bioihbjkbjnl/mcl2 huggingface dataset:
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Condition: Hereditary Inclusion Body Myopathy; Distal Myopathy With Rimmed Vacuoles; Distal Myopathy, Nonaka Type; GNE Myopathy
MeSH terms: Muscular Diseases; Distal Myopathies
Study type: Interventional
Phase: Phase 3

Summary: 
      The primary objective of this study is to evaluate the effect of 6 g/day aceneuramic acid
      extended-release (Ace-ER) treatment of participants with GNEM on upper extremity muscle
      strength (upper extremity composite [UEC] score) as measured by dynamometry.
    


Intervention name(s): aceneuramic acid extended-release (Ace-ER); Placebo
Intervention type(s): Drug; Drug
Intervention description(s): tablets for oral use; tablets for oral use
Other intervention name(s): UX001; sialic acid extended-release (SA-ER)
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: GNE Myopathy; GNEM; HIBM; Nonaka; Hereditary Inclusion Body Myopathy; DMRV
Pilot Study on Sleep Management for US Veterans
Condition: Insomnia
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The aim of the proposed pilot study is to scientifically evaluate the use of Mind-Body
      Bridging Program (MBBP) as an effective intervention for improving health outcomes in
      veterans exhibiting sleep disturbance in the Primary Care Clinic at the Veterans
      Administration Salt Lake City Health Care System (VASLCHCS).
    

Detailed description: 
      Participants will be randomly assigned to either MBBP or a treatment as usual sleep hygiene
      (SH) group. The interventions will last two weeks in which participants will attend 2 classes
      1-1.5 hr each over two consecutive weeks. Assessment of the effects of the treatments will be
      based on self-report outcome measures; participants will complete five questionnaires
      approximately one week prior to the start of the interventions (pre-treatment) and one week
      after the second session (post-treatment). The sleep quality assessment questionnaire will be
      additionally completed after the first session (Week 1).
    

Intervention name(s): Mind-Body Bridging Program; Sleep Hygiene
Intervention type(s): Behavioral; Behavioral
Intervention description(s): Bridging aims to reduce the impact of negative thought patterns that contribute to stress in the body.; Treatment as usual
Other intervention name(s): MBBP; SH
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Participant)
Primary purpose: Treatment
Keywords: Sleep problems; Awareness training program; Mind body bridging
Intravenous Plasma Treatment for Parkinson's Disease
Condition: Idiopathic Parkinson Disease
MeSH terms: Parkinson Disease
Study type: Interventional
Phase: Early Phase 1

Summary: 
      This study will do a preliminary evaluation of a possible treatment for mild-to-moderate
      Parkinson's disease. Patients will be treated with either transfusions of plasma from young
      donors. or with placebo transfusions. In the following several months, Parkinson's symptoms
      will be monitored and compared for the two groups.
    

Detailed description: 
      This is a double-blind placebo controlled trial, designed to evaluate the efficacy of plasma
      from volunteer donors of ages 18-25. This young fresh frozen plasma (yFFP) will be given
      intravenously in two doses of 12.5 ml/kg each, with 40-56 hours between doses. In the
      following 24 weeks, Parkinson's symptoms will be monitored using the short version of the
      Stanford Presenteeism Scale (SPS-6), and a modified version of the UPDRS scale.
    

Intervention name(s): [21CFR640.30] Plasma from 18 - 25 year old volunteer donors; Saline
Intervention type(s): Biological; Other
Intervention description(s): 12.5 ml/kg intravenous Spectrum Plasma yFFP, per each of two infusions (25 ml/kg total), in combination with ongoing normal standard treatment.; 12.5 ml/kg intravenous 0.9% sodium chloride with 0.1% riboflavin , per each of two infusions (25 ml/kg total), in combination with ongoing normal standard treatment.
Other intervention name(s): Young Fresh Frozen Plasma (yFFP); Normal saline
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Intervention model description: Placebo controlled trial
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary purpose: Treatment
A Study of the Effectiveness of an HIV Vaccine (ALVAC vCP205) to Boost Immune Functions in HIV-Negative Volunteers Who Have Already Received an HIV Vaccine
Condition: HIV Infections; HIV Seronegativity
MeSH terms: HIV Infections
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to see if it is safe to give an HIV vaccine (vCP205) to
      volunteers who received an HIV vaccine at least 2 years ago, and to study how the immune
      system responds to this vaccine.

      Vaccines are given to people to try to resist infection or prevent disease. There are a
      number of different HIV vaccines that are currently being tested. The vaccines that seem to
      be the most promising are canarypox vaccines, known as ALVAC vaccines; the vaccine tested in
      this study is ALVAC-HIV vCP205. This study will look at the safety of the vaccine and how the
      immune system responds to it.
    

Detailed description: 
      Vaccines may provide a route of therapy against HIV-1 infections by boosting the immune
      system responses. An artificially constructed HIV-1 vaccine (NYCBH), using vaccinia virus as
      its vector, has the advantage of conferring both cellular and humoral immune responses that
      are long-lived. Studies have shown that a second artificially constructed vector vaccine,
      HIV-1 canarypox (vCP205), also increases CD8+ cytotoxic T lymphocyte (CTL) activity, a
      cell-mediated immune response. Yet, immune responses are not boosted in volunteers previously
      vaccinated with vaccinia-based HIV-1 vaccines when a second vaccination with the same vaccine
      is given. One theory for vaccinia vaccine's failure is that immunologic barriers by
      antibodies to the vector itself may be responsible. This study examines the effectiveness of
      boosting the immune responses following vCP205 vaccination in the following: 1) volunteers
      who were previously immunized with vCP205 vaccine who may or may not have shown increased
      immune responses following the first immunization, and 2) volunteers who were previously
      immunized with NYCBH vaccine.

      Upon study entry volunteers receive one injection of ALVAC-HIV vCP205. Temperature and
      symptoms should be recorded by the volunteer each day for 2 days and reported to the clinic
      staff. Volunteers will have seven clinic visits for drawing blood, collecting urine
      specimens, and performing clinical evaluations. At Month 3 HIV testing will be done.
      Volunteers will be followed for 3 months, with a passive follow-up call at the end of a year
      and once or twice a year for the next 5 years. Counseling on avoidance of HIV infection and
      pregnancy will be done.
    

Intervention name(s): ALVAC-HIV MN120TMG (vCP205)
Intervention type(s): Biological
Primary purpose: Prevention
Keywords: Injections, Intramuscular; HIV-1; AIDS Vaccines; CD4-Positive T-Lymphocytes; CD8-Positive T-Lymphocytes; T-Lymphocytes, Cytotoxic; Gene Products, gag; HIV Seronegativity; Immunologic Memory; Antibodies, Viral; Genetic Vectors; Gene Products, env; Poxviridae; HIV Preventive Vaccine
A Study of RC48-ADC in Subjects With Advanced Non-small Cell Lung Cancer
Condition: Non Small Cell Lung Cancer
MeSH terms: Lung Neoplasms; Carcinoma, Non-Small-Cell Lung
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      This study will evaluate the efficacy and safety of RC48-ADC for injection in subjects with
      advanced non-small cell lung cancer with HER2 overexpression or HER2 mutation.
    


Intervention name(s): RC48
Intervention type(s): Drug
Intervention description(s): Participants will be treated with RC48-ADC at a dose of 2.0 mg/kg, every 2 weeks. They will continue the medication until one of the following conditions occurred: disease progression, intolerance of toxicity, investigators believed that the subject could no longer benefit from treatment, or this study ended.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Effect of Anesthesia Method on Neutrophil-Lymphocyte Ratio
Condition: Inflammation; Anesthesia
MeSH terms: Inflammation
Study type: Observational

Summary: 
      The neutrophil/lymphocyte ratio (NLR) is a simple and inexpensive marker of the inflammatory
      response. NLR is affected not only by surgical trauma but also by the anesthetic method. The
      method of anesthesia can affect NLR, thereby modulating the inflammatory response and
      surgical outcomes. In this study, it was aimed to evaluate the relationship between blood NLR
      and anesthesia techniques in patients undergoing forearm surgery, and the secondary aim was
      to evaluate the relationship between Platelet/lymphocyte ratio (TLR), Mean Platelet Volume
      (MPV) and anesthesia techniques.
    


Intervention name(s): peripheral nerve block
Intervention type(s): Procedure
Intervention description(s): local anesthetic application to the brachial plexus
Observational model: Cohort
Time perspective: Retrospective
Sampling method: Probability Sample
Study population: 
        Patients aged 18-65 years who have underwent forearm surgery
      
Keywords: General Anesthesia; Neutrophil Lymphocyte Ratio; Peripheral Nerve Block
Follow-up Study to Investigate Long Term Effect of Supplementing Pre- and Probiotics in Children Born by C-Section
Condition: Caesarean Section Born Children
Study type: Observational

Summary: 
      This study investigate the long term effect of supplementation of pre- and probiotics in
      early life in children born by Caesarean Section.
    

Detailed description: 
      This is an extension of investigation on the long term effect of supplementation of pre- and
      probiotics in early life in children born by Caesarean Section from an earlier completed
      study.
    

Intervention name(s): Prebiotics; Prebiotics & Probiotics
Intervention type(s): Other; Other
Intervention description(s): To study the long term effect of supplementation in early life; To study the long term effect of supplementation in early life
Observational model: Case-Control
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Children who had completed Juliu Sn study until 16 weeks of age.
      
Biospecimen description: 
      Stool samples will be collected from subjects at least once during study visits.

      Voluntary blood sample will be collected from subjects.
Nicotinic Cholinergic Modulation as a Novel Treatment Strategy for Aggression Associated With Autism
Condition: Autism Spectrum Disorder; Aggression; Irritability
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Aggression
Study type: Interventional
Phase: Phase 1

Summary: 
      Some individuals with autism spectrum disorders (ASD) also demonstrate irritability or
      aggression, which can interfere with functioning. The purpose of this pilot study is to test
      whether transdermal nicotine is effective for irritability and/or aggression in adults with
      ASD using a double-blind, placebo-controlled clinical trial. Subjects will participate in
      three visits. At the first visit, subjects are screened for eligibility and enrolled.
      Baseline measures include rating scales and a frustrative computerized task. They will then
      wear seven days of transdermal nicotine or placebo. Visit two is on day seven and the study
      measures are repeated, vital signs and side effects monitored. Subjects will return for a
      third and final visit on day 21 to repeat the study measures performed during visit two.
    


Intervention name(s): Transdermal nicotine; Transdermal placebo
Intervention type(s): Drug; Other
Intervention MeSH terms: Nicotine
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Autism spectrum disorder; Aggression; Irritability; Nicotine; Nicotinic acetylcholine receptor; Nicotinic agonist; Adult autism
A Non-inferiority Trial to Compare MVA-BN® Smallpox Vaccine to ACAM2000®
Condition: 18-42 Year Old Healthy Vaccinia-naïve Subjects
MeSH terms: Vaccinia; Smallpox
Study type: Interventional
Phase: Phase 3

Summary: 
      To demonstrate the efficacy of MVA-BN® in terms of vaccinia-specific Plaque Reduction
      Neutralization Test (PRNT) antibody response and by showing that vaccination prior to
      administration of ACAM2000® results in an attenuated take.
    

Detailed description: 
      To demonstrate the efficacy of MVA-BN® by assessing non-inferiority of MVA-BN® compared to
      ACAM2000® in terms of vaccinia-specific Plaque Reduction Neutralization Test (PRNT) antibody
      response at the Peak Visits (Day 42 for Group 1 and Day 28 for Group 2) and by showing that
      vaccination with MVA-BN® prior to administration of ACAM2000® results in an attenuation of
      take.
    

Intervention name(s): MVA BN®; ACAM2000®
Intervention type(s): Biological; Biological
Intervention description(s): 0.5 ml MVA BN® with a nominal titer of 1x10E8 TCID50, administered as a subcutaneous injection; 0.0025 ml ACAM2000®, consisting of 2.5-12.5x10E5 plaque forming units of live vaccinia virus (VACV). Picked up with a bifurcated needle and administered by the percutaneous route (scarification) using 15 jabs of that bifurcated needle.
Other intervention name(s): IMVAMUNE; IMVANEX
Intervention MeSH terms: Smallpox and monkeypox vaccine modified vaccinia ankara-bavarian nordic
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Prevention
The Safety and Pharmacokinetics Study of TAKC-02 Inhalation Solution in Healthy Adult Males.
Condition: Healthy Volunteer
MeSH terms: Respiratory Aspiration
Study type: Interventional
Phase: Phase 1

Summary: 
      MEX3B is an RNA-binding protein that is conserved in many animal species and has wide range
      of biological function. The MEX3B protein is deeply involved in the expression of various
      cytokines associated with the onset and exacerbation of several diseases such as inflammatory
      diseases, metabolic diseases, and malignant tumors. TAKC-02 is a nucleic acid medicine,
      antisense oligonucleotide, inhibits the MEX3B synthesis expected to have potential as new
      medication.

      This plans to evaluate the safety profile of the inhalation solution in order to develop
      TAKC-02 for severe asthma.

      The study is a double-blind, randomized, placebo-controlled Phase I study. The primary
      objective of the study is to assess the safety and tolerability of single and multiple
      inhaled doses of TAKC-02 in healthy male subjects. The study is a Single Ascending Dose (Step
      1) followed by a Multiple Comparative Dose (Step 2).
    

Detailed description: 
      This initial Phase I study will be conducted in normal healthy male volunteers to obtain an
      unconfounded understanding of the safety, tolerability and pharmacokinetics of TAKC-02
      inhalation solution. TAKC-02 is being developed as a medication for uncontrolled severe
      asthma. The first-in-human, randomized, double-blind trial will evaluate single ascending
      doses (SAD) and subsequently multiple ascending doses (MAD) of TAKC-02 and placebo.

      Step 1 (SAD) :

      There will be 5 cohort of 6 (Cohort 1, 2, 3) or 8 (Cohort 4, 5) subjects each. Subjects in
      each cohort are randomized in a 2:1 ratio (Cohort 1, 2, 3) and 3:1 ratio to receive a single
      dose of the study drugs, TAKC-02 or Placebo. The SAD study will evaluate the safety and
      tolerability of a single dose of TAKC-02 and determine the starting and maximum doses for
      Step 2. Pharmacokinetics will be also analyzed. One cohort (cohort 4.5) maybe added depending
      on the incidence of adverse effect in the lower dosing group.

      Step 2 There will be 2 cohort f 8 subjects each. Subjects in both cohorts are randomized in a
      3:1 ratio to receive multiple doses of the study drugs, TAKC-02 or Placebo. The safety and
      tolerability of repeated administration of TAKC-02 for 2 weeks will be evaluated, and the
      maximum dose of this drug in the next phase will be estimated. Pharmacokinetics will be also
      analyzed.
    

Intervention name(s): TAKC-02; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Inhalation Solution; Inhalation Solution
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: antisense oligonucleotides; anti asthmatic agents
Hospital-based Home Care for Children With Cancer
Condition: Neoplasms; Children
Study type: Interventional

Summary: 
      The purpose of this non-randomized controlled intervention study was to evaluate the effects
      of a hospital-based home care program for children with cancer at a university hospital in
      Denmark. The hypothesis was that hospital-based home care could replace an out-patient visit
      or an in-patient admission without increasing the incidence of adverse events and costs.
      Furthermore, to enhance the children's quality of life and the psychosocial impact on the
      family.
    

Detailed description: 
      Hospital-based home care for children with cancer is widely used but controlled studies are
      relatively rare and the evidence base is limited. This prospective non-randomized controlled
      intervention study aimed to evaluate hospital-based home care for children with cancer.

      A hospital-based home care program was conducted August 2008 - December 2009. Participants
      were children with cancer and their parents. The intervention group received part of their
      therapy, such as intravenous chemotherapy and antibiotics, as home care by a hospital-based
      home care nurse with paediatric-oncology experience. Children in the control group received
      all their treatment at the hospital.

      Fifty-one children between 0 and 18 years were included in the home care program, and 47
      children were included in the control group. After each home visit the parents answered a
      questionnaire. The results from 657 questionnaires showed that 100% were very satisfied or
      satisfied with home care and all would choose home care again, if offered as an option. There
      were no deaths or adverse effects of intravenous chemotherapy or antibiotics. A cost analysis
      based on the expenses associated with home care in correlation with standard cost of
      inpatient admissions and outpatient visits showed that the hospital-based home care programme
      was economically equal. The children's and the families' quality of life may be enhanced. The
      results indicate that the hospital-based home care program is safe, economically neutral and
      the parents' satisfaction with the program is major.
    

Intervention name(s): Hospital-based home care
Intervention type(s): Other
Intervention description(s): Low-intensive intravenous antibiotics b) intravenous low-toxic chemotherapy c) blood samples drawn from the central venous catheter or peripheral vein d) subcutaneous injections e) nutrition treatment f) pain management (e.g. controlling an intravenous morphine pump g) supportive care e.g. changing dressings.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Supportive Care
Keywords: Hospital-based home care; Pediatric oncology
An Investigation Of The Absorption And Pharmacokinetics Of Multiple Doses Of Two Controlled Release Pregabalin Tablets As Compared To Multiple Doses Of The Immediate Release Pregabalin Capsule In Healthy Volunteers
Condition: Healthy
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to 1) evaluate the extent of absorption of multiple doses of two
      pregabalin controlled release tablets as compared to multiple doses of the pregabalin
      immediate release capsule, 2) evaluate the pharmacokinetics of multiple doses of two
      pregabalin controlled release tablets as compared to multiple doses of pregabalin immediate
      release capsule and 3) evaluate the safety and tolerability of multiple doses of two
      pregabalin controlled release tablets as compared to multiple doses of the pregabalin
      immediate release capsule.
    

Detailed description: 
      Evaluate the absorption, pharmacokinetics, safety/tolerability of multiple doses of two
      pregabalin controlled release tablets as compared to multiple doses of pregabalin immediate
      release capsule
    

Intervention name(s): Pregabalin controlled release, 82.5 mg; Pregabalin controlled release, 165 mg; Pregabalin immediate release, 75mg
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Two tablets of 82.5 mg controlled release (administered concurrently) once daily for four days.; 165 mg controlled release tablet administered once daily for four days.; 75 mg immediate release capsule administered every 12 hours for four days
Intervention MeSH terms: Pregabalin
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Other
Keywords: pregabalin; pharmacokinetics; bioavailability; bioequivalence
Lifestyle Intervention and Physical Capacity in Patients With Morbid Obesity
Condition: Physical Capacity
MeSH terms: Obesity, Morbid
Study type: Observational

Summary: 
      Lifestyle intervention and physical capacity in patients with morbid Obesity (LEICO)
    

Detailed description: 
      It is still uncertainty about which is the most effective lifestyle treatment to reducing
      body weight and increased quality of life. Increased physical capacity and muscle mass lead
      to increased energy expenditure, thus explaining parts of the difference in weight loss
      achieved after lifestyle treatment. However, literature in this area is limited and need
      better documentation of how, and to what extent increased physical capacity can affect the
      effect of lifestyle treatment offered to patients in the health service.
    

Intervention name(s): Lifestyle
Intervention type(s): Other
Intervention description(s): Lifestyle intervention an effect on weight and physical capacity
Observational model: Cohort
Time perspective: Retrospective
Sampling method: Probability Sample
Study population: 
        Obese patient seeking lifestyle intervention program