bioihbjkbjnl/mcl2 huggingface dataset:
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Condition: Spinal Muscular Atrophy
MeSH terms: Muscular Atrophy; Muscular Atrophy, Spinal; Atrophy
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal
      Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants. A
      variety of outcome measures will be performed at each study visit to follow the course of the
      disease. Total duration of the study for type I infants will be 18 months, for type II
      infants, 24 months.
    

Detailed description: 
      Perform a phase I/II study to evaluate effects of Phenyl Butyrate (PBA) in a cohort of 12
      presymptomatic infants. These infants are predicted to have either SMA 1 or 2 given genotype
      and family history of an older sibling with the respective SMA type. Our goal is twofold: 1)
      to collect additional safety and pharmacokinetic data in neonates and young infants
      administered this compound, within the dosing guidelines already in use for urea cycle
      disorder therapy, and 2) to determine possible benefit of early treatment intervention with
      regard to status of denervation and functional motor status at specific time points for which
      we have matched natural history data to perform a comparison. Data obtained from this aim
      will guide future trials designed to determine the efficacy of PBA or other butyrate analogs
      in attenuating disease progression in SMA subjects identified in the presymptomatic period.
    

Intervention name(s): Sodium phenylbutyrate (NaPB)
Intervention type(s): Drug
Intervention description(s): The powder form of the drug will be dispensed. The target NaPB dosing is 450-600 mg/kg/day, divided into four doses. For cohort 1, we propose to continue treatment for 18 months. For cohort 2, we propose to continue treatment for 24 months.
Intervention MeSH terms: 4-phenylbutyric acid
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Spinal Muscular Atrophy; SMA; Sodium Phenylbutyrate
Single Ascending Dose Tolerability Study of DG3173
Condition: Healthy
Study type: Interventional
Phase: Phase 1

Summary: 
      This clinical trial investigated the safety, tolerability and pharmacokinetic profile of
      DG3173 in a double-blind, randomized, placebo-controlled, single dose escalation Phase 1
      study involving 72 healthy volunteers. Individuals received up to 2000 µg of DG3173 via
      single dose subcutaneous injection.
    


Intervention name(s): DG3173; Placebo
Intervention type(s): Drug; Drug
Allocation: Randomized
Intervention model: Single Group Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
A Clinical Study in Healthy Adults Who Sometimes Take Drugs for Pleasure Which Aims to Evaluate Whether GRT0151Y is Likely to be Abused
Condition: Pain; Acute Pain
MeSH terms: Acute Pain
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this clinical study was to evaluate whether healthy adults, who sometimes take
      drugs for pleasure, are likely to abuse GRT0151Y. This abuse potential is assessed at three
      different doses of GRT0151Y.

      During a Qualification Phase, a single dose of hydromorphone IR 8 mg and a single dose of
      placebo were separately administered orally over 4 days in a randomized, double-blinded
      manner.

      During the Treatment Phase, single doses of GRT0151Y free base (100 mg, 200 mg and 400 mg),
      hydromorphone Immediate-release (IR) (4 mg, 8 mg, and 16 mg), and placebo were administered
      orally over 7 Treatment Periods.

      Participants received the treatments according to a 7-sequence, 7-period balanced design.
    


Intervention name(s): GRT0151Y 50 mg capsule; Hydromorphone IR 4 mg; Matching placebo
Intervention type(s): Drug; Drug; Drug
Intervention description(s): GRT0151Y 50 mg capsule; Hydromorphone IR 4 mg tablet (encapsulated); Matching placebo
Intervention MeSH terms: Hydromorphone
Allocation: Randomized
Intervention model: Single Group Assignment
Intervention model description: Qualified participants for the treatment phase will be randomized to 1 of 7 treatment-sequences based on a computer generated randomization schedule. Participants will receive all 7 treatments in the order specified by the treatment sequence according to a 7-sequence, 7-period balanced design.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
A Trial to Evaluate the Safety of a Single Intravenous Infusion of MGAWN1 in Healthy Adults
Condition: West Nile Virus
Study type: Interventional
Phase: Phase 1

Summary: 
      The primary purpose of this phase 1, double-blind, cohort study is to evaluate the safety,
      tolerability, and pharmacokinetics of escalating doses of MGAWN1 administered as a single
      intravenous (IV) infusion to healthy adults. Subjects will be enrolled sequentially into one
      of 5 dose-level cohorts, with 8 subjects in each cohort. Six subjects in each cohort will
      receive MGAWN1 (a Neutralizing, Humanized, Monoclonal Antibody (IgG1k) to West Nile Virus)
      and 2 will receive a saline control.
    


Intervention name(s): MGAWN1; MGAWN1; Placebo
Intervention type(s): Drug; Drug; Other
Intervention description(s): Eight subjects per cohort, each receives a single IV infusion of MGAWN1 of saline control on study day 0 (6 MGAWN1, 2 saline control.
Cohort 1 - 0.3 mg/kg Cohort 2 - 1 mg/kg Cohort 3 - 3 mg/kg Cohort 4 - 10 mg/kg Cohort 5 - 30 mg/kg; Single IV dose; Single IV dose
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Randomized; Double-Blind; Dose-Escalation; Cohort Study
Patient-ventilator Interactions in Long Term Non-invasive Ventilation: Influence of Back-up Frequency
Condition: Obesity; Non Invasive Ventilation
Study type: Interventional

Summary: 
      In patients under bi-level pressure support ventilation for obesity hypoventilation syndrome,
      this study aims to determine if it is better, in terms of efficacy and patient-ventilator
      synchronisation to use

        -  Spontaneous mode (S)

        -  Spontaneous/Timed mode (ST) with an intermediate back-up rate, slightly below the
           respiratory rate of the patient

        -  Timed mode (T), with a ventilator respiratory rate above that of the patient. This issue
           has to our knowledge only been marginally studied without any consensus.
    

Detailed description: 
      Eligibility: patients under home bi-level pressure support ventilation for obesity
      hypoventilation in a stable clinical condition, aged over 18

      Methods:

      Three consecutive sleep studies in a randomised sequence with usual parameters Inspiratory
      Positive Airway Pressure(IPAP), Expiratory Positive Airway Pressure (EPAP) and change of only
      back up respiratory rate (RR): one night in a spontaneous mode, one with an intermediate RR,
      and one with a RR above that of the patient

      Endpoints:

      Quality of sleep and sleep structure (polysomnography), patient-ventilator synchronisation
      and respiratory events under NIV, efficacy of ventilation: oxygen saturation measured by
      pulse oxymetry (SaO2); transcutaneous capnography (PtcCO2), and comfort.
    

Intervention name(s): Adjustment of back-up respiratory rate on bi-level ventilator
Intervention type(s): Device
Intervention description(s): 3 consecutive nights in randomized order, in either "S" (Spontaneous) mode, "ST" (Spontaneous/Timed) mode with a back-up rate under spontaneous respiratory rate of the patient, or in "ST" (Spontaneous/Timed) mode with a back-up rate at least 2 cycles/minute above spontaneous nocturnal respiratory rate. Each assessment will include complete polysomnographic recording (sleep study) as well as SpO2, PtcCO2, pneumotachograph (flow), mask pressure, quantitative assessment of patient-ventilator synchronisation, central or obstructive apnea and hypopnea, data of built-in software, and rating of patient comfort
Intervention model: Single Group Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Supportive Care
Improving Maintenance Following Very Low Calorie Diet: The Sustain Lost Weight (SLW) Intervention
Condition: Obesity
Study type: Interventional

Summary: 
      Obtain a preliminary assessment of the feasibility and acceptability of delivering the
      sustained lost weight (SLW) intervention in a real-world clinical setting for patients who
      have completed a 21-week very low calorie diet (VLCD).

      Hypothesis: Intervention will be feasible to implement as evidenced by the ability to recruit
      and retain participants and acceptable to participants as evidenced by self-report of
      satisfaction with the program and adoption of weight maintenance behaviors.

      Secondary Aim: Obtain a preliminary assessment of the effectiveness of the sustained lost
      weight (SLW) intervention for patients who have completed 21 weeks of a very low calorie diet
      (VLCD).

      Hypothesis: Greater than 75% of participants who receive this novel approach, where
      maintenance behaviors are individualized based on resting metabolic rate and a physical
      activity factor, will regain greater or equal to 10 kg. at week 52 follow-up.
    


Intervention name(s): Optifast Products
Intervention type(s): Dietary Supplement
Intervention description(s): Optional Optifast products offered once every other week X 6 months; then monthly.
Other intervention name(s): Optifast Dietary Supplements
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Supportive Care
Evolution With CPAP Treatment of a Cohort of Type 2 Diabetic Patients With Apnea-hypopnea Syndrome
Condition: Sleep Apnea; Type 2 Diabetes
MeSH terms: Apnea
Study type: Interventional

Summary: 
      The main purpose of this study is to measure the evolution of hemoglobin A1c level after
      treatment with CPAP in a cohort of type 2 diabetes mellitus patients with poor glycemic
      control and associated moderate or severe obstructive sleep apnea.
    

Detailed description: 
      Subjects with poorly controlled type 2 diabetes are a high cardiovascular risk group in which
      a high prevalence of moderate to severe sleep apnea is expected. Studies based on
      interstitial glycemic measurement demonstrate a reduction in glucose levels when treating
      sleep apnea with CPAP. Nevertheless, the effectiveness of CPAP in improving glycemic control
      has been questioned as most studies have failed to demonstrate a reduction in hemoglobin A1c
      (HbA1C) level over time. Most of these studies have limitations such as a short follow-up or
      a suboptimal fulfillment of CPAP treatment. We hypothesize that treating moderate to severe
      sleep apnea with CPAP will improve glycemic control (measured by HbA1C) at 14 weeks in good
      compliers and that this improvement will be sustained at one year. We aim to test this
      hypothesis in consecutive type 2 diabetes patients on stable treatment with HbA1c ≥7% in
      routine outpatient visits in our Diabetes, Nutrition and Endocrinology Unit. After providing
      informed consent, patients will be screened for sleep apnea by nocturnal oximetry followed by
      a diagnostic respiratory polygraphy. Those patients with obstructive sleep apnea with an
      apnea-hypopnea index ≥20 will be invited to enter the study. After a 3-month observation
      period without any intervention to rule out a potential influence of entering the study on
      HbA1C levels, patients will be treated with CPAP. HbA1C levels will be measured at baseline,
      after 14 weeks, and thereafter every 14 weeks until completing one year of treatment. Other
      endocrine, metabolic and cardiovascular risk variables will be determined at baseline and at
      14 weeks of the intervention.
    

Intervention name(s): Continuous Positive Airway Pressure CPAP
Intervention type(s): Device
Intervention description(s): CPAP treatment
Other intervention name(s): no other name
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Sublingual Misoprostol in Decreasing Blood Loss Prior to Myomectomy
Condition: Blood Loss in Myomectomy
MeSH terms: Hemorrhage
Study type: Interventional
Phase: Phase 3

Summary: 
      The aim of the study is to compare the effectiveness of sublingual administered prostaglandin
      E1 synthetic analogue (misoprostol) 400 microgram versus placebo before myomectomy to
      decrease blood loss during and after the operation.
    


Intervention name(s): Misoprostol 400 sublingual; placebo 400 sublingual
Intervention type(s): Drug; Drug
Intervention description(s): patients will take 2 tablets of sublingual misoprostol 400 microgram 3 hours and 1 hour before the surgery; patients will take 2 tablets of sublingual placebo 400 microgram 1 hour before the surgery
Intervention MeSH terms: Misoprostol
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Prevention
Comparison of the Effects of Home Exercise and Isokinetic Exercise Program in Subacromial Impingement Syndrome Patients
Condition: Home Exercise; Quality of Life
MeSH terms: Shoulder Impingement Syndrome
Study type: Interventional

Summary: 
      The aim of this study is to evaluate and compare the effects of home exercise program and
      isokinetic exercise program on pain, muscle strength, functionality, proprioception sensation
      and quality of life in patients with subacromial impingement syndrome.
    

Detailed description: 
      The study was designed prospectively. A total of 50 patients with subacromial impingement
      syndrome according to clinical and MRI findings with shoulder pain for at least 3 months were
      included in the study. Patients were randomly divided into two groups. The first group
      (isokinetic exercise group, n = 25) was given isokinetic exercise. The second group(home
      exercise group, n=25) was given home exercise program. Patients were evaluated before and
      after treatment and 3 months after the end of treatment. In the evaluation, VAS, Shoulder
      Pain and Disability Index, Constant Murley Scoring, Shoulder Disability Questionnaire, Health
      Assessment Questionnaire, Pittsburgh Sleep Quality Index, Humic Norm II isokinetic device
      were used. At statistically evaluation of data Student T Test and Mann Whitney U tests were
      used. In qualitative datas comparison Ki-Kare Test was used. Significance was evaluated at
      the level of p&lt; 0.05.
    

Intervention name(s): Exercises of rehabilitation
Intervention type(s): Device
Intervention description(s): Compare and evaluate of the effects of the routin physical medicine and rehabilitation programs in impingement patients
Other intervention name(s): Isokinetic cybex dynamometer
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Evaluation of Medium Chain Triglycerides in a Mixed Racial Population of Patients: a Feasibility Study
Condition: Insulin Resistance
MeSH terms: Insulin Resistance
Study type: Interventional

Summary: 
      The study team will investigate the racial differences in the metabolic and clinical
      responses to Medium chain triglycerides (MCT) between African American and Caucasian American
      subjects.
    

Detailed description: 
      It is generally accepted that type 2 diabetes (T2D) arises from the progression of insulin
      resistance (IR), with hyperinsulinemia (HI) as a compensatory response. The possibility that
      HI can precede and contribute to insulin resistance (IR) and metabolic syndrome (MS) has been
      suggested but not tested in humans. While IR and HI are closely associated, demonstrating a
      primary role for HI in T2D is key to the development of new treatment strategies for this
      disease. One group in which HI could play a bigger role in T2D is African Americans (AA) who
      are known to be more hyperinsulinemic than Caucasian Americans (CA). Racial disparities in
      T2D treatment outcomes adversely affect AA. Our main hypothesis is that suppression of HI
      will contribute to the prevention and treatment of T2D, especially among AA. Our goal in this
      pilot study is to show that consumption of medium chain triglycerides (MCT) in the diet will
      decrease basal insulin secretion and HI, and will lead to improvement in the insulin
      sensitivity index (Si). 24 subjects (12 AA, 12 CA) will participate in a clinical trial in
      which they will receive MCT for 6 weeks. Insulin secretion dynamics and insulin sensitivity
      will be assessed by use of the frequently sampled intravenous glucose tolerance test
      (FSIVGTT) and Bergman's minimal model analysis.
    

Intervention name(s): Medium chain triglycerides (MCT)
Intervention type(s): Dietary Supplement
Intervention description(s): Subjects will consume 30 grams per 2000 kilocalories daily of medium chain triglyceride oil by mouth
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Other
Keywords: Hyperinsulinemia
Stem Cell Therapy for Outer Retinal Degenerations
Condition: Age Related Macular Degeneration; Stargardt's Disease; Exudative Age-related Macular Degeneration
MeSH terms: Macular Degeneration; Retinal Degeneration; Stargardt Disease
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      This study is a Phase I/II , open label,non randomized, prospective study to determine the
      safety of human embryonic stem cell derived Retinal pigmented epithelium (hESC RPE) sub
      retinal injections versus hESC RPE seeded on a polymeric substrate implanted in the sub
      retinal space,
    

Detailed description: 
      To determine whether the surgical implantation of a human embryonic stem cell-derived retinal
      pigmented epithelium (hESC-RPE) monolayer seeded onto a polymeric versus hESC-RPE injections
      into the sub retinal space is a safe procedure.

      6 Patients will receive hESC-RPE cell injections (100000 cells) in the sub retinal space (2
      Dry Age-related macular degeneration (AMD), 2 Wet AMD with disciform scar and 2 with
      Stargardt's disease).

      Also 5 patients Dry AMD, 5 patients with Wet AMD with disciform scar and 5 patients with
      Stargardt's disease with receive a subretinal implantation of the hESC-RPE seeded in a
      monolayer in a polymeric substrate.

      Patients will be enrolled sequentially, and after the procedure the patients will be followed
      for 1 year.
    

Intervention name(s): injection of hESC-RPE in suspension; injection hESC-RPE seeded in a substrate
Intervention type(s): Procedure; Procedure
Intervention description(s): The first six patients will receive a sub retinal injection of hESC-RPE in solution after a complete pars plana vitrectomy to access safety of the cell implant alone.; Fifteen patients will receive a sub-retinal implantation of embryonic stem cell derived retinal pigmented epithelium seeded in a polymeric substrate to access safety of substrate seeded with RPE
Other intervention name(s): Human Embryonic Stem Cell - Retinal Pigmented Epithelium; Human Embryonic Stem Cell seeded in a polymeric substrate
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Balanced Propofol Sedation During Upper Endoscopy in Morbidly Obese Patients
Condition: Obesity
MeSH terms: Obesity
Study type: Observational

Summary: 
      The purpose of this study is to evaluate the safety of propofol-based,
      gastroenterologist-administered sedation in severely obese patients (BMI≥35) undergoing upper
      endoscopy.

      The investigators aim to test the hypothesis that it is safe to use balanced-propofol,
      gastroenterologist-administered sedation in obese patients.
    

Detailed description: 
      The purpose of this study is to evaluate the safety of propofol-based,
      gastroenterologist-administered sedation in severely obese patients (BMI≥35) undergoing upper
      endoscopy. We will use changes in pulmonary and hemodynamic parameters as the primary safety
      measure in this study. We will compare these results in 30 non-obese patients and in 30 obese
      patients.

      The term balanced propofol sedation refers to using a low dose of propofol in combination
      with small amounts of an opioid and midazolam.
    

Observational model: Case-Control
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        The research subjects will be selected from a private practice.
      
Keywords: propofol; sedation; safety; obesity
Pharmacokinetics and Safety of a Single Intravenous Infusion of BAY 79-4980
Condition: Hemophilia A
MeSH terms: Hemophilia A
Study type: Interventional
Phase: Phase 1

Summary: 
      The primary objective of this study is to determine the pharmacokinetic profile after single
      administration of two doses of BAY 79-4980 (high and low: 35 IU FVIII/Kg reconstituted in 22
      mg and 13 mg of liposomes/Kg, respectively) compared to rFVIII-FS (35 IU/Kg reconstituted in
      2.5 mL WFI/1000 IU) in PTPs aged 12 to 60 years with severe hemophilia A.
    


Intervention name(s): Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980); Recomb. Factor VIII (Kogenate FS Liposome, BAY79-4980); Recombinant Factor VIII (Kogenate FS, BAY14-2222); Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Intervention type(s): Biological; Biological; Biological; Biological
Intervention description(s): Low dose of BAY 79-4980 [13mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU).; High dose of BAY 79-4980 [22mg of liposomes/kg] then cross over to rFVIII-FS (35 IU/kg reconstituted in 2.5 mL WFI / 1000 IU); rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to low dose of BAY 79-4980 [13mg of liposomes/kg]; rFVIII-FS (35 IU/kg reconstituted in 2.5mL WFI /1000 IU) then cross over to high dose of BAY 79-4980 [22mg of liposomes/kg]
Intervention MeSH terms: Factor VIII
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: FVIII; Long acting; pk
Correlation of Calcitonin Gene-Related Peptide (CGRP) Levels in Saliva With the Evolution of an Attack of Migraine
Condition: Headache, Migraine
MeSH terms: Migraine Disorders; Headache
Study type: Observational

Summary: 
      To assess CGRP levels in saliva through the evolution of migraine.
    

Detailed description: 
      The release of CGRP is assumed to be initiated early in the migraine process and increases as
      the headache intensifies. Levels of CGRP will be measured during the premonitory, mild,
      moderate, and severe phases of a single migraine attack and compared to the baseline value
      determined when the subject was headache free. Understanding of the clinical evolution of
      migraine CGRP release may have significant implications in acute treatment.
    

Intervention name(s): subjects personal triptan
Intervention type(s): Other
Intervention description(s): Subjects will treat 1 migraine attack with their usual triptan
Intervention MeSH terms: Serotonin 5-HT1 Receptor Agonists
Observational model: Case-Only
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients at a mid west headache specialty clinic
      
Biospecimen description: 
      saliva