bioihbjkbjnl/mcl2 huggingface dataset:
Efficacy Confirmation Study of NPC-09
Condition: GNE Myopathy; Distal Myopathy With Rimmed Vacuoles (DMRV); Hereditary Inclusion Body Myopathy (hIBM); Nonaka Disease
MeSH terms: Muscular Diseases; Distal Myopathies
Study type: Interventional
Phase: Phase 3

Summary: 
      GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene
      that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid).
      The investigators will examine the efficacy and safety of aceneuramic acid (SA-ER tablets) 6g
      daily for 48 weeks in patients with GNE myopathy in a placebo-controlled, double-blind,
      controlled trial.
    

Detailed description: 
      GNE myopathy is a distal myopathy that is thought to be caused by a mutation in the GNE gene
      that encodes an enzyme in the biosynthetic process of aceneuramic acid (typical sialic acid).
      It is an extremely rare progressive muscle disease, often occurring in the late teens to 30s,
      usually affecting the distal lower leg, especially the extensor muscles such as the tibialis
      anterior muscle, first, and weakness in both upper and lower limbs. However, the weakness of
      the quadriceps femoris is usually gradual. Although there are large individual differences,
      health management and deterioration of QOL will eventually become a problem, and in severe
      cases, constant assistance is required in daily life.

      A double-blind comparative study conducted in Japan provided results suggesting efficacy, but
      the efficacy could not be confirmed in a large-scale international clinical trial, so this
      study is decided to be conducted. Oral administration of aceneuramic acid tablets 500 mg
      (SA-ER tablets) or placebo tablets of the same appearance, 4 tablets at a time, 3 times a day
      for 48 weeks will be examined for differences in efficacy. The target was a total of 10
      cases, 7 cases in the active drug group, and 3 in the placebo group. The amount of change in
      upper extremity composite score (the sum of the average of the right and left HHD scores for
      grip, shoulder abductors, elbow flexors and elbow extensors) was used, and the secondary
      endpoints were the doctor's comprehensive evaluation and the GNE myopathy functional activity
      scale.
    

Intervention name(s): NPC-09; NPC-09 placebo
Intervention type(s): Drug; Drug
Intervention description(s): The drug will be administered by the oral route with the same manner; The drug will be administered by the oral route with the same manner
Other intervention name(s): Aceneuramic acid tablets; Aceneuramic acid matching placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: GNE myopathy; Distal myopathy with rimmed vacuoles (DMRV); Hereditary inclusion body myopathy (hIBM); Nonaka disease; sialic acid; aceneuramic acid; N-acetylneuraminic acid
To Compare the Efficacy of Combined Tenofovir Plus Telbivudine vs Tenofovir Alone in Patients With Spontaneous Reactivation of Hepatitis B
Condition: Spontaneous Reactivation of Hepatitis B
MeSH terms: Hepatitis A; Hepatitis B; Hepatitis
Study type: Interventional

Summary: 
      The relevant data will be prospectively collected included patient demographics, clinical,
      all laboratory variables including virological tests, genotyping by direct sequencing,
      abdominal ultrasound, and upper gastrointestinal (GI) endoscopy. Trans jugular liver biopsy
      (TJLB) and hepatic venous pressure gradient (HVPG) will be done in patients when it was not
      evident whether the underlying liver disease was chronic based on clinical, biochemical,
      radiological investigations, and upper GI endoscopy. Severity of the liver disease will be
      assessed by Child-Turcotte Pugh score (CTP) and model for end stage liver disease (MELD)
      score.
    


Intervention name(s): Tenofovir + Telbivudine; Tenofovir
Intervention type(s): Drug; Drug
Intervention description(s): Tenofovir (300 mg/day) plus telbivudine (600 mg/day).; In tenofovir arm subjects will receive tenofovir (300 mg) once daily.
Intervention MeSH terms: Tenofovir; Telbivudine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Text Messaging for Adherence in Adolescent Liver Transplant Recipients
Condition: Liver Transplant; Adherence
Study type: Interventional

Summary: 
      Text message reminders will improve adherence to lab tests in adolescent liver transplant
      recipients.
    

Detailed description: 
      Adolescent patients who received a liver transplant will be recruited to participate in
      receiving text message reminders for lab tests. They will be send de-identified messages
      according to their required lab schedule. The investigators will monitor their adherence to
      lab tests as well as medication and clinic visits. They will also have a questionnaire about
      their medical regimen before and at the end of the study.
    

Intervention name(s): Text Message Reminder
Intervention type(s): Behavioral
Intervention description(s): Sent TM reminder at beginning and end of each month that corresponded to required lab frequency. Patients were able to send autoresponses.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Health Services Research
Efficacy, Tolerability, and Safety of DFN-15
Condition: Migraine Headache
MeSH terms: Migraine Disorders; Headache
Study type: Interventional
Phase: Phase 3

Summary: 
      Efficacy, Tolerability, and Safety of DFN-15 in episodic migraine with or without aura, being
      conducted at multiple centers in the United States
    


Intervention name(s): DFN-15 Active; DFN-15 Placebo
Intervention type(s): Drug; Other
Other intervention name(s): Celecoxib Oral Solution
Intervention MeSH terms: Celecoxib
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Binocular Dig Rush Game Treatment for Amblyopia
Condition: Amblyopia
MeSH terms: Amblyopia
Study type: Interventional

Summary: 
      To compare the efficacy of 1 hour/day of binocular game play 5 days per week plus spectacle
      correction with spectacle correction only, for treatment of amblyopia in children 4 to <13
      years of age.
    

Detailed description: 
      The purpose of the study is to compare the efficacy of 1 hour/day of binocular game play 5
      days per week plus spectacle correction with spectacle correction only, for treatment of
      amblyopia in children 4 to <13 years of age.
    

Intervention name(s): iPad®; Spectacle correction
Intervention type(s): Other; Other
Intervention description(s): Binocular therapy using a Dig Rush application on an iPad®; Spectacle correction for all waking hours, 7 days per week
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Treatment
Keywords: Amblyopia
Magnesium Sulphate in Perinatal Asphyxia
Condition: Perinatal Asphyxia , Moderate to Severe HIE
MeSH terms: Asphyxia Neonatorum; Asphyxia
Study type: Interventional
Phase: Phase 3

Summary: 
      Magnesium sulphate has been shown to be neuroprotective. The investigators hypothesize that
      magnesium sulphate infusion given to babies with perinatal asphyxia should improve outcome in
      the immediate neonatal period.
    

Detailed description: 
      Magnesium sulphate has a neuroprotective potential as has been shown by many studied in
      pregnant ladies with eclampsia where it helped neonates also and in mothers with preterm
      labour where the incidence of cerebral palsy was less. We designed a randomised controlled
      trial on 40 neonates with severe perinatal asphyxia to see whether it helps in the short term
      outcome of these neonates.
    

Intervention name(s): Magnesium sulphate; Normal saline
Intervention type(s): Drug; Drug
Intervention description(s): 250 mg/kg/dose in 1 ml normal saline; 1 ml/kg/dose
Intervention MeSH terms: Magnesium Sulfate
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Asphyxia; perinatal; magnesium sulphate
Pharmacokinetic Properties of Lu AA21004 (Vortioxetine) in Healthy Young Chinese Men and Women
Condition: Healthy
Study type: Interventional
Phase: Phase 1

Summary: 
      The rationale for this pharmacokinetic (PK) study is to determine the PK parameters for Lu
      AA21004 (Vortioxetine) in the Chinese population.
    


Intervention name(s): Lu AA21004
Intervention type(s): Drug
Intervention description(s): 10 mg immediate-release (IR), one single dose, orally
Other intervention name(s): Vortioxetine
Intervention MeSH terms: Vortioxetine
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
TAX + Cisplatin + 5 F/U vs Cisplatin + 5 F/U in SCCHN
Condition: Carcinoma, Squamous Cell
MeSH terms: Carcinoma, Squamous Cell
Study type: Interventional
Phase: Phase 3

Summary: 
      To describe the clinical response rate in two groups (Docetaxel plus cisplatin plus 5-FU,
      versus Cisplatin plus 5-FU) after 3 cycle of neoadjuvant chemotherapy.
    


Intervention name(s): Docetaxel
Intervention type(s): Drug
Intervention MeSH terms: Docetaxel
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
A Study to Evaluate Hormone Patterns and Ovarian Follicular Activity With DR-1021
Condition: Healthy
Study type: Interventional
Phase: Phase 3

Summary: 
      This is a multi-center study to evaluate hormone levels with the oral contraceptive regimen
      DR-1021.
    

Detailed description: 
      Female volunteers, aged 18-35 years old who met all Inclusion and no Exclusion Criteria, were
      enrolled in this study. All participants were current users of a standard 21/7 oral
      contraceptive regimen (21 days of combination progestin/estrogen followed by 7 days placebo)
      and had completed at least one 28-day cycle prior to beginning the Cycle 1 baseline cycle.
      After completing screening, all enrolled participants continued to receive the same regimen
      of 150 μg DSG /20 μg EE combination pills once daily for 21 days followed by placebo once
      daily for 7 days during Cycle 1 (the Run-In phase). Following completion of Cycle 1,
      participants were randomly assigned to receive either DR-1021 or Mircette during Cycle 2. All
      participants who completed Cycle 2 were to receive Kariva during Cycle 3; however,
      participants were only followed for the first 21 days of this 28-day regimen, after which
      they were considered study completers.
    

Intervention name(s): DR-1021; Mircette®; Kariva®
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Twenty-one 150 μg desogestrel/20 μg ethinyl estradiol (EE) combination tablets plus seven 10 μg EE tablets.; Twenty-one 150 μg desogestrel/20 μg ethinyl estradiol (EE) combination tablets plus two placebo tablets plus five 10 μg EE tablets.; Twenty-one 150 μg desogestrel/20 μg ethinyl estradiol (EE) combination tablets plus two placebo tablets plus five 10 μg EE tablets.
Other intervention name(s): desogestrel/ethinyl estradiol; desogestrel/ethinyl estradiol; desogestrel/ethinyl estradiol
Intervention MeSH terms: Estradiol 17 beta-cypionate; Estradiol 3-benzoate; Ethinyl Estradiol; Desogestrel; Oviol; Estradiol; Polyestradiol phosphate
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
The Effect of Type 1 Diabetes on Pan-Arterial Vascular Function and Insulin Sensitivity in Humans
Condition: Diabetes Type 1
MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1; Insulin Resistance
Study type: Observational

Summary: 
      Arterial vascular disease is the major cause of morbidity and mortality for Type 1 diabetic
      patients (DM1). Metabolic insulin resistance (metIR), even in the absence of hyperglycemia,
      conveys a 1.5 to 3-fold increased CVD risk in the general population. Metabolic Insulin
      Resistance (MetIR) has been repeatedly shown to be prevalent in adults and adolescents with
      DM1. MetIR in obesity and DM2 are accompanied by vascular insulin resistance (vasIR) which is
      characterized by impaired vasodilatory action of insulin on resistance or microvascular
      vessels. VasIR has not been systematically studied in DM1. We hypothesize that in young
      adults DM1 impairs both baseline and insulin-responsive vascular function throughout the
      arterial vasculature.
    

Detailed description: 
      In our study, 20 healthy control subjects will be compared to 20 DM1 patients (18-40 yrs). We
      will assess function in conduit (pulse wave velocity-PWV, flow-mediated dilation-FMD and
      augmentation index-AI), resistance (post-ischemic flow velocity-PIFV) and heart and skeletal
      muscle microvascular (contrast enhanced ultrasound-CEU) vessels before and after 2 hrs of a
      euglycemic insulin clamp.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Type 1 Diabetes Healthy Controls
      
Keywords: Diabetes Type 1; Vascular Function
Safety, Tolerability and Efficacy of SPD503 in Treating ADHD in Children Aged 6-17
Condition: Attention Deficit Disorder With Hyperactivity
MeSH terms: Hyperkinesis; Attention Deficit Disorder with Hyperactivity
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to determine the effects of SPD503 compared to placebo on tasks
      of sustained attention in children and adolescents aged 6-17 diagnosed with ADHD.
    


Intervention name(s): SPD503 (Guanfacine HCl); Placebo
Intervention type(s): Drug; Drug
Intervention MeSH terms: Guanfacine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
Intermittent Preventive Treatment With Azithromycin-containing Regimens in Pregnant Women in Papua New Guinea
Condition: Malaria in Pregnancy; Sexually Transmitted Infections; Anaemia
MeSH terms: Infections; Sexually Transmitted Diseases; Anemia
Study type: Interventional
Phase: Phase 3

Summary: 
      The purpose of this study is to determine whether repeated courses of
      sulphadoxine-pyrimethamine (SP) in combination with azithromycin given at Antenatal Clinic,
      leads to lower rates of low birth weight deliveries (<2.5 kg) among Papua New Guinean women,
      than the current standard treatment of SP and chloroquine.
    


Intervention name(s): chloroquine, sulphadoxine pyrimethamine, LLIN; azithromycin, sulphadoxine pyrimethamine, LLIN
Intervention type(s): Drug; Drug
Intervention description(s): > 50Kg: chloroquine base 150 mg 4 tablets daily for 3 days, plus sulphadoxine pyrimethamine 1500/75 mg single dose.
< 50 Kg: chloroquine base 150 mg 3 tablets daily for 3 days, plus sulphadoxine pyrimethamine 1500/75 mg single dose.
Given at enrolment, 14-26 weeks gestation, by mouth.; sulphadoxine pyrimethamine (1500 mg/75 mg as single dose) plus azithromycin (1 g twice daily for 2 days).
Given three times by mouth at monthly intervals, commencing at between 14 and 26 weeks gestation.
Other intervention name(s): sulfadoxine-pyrimethamine; Zithromax; sulfadoxine-pyrimethamine
Intervention MeSH terms: Azithromycin; Chloroquine; Pyrimethamine; Sulfadoxine; Fanasil, pyrimethamine drug combination
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Primary purpose: Prevention
Keywords: Plasmodium falciparum; Plasmodium vivax; azithromycin; sulphadoxine pyrimethamine; low birth weight; haemoglobin; Chlamydia trachomatis; Neisseria gonorrhoeae; Treponema pallidum
Diagnostic Breath Analysis Study to Detect Sepsis
Condition: Puerperal: [Major Infection] or [Sepsis]
MeSH terms: Sepsis; Toxemia
Study type: Observational

Summary: 
      Analysis of exhaled breath samples using a high-speed gas chromatography medical device will
      identify Volatile Organic Compounds (VOC) that are specifically associated with SIRS and
      Sepsis.

      Primary outcome measures will include the assessment of the zNose Diagnostic Breath Analysis
      System, which includes high-speed gas chromatography, in the early detection of SIRS and
      Sepsis as compared with current SIRS and Sepsis evaluation methods.
    

Detailed description: 
      Exhaled breath samples were collected in a Tedlar bag or with a proprietary collection tube.
      The collection devices are clean and intended for single-use. They are manufactured with
      approved medical grade materials. Patients were asked to breath into a single-use, medical
      grade mouthpiece which is connected to the collection bag or tube. The design of the
      collection devices does not allow the patient to rebreathe the exhaled gas sample. Patients
      enrolled in the clinical study were asked to breathe into the breath collection device for 2
      to 4 breaths. Breath collection is completely non-invasive. Breath samples were held for 30
      days and retested at the end of the 30 day period to determine if the VOC profile has changed
      over time.
    

Intervention name(s): Diagnostic Breath Analysis
Intervention type(s): Device
Intervention description(s): Conduct exhaled breath samples
Observational model: Case-Only
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients enter through Emergency Room, are identified at risk for SIRS
      
Biospecimen description: 
      Exhaled breath saved for re-test in thirty days
Safety and Effectiveness of Propagermanium in Focal Segmental Glomerulosclerosis Participants Receiving Irbesartan
Condition: Focal Segmental Glomerulosclerosis
MeSH terms: Glomerulosclerosis, Focal Segmental
Study type: Interventional
Phase: Phase 2

Summary: 
      This study will be evaluating the safety and efficacy of propagermanium for the treatment of
      participants with FSGS who are already taking irbesartan by:

        -  monitoring symptoms that participants may experience while on the study,

        -  measuring levels of protein in participant's urine and kidney function during the course
           of the study,

        -  measuring the levels of propagermanium and irbesartan that enters into participant's
           urine and blood, and

        -  comparing the propagermanium outcomes to participants' pre-study and placebo outcomes.

      Eligible participants will randomly be assigned to one of two arms to receive both the
      propagermanium and placebo in different orders as follows, either:

      Treatment Period 1 taking a propagermanium capsule twice a day for 16 weeks, followed by a
      six week washout period followed by Treatment Period 2 taking a placebo capsule twice a day
      for 16 weeks.

      OR Treatment Period 1 taking a placebo capsule twice a day for 16 weeks, followed by a six
      week washout period followed by Treatment Period 2 taking a propagermanium capsule twice a
      day for 16 weeks.
    


Intervention name(s): Propagermanium; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Immediate release capsule; Placebo capsule
Other intervention name(s): PPG; repagermanium; DMX-200
Intervention MeSH terms: Propagermanium
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: Double-blind, Randomised, Placebo-Controlled, Crossover Study
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Treatment of Bacterial Vaginosis Combined With Human Lactobacilli
Condition: Bacterial Vaginosis
MeSH terms: Vaginosis, Bacterial; Vaginal Diseases
Study type: Interventional
Phase: Phase 4

Summary: 
      The primary objectives of this study were to characterize lactobacilli of human vaginal
      origin and to investigate if more extended antibiotic treatment against Bacterial Vaginosis,
      together with adjuvant lactobacilli treatment, could increase the cure rate and furthermore,
      to investigate factors that could influence relapse.
    

Detailed description: 
      Nine of the vaginal Lactobacillus strains isolated from healthy Swedish women and
      characterized in the present study were selected for the clinical trial.All women with
      symptomatic BV fulfilling the inclusion criteria were consecutively offered to participate in
      a prospective study of adjuvant lactobacilli given in addition to antibiotics. Women included
      were regularly menstruating women, 18 years or older, with normal gynaecological status, not
      pregnant or breast-feeding and without signs of other genital tract infections. Exclusion
      criteria were patients with hormonal intrauterine device without regular menstruation; women
      infected with Chlamydia trachomatis or with Trichomonas vaginalis, or with a clinical candida
      infection.
    

Intervention name(s): lactobacilli
Intervention type(s): Drug
Intervention description(s): women were given a seven days course of daily 2% vaginal clindamycin cream (Dalacin vaginal cream 2%, Pfizer Norway Ltd) together with oral clindamycin 300 mg BID for 7 days (Dalacin 300 mg, Pfizer Norway Ltd). Oral clindamycin treatment was also given to the patient's sexual partner.
Other intervention name(s): Treatments with differnt lactobacilli two zidovaltreatments
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: treatment, lactobacilli
Prebiotic Fibre Supplementation and Gut Microbiota in Non-alcoholic Fatty Liver Disease
Condition: Non-alcoholic Fatty Liver Disease
MeSH terms: Liver Diseases; Fatty Liver; Non-alcoholic Fatty Liver Disease
Study type: Interventional

Summary: 
      Non-alcoholic fatty liver disease (NAFLD) is a condition where accumulation of fat in the
      liver leads to metabolic dysfunction. Currently there are no approved treatments for NAFLD.
      Part of the metabolic dysfunction may arise through changes in the gut microbiota. Prebiotic
      fibres have beneficial effects on glucose tolerance, body weight, and gut microbiota;
      therefore they may have potential as part of a dietary strategy for NAFLD treatment.
    

Detailed description: 
      The main objective of this study is to assess the effect of prebiotic fibre supplementation,
      in conjunction with diet-induced weight loss, on reduction in liver fat and injury.

      Primary Objective - determine the change in hepatic injury (fibrosis and inflammation) and
      hepatic fat (percent fat) over 6 months in NAFLD patients treated with prebiotic or placebo
      during weight loss.

      Secondary Objectives - determine the changes in appetite, body composition, glycemic and
      insulinemic responses, quality of life with prebiotic or placebo during weight loss, and
      examine mechanisms related to prebiotic-induced changes in gut microbiota and lipogenesis.
    

Intervention name(s): Prebiotic fibre; Placebo; Weight Loss
Intervention type(s): Dietary Supplement; Dietary Supplement; Behavioral
Intervention description(s): Oligofructose-enriched inulin (Synergy1); Maltodextrin; All participants will receive 10 one-on-one sessions with a Registered Dietitian designed to achieve 10% weight loss over 6 months. The sessions will focus on nutrition education and behavior counseling to reduce food intake and improve dietary quality.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Dietary intervention; Prebiotic fibre; Weight loss; Gut microbiota; Obesity; Liver fat; Hepatic fibrosis