bioihbjkbjnl/mcl2 huggingface dataset:
A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype
Condition: Beta-Thalassemia
MeSH terms: Thalassemia; beta-Thalassemia
Study type: Interventional
Phase: Phase 3

Summary: 
      This is a single-arm, multi-site, single-dose, Phase 3 study in 23 participants less than or
      equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (TDT), also known as
      β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (HBB)
      gene. The study will evaluate the efficacy and safety of autologous hematopoietic stem cell
      transplantation (HSCT) using LentiGlobin BB305 Drug Product.
    


Intervention name(s): LentiGlobin BB305 Drug Product
Intervention type(s): Genetic
Intervention description(s): LentiGlobin BB305 Drug Product is administered by IV infusion following myeloablative conditioning with busulfan.
Other intervention name(s): betibeglogene autotemcel
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
GTx-024 as a Treatment for Stress Urinary Incontinence in Women
Condition: Stress Urinary Incontinence
MeSH terms: Urinary Incontinence; Enuresis; Urinary Incontinence, Stress
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to determine whether GTx-024 is safe and effective in the
      treatment of stress urinary incontinence in postmenopausal women.
    


Intervention name(s): GTx-024
Intervention type(s): Drug
Intervention description(s): GTx-024 softgel capsules will be administered once-daily to a total dose of 3 mg
Other intervention name(s): enobosarm
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Contrast Enhanced MRI of the Prostate
Condition: Prostatic Neoplasms
MeSH terms: Prostatic Neoplasms
Study type: Observational

Summary: 
      Prostate cancer detection and disease progression monitoring relies on systematic multi-core
      biopsies. Minimal invasive imaging capacities for lesion targeting and monitoring are badly
      needed. The purpose of this study is to determine if a new MRI technique can identify and
      monitor prostate disease progression. If so biopsy core number can be reduced to as small as
      one, and frequency for biopsy repeat can be reduced. The new technique is about a new way of
      analyzing the pictures taken as part of prostate MRI exam. This part is called dynamic
      contrast-enhanced MRI. It involves an injection of contrast reagent (or dye) through the arm
      vein during a time period when prostate MRI pictures are continuously taken.
    

Detailed description: 
      The aim of the study is to perform contrast-enhanced (CE) magnetic resonance imaging (MRI) at
      OHSU on subjects with diagnosed prostate cancer. This pilot project will study 20 subjects
      prior to their next scheduled clinical procedures (biopsy or radical prostatectomy). Data
      from this project will be used to explore new capabilities of the software platform developed
      under current SBIR funding. CE-MRI will involve the use of an extracellular (Gadolinium
      based, Gd) contrast agent. When Gd is used, (dynamic-contrast-enhanced) DCE-MRI signal
      intensity time-course data will be analyzed analytically using the so called "shutter-speed"
      paradigm which takes into account of the effects of finite water exchange kinetics. Region of
      interest (ROI) and high resolution (~millimeter) parametric maps of pathophysiologic
      quantities, such as tumor vessel permeability, tumor perfusion, extracellular extravascular
      volume fraction, will be generated from the DCE-MRI data. All parameters will be compared to
      literature results for software validation and correlated with pathology for clinical
      potential.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        A total of 20 subjects will be studied under this protocol. Subjects will be selected from
        patients scheduled for prostate biopsies with known prostate lesions. Subjects will also be
        recruited from prostate cancer patients who choose radical prostatectomy as disease
        management plan. The research project will be introduced to a potential subject by a
        urologist at OHSU. If interested, the subject will then be contacted through phone to
        participate in the study by an investigator or a research assistant on the research team.
A Study to Compare the Dose-proportionality of Tapentadol (CG5503) in Healthy Japanese and Korean Male Volunteers
Condition: Healthy Volunteer
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to evaluate the dose-proportionality of the pharmacokinetics of
      tapentadol (CG5503) in healthy Japanese and Korean adult male participants.
    

Detailed description: 
      This is a single-dose, open-label, single-center, randomized, four-way crossover study, ie,
      participants and investigator know the identity of the drug, which is assigned by chance,
      like flipping a coin, and participants may receive different interventions sequentially
      during the study. The study will consist of 4 treatment periods. A single dose of tapentadol
      (CG5503) will be administered orally with 240 mL of water under fasted conditions. All
      participants will receive 1 of the following 4 oral treatments in each period.

      Treatment A: One 25-mg tablet (25 mg) of tapentadol (CG5503) Treatment B: One 50-mg tablet
      (50 mg) of tapentadol (CG5503) Treatment C: One 100-mg tablet (100 mg) of tapentadol (CG5503)
      Treatment D: Two 100-mg tablets (200 mg) of tapentadol (CG5503) Each period will be separated
      by 7 - 14 days.
    

Intervention name(s): tapentadol (CG5503) ER two 100-mg TRF; tapentadol (CG5503) ER 50-mg TRF; tapentadol (CG5503) ER 25-mg TRF; tapentadol (CG5503) ER 100-mg TRF
Intervention type(s): Drug; Drug; Drug; Drug
Intervention description(s): 200mg TRF single oral dose; 50mg TRF single oral dose; 25mg TRF single oral dose; 100mg TRF single oral dose
Intervention MeSH terms: Tapentadol
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Keywords: tapentadol; NUCYNTA; CG5503; R331333; pharmacokinetics; healthy volunteer
Protein Requirements in Endurance-trained Athlete
Condition: Increased Metabolic Requirement; Healthy
Study type: Interventional
Phase: Early Phase 1

Summary: 
      Protein requirements in individuals who participate in endurance-based exercise training have
      been suggested to be greater than the current recommended dietary allowance (RDA).

      Nutritional requirements for dietary amino acids in adults have traditionally been determined
      utilizing the nitrogen balance technique, which is prone to underestimating protein
      requirements. As a result, there is a need to re-evaluate recommendations in order to
      characterize how dietary amino acid needs may be modulated by physical activity.

      Recent studies using the minimally invasive indicator amino acid oxidation (IAAO) technique
      have suggested that protein requirements in young men are at least 50% higher than RDA based
      on nitrogen balance data.

      Therefore, the purpose of this study is to measure the protein requirement in endurance
      trained adults using the IAAO technique.
    


Intervention name(s): amino acids intake
Intervention type(s): Dietary Supplement
Intervention description(s): amino acids intakes will be varied at 0.2-2.8 g/kg/day
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Basic Science
Effect Of The System In The Enriched Fat Metabolism Of The Child Reached Mitochondrial Cytopathy
Condition: Mitochondrial Cytopathies Disorders
MeSH terms: Kearns-Sayre Syndrome
Study type: Interventional

Summary: 
      The aim of the present study is to compare Resting Energy Expenditure (REE) between normal
      fat diet (NFD) as a control vs high fat diet (HFD) as a treatment in Mitochondrial
      cytopathies disorders ( MID's ) patients. Secondary objectives is to compare diet induced
      thermogenesis (DIT) and body composition between NFD vs HFD.

      This study is a randomized cross-over study. Normal fat diet (ie 10 % proteins, 45 % lipids,
      45 % carbohydrates) is the imposed diet at baseline, high fat diet (ie 10 % proteins, 30 %
      lipids, 60 % carbohydrates is used according to the cross-over design trial. Main evaluation
      criteria is REE and second evaluation criteria is DIT both measured by indirect calorimetry
      ). 36 included MID subjects will be included in this study. Main evaluation criteria and
      second evaluation criteria will be measured at baseline, 1 month, 2 month and 3 month.
    

Detailed description: 
      REE and DIT are measured by indirect calorimetry . The device used is a using an open-circuit
      ventilated hood system (QUARK RMR®, Cosmed, Pavona; Italy). The respiration quo- tient (RQ)
      and flow settings are calibrated by reference to alcohol combustion every six months. Before
      each test, the calorimeter is calibrated with a reference gas mixture (5% CO2, 95% O2 v/v).
      Expired carbon di- oxide (VCO2) and inspired oxygen (VO2) -flows are recorded, as well as the
      RQ. EE is calculated every minute from oxygen consumption (VO2 in ml/min) and carbon dioxide
      production (VCO2 in ml/min) using the Weir formula without protein correction. DIT is
      measured during 6 hours after a calibrated breakfast (normal fat diet or high fat diet
      according to the cross-over design) as 30% of theoric energy intake.
    

Intervention name(s): high fat diet; normal fat diet
Intervention type(s): Other; Other
Intervention description(s): high fat diet as 10 % proteins, 30 % lipids, 60 % carbohydrates; high fat diet as 10 % proteins, 45 % lipids,45 % carbohydrates
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Simulated Driving Performance, Daytime Sedation and Cognition in Healthy Volunteers Taking Gralise, Neurontin or Lyrica
Condition: Healthy
Study type: Interventional
Phase: Phase 4

Summary: 
      Phase 4, double-blind, placebo-controlled, four treatment, four sequence crossover study
      comparing simulated driving performance, daytime sedation and cognition in healthy volunteers
      administered therapeutic doses of Gralise® (Treatment A), Neurontin® (Treatment B), Lyrica®
      (Treatment C) and placebo (Treatment D). All doses were administered under fed conditions.
    


Intervention name(s): Gabapentin; Pregabalin; Placebo
Intervention type(s): Drug; Drug; Other
Other intervention name(s): Gralise®, Neurontin®; Lyrica®; Sugar Pill
Intervention MeSH terms: Pregabalin; Gabapentin
Allocation: Randomized
Intervention model: Crossover Assignment
Primary purpose: Other
Keywords: Volunteer; Pharmacodynamics; Pharmacokinetics
Phase 1/2 Study of HQK-1001 in Patients With Beta Thalassemia
Condition: Beta Thalassemia
MeSH terms: Thalassemia; beta-Thalassemia
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to evaluate the safety and tolerability of HQK-1001 administered
      daily for 8 weeks in subjects with beta thalassemia intermedia
    


Intervention name(s): HQK-1001; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): HQK-1001 capsules (10, 20, 30 or 40 mg) administered once a day, orally, for 56 days; Matching placebo capsules administered once a day, orally, for 56 days
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
2nd_Computerized Asthma Specific Quality of Life(cAQOL)
Condition: Persistent Asthma Patient
MeSH terms: Asthma
Study type: Observational

Summary: 
      Comparison of the responsiveness of two different asthma-specific QOL measures (AQLQ and
      cAQOL) in Korean patients with persistent asthma
    


Sampling method: Probability Sample
Study population: 
        asthma control patient
      
Biospecimen description: 
      Sub-group of ADRB2 and NK2R genotypes
    
Keywords: AQLQ and cAQOL
Smartphone Delivered Acceptance and Commitment Therapy for Adolescents With Longstanding Pain
Condition: Chronic Pain
MeSH terms: Chronic Pain
Study type: Interventional

Summary: 
      Intervention study with uncontrolled design investigating the effects of smartphone delivered
      Acceptance and Commitment Therapy for longstanding pain in adolescents. Primary outcome is
      pain interference. Internet-based parental support will be offered to the adolescents'
      caregivers.
    


Intervention name(s): Acceptance and Commitment Therapy
Intervention type(s): Behavioral
Intervention description(s): Eight weeks of smartphone delivered Acceptance and Commitment Therapy for the adolescent and eight weeks of internet delivered parental support to one or two parents of the adolescent.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Adolescents; Acceptance and Commitment Therapy; Smartphone
Evaluation of the Efficacy and Safety of Zinc in Viral Infections
Condition: SARS-CoV2 Infection
MeSH terms: Infections; Communicable Diseases; COVID-19; Virus Diseases
Study type: Interventional

Summary: 
      The main objective of the clinical study is to evaluate the efficacy of Zinc supplementation
      in non-critically ill Covid-19 patients..
    

Detailed description: 
      The VIZIR study is a national, longitudinal, multi-centre study conducted over a 3-month
      follow-up period.

      The priori hypothesis was that supplementation with zinc would reduce 30-day mortality and
      need to intensive care unit (ICU) admission among non critically ill patients with Covid-19.

      The VIZIR study will be carried out in Tunisia at the level of consultations of general
      practitioners of both sectors : public and liberal, as well as in the emergency services of
      Monastir and Sousse.
    

Intervention name(s): Zinc; Placebo
Intervention type(s): Dietary Supplement; Dietary Supplement
Intervention description(s): For each patient included, a prescription regimen of zinc (in bisglycinate form) is proposed at a dose of 25 mgtwice a day for 15 days for the intervention arm. This dose of zinc is to be taken away from meals.; For each patient included, a prescription of an identical to the intervention arm concerning shape, smell, taste and color pill of placebo . This dose of zinc is to be taken away from meals.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary purpose: Treatment
Phosphate Metabolites in Brain of Humans Aged 50+
Condition: Basic Science
Study type: Interventional

Summary: 
      The objective of this research project is to determine in elderly subjects (>50 year old) by
      31P-MRS the brain concentration of phosphorous metabolites species and fluxes, before and
      after consumption of 2 commercially available oral nutrition products
    


Intervention name(s): Peptamen® 1.5 Vanilla; Boost Plus® Vanilla
Intervention type(s): Dietary Supplement; Dietary Supplement
Intervention description(s): Nutritionally complete formula with 10g MCT in 250 mL, commercially available; Product of same nutritional composition but without MCT
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Basic Science
Modified Ketogenic Diet in Patients With McArdle Disease Part A
Condition: McArdle Disease
MeSH terms: Glycogen Storage Disease Type V
Study type: Interventional

Summary: 
      McArdle disease, glycogen storage disease type V, is a rare metabolic disease. Affected
      individuals are unable to utilize sugar stored as glycogen in muscle.

      We hypothesize that a modified ketogenic diet could be a potential treatment option, by
      providing ketones as alternative fuel substrates for working muscle.

      In this open interventional pilot study we wish to investigate 3 different modified ketogenic
      diet regimes, to find an optimal composition of a modified ketogenic diet that ensures
      adequate degree of ketosis and at the same time is well tolerated for patients with McArdle
      disease.
    

Detailed description: 
      Open interventional study to investigate 3 different modified ketogenic diet regimes, to find
      an optimal composition of a modified ketogenic diet for patients with McArdle disease

      McArdle disease, glycogen storage disease type V, is a rare metabolic disease caused by
      mutations in the PYGM gene resulting in absence of the enzyme muscle phosphorylase. Affected
      individuals are unable to utilize sugar stored as glycogen in muscle, leading to exercise
      intolerance, exercise-induced muscle pain, contractures and rhabdomyolysis, which may cause
      renal failure. Currently, there are no satisfactory treatment options for McArdle disease.
      Ketones are feasible fuel alternatives to muscle glycogen when muscle glycogenolysis is
      blocked as in McArdle disease.

      A key element of alleviating symptoms in McArdle disease is to provide alternative fuels for
      energy metabolism. Ketosis can potentially provide alternative fuel substrates by provision
      of endogenous ketone bodies (KBs) which are desirable fuels for skeletal muscle and brain.
      Ketosis can be reached by fasting and can be induced by adhering to a modified ketogenic
      diet, which entails a high-fat, low-carbohydrate diet, which simulates the metabolic effects
      of fasting.

      The optimal modified ketogenic diet composition, that ensures adequate degree of ketosis and
      at the same time is well tolerated, has not been investigated in patients with McArdle
      disease. Therefore this pilot study seeks to investigate which of 3 different diets is the
      most optimal ad effective for patients with McArdle disease. The 3 different diets will have
      carbohydrate percentages ranging from 5-25%, and fat percentage from 60-80%.
    

Intervention name(s): Modified ketogenic diets
Intervention type(s): Other
Intervention description(s): Different diet composition
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Open interventional study to investigate the effects of 3 different ketogenic diet regimes
Masking: None (Open Label)
Primary purpose: Treatment
A Research Study to Look Into How Well Semaglutide Medicine Works at Different Doses in People With Type 2 Diabetes and Overweight
Condition: Diabetes Mellitus, Type 2
MeSH terms: Diabetes Mellitus; Overweight; Diabetes Mellitus, Type 2
Study type: Interventional
Phase: Phase 2

Summary: 
      This study compares how three doses of semaglutide work in participants with type 2 diabetes
      (T2D) and overweight who are taking metformin. The study will look mainly at how well
      participant's blood sugar and participant's body weight are controlled when they are taking
      the study medicine at different doses. Participants will either get semaglutide [2 milligrams
      (mg), 8 mg, or 16 mg] or semaglutide placebo (a dummy medicine). Participants will take the
      study medicine with an injection pen called NovoPen®4. The injection pen is a medical tool
      with a needle used to inject the study medicine under the skin. The study will last for about
      52 weeks. Participants will have 13 clinic visits and 4 phone calls.
    


Intervention name(s): Semaglutide; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Semaglutide s.c. injection once-weekly for 40 weeks. Dose gradually increased over 24 weeks, followed by a 16 week maintenance period.; Semaglutide placebo s.c. injection once-weekly for 40 weeks. Dose gradually increased over 24 weeks, followed by a 16 week maintenance period.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
The Role of Brown Adipose Tissue in Triglyceride Clearance in People
Condition: Obesity
Study type: Interventional

Summary: 
      The recent discovery of functional brown adipose tissue (BAT) in humans has led to a paradigm
      shift in adipose tissue biology; it is now believed that adipocytes may play a significant
      role in regulating substrate metabolism. Given that the resurgence in the interest in human
      BAT is still in its infancy, a number of fundamental questions pertaining to the role of BAT
      in human physiology remain unanswered. One area of particular importance, but poorly
      understood, is the potential effect of BAT on triglyceride (TG) metabolism. Data from a
      series of studies have found that BAT is inversely associated with adiposity, high blood
      lipids, and fatty liver in people. However, the role of BAT in the regulation of TG
      metabolism in people is not known. The overall goal of this study is to determine the
      physiological importance of the human BAT in TG metabolism. To this end, we are planning to
      study overweight/obese women with high amounts of BAT (BAT+, n=14) and with no/minimal BAT
      (BAT-, n=14) both under thermoneutrality and mild cold exposure (~2 weeks apart). The
      investigators hypothesize that BAT+ participants will demonstrate greater plasma very
      low-density lipoprotein triglycerides (VLDL-TG) clearance rate compared to BAT- participants,
      and higher expression of genes involved in lipid metabolism only in BAT (but not muscle and
      white adipose tissue). Infusion of stable isotope tracers and metabolic modeling techniques
      will be used to assess VLDL-TG kinetics. Positron emission tomography computed tomography
      will be used for the identification and quantification of BAT. Supraclavicular BAT, abdominal
      white adipose tissue, and skeletal muscle tissue biopsies during cold exposure and
      thermoneutral conditions in conjunction with molecular biology techniques will used to
      measure expression of genes involved in lipid metabolism.
    


Intervention name(s): Cold exposure; Exposure to thermoneutral conditions
Intervention type(s): Other; Other
Intervention description(s): During cold exposure intervention, we will use an individualized cold exposure protocol above the individual shivering threshold of each participant using liquid condition garments and a temperature controlled room.; For the thermoneutral conditions intervention, subjects will be exposed to thermoneutral conditions (~26-28°C).
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Other