bioihbjkbjnl/mcl2 huggingface dataset:
Finding the Optimum Regimen for Duchenne Muscular Dystrophy
Condition: Duchenne Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne
Study type: Interventional
Phase: Phase 3

Summary: 
      The Finding the Optimum Regimen for Duchenne Muscular Dystrophy (FOR DMD) study will compare
      three ways of giving corticosteroids to boys with Duchenne muscular dystrophy (DMD) to
      determine which of the three ways increases muscle strength the most, and which causes the
      fewest side effects. Using the results of this study, the investigators aim to provide
      patients and families with clearer information about the best way to take these drugs.
    

Detailed description: 
      Boys with Duchenne muscular dystrophy experience progressive muscle weakness as they grow up.
      Corticosteroids are currently the only medicine that has been shown to increase muscle
      strength in boys with DMD. Benefits include an increase in the length of time that boys could
      continue to walk, reduction in the development of curvature of the spine, a longer time of
      adequate breathing, and possible protection against the development of heart problems.

      Doctors have tried different ways of prescribing corticosteroids in order to decrease
      undesirable side effects of the drug. No controlled, long-term study has ever looked at the
      effects of different corticosteroids to see which one improves strength the most and which
      one causes the fewest side effects, over a period of time. Different doctors in different
      countries prescribe the drugs in different ways, and some do not prescribe corticosteroids at
      all.

      The FOR DMD study will enroll boys with DMD ages 4-7. The study will look at three ways of
      taking the following corticosteroids by the mouth to determine which increases muscle
      strength the most, and which causes the fewest side effects:

        1. Prednisone 0.75mg/kg/day

        2. Prednisone 0.75mg/kg/day switching between 10 days on and 10 days off treatment

        3. Deflazacort 0.9mg/kg/day.

      The study will take place at 40 academic medical centers in the United States, Canada, United
      Kingdom, Germany and Italy.
    

Intervention name(s): Prednisone; Prednisone; Deflazacort
Intervention type(s): Drug; Drug; Drug
Intervention description(s): daily prednisone (0.75 mg/kg/day) tablets for 36-60 months; intermittent prednisone (0.75 mg/kg/day, 10 days on, 10 days off) tablets for 36 to 60 months; daily deflazacort (0.9 mg/kg/day) tablets for 36-60 months
Intervention MeSH terms: Prednisone; Deflazacort
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: prednisone; deflazacort; muscular dystrophy; neuromuscular disease; multi-center
Spectacle Lens Visual Acuity Assessments Study
Condition: Myopia; Myopia Progression; Juvenile Myopia
MeSH terms: Myopia
Study type: Interventional

Summary: 
      This is a two-arm parallel group, non-dispensing study. Participants will be existing active
      CYPRESS Extension (CPRO-1802-002) subjects. Subjects will undergo additional visual
      performance assessments.
    


Intervention name(s): Novel spectacle lens design; Spectacle lenses
Intervention type(s): Device; Device
Intervention description(s): Use of single vision, impact resistant spectacle lenses may reduce the rate of progression of juvenile myopia; Single vision, impact resistant spectacle lenses
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Optimal Positioning of Nasopharyngeal Temperature Probes: A Prospective Cohort Study
Condition: Hypothermia
MeSH terms: Hypothermia
Study type: Interventional

Summary: 
      Low body temperature (hypothermia) is often observed in anesthetized patients. Mild
      hypothermia increases complications such as surgical blood loss, postanesthesia recovery and
      the duration of hospitalization. To assess body temperature and minimize hypothermia-related
      complications, it is important to have accurate and reliable methods of measuring
      intraoperative core temperature. Common practice is to insert a nasopharyngeal (back of the
      throat from the nose) probe through one of the nostrils. However, there is no consensus or
      guideline regarding how deep the nasopharyngeal probe needs to be inserted. This study is
      being done to determine the insertion depth (or range of depths) that best approximates core
      temperature, which is temperature of the vital organs, e.g. heart, liver and lungs.

      Participation in the trial will occur on the day of surgery. The subject will be asked to
      breathe through one nostril and then the other before receiving anesthesia. The less
      congested nostril will be selected for study. If there is no difference, then the
      investigator will use the right nostril.

      Once under anesthesia, an esophageal temperature probe will be inserted to serve as a
      reference core temperature, which is used routinely in surgery. Then the nasopharyngeal probe
      will be inserted into the nostril.

      Both nasopharyngeal and esophageal temperatures will initially be recorded 45 minutes after
      anesthetic induction. The nasopharyngeal probe will then be withdrawn 2 cm and after a
      3-minute equilibration period, nasopharyngeal and esophageal temperatures will again be
      recorded. The nasopharyngeal probe withdrawal sequence will be repeated, 2 cm at a time,
      until only 2 cm remains in the nostril. There will be a total of 10 sets of nasopharyngeal
      and esophageal temperatures obtained.
    


Intervention name(s): Nasopharyngeal
Intervention type(s): Procedure
Intervention model: Single Group Assignment
Masking: None (Open Label)
Long-Term Growth and Skeletal Effects of Early Growth Hormone Treatment in Turner Syndrome
Condition: Turner Syndrome
MeSH terms: Turner Syndrome; Gonadal Dysgenesis; Syndrome
Study type: Interventional
Phase: Phase 4

Summary: 
      This is an extension study that will gather long-term data on the effect of early growth
      hormone (GH) treatment on adult height and other aspects of health and development in girls
      with Turner syndrome. The main purpose is to determine whether girls who received 2 years of
      GH treatment before 6 years of age achieve taller adult height than girls who were untreated
      during this time. The study will also look at middle ear and hearing function, and cognitive
      and behavioral development. Protocol completion is defined as attainment of height velocity
      less than or equal to 1.0 cm/year, or bone age greater than or equal to 15 years.
    


Intervention name(s): Humatrope
Intervention type(s): Drug
Intervention description(s): According to investigator's clinical practice and guided by the approved package insert
Other intervention name(s): LY137998; Somatropin; Growth hormone
Intervention MeSH terms: Hormones
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Pharmacokinetic of Alirocumab SAR236553 (REGN727) Administered Subcutaneously at 3 Different Injection Sites in Healthy Subjects
Condition: Hypercholesterolemia
MeSH terms: Hypercholesterolemia
Study type: Interventional
Phase: Phase 1

Summary: 
      Primary Objective:

      To assess the relative bioavailability of a single subcutaneous dose of alirocumab SAR236553
      (REGN727) administered at 3 different injection sites in healthy subjects.

      Secondary Objectives:

        -  To assess the pharmacodynamic effect of a single subcutaneous dose of alirocumab
           SAR236553 (REGN727) on serum low-density lipoprotein cholesterol and other lipids and
           apolipoproteins.

        -  To assess the safety of a single subcutaneous dose of alirocumab SAR236553 (REGN727).

        -  To assess the immunogenicity of a single subcutaneous dose of alirocumab SAR236553
           (REGN727).
    

Detailed description: 
      Total duration of the study per subject is about 15 weeks.
    

Intervention name(s): alirocumab SAR236553 (REGN727)
Intervention type(s): Drug
Intervention description(s): Pharmaceutical form:Solution for injection Route of administration: Subcutaneous
Intervention MeSH terms: Alirocumab
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Basic Science
48 Weeks Combination Therapy for Patients With HBeAg-negative Chronic Hepatitis B Virus (HBV) Infection
Condition: Hepatitis B
MeSH terms: Hepatitis A; Hepatitis B; Hepatitis
Study type: Interventional
Phase: Phase 3

Summary: 
      The purpose of this study is to investigate whether in patients with chronic HBeAg-negative
      hepatitis B, PEG-IFN-ribavirin combination therapy for 1 year leads to enhanced response (HBV
      DNA <10E4 copies/ml and normal ALT 24 weeks after treatment discontinuation) in comparison
      with pegylated interferon (PEG-IFN) monotherapy.
    

Detailed description: 
      Despite the introduction of newer drugs for the treatment of chronic hepatitis B, there is
      still no optimal treatment. Pegylated interferon alfa has proven sustained efficacy in
      approximately 30-40% of patients with HBeAg-positive or HBeAg-negative chronic hepatitis B.
      It is likely that combination therapy of pegylated interferon alfa with ribavirin in chronic
      hepatitis B is more effective than pegylated interferon alfa monotherapy. In chronic
      hepatitis C, adding ribavirin to pegylated interferon therapy doubled the sustained response
      rate (29% vs. 56%) and has become the standard option of treatment.

      To investigate the effect of the treatment with pegylated interferon and ribavirin on the
      amount of inflammation and fibrosis in the liver, a liver biopsy will be performed within one
      year prior to screening and at the end of follow-up.

      When patients with chronic hepatitis B are treated outside any study with pegylated
      interferon, they visit the outpatient clinic approximately every month for blood samples. So
      in this study the amount of blood samples taken from every patient is not increased as
      compared with treatment outside a study.
    

Intervention name(s): Ribavirin; Peginterferon alpha 2a
Intervention type(s): Drug; Drug
Intervention description(s): 1200 mg a day, 48 weeks; 180 µg per week, 48 weeks
Intervention MeSH terms: Ribavirin; Peginterferon alfa-2a
Allocation: Randomized
Intervention model: Factorial Assignment
Masking: Double (Participant, Investigator)
Keywords: HBeAg-negative; chronic; hepatitis B; viral
A Study of ARRY-502 in Patients With Persistent Asthma
Condition: Asthma
MeSH terms: Asthma
Study type: Interventional
Phase: Phase 2

Summary: 
      This is a Phase 2 study, involving a 6-week treatment period, designed to evaluate the
      effectiveness of investigational study drug ARRY-502 in treating mild to moderate persistent
      asthma, and to further evaluate the safety of the drug. Approximately 180 patients from the
      US will be enrolled in this study.
    


Intervention name(s): ARRY-502, CRTh2 antagonist; oral; Placebo; oral
Intervention type(s): Drug; Drug
Intervention description(s): multiple dose, single schedule; matching placebo
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Feasibility, Acceptability and Directional Signal Effect on Blood Folate Levels of Iodized Salt Fortified With Folic Acid: Clinical Study
Condition: Neural Tube Defects; Folic Acid Deficiency; Fortification
MeSH terms: Neural Tube Defects; Spinal Dysraphism; Folic Acid Deficiency
Study type: Interventional

Summary: 
      Aim 1: Assess directional signal of effect of fortified salt with folic acid and iodine on
      blood serum folate levels.

      It will be accomplished by feasibility pre-test/post-test clinical study by estimating mean
      and variability of serum blood folate levels effects of folic acid fortified salt.

      Hypothesis: Fortified salt with folic acid and iodine will increase serum blood folate levels
      from baseline by month 1.

      Aim 2: Assess feasibility and acceptability of salt fortified with folic acid and iodine.

      Feasibility will be measured by 1) subjects' refusal rate of consent by 30 subjects during
      6-month enrollment period; 2) method of determining salt consumption (weighting the
      saltshaker pre and post); 3) completion rates of study protocols from baseline to follow up.
      Hypothesis 1a: Completion of all blood draw visits will be 90-100%.

      Acceptability will be measured by 1) completion rate of daily salt logs; 2) consumption of
      salt by weighting saltshakers; 3) attitude towards taste and color difference. Hypothesis 1b:
      Completion rate (90-100%) of daily salt logs will range from 90-100% of participants and 2)
      consumption of salt will range from 90-100% of participants.

      Dietary surveys will be employed to document dietary habits and estimated daily FA intake in
      the sample population. Feasibility and acceptability of dietary surveys will be measured by
      willingness to answer survey questions. Hypothesis 1c: Completion rate of dietary surveys
      will range 90-100%.
    


Intervention name(s): Salt with folic acid and iodine
Intervention type(s): Dietary Supplement
Intervention description(s): Assess serum and RBC folate levels in women at baseline and 1 month after ingesting salt with folic acid
Intervention MeSH terms: Folic Acid
Intervention model: Single Group Assignment
Intervention model description: Pre-test and post-test of serum folate levels after using salt with folic acid in the same group of women, each serving as their own control.
Masking: None (Open Label)
Primary purpose: Prevention
Keywords: Serum folate
Safety, Tolerability, and Pharmacokinetics of Fidaxomicin in Pediatric Subjects With Clostridium Difficile-associated Diarrhea (CDAD)
Condition: Clostridium Difficile-associated Diarrhea
MeSH terms: Clostridium Infections; Diarrhea
Study type: Interventional
Phase: Phase 2

Summary: 
      The purpose of this study is to determine the safety, tolerability, and pharmacokinetics of
      fidaxomicin in pediatric subjects with Clostridium difficile-associated diarrhea (CDAD).
    


Intervention name(s): fidaxomicin
Intervention type(s): Drug
Intervention description(s): 6 months-5 years 11 months: oral suspension, 32 mg/kg/day with a maximum dose of 400 mg/day, divided into two doses, every 12 hours for 10 days.
6 years-17 years 11 months: tablets, 200 mg every 12 hours for 10 days.
Other intervention name(s): Dificid, Dificlir, OPT-80; PAR-101
Intervention MeSH terms: Fidaxomicin
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Clostridium difficile-associated diarrhea; CDAD; Pediatric
Exercise and Inflammation
Condition: Cardiovascular Disease
MeSH terms: Cardiovascular Diseases; Inflammation
Study type: Interventional

Summary: 
      The purpose of this study is to test the hypothesis that aerobic exercise leads to
      attenuation of the inflammatory response to lipopolysaccharide (LPS) stimulation.
    

Detailed description: 
      Aerobic exercise - the most widely recommended health behavior - is recognized to reduce the
      risk of coronary heart disease, so much so that consensus panels routinely include it as part
      of a cardioprotective regimen for healthy people, but the physiological or mechanistic basis
      of this protection is uncertain. Understanding the mechanisms has considerable public health
      significance because it will allow development and testing of targeted interventions to
      produce comparable cardioprotective effects more directly or in cases where aerobic exercise
      is not possible. This application proposes to test the hypothesis that aerobic training leads
      to attenuation of the inflammatory response to LPS stimulation and to examine the role played
      by exercise-induced increases in vagal activity, improvements in mood, and decreased
      expression of Toll Receptor 4 (TLR4), the cognate receptor for endotoxin expressed by
      monocytes.
    

Intervention name(s): aerobic training; Wait list
Intervention type(s): Behavioral; Behavioral
Intervention description(s): 12 weeks of aerobic training, 4 times/week; wait list control condition
Other intervention name(s): Training, aerobic; Condition, wait list control
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Basic Science
Biomarkers of Diabetic Retinopathy Progression
Condition: Type 2 Diabetes Mellitus; Mild Nonproliferative Diabetic Retinopathy
MeSH terms: Retinal Diseases; Diabetic Retinopathy; Diabetes Mellitus; Diabetes Mellitus, Type 2
Study type: Observational

Summary: 
      The purpose of this study is to validate a predictive model of diabetic retinopathy
      progression in patients with diabetes mellitus type 2 patients to clinically significant
      macular edema (CSME) needing treatment either photocoagulation or intravitreal injections
      (ITV) using non-invasive techniques.
    


Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients with type 2 diabetes mellitus and mild nonproliferative diabetic retinopathy
      
Keywords: type 2 diabetes mellitus; nonproliferative diabetic retinopathy
T-IR- Study to Understand the Effects of Testosterone and Estrogen on the Body's Response to the Hormone Insulin
Condition: Insulin Resistance; Type 2 Diabetes Mellitus; Obesity; Androgen Deficiency; Metabolic Disease
MeSH terms: Diabetes Mellitus, Type 2; Insulin Resistance; Metabolic Diseases
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this research study is to understand the effects of testosterone and estrogen
      on the body's response to the hormone insulin.
    

Detailed description: 
      The investigators will examine the effects of testosterone on insulin sensitivity and body
      composition in men. This study may lend greater insight into the increased risk of diabetes
      evident in men with low circulating levels of testosterone. Three drugs will be used in this
      study: acyline, given by injection; testosterone (T) gel that is applied to the skin; and
      letrozole, which is an oral drug that blocks the conversion of androgens (male hormones) to
      estrogens (female hormones). Acyline inhibits the production of luteinizing hormone (LH) and
      follicle stimulating hormone (FSH). When acyline stops the production of these hormones, it
      blocks the signal from the brain that stimulates the testicles to make testosterone. Adding
      testosterone to acyline will restore physiologic levels of testosterone in some study
      participants. One group of men will receive T gel with letrozole, an aromatase inhibitor;
      these men will have normal levels of testosterone but low levels of estrogen in the blood.
      This design will enable determination of the respective metabolic effects of testosterone and
      estrogen.
    

Intervention name(s): Acyline; Testosterone 1.62% gel; Letrozole; Placebo gel (for Testosterone 1.62% gel); Placebo pill (for Letrozole)
Intervention type(s): Drug; Drug; Drug; Drug; Drug
Intervention description(s): 300 mcg/mL administered subcutaneously (at Day 0, Week 2); Transdermal Testosterone Gel (either 1.25g or 5g/d) for 4 weeks; Letrozole oral aromatase inhibitor 5mg daily for 4 weeks; placebo gel manufactured to mimic Testosterone 1.62% gel; Oral placebo aromatase inhibitor to mimic Letrozole 5mg/d
Other intervention name(s): Androgel; Femara
Intervention MeSH terms: Letrozole; Methyltestosterone; Acyline; Testosterone; Testosterone undecanoate; Testosterone enanthate; Testosterone 17 beta-cypionate
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary purpose: Treatment
Keywords: insulin; androgens; insulin resistance; testosterone; estradiol; obesity
Prospective Study Assessing Blood Pressure and Other Outcomes Post-treatment in Patients With Primary Aldosteronism
Condition: Primary Aldosteronism; Primary Aldosteronism Due to Aldosterone Producing Adenoma; Primary Aldosteronism Due to Adrenal Hyperplasia (Bilateral)
MeSH terms: Adenoma; Adrenal Hyperplasia, Congenital; Adrenogenital Syndrome; Hyperaldosteronism; Hyperplasia
Study type: Observational

Summary: 
      Majority of patients with hypertension have primary hypertension (without an underlying
      cause). Secondary hypertension (due to an underlying disease) is important to recognize, as
      treatment can lead to cure of hypertension. Primary aldosteronism (PA) is the most common
      cause of secondary hypertension, and can be found in 5-10% of patients locally.

      PA is caused by excessive release of a hormone (aldosterone) from the adrenal glands, which
      can be unilateral (one gland) or bilateral (both glands). Distinction between two is crucial
      as unilateral disease is treated with the aim of cure by surgery, and bilateral disease is
      treated by medication.

      It has been shown that excess aldosterone has other harmful effects in addition to
      hypertension, such as directly affecting the heart, blood vessels, kidneys, diabetes and
      quality of life. This is supported by studies showing reversal of these effects after
      treatment for PA. In addition, improvements after surgery appears to be superior to medical
      treatment, although studies have found variable results.

      Hence, the investigators aim to accurately subtype patients with PA into unilateral or
      bilateral disease and study the post-treatment response after both surgery and medicine with
      regards to the effects on blood pressure, cardiovascular, renal, metabolic and quality of
      life.
    


Intervention name(s): Adrenalectomy; Mineralocorticoid Receptor Antagonists
Intervention type(s): Procedure; Drug
Intervention description(s): Adrenalectomy for unilateral adrenal hyperplasia / adenoma; Medical treatment with MRA / amiloride
Other intervention name(s): Spironolactone; Eplerenone; Amiloride
Intervention MeSH terms: Eplerenone; Amiloride; Spironolactone; Mineralocorticoid Receptor Antagonists; Mineralocorticoids
Observational model: Cohort
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Patients with confirmed primary aldosteronism
      
Biospecimen description: 
      Peripheral blood samples Adrenal vein blood samples (during adrenal vein sampling) Adrenal
      tissue (post-adrenalectomy)
    
Keywords: Primary aldosteronism; Blood pressure; Adrenalectomy; Mineralocorticoid-receptor Antagonists; Cardiovascular; Endothelial; Renal