Kornimate/medical-research-clean huggingface dataset:
a study to assess the long-term safety and clinical activity of mrna-3927 in participants previously enrolled in the mrna-3927-p101 study

Conditions: Propionic Acidemia

Summary: the main purpose of this study is to evaluate the long-term safety of mrna-3927 administered to participants with propionic acidemia (pa) who have previously participated in study mrna-3927-p101 (nct04159103).

the study will assess long-term safety and clinical activity of mrna-3927. participants with pa who were previously enrolled and completed the end-of-treatment (eot) visit of the mrna-3927-p101 study will have the option to enroll into this extension study provided all eligibility criteria have been met. the study will include 2 periods: 1) treatment period and 2) follow-up period (up to 2 years after the last dose of study drug). all participants will enter the study receiving mrna-3927 at the same dose and at the same dosing interval last received in the mrna-3927-p101 study.

Eligibility criteria:
inclusion criteria: * participated in study mrna-3927-p101. * completed the eot visit in study mrna-3927-p101 within 10 days of first dose of mrna-3927 in current study. exclusion criteria: * not expected to receive clinical benefit from continued mrna-3927 administration, in the opinion of the investigator. * any clinical or laboratory abnormality or medical condition that, at the discretion of the investigator, may put the individual at increased risk by participating in this study. * history of liver and/or kidney transplant.

Keywords: mrna-3927 propionic aciduria metabolism, inborn errors genetic diseases inborn amino acid metabolism, inborn errors acidosis acid-base imbalance metabolic diseases organic acidemias moderna mrna
506u78 in treating patients with relapsed or refractory t-cell acute lymphoblastic leukemia or t-cell lymphoblastic lymphoma

Conditions: Leukemia; Lymphoma

Summary: rationale: drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. purpose: clinical trial to study the effectiveness of 506u78 in treating patients who have relapsed or refractory t-cell acute lymphoblastic leukemia or t-cell lymphoblastic lymphoma.

objectives: * provide an investigational agent, 506u78, to physicians for the management of individual patients with relapsed or refractory t-cell acute lymphoblastic (lymphocytic) leukemia or t-cell lymphoblastic lymphoma who are not candidates for entry onto ongoing research clinical trials of higher priority. * evaluate the toxic effects of 506u78 when administered as a 2 hour iv infusion 3 days a week every 21 days in these patients. * evaluate the antitumor efficacy of this regimen in terms of rate of complete responses in these patients. outline: patients receive 506u78 over 2 hours on days 1, 3, and 5. treatment repeats every 21 days for a maximum of 12 courses in the absence of unacceptable toxicity or disease progression. patients are followed every 3 months for one year and then every 6 months until death. projected accrual: a minimum of 25 patients will be accrued for this study.

Eligibility criteria:
disease characteristics: * histologically confirmed relapsed or refractory t-cell acute lymphoblastic leukemia or t-cell lymphoblastic lymphoma * tumor cells should exhibit phenotypic characteristics of these diseases * no cns involvement requiring intrathecal or craniospinal radiotherapy * must not be eligible for intergroup, cooperative group, or local research studies of higher priority patient characteristics: age: * 16 and over performance status: * ecog 0-3 or * karnofsky 40-100% life expectancy: * not specified hematopoietic: * not specified hepatic: * bilirubin no greater than 2.0 mg/dl renal: * creatinine no greater than 1.5 mg/dl or * creatinine clearance at least 50 ml/min other: * no neuropathy grade 2 or higher * no history of significant neurological toxicity (grade 2 or greater) associated with prior chemotherapy or radiotherapy * no active seizure disorder * no active infection * no other active concurrent malignancy except curatively treated basal cell carcinoma or carcinoma in situ of the cervix * not pregnant or nursing * fertile patients must use effective contraception prior concurrent therapy: biologic therapy: * prior bone marrow transplantation allowed * no concurrent allogeneic bone marrow transplantation chemotherapy: * at least 3 weeks since prior chemotherapy (6 weeks for nitrosourea or mitomycin) and recovered * no prior 506u78 endocrine therapy: * no concurrent systemic steroid therapy radiotherapy: * see disease characteristics * prior radiotherapy allowed surgery: * not specified other: * no other concurrent investigational therapy * no concurrent treatment for seizures

Keywords: recurrent adult acute lymphoblastic leukemia t-cell adult acute lymphoblastic leukemia recurrent adult lymphoblastic lymphoma
producing a fully asynchronous online savvy program

Conditions: Dementia

Summary: this is a pilot/feasibility study to develop and test a fully online, self-administered psychoeducation program to enhance the caregiving mastery of family and friends who provide unpaid care for persons living with alzheimer's and similar dementia disorders (plwd).

this study seeks to develop and test a fully online, self-administered psychoeducation program to enhance the caregiving mastery of family and friends who provide unpaid care for persons living with alzheimer's and similar dementia disorders (plwd). as the number of plwd rises in the united states from the perhaps 7 million today to possibly 15 million in 2050, the healthcare system will rely on those caregivers even more. it is well established that family caregiving is taxing and stressful and that managing the day-to-day life of plwd and handling the behavioral and psychological symptoms in dementia (bpsd) that they may exhibit are the main sources of stress. group-based psychoeducational programs such as the savvy caregiver program (scp) have demonstrated that the acquisition of skills, knowledge, and caregiving mastery can ameliorate caregiving stress - and enhance plwd quality of life. many factors, however, preclude caregivers' attendance in group-based programs and limit programs' scalability. to address the issue of access, the researchers have developed the tele-savvy program, an online version of scp that brings groups of caregivers together in facilitator-led synchronous groups and provides substantive educational augmentation through asynchronous e-mail-delivered video lessons. the first aim of this study is to develop a fully asynchronous online savvy program that incorporates learning activities that promote both knowledge and skill acquisition and develop and enhance caregivers' felt-sense of caregiving mastery. developing this education program will utilize input from clinicians, educators, tele-savvy facilitators, and caregiver advisors. the second study aim is to determine the feasibility, acceptability and preliminary efficacy of the created online program. this study will recruit 60 family caregivers to take part in a no-control trial of the program. data will be gathered at baseline, immediately upon course completion and at 3 months post-baseline to assess caregiver distress measures (depression, strain, burden, anxiety), caregiver competence/ mastery, and care recipient quality of life.

Eligibility criteria:
inclusion criteria: * primary unpaid caregiver for a community-dwelling plwd * provide at least 10 hours of direct care per week * able to read and understand english * be able to access the course online * naïve to savvy or tele-savvy exclusion criteria: * cannot provide consent * prisoners * cognitively impaired adult * not able to clearly understand english

Keywords: dementia caregiver
efficacy of topical lidocaine to decrease discomfort with intranasal midazolam administration

Conditions: Pain

Summary: the purpose of this study was to establish whether premedication with topical lidocaine would reduce the pain associated with in midazolam administration in children. the study was designed to be a double-blinded, randomized, placebo-controlled trial performed in an urban, academic pediatric emergency department.

this was a double-blinded, randomized, placebo-controlled trial performed in an urban, academic pediatric emergency department with an annual census of 67,000 patients. children 6-12 years old for whom in midazolam was ordered were eligible for enrollment. patients were randomly assigned an identical intranasal medication (4% lidocaine or 0.9% saline). patients were administered the study drug followed by in midazolam. patients then assigned a pain score for midazolam administration using the wong-baker faces pain rating scale. the primary endpoint of pain score was analyzed with a two-tailed mann-whitney u test, with p \< 0.05 considered statistically significant.

Eligibility criteria:
inclusion criteria: * ages 6-12 * previously healthy * attending physician concludes that the patient would benefit from administration of an anxiolysis medication prior to a minor procedure exclusion criteria: * moderate to severe asthma or other chronic lung disease * co-morbid conditions including cerebral palsy, developmental delay, or other chronic illness deemed by the attending physician to be unsafe to receive anxiolysis with versed. * any child presenting with a life-threatening condition.

Keywords: children midazolam lidocaine intranasal
to demonstrate the relative bioavailability of desipramine hydrochloride 50 mg tablets

Conditions: Depression

Summary: to demonstrate the relative bioavailability of desipramine hydrochloride 50 mg tablets.


Eligibility criteria:
inclusion criteria: * no clinically significant abnormal finding on physical exam, medical history, or clinical laboratory results on screening. exclusion criteria: * positive test results for hiv or hepatitis b or c. * treatment for drug or alcohol dependence.

Keywords: antidepressant
to evaluate the preliminary efficacy and safety of gr1501 injection in patients with active axial spondyloarthritis

Conditions: Axial Spondyloarthritis

Summary: dosage: 100mg or 200mg or 300mg administration frequency: q2w administration in the first 4 weeks (w0, w2, w4), and subsequent q4w administration (w8, w12) administration: subcutaneous injection specifications: 100mg/ 1ml/bottle or placebo 0mg/1ml/ bottle

this is a randomized, double-blind, placebo-controlled phase ii clinical trial evaluating the preliminary efficacy, safety, pharmacokinetic, and immunogenicity of gr1501 injection (il-17a antiboby) at different doses and dosing frequency in patients with active axial spinal arthritis. a total of 160 patients were enrolled in the planned trial, and after passing the screening period of 2 weeks, they were randomly assigned to the 100mg group, 200mg group, 300mg group or placebo group according to 1:1:1:1.after the completion of the 16-week administration period, the follow-up period (\~24 weeks) was initiated.

Eligibility criteria:
inclusion criteria: 1. according with the diagnosis of axial spondyloarthritis (axspa) by the assessment of spondyloarthritis international society (asas);, 2. during the screening period, the bath ankylosing spondylitis disease activity index (basdai) ≥ 4 points and the spinal pain (basdai article 2) nrs score ≥ 4 points; exclusion criteria: 1. previous or current crohn's disease; 2. suffering from ulcerative enteritis and requiring immunosuppressive therapy at present;

Keywords: il-17a axial spondyloarthritis
modified aortic root enlargement procedure

Conditions: Small Aortic Annulus; Aortic Root Enlargement Procedure

Summary: this study is a prospective observational clinical trial, in which severe as patients aged 50-70 years with saa choose modified are or tavr according to their wishes to investigate the safety and efficacy of modified are; compare the incidence and clinical outcomes of moderate to severe ppm after modified are and tavr surgery; evaluate the feasibility of postoperative mid valve tavr and explore the most ideal initial surgical plan for this type of patient from a full lifecycle perspective.

small aortic annulus (saa), which is prevalently observed in patients with severe aortic stenosis (as), constitutes a significant challenge in treatment and may give rise to post-operative prosthetic valve mismatch (ppm) and an elevated late mortality rate. the optimal approach for treating saa remains controversial. transcatheter aortic valve replacement (tavr) has yielded superior valvular hemodynamic outcomes in patients with saa; however, the incidence of ppm subsequent to surgery remains considerable. aortic root enlargement (are) represents an effective solution for saa, but the conventional method of aortic ring enlargement for are has restricted efficacy. in 2021, yang bo proposed an enhanced are approach, where the mitral curtain of the aortic valve was incised in an inverted y-shape along the left non-coronary junction, enabling the enlargement of the number 3-5 of the aortic ring

Eligibility criteria:
inclusion criteria: * 1) symptomatic severe as patients aged 50-70 years who are planning to undergo biological valve replacement (diagnostic criteria for echocardiography are: mean transvalvular pressure gradient ≥ 40mmhg or flow velocity ≥ 4.0 m/s, aortic valve area (ava) ≤ 0.8 cm2 or ieoa\<0.5 cm2/m2) 2) merge saa (defined as mean aortic annulus diameter ≤ 23 mm measured by ct) exclusion criteria: * 1) high surgical risk, sts score\>8% 2) severe lesions in multiple coronary arteries requiring pci or cabg 3) simultaneous surgical intervention is required for severe mitral and tricuspid valve disease 4) combining other complex cardiovascular diseases requires simultaneous surgical treatment 6) combined severe pulmonary arterial hypertension or left ventricular dysfunction (lvef\<40%) 7) severe respiratory, liver and kidney dysfunction, or other important organ dysfunction combined 8) concurrent infective endocarditis in active phase 9) previous history of cardiac surgery
phase 2 study of an ocular sirolimus (rapamycin) formulation in combination with lucentis® in patients with age-related macular degeneration

Conditions: Age-Related Macular Degeneration; Choroidal Neovascularization

Summary: the purpose of this study is to determine the safety and efficacy of an ocular sirolimus (rapamycin) formulation in combination with lucentis in patients with sub-foveal choroidal neovascularization secondary to age-related macular degeneration.


Eligibility criteria:
inclusion criteria: * diagnosed with sub-foveal choroidal neovascularization secondary to age-related macular degeneration within six months of initial study visit, and may have been treated with up to 3 lucentis® (ranibizumab) or 3 avastin® (bevacizumab) injections with the last injection administered at least 4 weeks prior to the initial study visit, or is treatment-naïve * visual acuity of 20/40 to 20/200 in the study eye exclusion criteria: * any other ocular disease that could compromise vision in the study eye * presence of other causes of choroidal neovascularization other than secondary to age-related macular degeneration

Keywords: wet amd
monitoring and evaluation study of project echo for ild

Conditions: Interstitial Lung Diseases

Summary: the study will utilize pre-post survey measures to evaluate project echo for ild with respect to an initial set of practice and clinical outcomes and relies on questionnaire data obtained from providers participating in project echo for ild at baseline, at 6 months, and at study end.

project echo (extension for community healthcare outcomes) is a knowledge-sharing model to expand the capacity of the health care workforce so that more people can get high quality care for their health conditions in or near the communities where they live. the model brings specialty disease expertise to community providers through its hub-and-spoke networks. the model relies on videoconferencing to connect local providers in non-urban or underserved communities (spoke sites) with an interdisciplinary team of specialist providers at academic medical centers (hubs) during virtual "teleecho" clinic sessions, which include brief educational lectures and case-based, experiential learning. it is a guided, technology-enabled collaborative practice model in which community providers become experts in an area of community need within the scope of their practice, operating with increasing independence as their skills and self-efficacy grow. the model establishes meaningful, ongoing relationships with hub specialists who serve as telementors for spoke providers and relies on case-based learning from presentations of real-world de-identified patient cases. the overarching goal of the project is to implement and evaluate an adaptation of the echo model to interstitial lung disease (ild) diagnosis and care among primary care physicians (pcps) and community pulmonologists in non-urban or underserved communities.

Eligibility criteria:
inclusion criteria: * pulmonologists and primary care physicians who participate in project echo for ild. exclusion criteria: -

Keywords: progressive fibrosing interstitial lung disease idiopathic pulmonary fibrosis interstitial lung disease associated with systemic sclerosis myositis-associated interstitial lung disease sarcoidosis hypersensitivity pneumonitis pulmonary fibrosis
pharmacokinetic study of drug-coated angioplasty balloons in the superficial femoral or popliteal arteries:

Conditions: Peripheral Artery Disease

Summary: to describe the pharmacokinetics of paclitaxel in the blood delivered from a paclitaxel coated percutaneous angioplasty balloon catheter as a result of treatment of de novo or restenotic lesion(s), occluded/stenotic or re-occluded/restenotic lesion(s).

this study investigates the inhibition of restenosis using the cvi paclitaxel-coated pta catheter in the treatment of de-novo occluded/stenotic or re-occluded/ restenotic superficial femoral or popliteal arteries. the proposed clinical study will be a prospective, non-randomized, single arm, multi-center, pharmacokinetic study. the objective of the study is to describe the pharmacokinetics of paclitaxel in the blood delivered from the cvi paclitaxel-coated pta catheter as a result of de novo occluded/restenotic or re-occluded/restenotic lesion(s).

Eligibility criteria:
inclusion criteria: * male or non-pregnant female greater than or equal to 18 years of age. * subjects with symptomatic leg ischemia, requiring treatment of the superficial femoral artery (sfa) or popliteal artery. exclusion criteria: * pregnant or lactating females. * known intolerance to study medications, paclitaxel or contrast agents that in the opinion of the investigator cannot be adequately pre-treated.

Keywords: paclitaxel percutaneous transluminal angioplasty balloon catheter pharmacokinetic peripheral artery disease pad
newborn infant parasympathetic evaluation (nipe) index after standardized tetanic stimulations under general anesthesia

Conditions: Anesthesia

Summary: nipe monitor is the recently developed pediatric version of the ani monitor, which is used in adults to monitor analgesia during general anesthesia. in adults, under general anesthesia, ani decreases after a nociceptive stimulation. the amplitude of this decrease is related to the intensity of the stim. this study aims to investigate if nipe index decreases in the same manner after stimulations in anesthetized children.


Eligibility criteria:
inclusion criteria: * elective surgery under general anesthesia requiring tracheal intubation and muscle relaxation * written informed consent obtained from parents (and from child if appropriate) exclusion criteria: * thoracic or cardiac surgery * cardiac arrhythmia, pace maker * analgesic medication less than 24 hours before surgery * chronic anticholinergic medication * contraindication to muscle relaxants or general anesthetics

Keywords: monitoring general anesthesia analgesia pain pediatric
rivastigmine monotherapy and combination therapy with memantine in patients with moderately severe alzheimer's disease who failed to benefit from previous cholinesterase inhibitor treatment

Conditions: Alzheimer's Disease

Summary: this is a prospective, multicenter, open-label study. following screening and baseline assessments, eligible patients will be switched to rivastigmine and will enter the 16 week run-in rivastigmine treatment phase. after completion of assessments at the end of the run-in phase, patients who were not sufficiently stabilized on rivastigmine alone will receive add-on memantine to their rivastigmine treatment; patients who were stabilized on rivastigmine alone will have completed and be discontinued from the study.


Eligibility criteria:
inclusion criteria: * outpatients who have probable alzheimer's disease according to the dsmiv criteria * patients treated with donepezil (5-10 mg ) or galantamine (16- 24 mg) for at least 6 months * patients, in the investigator's clinical judgment, not stabilized on treatment with donepezil or galantamine exclusion criteria: * patients with evidence of severe or unstable physical illness, i.e., acute and severe asthmatic conditions, severe or unstable cardiovascular disorders, active peptic ulcer disease, hypersensitivity to cholinesterase inhibitors or memantine, clinically significant laboratory abnormalities or any patient with a medical condition which would prohibit them from completing the clinical trial

Keywords: alzheimer's disease, switch, rivastigmine, memantine
therapeutic exercises and stabilization exercises given after manual therapy

Conditions: Patients With Non-Specific Chronic Neck Pain

Summary: the study was conducted to determine and compare the effects of the therapeutic exercises and stabilization exercises given to the patient after the manual therapy session on pain, neck range of motion, daily living activities and quality of life.


Eligibility criteria:
inclusion criteria: * between the ages of 18-65, * having neck pain for 3 months or longer, * was diagnosed with chronic neck pain by a specialist. exclusion criteria: * people with neurological diseases, * who had previous surgery in the cervical region, * who had medulla spinalis tumors, * vascular pathologies, * fractures, * osteoporosis, * rheumatoid arthritis problems were not included in the study.