Kornimate/medical-research-clean huggingface dataset:
haploidentical bone marrow stem cell transplantation in patients with multiple myeloma

Conditions: Multiple Myeloma

Summary: the aim of this phase 2 study is to demonstrate that kir-ligand mismatched haplobmt with post-transplant cyclophosphamide will improve progression free survival in poor risk multiple myeloma patients.

the goal of this study is to evaluate the effectiveness of a new treatment modality, the kir-ligand mismatched haploidentical stem cell transplantation (haplobmt), for poor risk multiple myeloma (mm) patients. mm is a malignancy of plasma cells that usually resides in the bone marrow. despite new treatment modalities that have been introduced in the last years, mm is still an incurable disease for most patients and median survival for the younger patients (\<65) is about 5 years. mm can be treated by several disease modifiers - classical chemotherapy, high dose chemotherapy and autologous stem cell transplantation (asct), immunomodulators like thalidomide and lenalidomide, and drugs like bortezomib that interact with relevant intracellular pathways of malignant plasma cells. though these treatment modalities have improved overall survival and quality of life, patients are not cured.

Eligibility criteria:
inclusion criteria: * patients with mm \<60 years. * poor prognosis mm patients, permissive for kir-ligand mismatch and with a kir-ligand mismatched haploidentical donor. poor prognosis is based on: * patients with early disease recurrence (within 12 months after first asct) or * patients after a minimum of three lines of chemotherapy (including high dose therapy followed by asct rescue therapy) or * poor risk based on the cytogenetic profile. * written informed consent * no hla identical related or 10/10 matched unrelated donor * permissive for kir-ligand mismatch * responsive after reinduction therapy * measurable disease exclusion criteria: * - patients with an full matched (10/10) donor, who will enroll in the hovon 96 study * active uncontrolled infections * uncontrolled cns involvement by the malignant disease * severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or symptomatic ischemic heart disease) * severe pulmonary dysfunction (ctcae grade iii-iv) * severe neurological or psychiatric disease * significant hepatic dysfunction (serum bilirubin or transaminases ≥ 3 times upper limit of normal) * significant renal dysfunction (creatinine clearance \< 30 ml/min after rehydration) * history of active malignancy during the past 5 years with the exception of basal carcinoma of the skin or stage 0 cervical carcinoma * any psychological, familial, lingual, sociological and geographical condition potentially hampering compliance with the study protocol and follow-up schedule * breast-feeding female patients. * concurrent severe and/or uncontrolled medical condition (dm, hypertension, cancer).

Keywords: m myeloma kir ligand mismatch haploidentical
reliability and validity of the turkish version of the functional assessment of chronic illness therapy - treatment satisfaction - general questionnaire

Conditions: Satisfaction, Patient

Summary: in the evaluation of the services provided in hospitals in recent years, importance has been given to the measurements based on the perception of the patients. today, the most frequently used criteria for this purpose are quality of life and patient satisfaction. the general opinion of the experts is that the information obtained with these criteria is valid and reliable.the researchers pointed out that patients who are dissatisfied with the health service provided comply with the recommendations of the healthcare personnel less and thus the primary and secondary preventive services are adversely affected.although treatment satisfaction is a very important factor in terms of patient adherence to treatment, there is no valid and reliable scale in turkish to evaluate this factor. this study was carried out to investigate the validity and reliability of the turkish version of the functional assessment of chronic illness therapy - treatment satisfaction - general questionnaire in the patients receiving health care.


Eligibility criteria:
inclusion criteria: getting at least 3 sessions of physiotherapy and rehabilitation services, to be at the cooperative level to understand and answer the surveys. volunteering to participate in the study. exclusion criteria:
spanish atrial fibrillation and resynchronization study

Conditions: Chronic Heart Failure; Cardiomyopathy, Dilated; Atrial Fibrillation

Summary: the aim of the present study is to compare the response to cardiac resynchronization therapy (crt) in patients with chronic advanced heart failure and permanent atrial fibrillation (af) depending on atrio ventricular junction (avj) is ablated or not.

cardiac resynchronization therapy (crt) improves the functional capacity and the quality of life and reduces the mortality of patients with dilated cardiomyopathy, low ejection fraction and wide qrs. only 2% of patients included in crt randomized trials were in af. to obtain a good response to crt, percentage \>90% of ventricular pacing must be obtained. based on observational studies, current guidelines of crt recommend the atrio ventricular junction (avj) ablation in those patients with permanent atrial fibrillation.

Eligibility criteria:
inclusion criteria: * dilated cardiomyopathy (lvedd \>56 mm) * wide qrs (\> 120 msec) * nyha iii-iv * ef\<35% * permanent af

Keywords: permanent atrial fibrillation resynchronization therapy ablation
safety and immunogenicity of a melan-a vlp vaccine in advanced stage melanoma patients

Conditions: Melanoma

Summary: the purpose of this study is to evaluate whether vaccination with a melan-a vlp vaccine leads to a specific cellular immune response in patients with malignant melanoma at advanced stage of the disease.


Eligibility criteria:
inclusion criteria: * able to provide written informed consent * able and willing to complete all protocol requirements * age: 18 years and older * histologically confirmed stage iii or iv melanoma * hla-a\*0201 haplotype * expected survival of at least 6 months * ecog performance status of 0 or 1 * at least one and no more than 2 previous systemic therapies for metastatic melanoma * able to undergo computed tomography (ct) scan/ magnetic resonance imaging (mri) scan for tumor assessment. * lack of response to or progression after most recent systemic therapy for metastatic melanoma. * adequate organ and bone marrow functions * all adverse events (aes) from prior anticancer therapy have resolved to ≤ grade 1 * sexually active males should use adequate contraception throughout the study period and 3 months thereafter. * females of child bearing potential should use adequate contraception throughout the study period and 3 months thereafter, that can be oral contraception or a double-barrier local contraception (intra-uterine device plus condom or spermicidal gel plus condom), and have a negative serum pregnancy test within 4 weeks prior to the first dose of the vaccine. exclusion criteria: * pregnant or nursing * use of an investigational drug within 30 days before enrollment * known or suspected brain metastases * active malignancy in the 5 years prior to enrollment other than melanoma, basal cell carcinoma or cervical carcinoma in situ. * major surgery within 4 weeks prior to enrollment. * current use of an immunosuppressive drug or any concomitant medication that could potentially interfere with the study drug. * presence of significant cardiovascular, renal, pulmonary, endocrine, infectious, or neurological disorders. * serum tests positive for hiv, hepatitis b virus (hbv), hepatitis c virus (hcv). * active autoimmune diseases or severe allergies. * known type 1 allergy. * current diagnosis or history of relevant and severe psychiatric disorder that compromises the patient's ability to understand the patient information, to give informed consent, to comply with the trial protocol or to complete the study. * blood donation or loss of \> 400ml within 8 weeks prior to inclusion. * hemoglobin (hb) \< 10g/dl * abuse of alcohol or other recreational drugs. * previous vaccination with a melan-a peptide analogue.

Keywords: metastatic malignant melanoma of the skin
a study on the regenerative treatment of tympanic membrane with npc-18 and fbg-18

Conditions: Tympanic Membrane Perforation

Summary: to assess the efficacy and safety of the regeneration treatment of tympanic membrane with npc-18 and fbg-18.


Eligibility criteria:
inclusion criteria: 1. written informed concent obtained 2. at the time of obtaining informed consent, ages ranged from over 20 to 80 3. at the time of obtaining informed consent, only those with tympanic membrane perforation in 1 ear (not both) for over 6 months (perforation in both ears is excluded) exclusion criteria: 1. tmp caused by burn or radiation therapy 2. in patients with chronic otitis media with ear drum perforation, tympanic cavity are not dry 3. inflammatory,infection or otorrhea in patient's eardrum,earcanal,middle ear and tympanic cavity 4. no invasion of epithelial and no cholesteatoma in tympanic cavity or around perforated edge 5. history of tympanoplasty 6. a part of the eardrum adhered to tympanic cavity 7. by temporal bone ct, soft tissue shadow in mastoid antrum or tympanic (using a ct image of less than 12 wks before patients registration) 8. abnormality in the chain and ear ossicles 9. air-bone gap difference more than 25db by patch hearing test 10. unable to see whole edge of tmp due to narrow external auditory canal 11. unable to wash out ear drops during the treatment period 12. presenting with uncontrolled diabetes (ngsp hba1c 6.9% and more) 13. presenting with autoimmune disease 14. history of malignancy within 3 years prior to obtained informed concent 15. administration of immunosuppressive agent or anti-cancer-agent 16. history of allergic reaction to local anesthetic(xylocaine), bfgf(fiblast spray), fibrin glue(beriplast p combi set, etc.), gelatin sponge formulation(spongel) and others 17. though out the period from screening to treatment, patient who is unable to wash out "thrombolytic agent", "anticoagulant", "anti-platelet agent", "procoagulant agent", "anti-line solvent"and"aprotinin formulation"
restricted intravenous fluid regime effects on immunological indicators of elderly patients operated for abdominal cancer

Conditions: Surgery; Perioperative Care; Immunization; Abdominal Neoplasms

Summary: the purpose of this study is to find out whether perioperative fluid restriction influence on postoperative immunological function. and discuss the probable mechanism that fluid restriction regime effect on clinical data.

perioperative fluid administration is a common therapy in clinical practice, and recent regime has a history about 50 years. recently, clinicians found that traditional regime had some disadvantages, for example, pulmonary edema, increased cardiac load, increased body weight. and thus, perioperative fluid restriction regimen has been suggested and proved could reduce cardiopulmonary complications and mortality rates after major elective gastrointestinal surgery. patients were divided into two groups randomly and underwent restricted fluid regimen or traditional fluid regimen. we determined the complications and immunological function in two groups, and analyzed the relationship between complications and immunological changes. based on this we decide the probable mechanism that perioperative fluid restriction effects on complications.

Eligibility criteria:
inclusion criteria: * all patients(age≧65y) who was admitted for gastrointestinal cancer surgery were considered eligible if they had no life-threatening systemic disease (asa groups 1\~3) exclusion criteria: * lactation * mental disorders * language problems * smoking within two weeks * diabetes mellitus * renal insufficiency * disseminated cancer * secondary cancers * inflammatory bowel disease, or diseases hindering epidural analgesia
safety and efficacy study of nggt002 in pku adult subjects

Conditions: Phenylketonurias

Summary: this investigator initiated trial is an open-label, dose escalation study to evaluate the safety, tolerability and efficacy of nggt002, an aav derived investigational gene therapy product expressing human pah enzyme in adult phenylketonuria (pku) subjects with pah deficiency. all participants will receive a single administration of nggt002 and will be followed for safety and efficacy for 5 years.

this study will evaluate the safety and efficacy of nggt002 gene therapy with three dose cohorts in adult subjects with diagnosis of pku characterized by pah deficiency with confirmed pah gene mutations. nggt002 will be administered through intravenous infusion. dose escalation will be initiated from the low dose of nggt002. after evaluating the safety and efficacy data, a decision will be made regarding the cohort expansion or the escalation to the next dose level. the same process will be followed for subsequent dose cohorts.

Eligibility criteria:
inclusion criteria: * voluntarily sign informed consent form; * male and female subjects with diagnosis of pku caused by confirmed pah mutation as per the "clinical practice guidelines for phenylketonuria 2020"; * age ≥ 18 years; * phe concentration ≥ 600 μmol/l at screening and ≥ 600 μmol/l at least once within 2 years prior to screening; * subjects who are willing and able to maintain their baseline diet throughout the study, regardless of phenylalanine restriction, except at the investigator's request; exclusion criteria: * presence of anti-aav8 neutralizing antibody * prior gene therapy * positive hepatitis b virus surface antigen, hepatitis c virus antibody, anti-human immunodeficiency virus antibody or treponema pallidum-specific antibody * hepatic function abnormal: alanine aminotransferase (alt) or aspartate aminotransferase (ast) \> 1.5 × uln; alkaline phosphatase (alp) \> 1.5 × uln; total bilirubin (tbil) \> 1.5 × uln; international normalized ratio (inr) \> 1.3

Keywords: pah deficiency
adjuvant chemotherapy for high-risk retinoblastoma after enucleation

Conditions: Retinoblastoma

Summary: the purpose of this study is to determine whether 3 cycles of chemotherapy(cev) is not inferior to 6 cycles of chemotherapy(cev) in the treatment of stage i enucleated retinoblastoma.

this study will be a phase ⅲ open label interventional case series. patients with unilateral stage i enucleated retinoblastoma will be randomized to receive chemotherapy(cev) of 3 cycles or 6 cycles every 21 days. patients will be followed for 60 months.

Eligibility criteria:
inclusion criteria: * ability to provide written informed consent and comply with study assessments for the full duration of the study. * definite pathology signs of retinoblastoma, stage i base on international retinoblastoma staging system. * received enucleation in the study eye. * monocular retinoblastoma. exclusion criteria: * any previous disease in the study eye. * previous participation in any studies of investigational drugs within 1 month preceding day 0 (excluding vitamins and minerals). * history of chemical intervention for retinoblastoma in the study eye.
blood flow reserve: effects after training with heavy inspiratory exercises

Conditions: Coronary Artery Disease

Summary: the goal of this single-site, parallel-group, double-blind, sham-controlled randomized control trial is to examine the effect of high-intensity inspiratory muscle strength training (imst) on coronary blood flow assessed using positron emission tomography coronary perfusion imaging in patients with coronary artery disease (cad). the main question it aims to answer are: • if high-intensity imst will improve coronary blood flow in patients with cad, which could be assessed using positron emission tomography coronary perfusion imaging. participants will be asked to complete the 8-week high-intensity or low-intensity imst. researchers will compare high and low-intensity imst groups to see if coronary blood flow increases after imst.

coronary artery disease (cad) is a leading cause of morbidity and mortality. with the aging population, increasing number of patients with cad has frailty and immobility. the health benefits of traditional aerobic exercise have been well-established; however, alternative exercise programs, such as inspiratory muscle training (imst), may provide greater merits. imst is a form of exercise that engages the diaphragm and accessory respiratory muscles to repeatedly inhale against resistance, which can be achieved in less time and widely applicable even for immobile or frail patients compared to conventional aerobic exercise. since barriers to conventional exercise training include immobility, lack of time, and access to facilities, imst may be a beneficial exercise form that can overcome those factors. a previous study has shown that high-intensity imt can lower blood pressure and improved vascular endothelial function. improvements in endothelial function of coronary arteries could improve coronary blood flow, leading to the improvement of anginal symptoms as well as quality of life. imst might offer a widely applicable, feasible, time-efficient form of training for cad patients. our study will examine the preliminary efficacy of imst on coronary blood flow in patients with cad.

Eligibility criteria:
inclusion criteria: 1. ≥18 years of age; 2. patients referred to pet at the university of ottawa heart institute; 3. able to perform a respiratory exercise testing; and, 4. patients with clinical stability, including no change in medications for the past one month. exclusion criteria: 1. unstable angina or established diagnosis of severe valvular heart disease, hypertrophic obstructive cardiomyopathy, chronic obstructive pulmonary disease; 2. unable to follow training/breathing instructions; 3. unable to return for follow-up visit; 4. presence of any major non-cardiac problem that would adversely affect survival during the study in the opinion of the investigator; or, 5. unable to provide informed consent.

Keywords: inspiratory muscle strength training coronary artery disease positron emission tomography myocardial blood flow
safety and efficacy study of rt001 to treat moderate to severe lateral canthal lines

Conditions: Lateral Canthal Lines; Crow's Feet; Facial Wrinkles

Summary: the purpose of this study is to evaluate the safety and efficacy of rt001 to treat moderate to severe lateral canthal lines in adults.

this multi-center study evaluates the safety and efficacy of four different doses of rt001 compared to vehicle applied as a single, bilateral topical application in at least 60 subjects with moderate to severe lateral canthal lines. subjects will be randomized to one of the five treatment groups in a 1:1:1:1:1 ratio. follow-up visits are scheduled through 28 days post-treatment. the safety and efficacy of cohort 1 (through the day 14 visit) will be assessed prior to the decision to enroll subjects into cohort 2 if additional doses or treatment regimens need to be evaluated.

Eligibility criteria:
inclusion criteria: * female or male; 30 to 55 years of age * bilateral lateral canthal lines rated as moderate or advanced * willing to refrain from receiving facial fillers, laser treatments, use of any product that affects remodeling or a product that may cause an active dermal response in the treatment area beginning at screening through end of study * women of childbearing potential must be practicing and willing to continue to use an effective method of birth control during the course of the study exclusion criteria: * muscle weakness or paralysis in the area receiving study treatment * active skin disease or irritation at the treatment areas * undergone any procedures that may affect the lateral canthal region during the past 12 months prior to screening * previous treatment with botulinum toxin (any serotype) in the head or neck area within 9 months prior to baseline (day 0) * use of a topical steroid on either of the treatment areas or use of medications that suppress the immune system 30 days prior to screening and continuing through end of study (day 28) * any abnormality on the electrocardiogram (ecg) at screening or any history of clinically significant arrhythmia, unstable angina, myocardial infarction or congestive heart failure

Keywords: lateral canthal lines crow's feet facial wrinkles
market research - acceptability study for new mct fat products

Conditions: Ketosis; Epilepsy; Fatty Acid Oxidation Disorder; Malabsorption; Ketogenic Diet; Medium Chain Triglycerides

Summary: this is a market research, observational study to evaluate the tolerability and acceptability of mct supplements for young children, young people and adults with intractable epilepsy, glut-1 or pdhd from 3 years to adulthood.


Eligibility criteria:
inclusion criteria: * diagnosis of epilepsy requiring a ketogenic or a specialist diet as part of their treatment. * subjects new to taking or who are already taking an mct product and are willing to try each of the 3 study products for 7 days each. 21 days taking product with 2 days between each new product trialled. * those patients who have been taking mct in their diet should do so for a minimum of 4 weeks before commencement of the study. * patients aged 3 years of age to adulthood. * written informed consent obtained from patient or parental caregiver. exclusion criteria: * presence of serious concurrent illness * investigator's uncertainty about the willingness or ability of the patient to comply with the protocol requirements * participation in any other studies involving investigational or marketed products concomitantly or within two weeks prior to entry into the study * any patients having taken antibiotics over the previous 2 weeks leading up to the study * patients already on and mct product who have been taking it in their diet for less than 4 weeks * patients less than 3 years of age * patients over 20 years.
safety and efficacy of gabapentin for neuropathic pain in fabry disease

Conditions: Fabry Disease; Neuropathic Pain

Summary: the purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain in patients with fabry disease, and reducing their use of opioid analgesics. the investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. the primary endpoint is percent reduction in patients' use of hydrocodone-acetaminophen.

fabry disease is an x-linked lysosomal storage disorder which results from reduced activity of the enzyme α-galactosidase a. this reduced enzyme activity results in accumulation of globotriaosylceramide (gl-3), which causes disturbances in multiple organ systems. patients often complain of acroparesthesia, caused by small-fiber neuropathy. limited information is available regarding effective treatments for small-fiber neuropathic pain in fabry disease, and no standard-of-care has yet been established. opioid analgesics are often used because of their pharmacokinetic properties. while effective, the use of opioids has complications such as constipation, physical dependence and addiction. the purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain and reducing the use of opioid analgesics. the investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. the primary endpoint is percent reduction in hydrocodone-acetaminophen use.

Eligibility criteria:
inclusion criteria: * diagnosis of fabry disease * age ≥ 18 years of age at study enrollment * current neuropathic pain at any severity level exclusion criteria: * known sensitivity or allergy to study drug * history of illicit drug use * pregnancy * suicidal thoughts at study enrollment as assess by the c-ssrs

Keywords: fabry disease neuropathic pain gabapentin