bioihbjkbjnl/mcl2 huggingface dataset:
Outcome Measures in Duchenne Muscular Dystrophy: A Natural History Study
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Observational

Summary: 
      Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding
      of DMD natural history. This study aim to assess the natural history of DMD through a
      composite assessment tool capable of capturing disease progression linking ambulant and
      non-ambulant phases of the disease.
    

Detailed description: 
      Novel emerging therapies for Duchenne Muscular Dystrophy (DMD) require a deeper understanding
      of DMD natural history. This study aim to assess the natural history of DMD through a
      composite assessment tool capable of capturing disease progression linking ambulant and
      non-ambulant phases of the disease.

      With a recruitment target of 80 DMD patients across 5 centres (London, Newcastle, Paris,
      Leiden, Nijmegen), subjects are assessed 6 monthly according to a shared protocol.
      Assessments include 6-minute walk distance (6MWD), North Star Ambulatory Assessment (NSAA),
      Performance of Upper Limb (PUL) and MyoSet (myogrip, myopinch and moviplate). Both ambulant
      and non-ambulant subjects undergo upper limb evaluation and respiratory function test
      including forced vital capacity (FVC), maximum inspiratory and expiratory pressures
      (MIP/MEP). A subgroup of patients performs annual whole body DEXA scan. An imaging sub-study
      will aim to characterize muscle (upper/lower limb) and brain MRI.

      The investigators will analyze the longitudinal data for the different assessment tools and
      explore correlations among them.

      This study will offer a comprehensive natural history of DMD including novel outcome
      measures, allowing to capture disease progression and explore the relationship between
      different assessment tools.
    

Intervention name(s): Set of assessment tools
Intervention type(s): Other
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients with Duchenne muscular dystrophy (DMD) recruited across 5 specialised
        neuromuscular centres (London, Newcastle, Paris, Leiden, Nijmegen)
      
Biospecimen description: 
      Single sample suitable for testing for SNP genotyping. This can be either a blood sample,
      saliva sample or already extracted DNA sample (including stored DNA samples from a
      laboratory.
    
Keywords: Duchenne muscular dystrophy; Natural history; Outcome measures; North Star Ambulatory Assessment (NSAA); 6 minute walk distance (6MWD); Myoset; Exon skipping
A Pharmacokinetic Interaction Study Between TMC435, Atorvastatin and Simvastatin in Healthy Participants
Condition: Healthy Participants
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to evaluate effects of steady-state concentrations (constant
      concentration of medication in the blood) of TMC435 on the single dose pharmacokinetics (what
      the body does to the medication) of atorvastatin, the active metabolites ortho- and
      parahydroxylated atorvastatin, simvastatin and the active metabolite simvastatin acid. Plasma
      hydroxy-3-methylglutaryl coenzyme A (HMG-CoA) reductase inhibitory activity after a single
      dose of atorvastatin or simvastatin, either alone or in combination with TMC435 will be also
      evaluated.
    

Detailed description: 
      This is a Phase I, 2-panel, open-label (all people know the identity of the intervention)
      study in healthy participants to investigate the pharmacokinetic interaction between TMC435
      and the HMG-CoA reductase inhibitors atorvastatin and simvastatin. The study consists of a
      screening period (21 days prior to the first intake of study medication), a 17-day treatment
      period and a 5-7 days follow-up period. Approximately thirty six healthy participants will be
      equally divided to 2 panels (n=18 per panel). Within each panel all participants will receive
      the same treatments in the same order. In Panel 1, each participant will be administered a
      single oral 40-mg atorvastatin dose on Day 1 and Day 13. The participants will receive TMC435
      at a dose of 150 mg once daily from Day 4 until Day 15. In Panel 2, each participant will be
      administered a single oral 40-mg simvastatin dose on Day 1 and Day 13. TMC435 at a dose of
      150 mg will be administered once daily from Day 4 until Day 15. Treatment duration (including
      Day -1) will be 17 days for both panels (Panel 1 and Panel 2). Safety and tolerability will
      be evaluated throughout the study.
    

Intervention name(s): TMC435; Atorvastatin; Simvastatin
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Type=exact number, unit=mg, number=150, form=capsule, route=oral. TMC435 will be administered at a dose of 150 mg once daily from Day 4 until Day 15.; Type=exact number, unit=mg, number=40, form=tablet, route=oral. Atorvastatin will be administered as a single oral 40-mg dose on Day 1 and Day 13.; Type=exact number, unit=mg, number=40, form=tablet, route=oral. Simvastatin will be administered as a single oral 40-mg on Day 1 and Day 13.
Intervention MeSH terms: Simeprevir; Atorvastatin; Simvastatin
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Healthy participants; TMC435; HMG-CoA reductase inhibitors; Atorvastatin; Simvastatin; Pharmacokinetic interaction
Sleep Intervention for Youth With Type 1 Diabetes
Condition: Diabetes
MeSH terms: Diabetes Mellitus
Study type: Interventional

Summary: 
      Sleep intervention for youth with diabetes
    

Detailed description: 
      It is known that sleep disturbances in healthy youth have negative effects on neurobehavioral
      functioning. Further, data support that individuals with diabetes have more sleep
      disturbances and compromised neurobehavioral functioning than individuals without diabetes.
      However, the joint impact of sleep and glucose on neurobehavioral functioning in youth with
      type 1 diabetes (T1DM) is not yet known. Therefore, our primary study aims are to: (1)
      determine the relative contributions of various sleep disturbances on glucose control in
      youth with T1DM; (2) examine the joint impact of glucose control and sleep disturbances on
      neurobehavioral outcomes in youth with T1DM; and (3) determine if increasing sleep duration
      relative to youth's typical schedules contributes to changes in glycemic control and
      neurobehavioral performance. These aims will be achieved by following 120 youth with T1DM
      ages 10 through 16 for six days of naturalistic sleep using polysomnography, actigraphy, and
      questionnaires to assess sleep; continuous glucose monitors and hemoglobin A1C values to
      assess glucose control; and standardized cognitive tasks and behavior rating scales to assess
      neurobehavioral functioning. Further, the proposed study is innovative in that it will extend
      existing research by moving from correlational findings to an experimental paradigm by
      randomizing youth with T1DM to either a Sleep Extension or Fixed Sleep Duration condition for
      an additional six days. Once our aims are achieved and a causal link is established, the
      proposed Sleep Extension intervention will advance knowledge about the role of sleep in
      diabetes management and provide a beneficial intervention to help youth with T1DM.
    

Intervention name(s): Sleep extension; Fixed Sleep Duration
Intervention type(s): Behavioral; Behavioral
Intervention description(s): There will be a consultation with families to help youth to be successful in extending their sleep.; There will be a consultation with families to instruct youth to maintain the same average nightly sleep duration during the modification week. The purpose is to control for time and attention.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary purpose: Supportive Care
Keywords: Diabetes; Sleep problems; School; Neurobehavioral
A Study of IL-7 to Restore Absolute Lymphocyte Counts in Sepsis Patients
Condition: Severe Sepsis With Septic Shock
MeSH terms: Sepsis; Toxemia
Study type: Interventional
Phase: Phase 2

Summary: 
      A multicenter, randomized, double-blinded, placebo-controlled study of two dosing frequencies
      of recombinant Interleukin-7 (CYT107) treatment to restore absolute lymphocyte counts in
      sepsis patients; IRIS-7B (Immune Reconstitution of Immunosuppressed Sepsis patients).

      A parallel study will be performed in France to allow a common statistical analysis of the
      primary end points and analysis for the enrolled patient population.
    

Detailed description: 
      Sepsis is the leading cause of death in critically ill patients in most intensive care units
      in Europe and the US. Recently, evidence has accumulated that sepsis progresses from a state
      of hyper-inflammation to a state of immunosuppression. This immunosuppressive phase is
      characterized by increased incidence of secondary infections often with relatively avirulent
      opportunistic type pathogens. Currently, new therapeutic approaches to sepsis are occurring
      using immuno-adjuvants that boost host immunity. One of the most promising agents
      Interleukin-7 is an essential, non-redundant, pluripotent cytokine produced mainly by bone
      marrow and thymic stromal cells that is required for T-cell survival.In addition to its
      anti-apoptotic properties, IL-7 induces potent proliferation of naïve and memory T-cells
      potentially supporting replenishment of the peripheral T-cell pool which is severely depleted
      during sepsis. These effects were confirmed in clinical trials at the National Cancer
      Institute and in HIV+ patients.

      This clinical study will test the ability of IL-7 to restore the absolute lymphocyte counts
      in septic patients who have markedly reduced levels of circulating lymphocytes. An effect
      already confirmed in preclinical models of sepsis.
    

Intervention name(s): Interleukin-7; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): IM (intra-muscular) administration of CYT107 recombinant glycosylated human IL-7 (SC administration for patients with INR (International Normalized Ratio) >2.5 or platelet count < 35,000; IM administration of Placebo (SC administration for patients with INR>2.5 or platelet count < 35,000
Other intervention name(s): CYT107; NaCl 0.9%
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: sepsis lymphocytopenia IL-7; immune reconstitution
Effect of in Vitro Blocking the Common Beta Chain on Cell Viability in Asthma
Condition: Asthma
MeSH terms: Asthma
Study type: Observational

Summary: 
      This study examines in vitro blockade of signaling through the β-chain, on viability,
      activation and differentiation of eosinophils and their progenitors collected in sputum,
      blood and bone marrow samples pre and post-allergen challenge from mild atopic asthmatic
      subjects.
    

Detailed description: 
      The experiments will use sputum samples induced from subjects with mild asthma, undergoing
      allergen inhalation challenges. In general, each sample will be composed of >10% neutrophils
      and >10% macrophages. Samples collected pre-allergen will have a low frequency of eosinophils
      and lymphocytes (<1%), however the percentage of eosinophils will increase to approximately
      12% following allergen challenges. The sputum samples will be processed in DPBS (without
      dithiothreitol), and the cell suspension will be adjusted to 1 million cells/ml in DMEM with
      penicillin and streptomycin. A cytospin will be made for differential cell counts. The mixed
      cell population at 5 million cells/ml will be incubated for 48 hours at 37 degrees Celcius ±
      β-chain MAb at a concentration of 100 mcg/ml. After 48 hours the cell culture medium will be
      removed for assay of cytokines and chemokines by ELISA. Cells will be resuspended in PBS and
      Binding Buffer (BD Pharmingen, Cat no. 556454),stained for assessments by flow cytometry, and
      analyzed in duplicate.

      Experiments will use blood (80 ml) and bone marrow aspirates (5ml) from atopic asthmatics
      taken pre and 24hr post allergen challenge. Methylcult micro-culture colonogenic assays will
      be performed to enumerate outgrowth of Eo/Baso-CFU and GM-CFU from CD34+ cells populations
      collected from the blood and bone marrow samples. Methylcult assays will be performed with
      CD34+ enriched cell populations in the presence of IL-5,IL-3 and GM-CSF +/- CSL311. Following
      14 days culture, colonies will be enumerated.
    

Intervention name(s): Allergen
Intervention type(s): Other
Intervention description(s): Allergen extract is administered by inhalation.
Observational model: Cohort
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Subjects with mild allergic asthma.
      
Biospecimen description: 
      Blood, bone marrow and induced sputum.
    
Keywords: Asthma; β-chain signaling; allergen inhalation challenges; sputum; bone marrow aspirate; blood collection; -methacholine challenge
PK/PD of Ertapenem In Patients With TB
Condition: PK of Ertapenem in TB Patients
MeSH terms: Tuberculosis
Study type: Interventional
Phase: Phase 2

Summary: 
      Rationale:

      Treatment of multidrug or extensively drug resistant tuberculosis (MDR/XDR-TB) is a real
      challenge as failure in response to treatment and serious side-effects are frequently
      encountered. New, more effective drugs with less side effects are therefore urgently needed
      to solve this problem. Although several new drugs against TB are in the pipeline, physicians
      currently have limited treatment options for treatment of complicated MDR/XDR-TB cases.
      Therefore, drugs developed and labeled for other infectious diseases are evaluated for TB.

      Objective:

      The main objective of this prospective clinical trial is to evaluate pharmacokinetics of a
      standard dose (2000mg) of ertapenem in TB patients. This clinical trial will provide
      important information on PK of ertapenem in TB patients for future studies. Data can be used
      for limited sampling strategies for ertapenem based on a pharmacokinetic population model
      constructed from the full PK curves of the patients.

      Study design:

      A prospective pharmacokinetic study.

      Study population: 12 TB patients.

      Intervention: Single dose of 2000mg in a 30 minutes intravenous infusion.

      Main study parameters/endpoints:

      The pharmacokinetic parameters (Vd, Cl, AUC, etc) of ertapenem are the primary endpoints of
      the study. The T>MIC and AUC0-24h/Minimal inhibitory concentration (MIC) ratio are most
      likely the best predictive parameters for efficacy of ertapenem treatment and will be
      calculated for a range of M tuberculosis isolates.
    


Intervention name(s): ertapenem
Intervention type(s): Drug
Intervention description(s): single dose of 2000mg ertapenem IV
Intervention MeSH terms: Ertapenem
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Other
Keywords: ertapenem; tuberculosis; pharmacokinetics
Comparison of Intraneural and Extraneural Young's Modulus Using Shear Wave Elastography
Condition: Regional Anaesthesia
Study type: Observational

Summary: 
      The investigators would like to compare the stiffness inside and outside nerves using a
      special type of ultrasound imaging called shear wave elastography. Shear wave elastography is
      a special form of ultrasound as the pictures taken are in colour. An ultrasound machine has
      different ways of taking pictures of inside the body: one is by measuring the brightness of
      different body parts (often referred to as the B-Mode scan), the pictures taken in this way
      are the same as those you may have seen of unborn babies inside their mum's tummies; another
      way is by measuring the stiffness of different body structures which is how shear wave
      elastography works. The investigators hope that this new technology will help doctors to see
      the parts of the body that are important to them.
    

Detailed description: 
      Shear wave elastography is a quantitative ultrasound modality increasingly used to
      differentiate between "hard" breast cancer masses and "soft" normal tissue. Unlike strain
      elastography, shear wave elastography applies a non-compressive longitudinal acoustic
      radiation force to underlying tissues, inducing transverse shear waves. Studies in Thiel
      embalmed human cadavers have shown significant differences in Young's modulus between
      intraneural and extraneural tissue, and ready colour differentiation between tissues.

      The investigators own pilot studies have shown a 3-fold greater Young's modulus within nerve
      in Thiel embalmed cadavers and human volunteers. The investigators hypothesis is that shear
      wave elastography can differentiate between nerve and adjacent tissue in patients before
      nerve block for surgery. If so, this technology has the potential to reduce the incidence of
      complications with UGRA and encourage parallel applications such as cancer node biopsy.
    

Intervention name(s): Shear Wave Elastography
Intervention type(s): Device
Intervention description(s): Shear wave elastography
Other intervention name(s): Siemens 3000 shear wave utrasound machine
Observational model: Case-Only
Time perspective: Cross-Sectional
Sampling method: Non-Probability Sample
Study population: 
        Patients undergoing routine ultrasound-guided regional anaesthesia for elective orthopaedic
        surgery at Ninewells Hospital & Medical School, Dundee.
      
Keywords: Shear wave; Elastography; Repeatability and Reproducibility
Clinical Trial to Investigate the Pharmacokinetics and Safety After Oral Administration of CKD-378 and Co-administration of D745, D150, D029 in Healthy Adults
Condition: Type II Diabetes
MeSH terms: Diabetes Mellitus, Type 2
Study type: Interventional
Phase: Phase 1

Summary: 
      A clinical trial to compare and evaluate the safety and pharmacokinetics of CKD-378
    

Detailed description: 
      A Randomized, Open-label, Single-dose, Two-way Crossover Clinical Trial to Investigate the
      Pharmacokinetics and Safety after Oral Administration of CKD-378 and Co-administration of
      D745, D150, D029 in Healthy Adults
    

Intervention name(s): CKD-378 (low-dose)
Intervention type(s): Drug
Intervention description(s): Quaque day (QD), PO
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Trunk Extension Endurance Among Physical Therapy Students
Condition: Spine Injury
MeSH terms: Spinal Injuries
Study type: Interventional

Summary: 
      College students from physical therapy department will be assigned into four groups.
      Participants will be tested for trunk extensors endurance and will be given endurance
      training program to do at home for 6 to 8 weeks. Spinal mobility and lower extremity mobility
      will be measured. Isokinetic outcome measures for extension and flexion at two different
      speeds will also be measured at baseline, after 6 and 8 weeks.
    

Detailed description: 
      Active physical therapy students who are healthy and free from any significant pain at the
      spine will be recruited to participate in intervention endurance training. Participants will
      be assigned into four equal groups; doing trunk extension form lying on stomach while
      breathing normally and arching trunk backwards as long as tolerated. Group one will have arms
      above head, group two will have hands behind head, group three will have hands parallel to
      the trunk, and group four will receive instructions only and will act as control group.
      Spinal mobility of forward bending, backwards bending, right and left side bending will be
      taken. Straight leg raising and quadriceps flexibility will also be taken. Isokinetic
      measurements including peak torque, peak torque/body weight, average peak torque, work and
      average power will be taken at baseline, 6 and 8 weeks. Participants will have a training
      volume of 2 sets of 10 repetitions, 2 minutes rest in between, 2x/day for 6 to 8 weeks.
      Endurance timing will be measured in seconds using stop watch. If participant managed to
      maintain the trunk extension position for 4 minutes, time is recorded and it is considered as
      ceiling time.
    

Intervention name(s): trunk extensors endurance training
Intervention type(s): Other
Intervention description(s): endurance training
Other intervention name(s): trunk endurance
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Four equal groups of physical therapy students
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Prevention
Keywords: trunk extensors; endurance, peak torque, power, work