bioihbjkbjnl/mcl2 huggingface dataset:
Sialic Acid Supplementation in N-Acetylneuraminic Acid Synthase (NANS) Deficiency
Condition: N-Acetylneuraminic Acid Storage Disease
MeSH terms: Sialic Acid Storage Disease
Study type: Interventional

Summary: 
      This study is aimed at assessing the impact of short-term (3 days) exogenous sialic acid
      supplementation on endogenous biomarkers of sialic acid metabolism in NANS deficient
      patients.
    

Detailed description: 
      NANS deficiency is a genetic disorder presenting clinically with intellectual development
      disorder, skeletal dysplasia and dysmorphic features. It has recently been described in 9
      patients (4 children and 5 adults). Biallelic mutations in the NANS (N-Acetylneuraminic acid
      synthase) gene cause a block in the endogenous synthesis of sialic acid and accumulation of
      the precursor, N-acetyl mannosamine (ManNAc). In a cell culture model, this block results in
      hyposialylation of glycoproteins and glycolipids. It seems likely that in human, this enzyme
      deficiency impairs the sialylation of glycolipids and glycoproteins, known to be essential
      for brain development. Exogenously added sialic acid partially rescued the phenotype of
      NANS-deficient zebra fish. Currently there is no approved treatment for patients with NANS
      deficiency. The investigators concluded that exogenous sialic acid supplementation might be
      useful for NANS patients. Given that sialic acid is found as both, a free sugar and in a
      bound form in standard nutrition as well as in high quantities in breast milk, it can be
      considered as a safe nutritional ingredient; this notion is fully supported by animal
      toxicity studies.

      The use of sialic acid in NANS deficiency is in line with oral supplementation of specific
      sugars for treatment of other glycosylation and sialylation defects such as congenital
      disorders of glycosylation (CDG) and myopathy with mutation in the gene GNE. This novel
      monosaccharide therapy represents an opportunity to address fundamental biochemical questions
      about the relative contribution of endogenous and dietary sources on sialic acid metabolism
      and its potential role as a future therapy for NANS patients.
    

Intervention name(s): Neu5Ac supplementation
Intervention type(s): Other
Intervention description(s): Sialic acid as N-Acetyl-neuraminic acid dehydrate (Neu5Ac) 150 mg/kg/d (max 12g/d) in three doses orally in subjects with NANS deficiency compared to controls
Intervention model: Single Group Assignment
Intervention model description: Open label individual patient clinical trial study of small sample size
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Sialic acid; Monosaccharide
Efficacy and Satisfaction With Remifentanil Analgesia in Parturients During the Childbirth
Condition: Complication of Labor and/or Delivery
MeSH terms: Obstetric Labor Complications
Study type: Observational

Summary: 
      Retrospective observational study to determine the efficacy of remifentanil during the
      delivery. Authors will search the data in period between 2010 and 2014 in cohort of 100
      parturients and obtain data of analgesic efficacy (VAS score after remifentanil analgesia
      induction) and parturients satisfaction with the method.
    

Detailed description: 
      In retrospective observational study will authors search the documentation of parturients.
      The cohort size is estimated to 100 subjects, in period between 2010-2014. Excluded will be
      all patients who did not signed the informed consent for processing with their medical
      data.The main aim is to evaluate the efficacy of the remifentanil analgesia in parturients in
      the first phase of delivery.

      The analgesic efficacy will be evaluated by the change in VAS (visual analogue scale) score
      after induction of remifentanil analgesia Satisfaction will be evaluated by the questionary.
    

Intervention name(s): remifentanil
Intervention type(s): Drug
Intervention description(s): Remifentanil analgesia during delivery
Intervention MeSH terms: Remifentanil
Observational model: Cohort
Time perspective: Retrospective
Sampling method: Non-Probability Sample
Study population: 
        Parturients during the delivery
      
Keywords: Remifentanil, delivery, childbirth, analgesia, parturition, confinement
Theta Burst Stimulation in Young Adults With Depression
Condition: Depression
MeSH terms: Depression; Depressive Disorder
Study type: Interventional

Summary: 
      For the proposed 2-year study, the investigators will conduct a within-subject,
      counterbalanced investigation using functional magnetic resonance imaging (fMRI) and
      transcranial magnetic stimulation (TMS) to examine the acute effects of theta-burst
      stimulation (TBS) on function in dorsomedial prefrontal cortex (dmPFC) in 35 young adults
      with depression (18-25 years, 50% female).
    

Detailed description: 
      Each participant will undergo 3 sessions of TMS, one each of continuous and intermittent
      TBS--with the goal of decreasing or increasing dmPFC responding, respectively--and one of
      sham TBS. Session order will be counterbalanced, with a double-blind approach to condition.
      Brain function, behavior, and mood will be assessed before and after each TBS session.
      Broadly, the investigators predict that inhibitory TBS to the dmPFC will enhance neural,
      behavioral, and subjective aspects of reward function by reducing dmPFC function and dmPFC
      connectivity with the ventral striatum (VS).
    

Intervention name(s): Intermittent Theta Burst Stimulation; Continuous Theta Burst Stimulation; Sham Theta Burst Stimulation
Intervention type(s): Device; Device; Device
Intervention description(s): Theta Burst Stimulation, a form of Transcranial Magnetic Stimulation, will be applied to the dorsomedial prefrontal cortex. will include 600 pulses delivered in brief bursts of three pulses with a frequency of 50 Hz, at an intensity of 110% of resting motor threshold, and administered every 200 ms (5 Hz). Bursts will be delivered without interruption for a total duration of 40 seconds.; Theta Burst Stimulation, a form of Transcranial Magnetic Stimulation, will be applied to the dorsomedial prefrontal cortex. will include 600 pulses delivered in brief bursts of three pulses with a frequency of 50 Hz, at an intensity of 110% of resting motor threshold, and administered every 200 ms (5 Hz). Bursts will be delivered during 2 second periods (10 bursts/period) interleaved with 8-second stimulation-free intervals, for a total duration of 190 seconds.; For the sham of theta burst stimulation, the device providing Theta Burst Stimulation can be placed in the same position and turned on, creating a similar experience for the participant, without providing any neural stimulation. Sham TBS will be delivered with a Cool-B65 Active/Placebo Coil, which includes a sham setting, and MagLink research software.
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: All participants will receive intermittent Theta Burst Stimulation (TBS), continuous TBS, and sham TBS mimicking stimulation, in randomized, double-blind order across three sessions.
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Basic Science
Committed Actions for Successful Aging
Condition: Brain Aging
Study type: Interventional

Summary: 
      The investigators are planning to recruit approximately 30 study participants aged 50 years
      and older that will undergo a 4 week attention and emotion regulation intervention. The
      intervention systematically targets thoughts, emotions and behavior in order to promote
      quality of life and advance successful brain aging. The investigators will assess the
      feasibility of recruiting and retaining participants for an attention and emotion regulation
      intervention. In addition, the investigators will determine the efficacy of the intervention
      to enhance and promote quality of life, sustained attention, emotions, motivation and
      cognitive health.
    

Detailed description: 
      The CASA model is developed here in the investigators program (Geda et al) based on
      neuroscience and Acceptance and Commitment Therapy (ACT). The rationale for the study is that
      the cortico-cortical network linking the prefrontal cortex with the mesial temporal lobe is
      susceptible to the wear and tear of day to day life such as stress and emotional distress.
      The investigators hypothesize that the CASA intervention will promote emotional and cognitive
      health by targeting this network.
    

Intervention name(s): CASA
Intervention type(s): Behavioral
Intervention description(s): Each participant serves as his/her own control. Measures administered before and after the behavioral intervention.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Prevention
A Study to Evaluate Efalizumab in Adults With Plaque Psoriasis, Including Those Who Are Receiving Concomitant Antipsoriatic Therapies
Condition: Psoriasis
MeSH terms: Psoriasis
Study type: Interventional
Phase: Phase 3

Summary: 
      This is a Phase IIIb, open label, uncontrolled, multicenter study designed to evaluate the
      safety and tolerability of 12 weeks of subcutaneously administered efalizumab in subjects
      with moderate to severe plaque psoriasis who are candidates for systemic therapy and may also
      be receiving topical therapies, PUVA, or UVB phototherapy during the efalizumab treatment
      period.
    


Intervention name(s): efalizumab
Intervention type(s): Drug
Primary purpose: Treatment
Keywords: Plaque psoriasis; Antipsoriatic
PRAGMA (Prostate Radio Ablation Guided by Magnetic Resonance Imaging Acquisition) in Metastatic Prostate Cancer
Condition: Metastatic Prostate Cancer
MeSH terms: Prostatic Neoplasms
Study type: Interventional

Summary: 
      Patients with metastatic prostate cancer can undergo MRI-guided prostate Stereotactic body
      radiation therapy (SBRT) without significant adverse events, similar to what has been
      reported for patients with localized prostate cancer. We hypothesize that prostate SBRT will
      be well-tolerated in metastatic prostate cancer patients, with quality of life outcomes
      similar to what has been reported in non-metastatic prostate cancer patients.
    

Detailed description: 
      The MRidian ViewRay offers delivery of prostate SBRT with real-time MR guidance, which
      provides superior soft-tissue differentiation with excellent visualization of the prostate.
      This ViewRay platform offers the ideal setting for this study, that aims at precisely
      delivering prostate SBRT with a simultaneous integrated boost (when indicated) to visible
      nodules.

      In this study, we hope to demonstrate the safety and feasibility of using 36.25 Gy in 5
      fractions, plus a simultaneous integrated boost (when indicated), in patients with metastatic
      prostate cancer.
    

Intervention name(s): MR-Guided Prostate SBRT
Intervention type(s): Radiation
Intervention description(s): Prostate SBRT has become a standard of care for the treatment of localized prostate cancer. Radiation is delivered to the prostate and seminal vesicles in 5 treatment sessions (fractions). Doses ranging from 35-45 Gy in 5 fractions have demonstrated good outcomes with acceptable toxicity when planning is delivered appropriately.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
A Comparative Study of ZG-801 and Placebo in Patients With Hyperkalemia
Condition: Hyperkalemia
MeSH terms: Hyperkalemia
Study type: Interventional
Phase: Phase 3

Summary: 
      The purpose of this study is to evaluate the superiority of ZG-801 to placebo in Japanese
      hyperkalemia patients whose serum potassium value becomes normal in the Run-in period by
      comparing the change in the value at the Double-blind period week 4, and to further evaluate
      the efficacy and safety of ZG-801 in Japanese hyperkalemia patients administrated ZG-801 for
      maximum 9 weeks.
    


Intervention name(s): Calcium, hydrolysed divinylbenzene-Me 2-fluoro-2-propenoate-1, 7-octadiene polymer sorbitol complexes; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): ZG-801 is powder for suspension. In Run-in period, ZG-801 is taken once a day for 4 or 5 weeks. In Double-blind period, ZG-801 is taken once a day for 4 weeks.; Placebo is powder for suspension. In Double-blind period, Placebo is taken once a day for 4 weeks.
Intervention MeSH terms: Sorbitol; Calcium
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Vestibular Rehabilitation and Severe Traumatic Brain Injury
Condition: Severe Traumatic Brain Injury; Gait Disorders, Neurologic
MeSH terms: Brain Injuries; Brain Injuries, Traumatic; Nervous System Diseases; Gait Disorders, Neurologic; Wounds and Injuries
Study type: Interventional

Summary: 
      Severe brain injury (sTBI) is one of the most common causes of long-term disability and is
      considered the most frequent cause of mortality and serious disability in young adults in
      industrialized countries. It is defined as an alteration of brain function with loss of
      consciousness in the acute phase for at least 24 hours (Glasgow Coma Scale (GCS) <8) and it
      can induce a wide range of deficit, including cognitive-behavioural, motors, psychics,
      language, vision, coordination and balance impairments. Chronic vestibular symptoms such as
      dizziness and balance deficits (both static and dynamic postural instability) are present in
      patients with brain injury. These aspects can cause functions limitation and psychological
      distress, negatively impacting negatively on subjects' quality of life and social
      reintegration and are considered unfavourable prognostic factors of the recovery process. The
      literature supports the use of vestibular rehabilitation techniques in patients with mild and
      moderate brain injury, however, to date, no studies investigated the effect of vestibular
      rehabilitation in sTBI patients. The main aim of this randomized controlled trail is to
      verify the effect of a personalized vestibular training on balance and gait disorders in sTBI
      patients.
    


Intervention name(s): Vestibular Rehabilitation (VR); Conventional Neurorehabilitation
Intervention type(s): Other; Other
Intervention description(s): VR consisted of two types of exercises, i.e., those for gaze stability and those for postural stability
Gaze stability exercises The patients will perform the exercises while holding their gaze on a firm target (VORx1) during active horizontal and vertical head movements (one minute for each axis).
Postural stability exercises March in Place Each patient will ask to get on a foam cushion of 10 cm in height and then will blindfold. Treadmill Training As preparation for training, all subjects underwent a 1-minute walk on treadmill with open eyes using preferred walking speed. Immediately after preparation, patients will blindfold and will ask to walk on treadmill without support of hands for 4 minutes.
When patients made the mistake of changing direction, the physiotherapist help them to keep the right position using verbal cues (e.g., you are turning left or right).; muscles stretching, active and assisted limbs mobilization, four limbs coordination exercises, balance training on instable platform and gait training
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Treatment
The Efficacy of Distortion Banding on Outcome Measures in Children With Cerebral Palsy
Condition: Cerebral Palsy, Spastic
MeSH terms: Paralysis; Cerebral Palsy
Study type: Interventional

Summary: 
      The aim of the study is to compare the conventional rehabilitation program and the distortion
      taping applied with Kinesio Tex tape in addition to conventional rehabilitation in children
      with hemiplegic or monoplegic cerebral palsy with ankle varus deformity.
    

Detailed description: 
      Conventional rehabilitation practices will be applied to the control group. In addition to
      Kinesio Tex tape distortion taping, conventional rehabilitation will be applied to the
      intervention group. It is aimed to evaluate the patients' balance, quality of life,
      performance test, spasticity level and gait quality.
    

Intervention name(s): Kinesio Taping; Conventional Rehabilitation
Intervention type(s): Other; Other
Intervention description(s): In addition to Kinesio Tex tape distortion taping, conventional rehabilitation is applied.; Conventional rehabilitation practices are carried out
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Randomised Controlled Trial
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Cerebral palsy; Kinesio taping
Longitudinal Study of the Human Intestinal Microbiome
Condition: Dysbacteriosis
MeSH terms: Dysbiosis
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to identify the human intestinal microbiota (microbes that live
      inside and on human bodies) in healthy adults over a 6-month period and to study the effect
      of an antibiotic on the intestinal microbiota. Participants will include up to 60 healthy
      adult subjects, ages 18-45 years, from the Baltimore and University of Maryland communities.
      Study procedures will include providing multiple stool samples throughout the study.
      Participants will take a licensed antibiotic, Ciprofloxacin, for 3 days. Participants may be
      involved in study related procedures for up to 7 months.
    

Detailed description: 
      The purpose of this study is to describe the intestinal microbiomes of a cohort of healthy
      adult subjects over a 6-month period of time. Many questions about the human microbiota
      exist. Previous studies have shown that the differences among individuals are greater than
      the differences among different sampling sites in a single individual. Up to 60 healthy adult
      subjects, ages 18-45 years, from the Baltimore and University of Maryland Baltimore
      communities will be recruited and screened to document their health status. A brief medical
      history, including recent travel and antibiotic use, will be recorded. Subjects will provide
      a stool specimen for genomic analysis of the intestinal microbiome over a 6-month period at
      the following intervals: Day 0, Week 1, Week 2, Week 3, Week 4, Week 8, Week 12, Week 13,
      Week 14, Week 15, Week 16, Week 20, and Week 24. At Week 12, subjects will receive a 3-day
      course of oral Ciprofloxacin 500 mg every12 hours. Weekly stool specimens will be obtained
      beginning with the first day of antibiotic use and for 4 Weeks thereafter. Then monthly
      specimens of stool and other sites will be resumed at Week 16. The primary objective is to
      define the human intestinal microbiome in healthy adults in a longitudinal fashion. The
      secondary objective is to define the re-colonization of the intestine after treatment with a
      broad-spectrum antibiotic using comprehensive genomic techniques. Each subject will
      participate in the study for up to 7 months.
    

Intervention name(s): Ciprofloxacin
Intervention type(s): Drug
Intervention description(s): Licensed medication, dose: 500 mg every 12 hours for 3 days.
Intervention MeSH terms: Ciprofloxacin
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Other
Keywords: genomic techniques; intestinal microbiome
A Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Multiple Subcutaneous Injections of ABT-981 in Patients With Osteoarthritis of the Knee
Condition: Osteoarthritis
MeSH terms: Osteoarthritis; Osteoarthritis, Knee
Study type: Interventional
Phase: Phase 1

Summary: 
      To assess the safety, tolerability and pharmacokinetics of ABT-981 in patients with
      osteoarthritis of the knee.
    

Detailed description: 
      This is a phase 1, randomized, double-blind, placebo-controlled, multiple ascending dose
      study. Thirty-six patients with osteoarthritis of the knee will be selected to participate.
      Patients will be randomized to receive either ABT-981 or placebo. ABT-981 or placebo will be
      administered as subcutaneous (under the skin) injections in four dosing groups. Subjects will
      be administered subcutaneous injections of ABT-981 for up to 8 weeks.
    

Intervention name(s): ABT-981; Placebo
Intervention type(s): Biological; Biological
Intervention description(s): Injection; Placebo Injection
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Immunogenicity; Safety; Tolerability; Osteoarthritis; Pharmacokinetics
OPUS School Meal Study
Condition: Metabolic Syndrome X
MeSH terms: Microvascular Angina; Metabolic Syndrome
Study type: Interventional

Summary: 
      The aim of this randomized crossover study, is to investigate the effect of school meals
      based on a sustainable New Nordic Diet on growth, learning abilities, well-being, sleep
      quality, physical activity and risk markers of cardiovascular disease (CVD), type II diabetes
      and osteoporosis in 8-11 year old Danish school children.

      The participating 3rd and 4th grades on 9 selected primary schools will be randomized to
      either start receiving New Nordic Diet school meals for 3 month(mo) or to eat their habitual
      school lunch for 3 mo and then crossover for additional 3 mo. All participants will be
      examined three times; at baseline, after 3 mo and after 6 mo.
    


Intervention name(s): School meals based on the principles of the New Nordic Diet
Intervention type(s): Other
Intervention description(s): The study participants are provided with three daily school meals for 3 month
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Basic Science
Keywords: Metabolic Syndrome X; Fatty Acids, Omega-3; Learning; Attention; Nutritional Status; Nutrition Assessments; Body Weight; Physical Activity; Sleep; Child Nutrition Sciences