Kornimate/medical-research-clean huggingface dataset:
a real-world long-term safety and immunogenicity study of olipudase alfa therapy in pediatric patients less than 2 years of age with acid sphingomyelinase deficiency (asmd)

Conditions: Niemann-Pick Diseases; Acid Sphingomyelinase Deficiency

Summary: us, multicenter, cohort, open label observational study with primary data collection. ancillary protocol-specified procedures to address the study objectives (eg, assessment of ada) may be considered outside the standard of care for acid sphingomyelinase deficiency (asmd), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment. the total overall study duration will be 5 years. the follow-up period will be a minimum of 1 year to a maximum of 3 years. the enrollment period will be up to 4 years, to allow a minimum of 1 year of follow-up for the last participant enrolled.

this is a hybrid study design. patients need not travel to existing study sites in order to enroll.

Eligibility criteria:
inclusion criteria: * the participant must have asmd type a/b or b and must be \<2 years of age at the time of treatment initiation, or asmd type a (without age restriction). * the participant must weigh ≥ 2 kg \[the united states prescribing information (uspi)\] for olipudase alfa specifies this minimum weight for infants receiving olipudase alfa). * the participant must have documented asmd, as determined in peripheral leukocytes, cultured fibroblasts, or lymphocytes and/or by genotype determination. * signed informed consent must be provided by the participant's parent(s)/legal guardian(s), including compliance with the requirements and restrictions listed in the informed consent form (icf) and in this protocol. the signed icf must be provided before any protocol-related procedures are performed. * the participant is eligible to start olipudase alfa enzyme replacement therapy or has received the first dose (and no more) of olipudase alfa, and has retrievable clinical, laboratory, and ada data. exclusion criteria: * the participant has received an investigational drug within 30 days or 5 drug half-lives before signature of the icf and study enrollment. * the participant is not suitable for participation for reasons determined by the investigator, including medical or clinical conditions, or potential risk of noncompliance with study procedures. * the participant is an immediate family member of employees of the study site or other individuals directly involved in study conduct, in conjunction with section 1.61 of ich-gcp ordinance e6.
quality of life (qol) registry for patients with al amyloidosis

Conditions: AL Amyloidosis; Amyloidosis

Summary: this is an online registry to document the psychometric properties of sf-36v2 among patients with al amyloidosis, to document patients' burden of disease, to better understand the patient's experience and to follow quality of life issues using a variety of qol measures.


Eligibility criteria:
inclusion criteria: * patients must have al amyloidosis exclusion criteria:
the predictors and patient versus parental perceptions of health-related quality of life in pediatric chronic pain

Conditions: Pediatric Chronic Pain

Summary: this study is intended to provide additional insight into the factors affecting health-related quality of life with pediatric chronic pain as perceived by the patient versus his or her parents.in doing so, additional insight into the subjective interpretation the pediatric chronic pain experience will be gained. the four objectives of this study will be (a) to examine further the relationship between patient self-reported health-related quality of life and parent proxy-reported health-related quality; (b) to identify what biological, psychological, and/or social factors are the strongest predictors of a pediatric chronic pain patient's self-reported pain intensity and self-reported health-related quality of life, in a diverse cohort of patients referred to and subsequently treated by an anesthesiology-based yet interdisciplinary pediatric chronic pain medicine program; (c) to assess the effect of patient-specific, pain-focused biopsychosocial treatment regimen on pain intensity and health-related quality of life; and (d) to determine the effect of patient/parental satisfaction with on-going health care on their compliance with and uptake of the pain treatment regimen.


Eligibility criteria:
inclusion criteria: * outpatients ranging between 8 years and 18 years of age directly referral from either their primary care physician or another specialist physician exclusion criteria: * patients in whose biologic families english is not the primary, native language * patients suffering from severe cognitive dysfunction (i.e., mental retardation) * patients with a life-expectancy of less than three months at the time of initial clinical evaluation
aggrastat to zocor (atoz) - the use of two approved drugs to treat patients who have experienced chest pain or a heart attack (0733-180)

Conditions: Acute Coronary Syndrome

Summary: a-phase: evaluating patients with chest pain who are receiving approved drugs, to estimate the effectiveness of one type of blood thinner as compared to another type of blood thinner. z-phase: to evaluate early treatment of patients with long term chest pain (using an approved drug for 30 days, followed by an increased dose of the drug) as compared to patients (treated with diet and 4 months placebo followed by diet and approved drug) in patients who have experienced acute chest pain or heart attack.


Eligibility criteria:
inclusion criteria: * a-phase: chest pain at rest for 10 minutes, ekg changes or elevated cardiac blood work * z-phase: elevated cholesterol , plus at least one risk factor ( \> 70 years old, diabetes, history of prior heart or blood vessel disease, chest pain with ekg changes, elevation of cardiac lab work or positive cardiac tests , at least 2 heart vessels blocked \[one \>= 75% and one \>= 50%\]) exclusion criteria: * a-phase: use of some specific cardiac drugs, high risk bleeding, prior blood clotting disorders * z-phase: elevation in certain cardiac blood tests, no significant heart damage at catheterization, planned cardiac surgery or specific cardiac drugs that lower cholesterol levels, within 6 weeks of enrollment
paracetamol uniflash for buccal use phase iii trial in acute pain due to a tooth extraction

Conditions: Acute Pain

Summary: this study aims to evaluate the analgesic efficacy of single dose of a new paracetamol formulation (paracetamol uniflash) for buccal use in comparison to two different doses of an oral paracetamol form controlled versus placebo.


Eligibility criteria:
inclusion criteria: * male and female patients aged from 18 years of age * patients scheduled to undergo the removal of one third soft-tissue impacted or not-impacted mandibular molar (associated or not to an ipsilateral erupted maxillary molar) under short-acting local anaesthetic preoperatively; * patients weighing \> 50 kg; additional inclusion criteria * patients experiencing a moderate pain within 4 hours after the dental extraction, defined by a baseline pain intensity visual analogic scale (vas) score ≥ 40 mm and ≤ 60 mm; * third mandibular molar extraction completed without any immediate complication, that in the opinion of the investigator, would interfere with the study conduct and/or assessments (e.g., suspected neurosensory complication, incomplete removal of tooth). exclusion criteria: * patient who undergoes an extraction of contralateral molar in the same procedure or a bony-impacted molar; * patients treated by analgesics or nonsteroidal anti-inflammatory drugs (nsaids) within the 3 days prior to the day of randomization (or within 5 times the elimination half-life whichever the longest); * patient who received other analgesic than short-acting preoperative or intraoperative local anaesthetic agents within 12 hours before the start of the surgery or peri-operatively until randomization; * patients with any known hypersensitivity to paracetamol, ibuprofen or ingredients contained in imps and non-investigational medicinal product (nimp);

Keywords: paracetamol
micropulse transscleral laser therapy and its short-term impact on ocular surface

Conditions: Conjunctiva; Cornea

Summary: micropulse transscleral laser therapy (tlt) is proven to be effective in reducing intraocular pressure with minimal complications in either primary or secondary glaucoma. however, its impact on the human ocular surface remains unexplored. this study aims to bridge this gap by examining the clinical and histopathological effects of micropulse tlt on the ocular surface.

this prospective study, conducted at mohamed taher maamouri university hospital in nabeul, tunisia, investigates the clinical and histopathological short-term effects of micropulse transscleral laser therapy (tlt) on the ocular surface. the study involves 16 eyes from 15 adult patients. participants underwent micropulse tlt, followed by conjunctival biopsies for the analysis of inflammation and fibrosis. participants also benefited from ophthalmological examination and responded to the ocular surface disease index (osdi) questionnaire before and after micropulse tlt.

Eligibility criteria:
inclusion criteria: * age \> 18 years old * non-controlled open-angle glaucoma despite undergoing maximal medical therapy * poor therapeutic adherence * local or general intolerance to topical treatments. exclusion criteria: * ophthalmic or general diseases that could potentially impact the conjunctiva such as symblepharon, ocular injury, or prior eye surgery * pevious transscleral laser therapy applied to the same eye * angle-closure glaucoma * active ocular inflammation * a non-functional eye (absence of light perception) * scleral thinning

Keywords: micropulse transscleral laser therapy ocular surface inflammation fibrosis glaucoma
administration of platelet-rich fibrin to autologous fat tissue in injection laryngoplasty for vocal cord paralysis

Conditions: Vocal Cord Paralysis

Summary: the study tries to see the effect of using a combination of platelet-rich fibrin (prf) and autologous fat as a filler for injection laryngoplasty procedures to treat unilateral adductor vocal cord paralysis.

the vocal cord in humans is essential in producing voice used in communication and interaction between us. vocal cord paralysis causes dysphonia, which interferes with communication, causing disruptions towards social activity and daily activities. one of the managements for vocal cord paralysis is medialisation and augmentation of the vocal cord through injection laryngoplasty. autologous fat is one of the best fillers that can be used in this procedure, but it is highly absorbable and can be reabsorbed very quickly when injected to body tissues. platelet rich fibrin (prf) is a biomaterial consisting of growth factors that are thought to improve fat tissue longevity through increase of adipogenesis and angiogenesis. improvement in fat longevity will improve clinical outcomes after laryngoplasty procedure potentially reducing number of repeated injections needed to achieve satisfactory resolution to vocal cord paralysis. the study evaluates a combination of prf and autologous microlobular fat compared with autologous microlobular fat alone on laryngoplasty. subjective evaluation was done by using voice handicap index (vhi-30) questionnaire, while objective evaluation was conducted via computerized acoustic analysis/multidimensional voice program (mdvp), videostroboscopy, and maximum phonation time.

Eligibility criteria:
inclusion criteria: * subject is diagnosed with unilateral vocal cord paralysis in paramedian position or 3 months lateral onset without movement and mucosal waves of the vocal cords on videostroboscopy. * willing to give consent exclusion criteria: * has a history of malignancy of the larynx or lung * subject isn't able to undergo injection laryngoplasty procedure under general anesthesia * subject with thrombositopenia

Keywords: platelet-rich fibrin vocal cord paralysis dysphonia injection laryngoplasty
clinical study for the treatment of peripheral nerve defects with neuromaix

Conditions: Patients With a Scheduled (Diagnostic) Nervus Suralis Biopsy

Summary: the aim of this study is the development and initial clinical application of the nerve guide neuromaix in humans to provide evidence for the safety and performance of the device. neuromaix is intended to be used as a guiding structure to bridge a peripheral nerve discontinuity, and to create a conduit for axonal growth across the nerve gap.

the neuromaix nerve guide is an absorbable implant composed of porcine collagen, used to bridge a peripheral nerve discontinuity, and to create a conduit for axonal growth across the nerve gap. neuromaix provides a protective environment for peripheral nerve regeneration after injury. neuromaix connects the proximal and distal ends of a transected nerve, allowing regenerating axons to grow through the scaffold, into the distal nerve tissue towards the target muscle or skin. neuromaix is composed of two parts. the epimaix part provides the structural characteristics needed to suture the nerve guide in place and to prevent ingrowth of scar tissue. the perimaix part provides a structure that mimics endoneurial tubes, providing guidance for the regenerating axons while bridging the nerve gap.

Eligibility criteria:
inclusion criteria: * both genders in the age between 18 und 70 years * patients who are scheduled for a nerve biopsy exclusion criteria: * alcohol-related polyneuropathy * paraneoplastic polyneuropathy * present: immunosuppressive therapy * present: malignant tumor * peripheral vascular diseases * collagen diseases (e.g. existing keloid scars) * patients with an increased wound healing disorder (e.g. diabetics) * patients with chronic venous insufficiency (vein thrombosis, skin diseases) * patients with coagulation and bleeding disorders (asa- or marcumar-patients) * present: pregnancy * hiv, hepatitis b or hepatitis c infection

Keywords: nerve biopsy nerve regeneration neuroma pain loss of sensation
randomized controlled trial of virtual reality

Conditions: Muscle Spasticity

Summary: measure pain and anxiety during a putative painful medical procedure (i.e., botulinum toxin injections) when virtual reality is employed compared to standard of care only using a randomized controlled trial study design.


Eligibility criteria:
inclusion criteria: * patients receiving botulinum toxin injections at gillette children's specialty healthcare exclusion criteria: * patients who are fully anesthetized during their medical procedure * non-english speaking parents/patients * patients with history of motion sickness * patients with epilepsy * patients with a ventricular shunt * patients who have surgery during the study time period

Keywords: botulinum toxin injections virtual reality
maat for young adult cancer survivors

Conditions: Cancer

Summary: the goal of this clinical trial is to pilot-test a modified version of telehealth-delivered memory and attention adaption training (maat) that is tailored to young adult childhood cancer survivors (ages 18-39; maat-ys) with cancer-related cognitive impairment (crci). maat-ys consists of 8 weekly visits (45-minutes in duration) and participants use a survivor workbook and complete homework between visits. participants in this single-group pilot trial (n=9) will complete online self-report measures of cognitive symptoms, quality of life, treatment satisfaction and a brief online neuropsychological test battery at baseline and post-maat-ys timepoints.

the goal of this clinical trial is to pilot-test a modified version of telehealth-delivered memory and attention adaption training (maat) that is tailored to young adult childhood cancer survivors (ages 18-39; maat-ys) with cancer-related cognitive impairment (crci). maat-ys consists of 8 weekly visits (45-minutes in duration) and participants use a survivor workbook and complete homework between visits. this pilot trial will determine maat-ys feasibility, treatment satisfaction and size of effect in self-report and objective measures of neurocognitive function. eligible participants will be 9 individuals who were diagnosed with non-central-nervous system (cns) cancer (including leukemia/lymphoma without known cns involvement) prior to the age of 18, are at least 1-year from completion of cancer treatment, including chemotherapy. participants will complete online self-report measures of cognitive symptoms, quality of life, treatment satisfaction and a brief online neuropsychological test battery at baseline and post-maat-ys timepoints. if successful, further research is planned with a larger, multi-site randomized controlled trial of maat-ys.

Eligibility criteria:
inclusion criteria: 1. adults aged 18-39 2. diagnosed prior to 18 years of age with non-cns solid tumor or leukemia/lymphoma without known cns involvement 3. at least 1 year after completion of treatment including chemotherapy, cancer-free 4. english fluent 5. memory or other cognitive problems attributed to cancer and/or cancer treatment 6. score of \<10 on the fact-cog impact on quality of life scale 7. willing to use telehealth with internet access 8. willing to provide informed consent to participation exclusion criteria: 1. severe non-cancer brain injury such as severe traumatic brain injury, stroke or toxic injury causing memory impairments; 2. currently meeting diagnostic and statistical manual-5 (dsm-5) criteria for a severe psychiatric disorder, including substance abuse, mood, anxiety, or psychotic disorders, as assessed by the prime-md; 3. scoring 3 or below on the 6-item cognitive screen designed to detect severe memory disorders;59 4. severe uncorrected sensory impairment (severe hearing or visual impairment).

Keywords: cancer cancer survivorship cancer-related cognitive impairment crci cognitive-behavioral therapy memory attention and adaptation training
effects of n-acetyl cystein (nac) supplementation in g6pd deficient individuals after acute exercise

Conditions: G6PD Deficiency

Summary: the purpose of this study is to investigate the effects of n-acetyl cystein (nac) supplementation on redox status, physiological and biochemical parameters in g6pd deficient individuals after acute exercise.

in a randomized double-blind, crossover design, 12 adult volunteers with g6pd deficiency of both sexes will be supplemented with either 10 mg/kg of nac (experimental condition - ec) or placebo (control condition - cc) every day for 4 weeks. before intervention, all participants will be informed about the study protocol, fill a medical history questionnaire and sign an informed consent form. moreover, measurements of anthropometric characteristics and physiological parameters, as well as a vo2max test will be performed. participants will perform 4 trials of exercise (70% vo2max for 45min and 90% till exhaustion) before and after each condition. blood samples will be collected before, immediately after and 1 hour after exercise. moreover, measurements of anthropometric characteristics and physiological parameters will be performed before and after each condition. there will be a washout period of at 4 weeks between conditions.

Eligibility criteria:
inclusion criteria: * g6pd deficiency exclusion criteria: * any uncontrolled health condition for which exercise is contraindicated * current use of dietary supplements or drugs * pregnant, pregnancy intention or breast feeding
indocyanine green versus blue dye for detection of sentinel lymph node in endometrial cancer

Conditions: Endometrial Neoplasms

Summary: to determine the difference in the proportion of hemipelves with successful detection of sentinel lymph node according to the dye used (indocyanine green with near-infrared imaging vs blue dye) in women with endometrial cancer.

in the past few years, sentinel lymph node (sln) procedure has emerged as an interesting solution to the debate on lymphadenectomy in endometrial cancer. using blue-dye is inexpensive but results in an unsatisfactory detection rate of sln. indocyanine green (icg) with near-infrared imaging is a novel technology for sln mapping and initial reports on the use of icg have shown high detection rate. in the present study, we aim to determine precisely the increase in detection rate of sln associated with the use of icg instead of blue dye. this randomized controlled trial will include patients with preoperative stage i endometrial carcinoma. all included women will undergo sln mapping with blue dye in one hemipelvis and icg in the other hemipelvis. randomization will concern the side of the hemipelvis in which blue dye vs icg mapping is used, so that the patient's contralateral hemipelvis will serve as a control to her ipsilateral hemipelvis. the primary endpoint is the difference in the proportion of hemipelves with successful detection of sln according to the dye used. the unique precise information gained from the present study will contribute to determine whether the benefit of the use of icg over blue dye is sufficient to justify the expense of its use.

Eligibility criteria:
inclusion criteria: * all patients with endometrial carcinoma proven by endometrial biopsy or curettage and * preoperative figo (international federation of gynecology and obstetrics) stage i and * intended for staging via laparoscopic or robotic surgery and * patients must be older than 18 year-old, able to read french or english. exclusion criteria: * preoperative figo stages ii to iv * previous pelvic or paraaortic lymphadenectomy or radiotherapy * surgery that could change the uterine lymphatic drainage (myomectomy) * iodine allergy * pregnancy.

Keywords: indocyanine green sentinel lymph node biopsy