Kornimate/medical-research-clean huggingface dataset:
safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of sebelipase alfa in children with growth failure due to lysosomal acid lipase deficiency

Conditions: Lysosomal Acid Lipase Deficiency; Wolman Disease

Summary: this was an open-label, repeat-dose, intra-participant dose-escalation study of sbc-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (lal) deficiency. eligible participants received once-weekly (qw) infusions of sebelipase alfa for up to 5 years.

lal deficiency is a rare autosomal-recessive lipid storage disorder that is caused by a marked decrease or almost complete absence of lal, leading to the accumulation of lipids, predominately cholesteryl esters and triglycerides, in various tissues and cell types. in the liver, accumulation of lipids leads to hepatomegaly, liver dysfunction, and hepatic failure. although a single disease, lal deficiency presents as a clinical continuum with 2 major phenotypes, cholesteryl ester storage disease (cesd) and wolman disease. early-onset lal deficiency (wolman disease) is extremely rare, with an estimated incidence of less than 2 lives per million. it is characterized by profound malabsorption, growth failure, and hepatic failure, and is usually fatal in the first year of life.

Eligibility criteria:
inclusion criteria: * participant's parent or legal guardian provided written informed consent/permission prior to any study procedures. * male or female child with documented decreased lal activity relative to the normal range of the laboratory performing the assay or documented result of molecular genetic testing (2 mutations) confirming a diagnosis. * growth failure with onset before 6 months of age. exclusion criteria: * clinically important concurrent disease or comorbidities. * had received an investigational product other than sebelipase alfa within 14 days prior to the first dose. * participant was older than 24 months of age. * myeloablative preparation, or other systemic pre-transplant conditioning, for hematopoietic stem cell or liver transplant. * previous hematopoietic stem cell or liver transplant. * known hypersensitivity to eggs.

Keywords: lipa wolman disease wolman phenotype acid lipase deficiency acid cholesteryl hydrolase acid lipase disease deficiency, type 2 cholesteryl ester storage disease (cesd) cholesteryl ester hydrolase deficiency early onset lysosomal acid lipase deficiency (wolman disease) lal deficiency late onset lysosomal acid lipase deficiency (cesd) wolman disease (early onset lal deficiency) related disorders: non-alcoholic fatty liver disease (nafld) non-alcoholic steatohepatitis (nash) alcoholic liver disease cryptogenic cirrhosis niemann-pick disease (npd) type c chanarin dorfman syndrome
clinical impact of orsiro™ stent in patient with chronic kidney disease

Conditions: Chronic Kidney Diseases; Coronary Artery Disease

Summary: orsiro™ or orsiro_mission™ stents have the biodegradable polymer and the thinnest stent strut (60㎛) in commercially available stents, which could minimize chronic inflammation and incomplete endothelialization. in several prior studies, ultrathin strut sirolimus-eluting stent (orsiro®) showed better outcomes in terms of target lesion failure or target lesion revascularization compared to other contemporary stents. chronic kidney disease (ckd) is known to be associated with adverse cardiovascular outcome including restenosis or stent thrombosis following percutaneous coronary interventin (pci), because the patients with ckd have more complex coronary lesion characteristics, severe calcification, and diffuse plaque of the coronary artery. however, there is a paucity of data regarding clinical efficacy of orsiro® in ckd population. therefore, this study aims to investigate the differences in clinical outcomes according to renal function in patients undergoing pci with orsiro™ or orsiro_mission™ stents. after patients will be divided into three groups according to the renal function; normal or mild renal dysfunction (egfr ≥ 60 ml·min - 1·1.73㎡), moderate renal dysfunction ( 30≤ egfr\< 60 ml·min - 1·1.73㎡), and severe renal dysfunction (egfr \< 30 ml·min -1·1.73㎡), the clinical outcome will be investigated among groups.


Eligibility criteria:
inclusion criteria: * 19 years of age or older * patient undergoing percutaneous coronary intervention with orsiro™ or orsiro_mission™ stents exclusion criteria: * patient with a life expectancy of less than one year * patient who used another drug-eluting stent for percutaneous coronary intervention at study registration time * cardiogenic shock * pregnancy

Keywords: chronic kidney diseases coronary artery disease orsiro stent
economic evaluation of stream™ platform

Conditions: Anastomotic Leak

Summary: this economic evaluation study is a multi-center, two-arm, non-randomized, interventional trial. the purpose of this study is to evaluate the economic and patient outcomes of using the stream™ platform for early detection of anastomotic leakage after gastrointestinal surgery. the trial will track patient outcomes and healthcare costs in two groups: the intervention group, where the stream platform is deployed, and the control group, before the integration of the stream™ platform. the data collected from both groups will be compared to assess the impact of implementing the stream™ platform and early leak detection on overall patient outcomes and healthcare costs.this trial will include hepatobiliary, colorectal, and trauma/acute care patient populations.


Eligibility criteria:
inclusion criteria: * age ≥ 18 years - male or female * subject understands and has voluntarily signed and dated the informed consent form (icf). * subjects must be willing to comply with trial requirements * subject has performed an open or laparoscopic surgery with peritoneal or pelvic drainage exclusion criteria: * subject is pregnant * subject will be discharged \< 8 hours post-surgery * 24 or more have passed since the end of the subject's surgery * involvement in the planning and conduct of the clinical investigation * subject is allergic to contrast medium * participation in another investigational drug or device study which would interfere with the endpoints of this study * device does not attach to drain used on the subject
a comparative study of salvage treatment with combination of docetaxel and epirubicin

Conditions: Metastatic Advanced Gastric Cancer

Summary: a salvage treatment with combination of docetaxel and epirubicin in patients with unresectable, metastatic gastric cancer after fluoropyrimidine failure : a her2 status-based study


Eligibility criteria:
inclusion criteria: 1. histologically confirmed metastatic, or recurrent agc 2. previous use of fluoropyrimidine as palliative chemotherapy. concurrent use of platinum is permitted. patient with recurred disease within 6 months after the end of adjuvant therapy can participate to this trial. 3. documented her2 expression status. 4. radiologically proven progression of disease. 5. age \> 20 years 6. ecog ps 0 - 2 7. life expectancy more than 3 months 8. adequate organ function for treatment including liver, kidney and bone marrow (absolute neutrophil count (anc) ≥ 1,500/ul, platelets ≥ 100,000/ul, ast/alt ≤ 2.5 x uln, ≤ 5.0 x uln if liver involvement, total serum bilirubin ≤ 2.0 mg/dl) 9. written informed consent exclusion criteria: 1. prior administration of taxane or anthracycline as palliative chemotherapy. if those agents were used as adjuvant or neoadjuvant aim, at least 6 months should be passed. 2. uncontrolled medical comorbidities congestive heart failure (nyha class iii or iv) recent (within 6 months) unstable angina or myocardial infarct clinically significant cardiac arrhythmia lvef \< 45% liver cirrhosis (child-pugh class b or c) interstitial pneumonia psychiatric illness/social situations that would limit compliance with study requirements 3. active cns metastasis not controllable with radiation or steroid 4. concurrent administration of systemic chemotherapy or radiotherapy. palliative radiotherapy for pain control more than 2 weeks ago is acceptable. 5. pregnant or lactating women. 6. peripheral neuropathy g3\~4. 7. metachronous malignancy other than adequately treated cin of uterine cervix, basal cell or squamous cell carcinoma of the skin.

Keywords: agc, second-line, docetaxel, epirubicin
safety, pk and pd of kamada anti-sars-cov-2 in covid-19

Conditions: Covid19; Pneumonia, Viral

Summary: evaluate the safety pharmacokinetics and pharmacodynamics (pk/pd)of a single dose of kamada anti-severe acute respiratory syndrome (sars)- cov-2 in patients hospitalized with covid-19 caused pneumonia


Eligibility criteria:
inclusion criteria: 1. age ≥ 18 years 2. laboratory confirmed sars-cov-2 infection by nasopharyngeal swab real time polymerase chain reaction (rt-pcr) 3. hospitalized for covid-19 pneumonia 4. dosing should be within 10 days of symptom start 5. able and willing to sign informed consent form exclusion criteria: 1. history of hypersensitivity to plasma products and/or severe immunoglobulin a deficiency (\< 7 mg/dl) 2. requirement of high flow oxygen devices, or non-invasive ventilation or mechanical ventilation, or extracorporeal membrane oxygenation (ecmo) at screening 3. cardiovascular instability 4. history of thrombo-embolic events 5. acute renal failure or creatinine \>2 mg/dl or estimated estimated glomerular filtration rate (egfr) \<30 ml/min 6. history of lung transplantation 7. major surgery (abdominal and chest) within the last 4 weeks 8. severe chronic background disease, per investigator's judgement for example, cirrhosis grade c, dialysis, cardiac insufficiency (nyha iii), pulmonary disease (fev1\<50 percent of predicted) etc. 9. pregnancy or lactation 10. treatment with plasma units or immunoglobulin preparations within the last 4 weeks 11. participation in another pharmaceutical interventional clinical study within 4 weeks from screening
iron bioavailability from encapsulated ferrous sulphate

Conditions: Iron Deficiency

Summary: salt is one of the very few regularly purchased food item in all socioeconomic classes including poor remote areas with subsistence farming. therefore, it is a promising vehicle for fortification with micronutrients, such as iodine, iron, vitamin a, to alleviate the burden of micronutrient deficiencies. however, ensuring the bioavailability of iron and the sensory quality in fortified salt is difficult. water-soluble iron compounds, such as ferrous sulphate feso4, are the most bioavailable, but they react with moisture and impurities in salt, and cause unacceptable changes in colour. encapsulation can reduce iron-mediated colour change in fortified salt without significantly compromise bioavailability. in the present project we aim to investigate the iron bioavailability from salt fortified with feso4 using a new encapsulation type based on hyaluronic acid (ha) and a polymer from the eudragit family (epo).


Eligibility criteria:
inclusion criteria: * female, 18 to 40 years old * marginal iron status (ferritin \< 25 ng/ml) * body weight \< 65 kg * normal body mass index (18.5 - 25 kg/m2) * signed informed consent exclusion criteria: * pregnancy (assessed by a pregnancy test) / intention to become pregnant * lactating up to 6 weeks before study initiation * moderate or severe anaemia (hemoglobin \< 9.0 g/dl) * elevated c-reactive protein (\> 5.0 mg/l) * any metabolic, gastrointestinal kidney or chronic disease such as diabetes, hepatitis, hypertension, cancer or cardiovascular diseases (according to the participants own statement) * continuous/long-term use of medication during the whole study (except for contraceptives) * consumption of mineral and vitamin supplements within 2 weeks prior to 1st meal administration * blood transfusion, blood donation or significant blood loss (accident, surgery) over the past 4 months * earlier participation in a study using fe stable isotopes or participation in any clinical study within the last 30 days * participant who cannot be expected to comply with study protocol (e.g. not available on certain study appointments or difficulties with blood sampling)

Keywords: iron deficiency iron fortification iron encapsulation
efficacy of platelet rich plasma for treatment of muscle rupture with haematoma

Conditions: Muscle Injuries

Summary: this is a multicenter, simple blind, masking of outcomes assessors, parallel, randomised clinical trial in patient with muscle rupture and with hematoma production. the main hypothesis is that infiltration in the area of muscle injury in autologous platelet-rich plasma (prp)improves muscle regeneration and repair by shortening the time to complete recovery. the main objective is to evaluate the prp for healing muscular lesions 'tennis leg' type or distal rectus femoral. the secondary objectives are: to evaluate the risk of lesion recurrence; to evaluate the quality of lesion recovery process and evaluate intervention's safety. experimental treatment will be the administration of prp autologous (4-8 cc in a unique dose) by muscular infiltration en the empty space generated after the hematoma evacuation. control treatment will be hematoma evacuation. both treatment groups will use compressive bandage and they will recommend rest, extremity elevation, local ice and lately physiotherapy. size sample: 76 patients (38 in each group)


Eligibility criteria:
inclusion criteria: * adults over 18 years * lesion with haematoma at the gastrocnemius muscle or the lower portion of the rectus femoral muscle * acceptance to participate at the clinical trial * not indicated the surgical treatment of the muscle injury exclusion criteria * patients who do not meet the inclusion criteria * history of bleeding disorders * inability to follow-up the patient * the use of corticosteroids, acetylsalicylic acid(aspirin ®) and nonsteroidal antiinflammatory drugs during the study

Keywords: muscle injuries platelet-rich plasma autologous haematoma
neoadjuvant chemotherapy followed by radiotherapy alone in patients with breast cancer

Conditions: Breast Cancer; Radiation Therapy

Summary: after neoadjuvant chemotherapy, patients normally receive either conservative breast surgery or mastectomy followed by radiation therapy. some patients achieve a complete response after neoadjuvant chemotherapy. considering that radiation therapy is an effective treatment for subclinical microscopic disease, the question arises whether breast surgery before radiation therapy can be avoided in the subgroup of patients with complete response after neoadjuvant chemotherapy.


Eligibility criteria:
inclusion criteria: * age 40 years or older * pathologically confirmed unicentric invasive breast cancer * clinical stage t1-3 / n0 or n2 / m0 * clinical conditions to receive neoadjuvant chemotherapy exclusion criteria: * prior diagnosis of cancer (exception: in situ cervical tumor and non-melanoma skin) * patient is known to be pregnant * prior history of radiation therapy in the thoracic region * impossibility to perform magnetic resonance imaging
identification of risk factors for brain recurrence in patients with her2-positive localised breast cancer

Conditions: HER2-positive Breast Cancer

Summary: her2 gene amplification, detected in 20% to 30% of breast cancers, was a poor prognostic factor before the advent of anti-her2 therapies. in the early 2000s, trastuzumab revolutionised the management of patients with her2-positive (her2+) breast cancer in the metastatic and localised stages of the disease. at the time of diagnosis of metastatic disease, 7-11% of patients have brain metastases, with (70% of cases) or without symptoms (30% of cases). in the absence of brain metastases, 30% to 50% of patients will develop brain metastases within the first two years of treatment, depending on whether the disease is hormone receptor positive (hr+) or negative (hr-). the presence of brain metastases is the most important prognostic factor. the neurological symptoms caused by the presence of these lesions, but also by the local treatments offered, affect patients' quality of life, although improvements in surgical and radiotherapy techniques have significantly reduced the need for particularly toxic whole brain radiotherapy. international guidelines do not recommend systematic brain mri in the absence of neurological symptoms, either in the adjuvant or metastatic stages of this disease. however, there may be a role for more systematic and earlier screening for cerebral recurrence, as single cerebral recurrences without extracranial involvement are common and the new anti-her2 agents (i.e. tucatinib, an anti-her2 tyrosine kinase inhibitor, and t-dxd) have shown significant objective response rates in cerebral metastases. to date, no clinical or histological prognostic factor (proliferation index, hr expression, etc.) has been used to identify a population of patients at high risk of cerebral relapse, allowing monitoring and treatment to be personalised. new tools for these indications would significantly modify our clinical practice, allowing the identification of a subpopulation at high risk of cerebral recurrence, suitable for increased monitoring and therapeutic adjustment.


Eligibility criteria:
inclusion criteria: * age ≥ 18 years old * be a female patient * patients with histologically proven her2-positive invasive breast cancer (ihc 3+ or 2+ with positive sish), * neoadjuvant chemotherapy and intra-tumour clips indicated at the multidisciplinary consultation meeting (rcp). * signed informed consent form exclusion criteria: * pregnant or breast-feeding women * have had a haematoma requiring level ii analgesics at the time of the diagnostic biopsy. * known coagulation disorders * individual deprived of liberty or placed under the authority of a tutor, or a currator * not be affiliated to a social security regimen

Keywords: metastases central nervous system biopsy
rtms and rehabilitation for individuals with crps type 1

Conditions: Complex Regional Pain Syndrome Type I

Summary: subjects with complex regional pain syndrome (crps) type 1 will be randomized to receive repeated transcranial magnetic stimulation (rtms) followed by rehabilitation or sham rtms followed by rehabilitation. treatment will last for 4 weeks, with the first week including 4 rtms treatments and 2 rehabilitation treatments. subsequent weeks will include 2 rtms treatments followed by 2 rehabilitation treatments. outcome measures will include pain ratings, promis questionnaires, global rating of change, and grip strength or 1 repetition maximum leg press.

subjects will be randomized to receive either real or sham rtms, combined with rehabilitation. subjects will attend 10 sessions over 4 weeks. rtms will be delivered at 10hz using an intensity of 80% of the motor threshold. pulse trains will be delivered for 10 seconds, with a 30-second rest for 20 repetitions. this will result in a total of 2,000 pulses of magnetic stimulation and will take approximately 20 minutes. there will be 10 rtms sessions over the 4 weeks. sham rtms will be delivered using the same method as rtms, but a sham coil, that does not deliver magnetic stimulation, will be used. rehabilitation will include graded motor imagery, education, and graded functional activity. there will be 8 rehabilitation sessions over 4 weeks. outcomes will include questionnaires asking about pain and function, and either grip strength or leg press strength measurements.

Eligibility criteria:
inclusion criteria: * upper or lower extremity crps type 1 (also called rsd-reflex sympathetic dystrophy) of at least 6 months. * pain rating on nprs of at least 4/10 * no initiation of a new intervention (i.e., medication, rehab, injections) in the previous 2 months. * no plan to initiate a new intervention during the study treatment timeframe (4 weeks). exclusion criteria: * a history of seizures or epilepsy * intracranial metallic devices * pacemaker * intrathecal infusion pumps * brain or spinal cord stimulators with epidural electrodes * other ferromagnetic metallic intracranial implants * apparent mental or psychiatric disorder that prevents adequate informed consent * current pregnancy * non-english speaking
remote ischemic preconditioning in liver transplantation

Conditions: Liver Transplanatation

Summary: remote (upper arm) ischemic preconditioning in the donor of living donor liver transplanta to ameliorate the effect of reperfusion injury in recipients.

upper arm ischemia will be induced by elevation of nibp cuff pressure to 200 mm hg for 5 minutes repeated four times 5 mniutes apart, aiming to decrease the effect of ischemia reperfusion in the graft liver.

Eligibility criteria:
inclusion criteria: * all candidates for living donor liver transplantation exclusion criteria: * donors with peripheral vascular disease
axillary reverse mapping using near-infrared imaging in invasive breast cancer: predictors of nodal positivity

Conditions: Breast Cancer

Summary: the initial standard treatment of breast cancer is surgery. tumor involvement of lymph nodes is of paramount importance in the subsequent management of this cancer and surgery of invasive breast cancer (bc) involves axillary lymph node dissection (alnd). to preserve arm lymphatic drainage during alnd and avoid the risk of arm lymphedema, mapping the lymphatic drainage by axillary reverse mapping (arm) has been developed. but oncological safety is uncertain. the arm procedure presented here uses indocyanine green (icg) and fluorescence detection of draining lymphatics. the project aims to train surgeons to the technique and to identify predictive factors for metastatic arm nodes in invasive bc using tumor and axillary pathological parameters to better select patients who would not require removal of the arm node in the future


Eligibility criteria:
inclusion criteria: * adult patients over 18 years of age * preoperative diagnosis of invasive breast cancer * patient deemed for total mastectomy with axillary lymph node dissection (alnd) * subject capable of giving informed consent and participating in the process of consent * affiliated to the french social security exclusion criteria: * mastectomy without alnd * previous ipsilateral axillary radiotherapy * previous axillary surgery * pregnant women as determined by urinary or serum beta human chorionic gonadotropin (hcg) within 72 hours of surgery * breastfeeding * allergy to indocyanine green * patients with a known history of reaction to iodine or iodine-containing compounds. * no consent * impaired capacity to make informed medical decisions * patient on guardianship