bioihbjkbjnl/mcl2 huggingface dataset:
Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Condition: Gaucher's Disease
MeSH terms: Gaucher Disease
Study type: Interventional
Phase: Phase 1

Summary: 
      OBJECTIVES: I. Transfer the human glucocerebrosidase (GC) gene into peripheral blood stem
      cells (PBSC) obtained from patients with type I Gaucher disease using a retroviral vector.

      II. Transplant the autologous transduced PBSC in these patients. III. Measure the carriage
      and expression of the transferred gene and its duration in peripheral blood leukocytes.

      IV. Assess the clinical effects of transplanting genetically corrected PBSC.
    

Detailed description: 
      PROTOCOL OUTLINE: Patients undergo autologous transplantation using peripheral blood stems
      cells (PBSC) stimulated with filgrastim (G-CSF) and transduced with a retroviral vector
      containing the human glucocerebrosidase gene (R-GC). Patients may receive up to 4 transplants
      if a deficient glucocerebrosidase level is found in peripheral leukocytes 1 month following
      transplantation.

      The G-CSF-stimulated PBSC are enriched for CD34-positive stem cells and transduced with the
      R-GC vector. Stem cells with normal gene activity are selected for transplantation.

      Patients are followed every month for 6 months, every 6 months for 18 months, and then
      annually thereafter.
    

Intervention name(s): human glucocerebrosidase gene into autologous peripheral blood stem cells
Intervention type(s): Genetic
Primary purpose: Treatment
Keywords: Gaucher's disease; inborn errors of metabolism; rare disease; sphingolipidoses
Predictors of Poor Immune Response to Rotavirus Vaccine in Infants
Condition: Innate Immune Response
Study type: Interventional

Summary: 
      This will be a randomized study with the primary objective of assessing the impact of
      withholding versus encouraging breastfeeding on the immunogenicity of Rotarix® in infants.

      Enrolled subjects will all be administered Rotarix®, at ages 6 to 7 weeks, and at10 -14
      weeks. The subjects will be randomized into one of the two study arms. In one of the group
      caregivers will be advised to breastfeed immediately prior to and after each dose of the
      vaccine while in the other group caregivers will be advised to withhold breastfeeding for 30
      minutes before and after each dose of the vaccine. The other childhood vaccines i.e. OPV and
      Pentavalent will be administered at least 30 minutes after administration of Rotarix®, The
      enrolled subjects will be followed up for 4 weeks after the 2nd dose of Rotarix®.
    

Detailed description: 
      Infants ≤7 weeks and pregnant women will be identified through a household survey.Families of
      infants aged 6-7 weeks will be called to the study clinic for consenting, screening and
      enrollment.All enrolled subjects will be administered 2 doses of Rotarix® at 6 to 7 weeks and
      at 10-14 weeks of age. Other childhood vaccines will also be offered.

      At enrollment, a blood specimen and breast milk specimen will be obtained from the mother and
      a blood, salivary and stool specimens will be obtained from the subject before administering
      the Rotarix® vaccine.

      At the time of the 2nd administration, a breast milk specimen will be obtained from the
      mother and a salivary specimen will be obtained from the subject before administering the
      Rotarix® vaccine. At 28 days (±) 5 after the 2nd dose of Rotarix® vaccine, a blood, salivary
      and stool specimen will be obtained from the subject.

      Whenever the family comes to the study clinic at 6-7 weeks, consent will be administered and
      a clinical examination conducted.If the family is willing for the mother and the child to be
      part of the study and the child meets the eligibility criteria, the child will be enrolled.

      Depending on the group allocation the mothers will be advised to withhold or encourage
      breastfeeding for 30 minutes before and after receipt of Rotarix®.

      subjects will be observed for 30 minutes after receipt of the vaccines to assess for
      immediate adverse events.

      All minor illnesses that do not require hospital referral will be managed by the study
      physician using current practices.

      All hospitalizations and deaths will be reported and reviewed by an Independent Safety
      Monitor.
    

Intervention name(s): Breastfeeding
Intervention type(s): Other
Intervention description(s): breastfeeding encouraged versus withheld
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Basic Science
Keywords: immune response; infants; rotavirus vaccine
Vestibular Evoked Myogenic Potentials in Benign Paroxysmal Positional Vertigo (VEMP in BPPV)
Condition: Vertigo
MeSH terms: Vertigo; Benign Paroxysmal Positional Vertigo; Dizziness
Study type: Observational

Summary: 
      Benign Paroxysmal Positional Vertigo (BPPV) is the most frequent cause of vertigo of
      peripheral vestibular origin with life time incidence of 2.4%. BPPV is characterized by bouts
      of acute whirling vertigo lasting less than one minute provoked by changes in head position
      in relation to the gravitational vector. The vertigo is accompanied by typical rotational or
      horizontal nystagmus that is often demonstrated by the Dix-Hallpike maneuver and less
      frequently by testing for positional nystagmus. BPPV pathogenesis is currently explained by
      the fall of otoconia (calcium-carbonate crystals) or otoconial debris from the tectorial
      membrane of the otolithic organs into the dependant semicircular canals (canalithiasis) or
      adherence of such particles to the semicircular canal's cupula (cupulithiasis). Under these
      circumstances, the semicircular canal which normally responds only to angular velocity and
      acceleration is stimulated by gravity. Otoconial remnants as free floating particles inside
      the semicircular canal arms or attached to the cupula have been observed by few
      investigators. Although the presence of such particles explains most characteristics of the
      positioning nystagmus described in BPPV, it does not account for the dizziness and
      disequilibrium which are described by many patients even without changes in head position and
      the continuation of such symptoms after successful treatment of BPPV as evidenced by the
      resolution of positional vertigo and nystagmus.

      The study hypothesis is that otolithic pathology is an important component in the
      pathogenesis of BPPV explaining these symptoms, BPPV recurrence, and the refractoriness of
      some BPPV cases to the vastly employed particles repositioning treatments. In the present
      study the Vestibular Evoked Myogenic Potentials (VEMP) testing would be employed to measure
      the function of one of the otolithic organs - the saccule. The study objectives are: 1. To
      investigate possible malfunction of the saccule in patients suffering from BPPV. 2. To look
      for association between saccular pathology and BPPV recurrence and between such pathology and
      BPPV treatment failure. 3. To study possible relation between saccular pathology and
      continuation of dizziness and disequilibrium despite the resolution of positional vertigo.
    


Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Patients suffering fron Benign Paroxysmal Positional Vertigo
      
Keywords: Benign paroxysmal positional vertigo; Vestibular evoked myogenic potentials; Sacculo-colic reflex
Safety of Cat-PAD to Treat Cat Allergy in Cat Allergic Subjects With Controlled Asthma
Condition: Cat Allergy
MeSH terms: Hypersensitivity
Study type: Interventional
Phase: Phase 2

Summary: 
      Cat allergy is an increasingly prevalent condition, affecting 10-15% of patients with
      allergic rhinoconjunctivitis and/or asthma. Cat-PAD is a novel, synthetic, allergen-derived
      peptide desensitising vaccine, currently being developed for the treatment of cat allergy.

      This study will evaluate the safety and tolerability of multiple doses of Cat-PAD in
      controlled asthmatics treated with either inhaled salbutamol, inhaled corticosteroids or
      inhaled corticosteroids with a LABA and to explore the efficacy of Cat-PAD in these subjects
      using the Late Phase Skin Response, Early Phase Skin Response, and Conjunctival Provocation
      Test.
    

Detailed description: 
      This study is designed as a randomised, double-blind, placebo-controlled, parallel group,
      multiple dose study to evaluate the safety and tolerability of Cat-PAD in cat allergic
      subjects with allergic rhinoconjunctivitis and controlled asthma. The efficacy of Cat-PAD
      will also be explored in these subjects using the EPSR, LPSR, CPT, and levels of cat specific
      IgE. A single centre will be initiated first, with a second centre included as a backup, if
      needed, to enable recruitment numbers to be met.

      Three cohorts will be studied, depending on the treatment used to control the subject's
      asthma:Cohort 1 - inhaled salbutamol only, Cohort 2 - inhaled corticosteroid only, Cohort 3 -
      inhaled corticosteroid plus a LABA

      The study will consist of 3 study periods. In Period 1, Screening will be performed up to a
      maximum of 8 weeks before randomisation and may consist of one or two visits to the clinic,
      at the investigator's discretion. Baseline Challenge will consist of a single visit 1 to 4
      weeks before randomisation.

      In Period 2, subjects in each cohort complying with the inclusion/exclusion criteria will be
      randomised to either 3 nmol Cat-PAD or placebo. Treatments will be administered every 2 weeks
      (±2 days) for 14 week

      In Period 3, Post Treatment Challenge consist of a single visit 24-28 weeks after the first
      administration in the treatment period and assessments will be performed identical to those
      at the baseline challenge. Follow-up will be conducted 3-10 days after PTC.
    

Intervention name(s): Cat-PAD; Placebo
Intervention type(s): Biological; Biological
Intervention description(s): Cat-PAD dose 1x8 administrations 2 weeks apart; Placebo comparator, 1x8 administrations 2 weeks apart
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: Cat allergy; Immunotherapy; Cat-PAD
Effects of Dry Needling in Patients With Sleep Bruxism and Temporomandibular Disorders: a Prospective Case Series
Condition: Myofascial Pain
Study type: Interventional

Summary: 
      The objective of the present study was to assess the effectiveness of deep dry needling in
      the masseter and temporalis MTrPs on pain, pressure pain threshold (PPT), range of motion and
      disability in patients with sleep bruxism and myofascial temporomandibular disorders.
    


Intervention name(s): Deep Dry needling
Intervention type(s): Device
Intervention description(s): The muscles were repeatedly perforated by rapidly inserting and partially withdrawing the needle from the myofascial trigger point, eliciting local twitch responses in some insertions
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Distal Renal Denervation
Condition: Hypertension, Resistant to Conventional Therapy
MeSH terms: Hypertension
Study type: Interventional

Summary: 
      Objective of this study is to evaluate whether a distal mode of endovascular renal
      denervation with the treatment performed primarily in segmental branches of renal artery is
      more effective than conventional mode of the intervention with the treatment equally
      distributed within its main trunk for the treatment of drug-resistant hypertension.
    

Detailed description: 
      Recent spectacular failure of renal denervation (RDN) therapy in SymplicityHTN-3 trial in
      fact might be easily predicted from the very beginning. Conventional RDN done as 4-6 point
      treatments equally distributed within main trunk of renal artery (RA) may only be effective
      if renal plexus tightly surrounds the artery throughout its whole course with equal
      longitudinal and circumferential density of the nerve fibers. While this idea itself is
      unnaturally idealistic also a number of surgical studies demonstrated that proximally
      majority of renal nerves go at a distance from RA obliquely to its course and join the artery
      mainly in its distal part (fan-shaped renal plexus with wide base directed toward aorta and
      apex converging to renal gate). We developed a distal mode of RDN targeting segmental
      branches of RA and conducted a single-center double-blind randomized controlled parallel
      group study to compare its efficacy and safety to those of conventional RDN in patients with
      drug-resistant hypertension.
    

Intervention name(s): Distal renal denervation; Conventional renal denervation
Intervention type(s): Procedure; Procedure
Intervention description(s): Percutaneous endovascular intervention when catheter-based electrode (Symplicity Flex; Medtronic, Inc.) is used for stepwise radiofrequency energy delivery to segmental branches of the renal artery in a number of points distributed along the length and circumference of the vessels in order to ablate renal nerve plexus; Percutaneous endovascular intervention when catheter-based electrode (Symplicity Flex; Medtronic, Inc.) is used for stepwise radiofrequency energy delivery to the main trunk of the renal artery in a number of points equally distributed along its length and circumference in order to ablate renal nerve plexus
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Hypertension, Catheter Ablation, Renal Nerves
Exercise as a Strategy to Treat Cognitive Dysfunction in Persons With Multiple Sclerosis
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis; Cognitive Dysfunction
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      Many persons with Multiple Sclerosis ( MS) have problems with memory and thinking.Exercise
      has been shown to improve memory and thinking in persons with diseases such as Alzheimer's.
      This study will investigate whether a program of aerobic exercise can improve memory and
      thinking in persons with MS
    

Detailed description: 
      Persons with Multiple Sclerosis(MS)will be screened for eligibility and randomized to a
      protocol with two intervention arms. One group will have aerobic training on a recumbent
      cross trainer three times a week for 20-30 minutes/session. The other group will participate
      in stretching exercises at the same frequency and intensity.Total training time will be six
      months.Outcome measures will include tests of memory and thinking, and fitness measures.
    

Intervention name(s): exercise; stretching
Intervention type(s): Behavioral; Behavioral
Intervention description(s): subjects will participate in one of two forms of exercise; subjects will particiopate in supervised stretching as described in arm description.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Treatment
Keywords: Multiple Sclerosis; Exercise; Cognition
AT247, NovoRapid® and Fiasp® in Glucose Clamp Study
Condition: Diabetes Mellitus, Type 1
MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 1
Study type: Interventional
Phase: Phase 1

Summary: 
      A Phase I, randomised, single-centre, double-blind, single-dose, three period, balanced cross
      over study in a glucose clamp setting. The study compares the pharmacodynamic,
      pharmacokinetic and safety characteristics of AT247, NovoRapid® and Fiasp® in male
      participants with type I diabetes mellitus.
    


Intervention name(s): NovoRapid; Fiasp; AT247
Intervention type(s): Biological; Biological; Biological
Intervention description(s): Rapid acting prandial insulin aspart; Fast acting prandial insulin aspart; Ultra rapid acting prandial insulin aspart
Other intervention name(s): NovoLog
Intervention MeSH terms: Insulin Aspart
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Effects of Daily Walnut Intake Among the Subjects With Metabolic Syndrome
Condition: Metabolic Syndrome
MeSH terms: Metabolic Syndrome; Syndrome
Study type: Interventional

Summary: 
      Investigators planed to conduct a randomized, controlled, crossover trial to examine the
      effects of daily walnuts intake among Korean subjects with metabolic syndrome. Primary
      objective of this study was to investigate the effects of daily walnut intake on changes of
      metabolic syndrome indices in Koreans with metabolic syndrome age over 30 years old. This
      study was consisted of four periods: run-in, first intervention, wash-out, and second
      intervention phases. As 16 weeks of first intervention period began after the run-in phase,
      the subjects were randomly assigned into one of two groups: walnut group and control group.
      Participants in walnut group consumed 45 grams of walnuts on a daily basis and participants
      in control group were provided iso-caloric white bread for first 16 weeks. After the first
      intervention and wash-out period (6 weeks), second intervention which is crossover of first
      intervention was started during the next 16 weeks. Anthropometric measurements and
      biochemical analyses were done at the beginning and the end of each intervention (0, 16, 22,
      and 38 weeks of trial). Habitual diet was randomly observed using 3-day diet record once
      during each phase.

      Investigators expected to evaluate 1)effects of walnut ingestion on reverting metabolic
      syndrome to normal status especially by reducing waist circumference and improving serum
      levels of triglyceride and high-density lipoprotein, 2)beneficial effects of daily walnut
      consumption on changes of body composition in the subjects with metabolic syndrome, and
      3)regulatory effects of daily walnut intake on inflammatory markers and diabetic markers.
    


Intervention name(s): food
Intervention type(s): Other
Intervention description(s): 45 g of walnut / iso-caloric white bread
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Double (Investigator, Outcomes Assessor)
Primary purpose: Treatment
Educational Programme Impact on Serum Phosphate Control
Condition: Renal Insufficiency, Chronic
MeSH terms: Renal Insufficiency; Renal Insufficiency, Chronic
Study type: Interventional

Summary: 
      Background: In Chronic Kidney Disease patients, it is crucial to begin treatment as soon as
      possible in order to minimize the complication-related risks. Encouraging patients to adhere
      to their treatment plans is a great challenge for health care professionals. Objectives: The
      aim of this study is to evaluate the impact of a nutrition educational programme on the
      hyperphosphatemia using the transtheoretical model of eating behavior. Subjects and Methods:
      A prospective Interventional study is being conducted in a dialysis centre with 180 stage 5D
      Chronic Kidney Disease patients who exhibit phosphate serum levels > 5.5 mg/dL. The
      educational intervention consists of lectures and group dynamics sessions performed during
      dialysis sessions. Anthropometric, clinical, demographic and laboratory parameters (serum
      phosphorus concentration will be evaluated and the transtheoretical model of eating behavior
      (TMFB) will be applied pre- and post-intervention.
    

Detailed description: 
      Background: In Chronic Kidney Disease patients, it is crucial to begin treatment as soon as
      possible in order to minimize the complication-related risks. Encouraging patients to adhere
      to their treatment plans is a great challenge for health care professionals. Objectives: The
      aim of this study is to evaluate the impact of a nutrition educational programme on the
      hyperphosphatemia using the transtheoretical model of eating behavior. Subjects and Methods:
      A prospective Interventional study is being conducted in a dialysis centre with 180 stage 5D
      Chronic Kidney Disease patients who exhibit phosphate serum levels > 5.5 mg/dL. The
      educational intervention consists of lectures and group dynamics sessions performed during
      dialysis sessions. Anthropometric (weight, height, body mass index), clinical, demographic
      (sex, age, time on dialysis programme, literacy/schooling) and laboratory parameters
      (phosphate serum levels) will be evaluated and the transtheoretical model of eating behavior
      (TMFB) will be applied pre- and post-intervention. Reduction of phosphate serum levels and
      positive/negative behavioral changes will be the evaluated outcomes. Statistical Analyses
      will be carried out in order to compare the effect of educational programme on phosphate
      serum levels and on the behaviour changes.
    

Intervention name(s): Educational Programme
Intervention type(s): Other
Intervention description(s): Educational Programme consists of lectures and group dynamics about nutrition during hemodialysis sessions.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Health Services Research
Keywords: renal dialysis, hyperphosphatemia, educational programme
Assessment of Oxidative Stress Markers in the Upper and Lower Airways of Atopic Children Treated With Nebulized Beclomethasone
Condition: Asthma; Allergic Rhinitis
MeSH terms: Rhinitis; Rhinitis, Allergic
Study type: Interventional
Phase: Phase 3

Summary: 
      Although it is well known that the presence of uncontrolled inflammation in upper airways may
      compromise the control of asthma and may favor the progression of asthma toward more severe
      grades of disease, few studies addressed whether therapies aimed to control both upper and
      lower airway inflammation may be more effective in controlling asthma. Markers of oxidative
      stress and of inflammation such as Nitrotyrosine and IL-5 are increased in the airways of
      children with atopic asthma and correlated with the levels of oral and nasal FeNO, and with
      the grade of atopy. We hypothesize that the treatment with Beclometasone nebulized with a
      facial mask (for treating both upper and lower airways) will be able to reduce the production
      of oxidants as well as of IL5 in both districts thus promoting clinical and functional
      improvements in mild intermittent asthmatic children. The results provided by this study will
      contribute to further clarify the relationship between nasal and bronchial inflammation.
    


Intervention name(s): beclomethasone dipropionate; placebo
Intervention type(s): Drug; Drug
Intervention description(s): 400 mcg/1 ml b.i.d.; 1 ml b.i.d.
Intervention MeSH terms: Beclomethasone
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment