral dalfampridine on upper extremity function in patients with ms.

Eligibility criteria:
inclusion criteria: 1. the patient must have the ability to understand the purpose and risks of the study provide a signed and dated informed consent and authorize confidential health information to be examined in accordance with national and local subject privacy regulations. 2. the patient must have been diagnosed with clinically definite ms, at the time of informed consent. 3. the patient must be between 18-70 years of age, inclusive, at the time of informed consent. 4. the patient must have scored between 50 and 90 on the upper limb motricity index test, at the time of informed consent. this test evaluates strength during three essential movements (pinch grasp, elbow flexion and shoulder abduction). the selected score range criteria determine patients who suffer a moderate decline in function abilities of the upper limb. exclusion criteria: 1. onset of multiple sclerosis exacerbation within 60 days of screening. 2. history of seizures or evidence of epileptic form activity found on a screened electroencephalogram. 3. changes in concomitant medications to avoid related changes in multiple sclerosis symptoms during the study.
osteoradionecrosis rate in patients undergoing radiotherapy for head and neck cancer treatment.

Conditions: Head and Neck Cancer; Osteoradionecrosis; Radiotherapy Side Effect; Tooth Avulsion

Summary: objectives high-dose radiotherapy (rt) for head and neck cancer has significant adverse effects on maxillofacial tissues, among which osteoradionecrosis (orn) is the most severe and potentially life-threatening. although tooth extractions seem to be the main risk factor, few perspective studies evaluated protocols to minimize the orn risk due to extractions. the aim of this study is to evaluate incidence and risk factors of orn in a cohort of patients receiving tooth extractions before rt and evaluate an algorithm about extraction decision. methods one-hundred ten patients were consecutively recruited in this study: impacted third molars with radiographic sign of pericoronitis, teeth with periapical lesions, unrestorable teeth, periodontally compromised teeth (pocket probing depth \>5 mm, clinical attachment loss \> 8 mm, grade 2 tooth mobility, ii grade furcation involvement) were extracted under antibiotic prophylaxis. a 15-days interval between the last tooth extraction and the beginning of rt was recommended. patients were visited at 15 days, 1, 3 and 6 months after the beginning of rt. data of patients with a minimum of 6 months follow-up are presented in this report. orn was defined as irradiated exposed necrotic bone, without healing for 3 months, in absence of cancer recurrence. the protocol was approved by the ethic committee of catholic university - fondazione policlinico gemelli (prot. ohhn-1, id-2132).


Eligibility criteria:
inclusion criteria: * patients willing to sign the informed consent form * patients with diagnosis of hnc, with specific prescription of local radiotherapy, both for curative and palliative purpose, with or without chemotherapic treatment. * patient with diagnosis of hnc, with specific prescription of local radiotherapy as an adjuvant to surgical resection, with or without chemotherapic treatment. exclusion criteria: * patients participating to other clinical studies * patients who refuse to participate in the study * patients unable to attend the ambulatory visits scheduled by the protocol
pharmacokinetics of anti-epileptic drugs in obese children

Conditions: Epilepsy; Obese

Summary: the study is a prospective, multi-center, open-label clinical trial. study's purpose is to characterize the pharmacokinetics and safety of four oral anti-epileptics drugs (levetiracetam, valproic acid \[divalproex sodium er or immediate release formulation if inadequate enrollment}, topiramate, and oxcarbazepine) in a non-randomized sample of obese children and adolescents. the study's duration will be up to eleven days (up to seven days of screening and four days of pharmacokinetic sampling). eligible participants ages 2 to 18 years will be identified through outpatient clinic schedules and inpatient admissions at each clinic site. participants receiving at least one of the study drugs per local standard of care will have pharmacokinetic concentrations in plasma drawn according to the specific dosing schedule for each drug. other study measures include demographics, bmi, waist/hip ratio, medical history, concomitant medication history, documentation of study drug oral intake, adverse effects, and physical examination. the sample size will include 24 participants for each anti-epileptic drug (total 96).


Eligibility criteria:
inclusion criteria: * 2 years to \< 18 years at the time of enrollment * bmi ≥ 95th percentile for age and sex, based on cdc recommendations * informed consent/hipaa from the parent/legal guardian and assent (as applicable) * receiving ≥ 1 of the study drugs per local standard of care exclusion criteria: * known pregnancy as determined via interview or test results, if available

Keywords: epilepsy obese
shr-2004 for the prevention of venous thromboembolism in patients undergoing total knee arthroplasty

Conditions: Prevention of Venous Thromboembolism After Knee Arthroplasty

Summary: the purpose of the study is to evaluate the efficacy and safety of shr-2004 in preventing venous thromboembolism after elective unilateral total knee arthroplasty


Eligibility criteria:
inclusion criteria: 1. understand the research procedures and methods, volunteer to participate in the study, and sign the informed consent 2. scheduled to undergo elective unilateral total knee arthroplayts (tka) 3. male or female(≥ 40 years old and \< 80 years old) exclusion criteria: 1. unable to receive ct angiography of both lower limbs; 2. malignant tumor within one year of the screening ; 3. history of venous thromboembolism; 4. myocardial infarction, transient ischemic attack or ischemic stroke occurred within 6 months of the screening; 5. any medical history that may increase the risk of bleeding or any conditions that the investigator considers to increase the risk of bleeding 6. any of the laboratory test indicators meets the following criteria: ①estimated glomerular filtration rate \< 60 ml/min/1.73m2 ; ②alanine aminotransferase (alt) or aspartate aminotransferase (ast) \>3 times the upper limit of normal value (uln); ③total bilirubin was \> 2 times, etc 7. history of drug abuse; 8. pregnant or lactating women
lyso-gb1 as a long-term prognostic biomarker in gaucher disease

Conditions: Lysosomal Storage Diseases; Gaucher Disease; Sphingolipidoses

Summary: international, multicenter, epidemiological study to demonstrate the correlation and predictive value of lyso-gb1 concentration with the clinical severity of naïve, initially non-ert/srt gaucher disease type 1 and during the study ert/srt-newly started gaucher type 1 patients and to correlate lyso-gb1 concentration with the clinical improvement of ert or srt treated gaucher type 1 and the clinical course of non-treated patients based on gd-ds3

gaucher disease is an autosomal recessive inherited lysosomal storage disorder. the disease is caused by the hereditary deficiency of the glucocerebrosidase, a lysosomal enzyme that breaks down glucocerebroside into glucose and ceramide. to date a definitive diagnosis of gaucher's disease can only be made applying biochemical testing measuring the reduced enzymatic activity of the beta-glucosidase together with genetic confirmation. since numerous different mutations may be the cause of a particular lysosomal storage disease the sequencing of the entire beta-glucosidase gene is applied in gaucher's disease in order to confirm the genetic diagnosis. the use of primary storage molecules as biomarker was assessed for glucosylceramide (gb1) in plasma of gaucher's disease patients and compared to the level of gb1 in healthy individuals. in order to establish a sensitive and specific biomarker for gd, we compared mass spectra of the plasma of healthy controls and gd patients using hplc and tandem mass spectrometry. mass spectra that differed most between patients and controls were analysed in more detail. the resulting biomarker, which was patented in june 2011 (pct/ep2012/002409), was lyso - gb1. we identified this compound as a reliable, sensitive and specific biomarker for gd in a cohort of gd patients. furthermore, in a pilot study we evaluated whether lyso-gb1 is related to the specific genotypes and is reliable for long-term monitoring of the efficiency of therapy. the aim this study is therefore to investigate lyso-gb1 as a long-term prognostic marker in naïve, non-ert/srt gd type 1 patients by monitoring over the course of 36 months.

Eligibility criteria:
inclusion criteria: * male or female patients aged 6 months or older * patients with genetically confirmed diagnosis of gaucher disease type 1 * no prior treatment with enzyme replacement therapy or substrate reduction therapy ro no traetment for more than 24 months * signed informed consent by parents/legal guardian and patient exclusion criteria: * male or female patients being younger than 6 months * patients without genetically confirmed diagnosis of gaucher disease type 1 * gaucher disease 2 or 3 * patient is currently undergoing enzyme replacement therapy or substrate reduction therapy * missing signed informed consent

Keywords: gaucher disease type 1 lymphatic diseases lipid metabolism, inborn errors lipid metabolism disorders lipidoses
diagnosis of mgrs in patients with paraproteinemias: clinical, anatomopathological and pathophysiological study

Conditions: Monoclonal Gammopathy of Renal Significance

Summary: this is the first prospective study which evaluate patients with small b-cell clones to diagnose monoclonal gammopathy of renal significance (mgrs) and characterize it clinically, anatomopathologically and pathophysiologically taking int account a geriatric approach.


Eligibility criteria:
inclusion criteria: * paraproteinemias exclusion criteria: * multiple myeloma * waldenstrom macroglobulinemia * chronic lymphocytic leukemia * malignant lymphoma * monoclonal gammopathy of unknown significance (mgus) secondary to infections * mgus secondary to autoimmune diseases * uncompleted hematological evaluation * renal tumor except for cysts bosniak type i/ii * other malignant neoplasia * unresolved obstructive uropathy * patients with only one kidney except for renal transplant * co-morbidities which would limit the ability to receive aggressive therapeutic intervention * fragile old people * pregnancy * prison * impatient in psychiatric institutions.

Keywords: "paraproteinemias"[mesh] "kidney diseases"[mesh]
study of welgenaleucel (uwc19) in patients with relapsed or refractory b-cell lymphoma

Conditions: B-cell Non-Hodgkin Lymphoma

Summary: this is an open-label, single-center phase 1/2 study with a dose-escalation phase (part 1) and a cohort expansion phase (part 2) in patients with relapsed or refractory (r/r) b-cell non-hodgkin lymphoma (b-nhl).


Eligibility criteria:
inclusion criteria: have a primary diagnosis of b cell non-hodgkin lymphoma - histologically confirmed: diffuse large b cell lymphoma (dlbcl), primary mediastinal large b cell lymphoma (pmbcl), transformation follicular lymphoma (tfl), high grade b-cell lymphoma (hgbcl), mantle cell lymphoma (mant), burkitt lymphoma (burk), lymphoblastic lymphoma * chemotherapy-refractory disease, defined as one of more of the following no response to last line of therapy or refractory post-autologous stem cell transplant (asct) * individuals must have received adequate prior therapy including at a minimum: anti-cd20 monoclonal antibody unless investigator determines that tumor is cd20-negative and an anthracycline containing chemotherapy regimen for individual with transformed fl must have chemorefractory disease after transformation to dlbcl. * no active infection of hiv, htlv and syphilis * adequate renal function * adequate hepatic function * adequate cardiac function * adequate venous access for apheresis, and no other contraindications for leukapheresis * voluntary informed consent is given. exclusion criteria: a subject will not be eligible for inclusion in this study if any of the following criteria apply: * received allogeneic hematopoietic stem cell transplantation (allohsct) * body weight less than 30 kg * pregnant or lactating women. * uncontrolled active infection. * history of hepatitis b or hepatitis c infection. * previously treatment with any gene therapy products or cell therapy product in past 28 days. * hiv infection. * lymphoma with central nervous system (cns) involvement * have autoimmune disorders * have active infection or inflammatory disorders * prescreening test results in expansion rate less than 5 folds * an allergy to gentamycin and/or streptomycin

Keywords: cd19 non-hodgkin lymphoma car-t cells
interferon alfa plus sargramostim in treating patients with newly diagnosed chronic phase chronic myelogenous leukemia

Conditions: Leukemia

Summary: rationale: interferon alfa may interfere with the growth of cancer cells. colony-stimulating factors such as sargramostim may increase the number of immune cells found in bone marrow or peripheral blood and may help a person's immune system recover from the side effects of therapy. combining sargramostim with interferon alfa may kill more cancer cells. purpose: phase ii trial to study the effectiveness of sargramostim in treating patients who are receiving interferon alfa for chronic phase chronic myelogenous leukemia that is in remission.

objectives: i. estimate the rate of major cytogenetic responses to sargramostim (gm-csf) and interferon alfa in patients with newly diagnosed chronic phase chronic myeloid leukemia. ii. estimate the dosing, schedule, and toxic effects of gm-csf plus interferon alfa in these patients. outline: all patients are in hematologic remission on subcutaneous interferon alfa upon entering the study. once a complete hematologic response is achieved and the interferon alfa dose has been stable for 14 days, patients receive subcutaneous sargramostim (gm-csf) daily for 6 months. patients are followed at 3, 6, 9, and 12 months. projected accrual: approximately 48 patients will be accrued for this study.

Eligibility criteria:
disease characteristics: cytogenetically or molecularly proven chronic phase chronic myeloid leukemia (cml) that is philadelphia chromosome positive or philadelphia chromosome negative with evidence of the bcr-abl rearrangement or evidence of the p120 protein on interferon alfa therapy less than 6 months in complete hematologic response, defined as: wbc less than 10,000/mm3 platelet count less than 450,000/mm3 less than 5% circulating blasts no signs and symptoms of disease, including progressive splenomegaly patient characteristics: age: 18 and over performance status: not specified life expectancy: not specified hematopoietic: see disease characteristics hepatic: not specified renal: not specified other: not pregnant or nursing fertile patients must use effective contraception no history of intolerance to sargramostim (gm-csf) must be able to perform self injection prior concurrent therapy: biologic therapy: prior interferon alfa required chemotherapy: prior hydroxyurea and cytarabine allowed endocrine therapy: not specified radiotherapy: not specified surgery: not specified other: no other concurrent myelosuppressive drug therapy

Keywords: chronic phase chronic myelogenous leukemia philadelphia chromosome positive chronic myelogenous leukemia philadelphia chromosome negative chronic myelogenous leukemia
single ascending-dose study of the safety, tolerability, and pharmacokinetics of regn1908-1909 in allergic, adult subjects

Conditions: Allergy

Summary: the primary objective of the study is to assess the safety and tolerability of single ascending doses of subcutaneously (sc) administered regn1908-1909 in allergic, adult subjects.


Eligibility criteria:
inclusion criteria: 1. healthy men and women between the ages of 18 and 55 2. positive allergen skin prick test 3. willing and able to comply with clinic visits and study-related procedures 4. provide signed informed consent exclusion criteria: 1. persistent, chronic, or active recurring infection requiring treatment with antibiotics, antivirals, or antifungals within 4 weeks prior to the screening visit 2. any clinically significant (determined at the investigator's discretion) abnormalities observed during the screening physical examination 3. onset of a new exercise routine or major change to a previous exercise routine within 4 weeks prior to the screening visit. patients must be willing to maintain a similar level of exercise for the duration of the study and to refrain from unusually strenuous exercise for the duration of the trial 4. hospitalization for any reason within 60 days prior to the screening visit 5. participation in any clinical research study evaluating another investigational drug or therapy within 30 days or at least 5 half-lives (whichever is longer) of the investigational drug prior to the screening visit 6. any medical or psychiatric condition that in the opinion of the investigator or regeneron, would place the patient at risk, interfere with participation in the study or interfere with the interpretation of study results the information listed above is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial and not all inclusion/ exclusion criteria are listed.
team-based virtual pediatric cochlear implant clinic

Conditions: Hearing Loss

Summary: the purpose of this study is to see if remote visits for pediatric cochlear implant patients are possible. the investigator will be assessing whether a multi-disciplinary team approach can be achieved remotely for patients both undergoing the cochlear implant (ci) process and for those who have already been implanted.


Eligibility criteria:
inclusion criteria: new patients undergoing ci evaluation: 1. english speaking patients (parents can speak english and/or spanish) 2. ages 0-12 years who are cochlear implant candidates and undergoing the evaluation process established ci recipients: 1. english speaking patients (parents can speak english and/or spanish) 2. ages 1-12 with at least 6 months of ci experience with a company that has remote programming capacity exclusion criteria: 1. patients with families who speak languages other than english or spanish 2. patients who do not live in florida 3. patients who have any condition that, in the opinion of the investigator, would compromise the well-being of the patient or the study or prevent the patient from meeting or performing study requirements
sotorasib activity in subjects with advanced solid tumors with kras p.g12c mutation (codebreak 101)

Conditions: Advanced Solid Tumors; Kirsten Rat Sarcoma (KRAS) pG12C Mutation

Summary: to evaluate the safety and tolerability of sotorasib administered in investigational regimens in adult participants with kras p.g12c mutant advanced solid tumors.


Eligibility criteria:
inclusion criteria: * men or women greater than or equal to 18 years old. * pathologically documented, locally-advanced or metastatic malignancy with, kras p.g12c mutation identified through molecular testing performed according to in-country requirements. in the united states, this test must be performed in a clinical laboratory improvement amendments (clia)-certified laboratory. exclusion criteria: * primary brain tumor. * spinal cord compression, or untreated, or symptomatic, or active brain metastases, or leptomeningeal disease from non-brain tumors. * myocardial infarction within 6 months of study day 1. * gastrointestinal (gi) tract disease causing the inability to take oral medication.
linear focused shockwave treatment for erectile dysfunction

Conditions: Vasculogenic Erectile Dysfunction

Summary: the study is aimed at comparing the performance of protocols employing one and two sessions per week, respectively. the study hypothesis claims that there is no significant difference between the performances of the compared protocols.


Eligibility criteria:
inclusion criteria: 1. good general health 2. vasculogenic ed- erectile dysfunction, according to physician judgment, for at least 6 months and for no longer than 5 years. 3. international index of erectile function 6 (iief-ef) between 17 and 25 4. satisfactory response to the use of pde5 (phosphodiesterase type 5) inhibitors 5. stable sexual relationship for at least 3 months prior to treatment 6. minimum of two sexual attempts per month exclusion criteria: 1. hormonal, neurological or psychological pathology 2. past radical prostatectomy or extensive pelvic surgery 3. recovering from cancer during last year 4. any unstable medical, psychiatric, spinal cord injury and penile anatomical abnormalities 5. clinically significant chronic hematological disease 6. anti-androgens, oral or injectable androgens 7. past radiotherapy treatment of the pelvic region 8. international normalized ratio (inr) \> 3 for patients using blood thinners (such as coumadin)
pilot stair climbing study

Conditions: Prediabetic State

Summary: this pilot study seeks to demonstrate feasibility to conduct data collection and illustrate potential for effect of fractionalized bouts of stair climbing on glucose control in adults at risk for type 2 diabetes.

objective: compare the short-term effects of two different fractionalized stair climbing bout protocols on glucose control among adults at risk for type 2 diabetes. rationale: fractionalized physical activity is associated with reduced bmi, glycated hemoglobin, lower 2-hr plasma glucose and insulin sensitivity in cross-sectional studies. yet, few studies have examined the effects of fractionalized or intermittent physical activity in a prospective manner. furthermore, the research is limited to patients with diagnosed type 2 diabetes, or using exercise of a moderate intensity. the hypothesis is that stair climbing, a vigorous-intensity activity, may have a greater health effect. however, this has not been tested in short bouts of activity that are spread out during a day. implications: fractionalized physical activity may be more feasible for adults at risk for, or with diagnosed type 2 diabetes who are not currently active. these short bouts can increase energy expenditure throughout the day, rather than in one long continuous bout as currently recommended.

Eligibility criteria:
inclusion criteria: * age greater than 40 years but less than 65 years (working age adult with reduce physiologic capacity to regulate glucose) * bmi of overweight or obese (25.0 to 35.0) * answered "no" to all questions on the physical activity readiness questionnaire, or has physician approval to participate * hemoglobin a1c% value in the at-risk range of 5.7 to 6.4 exclusion criteria: * unable to physically perform stair climbing * currently exercising for 60 or more minutes weekly. * weight over 275 lbs (because stair climbing machine has a weight limit)