Kornimate/medical-research-clean huggingface dataset:
clinical benefit, safety, pk and pd study of at-007 in pediatric subjects with classic galactosemia

Conditions: Classic Galactosemia

Summary: this study is designed to assess the clinical benefit as well as the safety, pharmacokinetics (pk), and pharmacodynamics (pd) (reduction of galactitol levels) of at-007 in pediatric subjects with classic galactosemia (cg).

this is a randomized, double-blind, placebo-controlled study in pediatric subjects with cg. children with cg ages 2 through 17 years will be enrolled. the study is designed to assess the clinical benefit using validated, standardized clinical outcome assessments (coas) as well as the safety, pk, and pd (reduction of galactitol levels) of at-007 in pediatric subjects with cg. the study consists of 2 main parts, part a and part b, and an open-label extension (ole). part a is an intra-patient dose escalation evaluating multiple ascending doses (mad) of at-007. part b is designed to assess the clinical benefits of long-term administration of at-007 (at the optimum dose identified in part a) on validated, standardized coas. open-label extension (ole) is an active treatment extension for patients who received placebo treatment in part b.

Eligibility criteria:
inclusion: * male * female non-pregnant * female non-lactating subjects aged ≥2 to \<18 years. * diagnosis of classic galactosemia, confirmed by \<1% galt (galactose-1-phosphate uridyltransferase) enzyme activity in erythrocytes, or a historical record of diagnosis of \<1% galt enzyme activity. exclusion: * male/female with no significant health problems (other than classic galactosemia) * no other disease that would preclude participation in the study.

Keywords: galactosemia classic galactosemia
study of the effects of dopaminergic medications on dopamine transporter density in subjects with parkinson's disease

Conditions: Parkinson Disease; Parkinsonian Syndrome

Summary: this study investigates whether there is a change in 123iodine-2ß- carbomethoxy-3ß-(4-iodophenyl) tropane (\[123i\]ß-cit) uptake after short-term treatment with levodopa compared to either dopamine agonist or placebo.

this is a multi-center, open-label study of short-term treatment with levodopa or cabergoline on striatal datscan uptake in early parkinson's disease. approximately 120 parkinson's disease subjects will be randomized to receive either carbidopa/levodopa, cabergoline or no treatment during a twelve week period. subjects will undergo spect imaging with datscan at screening and after 12 weeks. after twelve weeks carbidopa/levodopa and cabergoline treatment will be withdrawn and all subjects will undergo spect imaging with datscan after 8 weeks (20 weeks after baseline).

Eligibility criteria:
inclusion criteria: * the subject is aged 40 years or older. * written informed consent is obtained. * subjects have a clinical diagnosis of idiopathic parkinson's disease. * hoehn and yahr stages for subjects are i-ii. exclusion criteria: * the subject has atypical or drug-induced parkinson's disease. * the subject has dementia. * the subject has clinically significant abnormal laboratory values, and/or clinically significant or unstable medical or psychiatric illness. * the subject is pregnant.

Keywords: parkinson brain imaging diagnosis
the application of cognitive rehabilitation therapy for amphetamine-type stimulants addiction

Conditions: Amphetamine Addiction

Summary: the computerized cognitive rehabilitation therapy will be used to treat amphetamine-type stimulant (ats) addiction.

the deficits in executive functions related to prefrontal-striatal circuits play a vital role in ats addiction and relapse, and proposed computerized cognitive rehabilitation therapy as the novel interventions. focused on evaluating and training executive functions including working memory, inhibitory control, cognitive flexibility, and congitive bias concerning prefrontal-striatal circuits, neuropsychological tests, electroencephalography tests, magnetic resonance spectroscopy (mrs) and functional mri will be used to evaluate the therapeutic effect of ats addiction treatment as well as investigate the mechanisms. the study will be very helpful to develop novel interventions in clinical practice and decrease ats-related harm for both the patients and their families.

Eligibility criteria:
inclusion criteria: 1. diagnosed ma dependence in accordance with the fourth edition of the american psychiatric association's diagnostic and statistical manual of mental disorders (dsm-iv) (association, 2000); 2. more than 9 years of education; 3. aged of 18-60 years old; 4. normal vision, audition and no color blindness; 5. receive no detoxification medications during treatment and 6. right handedness. exclusion criteria: 1. current medical diseases that required hospitalization or regular monitoring; 2. serious physical or neurological illness that required pharmacological treatment affecting cognitive function; 3. history of major psychiatric disorder such as bipolar disorder, schizophrenia, depression and disorders of high comorbidity with substance abuse/dependence; 4. neurological diseases such as stroke, seizure, migraine, head trauma 5. substance dependence other than nicotine, within the past 5 years; 6. intelligence quotient (iq) \<70.

Keywords: amphetamine-type stimulants prefrontal-striatal circuits cognitive rehabilitation therapy
myotonic dystrophy - vascular and cognition

Conditions: Myotonic Dystrophy 1; Diabetes; Carbohydrate Intolerance

Summary: the cognitive disorders of adult forms of myotonic dystrophies type 1 are heterogeneous (impairment of executive functions, visio construction and theory of the mind, which can progress to the stage of dementia). nevertheless, patients have very different degrees of cognitive impairment. expansion of ctg triplets disrupts the alternative splicing of mrnas of various proteins, including the insulin receptor and tau protein. type 2 diabetes, associated with peripheral insulin resistance, is therefore common in this pathology. type 2 diabetes,could to explain the cognitive impairment of patients, through the accelerated development of brain lesions (especially tauopathy and cerebral atrophy).


Eligibility criteria:
inclusion criteria: * molecularly proven type 1 myotonic dystrophy * voluntary, having given informed consent * socially insured patient * patient willing to comply with all study procedures and duration (3 hours + mri 35 minutes) * patient insured under the french social security system * signed consent form exclusion criteria: * neurological history other than neuropathy: epilepsy, stroke, dementia * pregnancy or breastfeeding or woman of childbearing age without effective contraception (a pregnancy test will be done) * contra indication to mri * person under guardianship or curators * persons of full age deprived of their liberties by a judicial or administrative decision * major comorbidity considered as a contraindication by the investigator (cancer, unstable angina, etc.).

Keywords: cerebral atrophy mri cognition metabolic syndrome tauopathy
celiac disease and diabetes- dietary intervention and evaluation trial

Conditions: Celiac Disease; Diabetes Mellitus, Type 1

Summary: currently no consensus exists on the optimal way to manage asymptomatic patients with celiac disease (cd) and type 1 diabetes (t1d). the impact of dietary treatment as it pertains to clinically relevant outcomes such as metabolic control, bone mineralization and wellbeing will be evaluated in this study. a randomized controlled study longitudinally evaluating hba1c and glycemic excursions using continuous glucose monitoring will rigorously determine the impact of a gluten-free diet (gfd) on blood glucose variability in patients with t1d.


Eligibility criteria:
inclusion criteria: subjects must meet each of the following criteria for inclusion into the study: 1. males and females age between 8 and 45 years. 2. diagnosis of t1d by american diabetes association (ada) criteria with duration of t1d greater than 1 year. 3. cd screen positive (positive ttg serology). 4. presence of a positive duodenal biopsy for cd (marsh score of 2 or greater). 5. ability of the subject or a legally authorized representative to speak and read english or french. 6. ability of the subject to participate in all aspects of this clinical trial. 7. written informed consent must be obtained and documented, with assent of the child if \<14 years of age. exclusion criteria: 1. prior diagnosis of cd. 2. symptoms or other evidence of overt cd defined by at least one of: * cd symptoms using the gastrointestinal symptom scale \[giss\] * impaired growth * anemia 3. presence of recurrent apthous ulcers (painful mouth ulcers involving mucous membranes) or dermatitis herpetiformis (blistering skin rash). mouth or skin lesions known to be related to diagnosed herpes are not included in this exclusion criteria. 4. conditions which in the opinion of the investigator may interfere with the subject's ability to participate in the study. 5. prior enrolment in the current study. 6. concurrent enrolment in a longitudinal intervention study. 7. previously diagnosed or treated osteoporosis. 8. pregnancy or lactation.

Keywords: pediatric dietary treatment celiac disease t1d
a comparison of two thoracic manipulation techniques to improve neck pain

Conditions: Neck Pain

Summary: the purpose of this study is to compare the short-term effects of two different thoracic spine thrust manipulation techniques on neck range-of-motion, pain, and self-reported disability in individuals experiencing neck pain.

the study will be conducted at the musculoskeletal clinical facility in the dept. of physical therapy and athletic training, northern arizona university, flagstaff az. the investigators aim to test a maximum of 76 participants in this study. a phone screening will determine initial eligibility. if the participant meets the inclusion criteria and is not excluded after the phone screen, an appointment will be scheduled. final eligibility will be determined in the initial session after the participant completes the outcome measures and is evaluated by a licensed physical therapist. participants will be involved in the study over a 4 week period. each participant will be asked to attend 4 sessions, with one week in between each session as follows: week 0 = baseline assessment and treatment 1; week 1= treatment 2; week 2 = treatment 3; and week 4 = follow-up assessment. after completion of the baseline assessment, qualified participants will be randomly assigned to either a supine or prone thoracic spine thrust manipulation intervention.

Eligibility criteria:
inclusion criteria: * between 20 and 50 years of age (inclusive) * primary complaint of neck pain with or without symptoms that spread down into one arm * overall rating of neck pain intensity is at least 3/10; the overall rating is the average of the participant's rating of current, least, and worst pain experienced over the previous 24 hours on separate numeric pain rating scales where 0 corresponds to "no pain" and 10 corresponds to "worst possible pain" * neck disability index (ndi) score \> 10 points (measure of self-reported disability with a 0 to 50-point scale where higher scores indicate higher levels of disability) * proficient in speaking and reading english to complete outcome questionnaires exclusion criteria: * neck pain related to a motor vehicle accident or other trauma within the previous 6 weeks * neck pain that spreads down into both arms * low back pain or thoracic origin of pain * nerve root involvement; defined as the presence of two or more neurological findings (e.g., decreased strength, diminished deep tendon reflex, or decreased sensation) at the same nerve root level * diagnosis of cervical spinal stenosis (narrowing of the central canal that contains the spinal cord) * history of spinal tumors, spinal infection, cervical spine fracture, or previous neck surgery * pending legal action related to current episode of neck pain * contraindications to thoracic spine thrust manipulation 1. serious pathologies or conditions (tumor, fracture, metabolic diseases, rheumatoid arthritis, osteoporosis, history of prolonged steroid use) 2. hyperreflexia 3. unsteadiness during gait 4. nystagmus 5. loss of visual acuity 6. impaired sensation of the face 7. altered taste 8. the presence of pathological reflexes 9. pregnancy or considering pregnancy
manganese in women with encephalopathy

Conditions: Hepatic Encephalopathy

Summary: objective: report manganese serum levels and mortality in encephalopathic patients. patients and participants: consecutive patients aged \> 18 years, with hepatic encephalopathy and informed consent signed by their families. interventions: patients' clinical characteristics as well as biochemical tests of renal function, hemoglobin, glucose and albumin levels were obtained as well as a blood sample to analyze manganese levels with a graphite furnace atomic absorption spectrometer. hypothesis: there is a difference in the manganese levels between male and female patients.


Eligibility criteria:
inclusion criteria: * consecutive patients aged \> 18 years * with hepatic encephalopathy * informed consent signed by their families exclusion criteria: * portal bypass surgery * cancer of any localization * known malignancy and other severe diseases which shorten life expectancy \< 6 months * psychiatric illness

Keywords: hepatic encephalopathy hepatitis c manganese mortality prognosis.
differential effects of zolpidem versus ramelteon in burned children

Conditions: Sleep; Burns

Summary: to examine sleep changes following therapeutic drug interventions designed to promote sleep.


Eligibility criteria:
inclusion criteria: * burn injury \> 20% total body surface area * between 3 and 18 years of age * \< 7 days from acute injury * written informed consent and hippa release signed exclusion criteria: * suspected anoxic brain injury or head injury * hepatic or endocrine disease * history of alcoholism or substance abuse * pre-existing neurological or primary psychiatric disorder * medical history of pre-existing sleep disorder or lactose deficiency * questionable survival (\<72 hrs) as decided by pi * receipt of drugs with known effects on sleep within 24 hrs of study entry * no informed consent/hippa release
hypofractionated boost vs conventionally fractionated boost for localized high risk prostate cancer

Conditions: Prostate Cancer

Summary: randomized trial comparing 2 external beam radiotherapy fractionation schemes in patients with localized high risk prostate cancer. primary endpoint is acute toxicity.

patients enrolled onto this study will be randomized to one of the following treatment arms: * standard fractionation: using 2 sequential imrt plans, the pelvic lymph nodes and prostate will initially be treated to 46 gy in 23 fractions, followed by a subsequent boost to the prostate to a total dose of 78 gy. * hypofractionation: using a one phase imrt plan, the pelvic lymph nodes will be treated to a dose of 48 gy in 25 fractions, while the prostate will be treated to a dose of 68 gy in 25 fractions concomitantly (simulataneous integrated boost). in addition, all patients receive 1.5- 3 years of androgen deprivation therapy.

Eligibility criteria:
inclusion criteria: * written informed consent obtained. * histologically confirmed diagnosis of adenocarcinoma of the prostate. * t1-2 n0 m0, gleason score \<= 7, psa 20 - 100 * t1-2 n0 m0, gleason score 8 - 10, psa \<= 100 * t3 n0 m0, any gleason score, psa \<= 100 exclusion criteria: * patients with unilateral or bilateral hip replacement. * patients with active collagen vascular disease. * patients with active inflammatory bowel disease. * patients with previous radiotherapy to the pelvis. * patients with ataxia telangiectasia. * patients with nodal or distant metastases

Keywords: high risk prostate cancer radiotherapy
the autonomic nervous system state and response to different stimuli among patients post stroke: a follow-up study

Conditions: Stroke

Summary: the study describes the autonomic control system function in subjects post stroke in comparison with healthy subjects. aims: to describe the autonomic hart rate control system function in post stroke subjects in different rehabilitation levels in comparison with healthy subjects. to examine the reflective reaction of the autonomic control system as reflected in the heart rate variability to stimulations subjects in different rehabilitation levels in comparison with healthy subjects. to examine the autonomic control system reaction as reflected in the heart rate variability to physical and cognitive action subjects in different rehabilitation levels in comparison with healthy subjects.

subjects will be examined 3 times: a week 1 and 3 month post stroke. control consist similar in description healthy subjects, will be examined once. each test includes 24 hours holter surveillance and an examine that include general check that reflects the autonomic reaction to different manipulation. the examination description: a. prior to the examination subjects will be asked to fill the mrs, nihss forms. b. subjects will receive an examination that includes * general neurological examination (strength, deep and superficial sensation, cognitive test and pain level.) c. holter will be place for 24 hours. and autonomic examination test will be conducted * grip test: emphases the sympathetic system. * breathing test: emphases the parasympathetic system. d. manipulation to examine the two sub systems. each manipulation lasts1 minute.

Eligibility criteria:
inclusion criteria: * post hemispheral ischemic stroke cognitively intact. exclusion criteria: * subjects with severe visual or hearing impairments, stroke location brain steam.

Keywords: autonomic nerve system post stroke focus of study: to describe the autonomic nerve system function post stroke autonomic nervous system
efficacy and tolerability of baf312 in patients with polymyositis and dermatomyositis

Conditions: Polymyositis; Dermatomyositis

Summary: this study determined the efficacy, safety, tolerability and the pk profile of baf312, a novel immunomodulator, in polymyositis and dermatomyositis patients who were not responsive to traditional immunosuppressive and/or corticosteroid therapy. the study consisted of a 12 week, randomized, placebo controlled period, followed by another 12 weeks where all subjects received baf312 treatment.


Eligibility criteria:
inclusion criteria: * patients with disease at least 3 months before study * muscle weakness * received corticosteroids with or with out disease modifying antirheumatic drugs at least 3 months before study however not responding to this therapy exclusion criteria: * other idiopathic inflammatory myopathies * myopathy other than polymyositis and dermatomyositis * patients with late stages of disease

Keywords: inflammatory muscle disease, muscle weakness, myositis, inflammatory myopathy, inclusion body myositis, skin rash, autoimmune, difficulty swallowing, dysphagia
recurrence of dyschezia in rectal prolapse, rectocele and elytrocele

Conditions: Rectal Prolapse

Summary: prolapse of the rectum is a pathology that preferentially affects older women with a significant impact on quality of life. a very large number of therapeutic approaches can be proposed (functional treatment, surgical techniques by perineal approach and surgical techniques by anterior approach). d'hoore and pennix described ventral rectopexy with prosthetic reinforcement which is accepted as a standard of treatment in much of europe for externalized prolapse but remains maligned in much of the world. due to the relatively recent appearance of this technique and the great variability in the management methods, the long-term results of ventral rectopexy have been little studied. this surgical technique is the preferred approach offered at chu grenoble alpes. pr faucheron have internationally recognized experience in surgical grip with a very high patient volume in recent years.

prolapse of the rectum is a pathology that preferentially affects older women with a significant impact on quality of life. a very large number of therapeutic approaches can be proposed (functional treatment, surgical techniques by perineal approach and surgical techniques by anterior approach). d'hoore and pennix described ventral rectopexy with prosthetic reinforcement which is accepted as a standard of treatment in much of europe for externalized prolapse but remains maligned in much of the world. due to the relatively recent appearance of this technique and the great variability in the management methods, the long-term results of ventral rectopexy have been little studied. this surgical technique is the preferred approach offered at chu grenoble alpes. pr faucheron have internationally recognized experience in surgical grip with a very high patient volume in recent years. the objective of this work is to describe the long-term results of this technique.

Eligibility criteria:
inclusion criteria: * patient operated for rectal prolapse or enterocele for 10 or more * age 18 and over * patients informed exclusion criteria: - patient opposition
pregabalin in the treatment of patients with generalized anxiety disorder (gad).

Conditions: Generalized Anxiety Disorder

Summary: the primary objective of this study is to evaluate the efficacy of pregabalin as compared to placebo in the treatment of patients with general anxiety disorder (gad). efficacy will be measured by the improvement in the total hamilton anxiety rating scale (ham-a) scores from baseline observed following 8 weeks of double-blind treatment or at earlier termination during the double-blind treatment phase and analyzed using a mixed linear model for repeated measures.

further enrollment in this study was stopped on january 28, 2008 based on the recommendation of an independent data monitoring committee. the recommendation to stop the study was not based on any safety findings.

Eligibility criteria:
inclusion criteria: * adult male and female subjects with primary dsm-iv diagnosis of gad, as confirmed by the mini structured interview. * historical failure to respond optimally to a gad treatment exclusion criteria: * current primary dsm-iv (diagnostic and statistical manual of mental disorders 4th edition) diagnosis of major depressive disorder with or without seasonal pattern, dysthmic disorder, depressive disorder nos (not otherwise specified), social phobia, panic disorder with or without agoraphobia, post traumatic stress disorder, dissociative disorder, borderline personality disorder, obsessive-compulsive disorder, antisocial personality disorder, as defined in the dsm-iv tr (diagnostic and statistical manual of mental disorders 4th edition, text revision). * past and/or current dsm-iv diagnosis of schizophrenia, schizoaffective disorder, other psychotic disorders, bipolar disorders (i or ii), factitious disorder or cognitive disorder (including delirium, dementia, and amnestic disorder).
study evaluating the safety, tolerability and activity of one dose of paz-417 given to healthy japanese subjects

Conditions: Alzheimer Disease; Healthy

Summary: this study will provide an initial assessment of the safety, tolerability, and pharmacokinetics (pk) of paz-417 after administration of ascending single oral doses to healthy young japanese male and healthy elderly japanese male subjects.


Eligibility criteria:
inclusion criteria 1. men aged 20 to 45 years inclusive (healthy young male subjects) and ≥65 years inclusive (healthy elderly male subjects) 2. nonsmoker or smoker of fewer than 10 cigarettes per day. 3. healthy as determined by the investigator on the basis of medical history, physical examination, clinical laboratory test results, vital signs, and 12-lead electrocardiogram (ecg). exclusion criteria 1. any significant cardiovascular, hepatic, renal, respiratory, gastrointestinal, endocrine, immunologic, dermatologic, hematologic, neurologic, or psychiatric disease. 2. subjects who are identified as being at risk for hypercoagulability 3. use of any investigational or prescription drug within 30 days before test article administration

Keywords: alzheimer disease