Kornimate/medical-research-clean huggingface dataset:
study of aro-ang3 in adults with mixed dyslipidemia

Conditions: Mixed Dyslipidemia

Summary: the purpose of aroang3-2001 is to evaluate the efficacy and safety of aro-ang3 in participants with mixed dyslipidemia. participants will initially receive 2 subcutaneous injections of aro-ang3 or placebo. participants who complete the double-blind treatment period may opt to continue in an open-label extension during which they will receive up to 8 doses of aro-ang3.


Eligibility criteria:
inclusion criteria: * based on medical history, evidence of tg ≥ 150 mg/dl but ≤ 499 mg/dl * fasting levels at screening of ldl-c ≥ 70 mg/dl or non-hdl-c ≥ 100 mg/dl after at least 4 weeks of stable diet and stable optimal statin therapy * mean fasting tg ≥ 150 mg/dl and ≤ 499 mg/dl during screening collected at two separate and consecutive visits and at least 7 days apart and not more than 17 days apart * willing to follow diet counseling and maintain a stable diet per investigator judgment based on local standard of care * participants of childbearing potential must agree to use highly-effective contraception during the study and for at least 24 weeks from last dose of study medication * women of childbearing potential must have a negative pregnancy test and cannot be breastfeeding * women of childbearing potential on hormonal contraceptives must be stable on the medication for ≥ 2 menstrual cycles prior to day 1 * men must not donate sperm during the study and for at least 24 weeks following the last dose of study medication * able and willing to provide written informed consent and to comply with study requirements exclusion criteria: * current use or use within 365 days from day 1 of any hepatocyte targeted sirna or antisense oligonucleotide molecule * active pancreatitis within 12 weeks prior to day 1 * any planned bariatric surgery or similar procedures to induce weight loss from consent to end of study * acute coronary syndrome event within 24 weeks of day 1 * major surgery within 12 weeks of day 1 or planned surgery during the study * planned coronary intervention (e.g., stent placement or heart bypass) during the study * uncontrolled hypertension * human immunodeficiency virus (hiv) infection, seropositive for hepatitis b (hbv), seropositive for hepatitis c (hcv) * uncontrolled hypothyroidism or hyperthyroidism * hemorrhagic stroke within 24 weeks of day 1 * history of bleeding diathesis or coagulopathy * current diagnosis of nephrotic syndrome * systemic use of corticosteroids or anabolic steroids within 4 weeks prior to day 1 or planned use during the study * malignancy within the last 2 years prior to date of consent requiring systemic treatment (some exceptions apply) note: additional inclusion/exclusion criteria may apply per protocol
evaluation of a new strategy for the diagnosis of peroxisomal diseases

Conditions: Peroxisomal Disorders; Diagnoses Disease

Summary: the principal aim of the study is to avoid the diagnostic wanderings of patients suffering from a peroxisomal disorder. for this purpose, a new diagnostic strategy is proposed. it rests on functional metabolic explorations and gene studies directly connected to a first-line enlarged physico-chemical detection of metabolites from peroxisomal origin in clinically suspect patients.


Eligibility criteria:
inclusion criteria: i - clinical/biological criteria for an efficient screening of the patients of the 4 contributing university hospitals (amiens, caen, lille and rouen), various inclusion criteria were selected: in general, the inclusion criterion is the existence of a positive biology or in turn the persistence of a clinical suspicion in spite of a negative biology. i a - children from 0 to 17 years: the clinical inclusion criterion is a positive biology or the persistence of a clinical suspicion in spite of a negative biology. this persistent clinical suspicion is left to the discretion of the clinician. it is essentially based on the existence of a family history of peroxisomal (or suspected) pathology and / or the combination of several clinical signs of craniofacial dysmorphism, skeletal abnormalities, encephalopathy (seizures, ataxia, hypotonia), demyelinating peripheral neuropathy, ophthalmopathy (retinopathy, cataract), hepatic impairment (hyperbilirubinemia, hepatomegaly, cholestasis) and growth retardation or i b - adults from 18 years the neurological symptoms of peroxisomal diseases in adulthood are numerous and non-specific. the three inclusion criteria selected for patient selection are as follows: * cognitive impairment (delayed acquisition or regression) and / or leukodystrophy and at least one of the following signs: cerebellar ataxia, spastic paraparesis, peripheral neuropathy, neurosensory deafness, retinitis pigmentosa, epilepsy, unexplained unexplained vigilance, peripheral corticotropic insufficiency +/- gonadotropic and * no evidence for an extraperoxisomal origin of the patient disease stated after the usual paraclinic explorations ii - non clinical criteria * social insurances * having understood the information note and having signed the informed consent form. * patients under guardianship or curatorship may be included, since peroxisomal diseases as a cause of neurological impairment may potentially lead to guardianship. exclusion criteria: * pregnant or nursing women * person deprived of liberty or in emergency situations
probiotics to prevent relapse after hospitalization for mania

Conditions: Bipolar Disorder; Schizoaffective Disorder

Summary: the purpose of this study is to determine if taking a probiotic supplement versus a placebo will lower rates of relapse and improve the clinical course among participants who have been hospitalized for mania. relapse and clinical course are measured by time to re-hospitalizations, new mood episodes, and changes in mood-related symptoms.


Eligibility criteria:
inclusion criteria: * age 18-65 * capacity for written informed consent * currently admitted to a sheppard pratt inpatient or day hospital for symptoms of mania * primary axis i diagnosis (dsm-iv) at time of admission of bipolar i disorder (single manic episode, most recent episode manic, or most recent episode mixed) or schizoaffective disorder, bipolar type (manic or mixed state) * proficient in the english language * available to come to sheppard pratt towson for follow-up visits * participated previously in one of our screening studies exclusion criteria: * diagnosis of mental retardation * symptoms of mania secondary to a general medical condition * any clinically significant or unstable medical disorder as determined by the investigators including congestive heart failure, abnormal liver function or disease, renal failure, acute pancreatitis, any diagnosis of cancer undergoing active treatment, hiv infection or other immunodeficiency condition * history of iv drug use * primary diagnosis of substance abuse or dependence according to dsm-iv criteria within the last 3 months, or has a positive drug toxicity screen * participated in any investigational drug trial in the past 30 days * pregnant or planning to become pregnant during the study period * receipt of antibiotic medication within the previous 24 hours (as anaerobic organisms residing in the gastrointestinal tract may be minimally affected by antibiotics) * documented celiac disease (as such persons should be on a gluten-free diet as this is the standard care). of note, we are not limiting the study to individuals with elevated levels of gliadin or casein antibodies as we intend to look at these levels as a predictor of response.

Keywords: mania manic episode mixed episode bipolar disorder schizoaffective disorder probiotics probiotic supplement relapse prevention new mood episode re-hospitalization
evaluation of preimplantation portal vein and hepatic artery flushing with tacrolimus

Conditions: Early Allograft Dysfunction; Ischemic Reperfusion Injury; Liver Transplantation; Hyperfibrinolysis

Summary: the purpose of this study is to determine whether the tacrolimus added to histidine-tryptophan-ketoglutarate (htk) solution given through intraportal and intraarterial infusion during back-table procedure is capable of reducing the degree of early allograft liver dysfunction, as assessed by postoperative levels of aspartate aminotransferase (ast), alanine aminotransferase (alt), during first 7 postoperative days and by serum and histochemical markers of liver injury and inflammation.

early allograft liver dysfunction remains a significant complication of cadaveric liver transplantation with resource consuming and costly treatment, increased risk of multiorgan failure and 6-months mortality. ischemic reperfusion injury (iri) is a main reason for early allograft liver dysfunction. inflammatory response to brain death in donor can precipitate the extent of dysfunction after reperfusion in recipient (1). clear inflammatory pathways in response to iri have been reported to be associated with early allograft liver dysfunction (2,3). it was shown that ex vivo intraportal tacrolimus perfusion suppressed inflammation and immune response in the transplanted liver on a genome-wide basis (4). we hypothesize that tacrolimus added to htk solution given through intraportal and intraarterial back-table infusion is capable of reducing the degree of early allograft liver dysfunction, as assessed by incidence of postreperfusion hyperfibrinolysis, postoperative levels of ast,alt, during 1-7 postoperative days as well as serum and histochemical markers of liver injury and inflammation compared to no intraportal and intraarterial back-table infusion.

Eligibility criteria:
inclusion criteria: * donor: age 15-65 years macrovesicular steatosis \< 40% (macroscopy or biopsy) sodium \<165 mmol/l icu stay and ventilation \< 11 days cold ischemia time \< 13 hours ast \< 200 u/l alt \< 200 u/l bilirubin \< 50 μmol/l application of norepinephrine is allowed * recipient age: 18-69 exclusion criteria: recipient: * live donor liver transplant * reduced and split grafts * multi organ failure

Keywords: liver transplantation early allograft dysfunction ischemic reperfusion injury portal and arterial ex vivo htk flush
safety and efficacy evaluation of et-01 transplantation in subjects with transfusion dependent β-thalassaemia

Conditions: Transfusion Dependent Beta-Thalassaemia

Summary: this is an open label, multi-center study to evaluate the safety and efficacy of et-01 transplantation in subjects with transfusion dependent β-thalassaemia.

after the proper subject is recruited, subject will go through steps generally as stem cell mobilization, apheresis, conditioning and et-01 infusion to complete therapy. two years follow-up will be carried out post-transplantation and related data will be collected. subjects who have been treated by et-01 will be asked to participate in a long-term follow up study. monitoring the long-term efficacy and safety up to 15 years after et-01 transplant.

Eligibility criteria:
inclusion criteria: * subject and/or subject's legal personal representative fully understand and voluntarily sign informed consent forms; * 6\~35 years old, all gender; * diagnosis of transfusion dependent β-thalassemia (β-tdt) as defined by protocol; * eligible for autologous stem cell transplant; * eligible for autologous stem cell transplant; * organs in good function. * other protocol defined inclusion criteria may apply. exclusion criteria: * subjects with associated α-thalassemia; * active bacterial, virus, fungal or parasitic infection, including hiv infection, hbsag and hbv dna positive, hcv dna positive, or treponema pallidum infection; * hla identical sibling or unrelated donors are available; * prior allo-hsct or gene therapy. * other protocol defined exclusion criteria may apply.

Keywords: beta-thalassaemia hematopoietic stem-cell transplantation
long-term follow-up of subjects with fanconi anaemia subtype a treated with ex vivo gene therapy

Conditions: Fanconi Anemia Complementation Group A; Fanconi Anemia

Summary: this is a long-term safety and efficacy follow-up study for subjects with fanconi anaemia subtype a who have been treated with ex vivo gene therapy on the fancolen-i trial. after completion of the fancolen-i study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. no investigational drug product will be administered during this study.

this long-term follow-up protocol will evaluate the long term safety and efficacy of the infusion of autologous cd34+ cells transduced with lentiviral vector (lv) carrying the fanca gene.

Eligibility criteria:
inclusion criteria: 1. enrolled in the fancolen-i study 2. treated with gene therapy in the fancolen-i study 3. able to adhere to the study visit schedule and protocol requirements 4. provided written informed consent and, as applicable, assent to participate exclusion criteria: * there are no exclusion criteria for this study

Keywords: gene therapy anaemia autologous hematopoeitic stem cell transplant safety bone marrow failure genetic diseases anemia
dante space for evaluation of subjects with intracranial vascular disease

Conditions: Intracranial Vascular Disease

Summary: purpose: the purpose of this study is to evaluate the delay alternating with nutation for tailored excitation (dante) space sequence in clinical studies to determine whether it can provide more useful information for clinical diagnosis. participants: 100 participants with concern for intracranial vascular disease scheduled to undergo a clinical vessel wall mri will be recruited. procedures (methods): patients with concern for intracranial vascular disease scheduled to undergo a clinical vessel wall mri who will have an additional non-fda approved sequence (dante space) added to their clinical scan. the investigational sequence requires less than 15 minutes and will be added following the standard mri sequence.


Eligibility criteria:
inclusion criteria: * ages 18 and older * concern for intracranial vascular disease * scheduled to undergo a clinical vessel wall mri exclusion criteria: * claustrophobia * implanted metallic devices, parts, vascular clips, or other foreign bodies. * known hypersensitivity to gadolinium contrast or to any component of gadolinium contrast refractory to standard medications (antihistamines, steroids) * impaired kidney function (serum creatinine level \> 1.8 mg/dl or a glomerular filtration rate \< 60 as approximated using serum creatinine levels) unless anuric and on dialysis. * any woman who is pregnant or has reason to believe she is pregnant via self report
omega-3 fatty acids and nutritional support in gastrointestinal cancer

Conditions: Sarcopenia

Summary: sarcopenia is a frequent complication in patients with cancer and chronic diseases, it is characterized by decreased muscle strength and fatigue due to reduced skeletal muscle mass, which is accompanied by atrophy and decreased quality of muscle tissue. in all cases, it negatively impacts treatment tolerance, clinical outcomes and survival, in consequence, quality of life of these patients decreases while morbidity, mortality and costs increase. in this context, appropriate nutritional screening and early nutrition support are extremely recommended, to this aim, in some cases, oral nutritional supplements (ons) are necessary; ons could have a standard formula or be enriched with specific nutrients (arginine, glutamine, branched chain amino acids, n-3 fatty acids, and nucleotides), which can modulate the activity of the immune system and provide an additional benefit beyond the nutritional support, this intervention type is called immunonutrition. despite these possible benefits, their utility has been proven in few clinical scenarios, for example in with patients with upper gastrointestinal cancer undergoing surgical resection; based on this, current guidelines recommend that patients should receive oral/enteral nutritional support with an specific formula enriched in immunonutrients (with arginine, n-3 fatty acids or nucleotides) , but there is a lack of evidence for supporting its use in other clinical conditions including patients with cancer that receive systemic treatment


Eligibility criteria:
inclusion criteria: * - patients with gastrointestinal tumor undergoing systemic treatment (chemo-radio-immuno therapy or their combination) * body weight loss \>5% in the previous three months or \>10% in the previous six months * both sexes * age between 18-85 y-old. exclusion criteria: * life expectancy \< 2 weeks * mdrd \< 15 ml/min * end-stage liver disease * any musculoskeletal, cardiovascular and/or neurological disorders that could affect exercising.

Keywords: cancer nutritional support omega-3 enriched nutritional supplements
mesenchymal stem cells (mscs) - like cell transplantation in women with primary ovarian insufficiency

Conditions: Primary Ovarian Insufficiency

Summary: there is a high incidence of women suffering from primary ovarian insufficiency (poi). so far, there was no treatment sufficient enough to cure poi. cell therapy is a rapidly developing field and have shown immense promise in the treatment of ovarian dysfunction. in this study, the investigator will evaluate the safety of msc-like cell therapy in women suffering from poi.

the purpose of this work is to explore the safety and preliminary efficacy of human embryonic stem cell derived msc-like cell transplantation in women with poi. msc-like cells were injected directly into bilateral ovaries under transvaginal ultrasound. patients are subjected into 3 groups, with low (0.2×107), medium (0.5×107) and high dosage (1.0×107) of cells injection for each ovary. the outcomes of patients were followed after the injection every 2 weeks. the investigator mainly follow the safety indexes, and the level of sex hormone and signs of follicle growth will also be monitored. the growing follicles will be stimulated by exogenous hormone, followed by oocyte retrieval and in vitro fertilization (ivf).

Eligibility criteria:
inclusion criteria: 1. under 40 years of age; 2. have established regular menstrual cycle, oligomenorrhea / amenorrhea ≥ 4 months; 3. fsh (follicle-stimulating hormone) \> 25 iu/ml; 4. bilateral ovaries are visible by ultrasound; 5. have fertility requirement, husband has sperms; 6. willing to sign the informed consent form. exclusion criteria: 1. primary amenorrhea; 2. history of serious drug allergy or allergic constitution; 3. thrombophlebitis and thromboembolia, past and present; 4. acute or chronic infection, hematologic disease, angiocardiopathy, pulmonary disease, liver disease, nephropathy, pancreas disease 5. autoimmune disease, history of severe familial genetic disease, etc. 6. anatomical abnormality of reproductive system; 7. contraindications for pregnancy; 8. spouse azoospermia; 9. hiv+, hepatitis b, c; 10. thyroid dysfunction; 11. history of previous malignant or ovarian tumors, history of ovarian therapy; 12. abnormal karyotyping (e.g. turner syndrome, fragile x syndrome); 13. during pregnancy or lactation; 14. alcohol or other substance abuse; 15. mental disease, communicate obstruction; 16. unwilling to comply with follow-up schedule or want to take other treatment during the follow-up period; 17. attending doctor consider inappropriate to take part in.

Keywords: mesenchymal stem cell transplantation human embryonic stem cell premature ovarian failure
reliability and validity of ios in ipf

Conditions: Idiopathic Pulmonary Fibrosis

Summary: idiopathic pulmonary fibrosis (ipf) is a condition where scar tissue (called fibrosis) builds up in the lungs. it usually gets worse over time. fibrosis causes the lungs to become stiff, and reduces the amount of oxygen that the lungs can take up. people with ipf complain of worsening breathlessness, which limits their day to day activities. lung function tests are breathing tests that measure how well the lungs are working, and are used by doctors to decide whether to start or stop medicines in people with ipf. however, people with ipf tell us that lung function tests require a lot of effort, can make them cough and feel very short of breath. about 1 in 5 people with ipf are unable to perform lung function results accurately. impulse oscillometry (ios) is a new type of breathing test. it uses sound waves to measure the stiffness of the lung. the test is very quick (30 seconds), does not require any effort from the patient, and only requires a patient to breathe in their usual way. ios has been used successfully in children who are unable to perform normal lung function tests. the investigators will assess whether people with ipf can perform ios tests accurately and to compare their experiences of having ios tests with their experience of performing current lung function tests. the investigators will also compare whether there is a relationship between ios tests and the information gathered from the tests currently used by doctors to measure the impact of ipf (lung function tests, exercise tests, lung scans and questionnaires that measure quality of life).


Eligibility criteria:
inclusion criteria: 1. diagnosis of idiopathic pulmonary fibrosis made by specialist multidisciplinary team according to international standards 2. able to provide written informed consent exclusion criteria: 1. contraindication to performing conventional lung function tests, e.g untreated tb, haemoptysis, pneumothorax, unstable cardiovascular status, recent eye, thoracic or abdominal surgery, thoracic, abdominal or cerebral aneurysm, acute illness (including diarrhoea and vomiting) 2. unable to create seal on mouthpiece
analgesic effect of ultrasound guided maxillary nerve block in trigeminal neuralgia

Conditions: Trigeminal Neuralgia

Summary: this is a study about the dynamics and effects of ultrasound guided maxillary nerve block in trigeminal neuralgia

standard pharmacological treatment for trigeminal neuralgia is often insufficient. in this setting, patients often suffer trough everyday activities and quality of life is disturbed substantially. in this prospective clinical study, analgesic effect of ultrasound (us) guided maxillary nerve block will be investigated, in order to prolong pain-free time, reduce the use of standard analgesics, as well as atypical ones (e.g. antiepileptic drugs and antidepressants). characteristics of us guided maxillary nerve block will pain investigated: reduction of pain (decrease of vas score), duration of pain-free period, pain level during the procedure.

Eligibility criteria:
inclusion criteria: * patients with trigeminal neuralgia exclusion criteria: * allergy to local anesthetics * local infection at the site of needle puncture * parotitis * serious liver disease * use of antiarrhythmics group iii (eg amiodarone) * anticoagulant or antithrombotic drugs * lactation

Keywords: trigeminal neuralgia maxillary nerve ultrasound guided nerve block
botulinum-assisted orbital fat augmentation with prp (platelet-rich-plasma)

Conditions: Orbital Atrophy

Summary: immobilization of yoked extra-ocular muscles in the anophthalmic orbit, in conjunction with platelet-rich-plasma, will improve integration of viable adipocytes injected into orbital fat

immobilization of tissue (graft, graft bed) is a prerequisite for tissue integration. since yoked extra-ocular muscles continue to be stimulated by movements of the seeing eye after fat injection into the anophthalmic orbit, the use of botulinum toxin(s) can be used to prevent movement of the orbital tissue bed(s) and improve anchoring of injected fat. platelet rich plasma may also improve fat cell anchoring by the action of fibrin on the adhesion process. this innovation builds on the results of irb protocols 10.27 and 12.01 as registered with clinical trials.gov

Eligibility criteria:
inclusion criteria: * anophthalmic enophthalmos in subjects within a 50 mile radius of new york city, grade 0-1 anesthesia risk, ages 21-75 exclusion criteria: * radiated orbits