bioihbjkbjnl/mcl2 huggingface dataset:
A Study to Explore the Effect of Sepranolone in Tourette Syndrome
Condition: Tourette Syndrome; Tourette Syndrome in Adolescence
MeSH terms: Tourette Syndrome; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      The study was an open-label, randomized, multicenter, parallel, Phase 2a study in adolescents
      and adult patients with Tourette syndrome that aimed to explore the efficacy of Sepranolone
      as a treatment for Tourette syndrome, by reducing the severity and frequency of tics. The
      total study duration from the Screening Visit to the final follow-up visit was approximately
      26 weeks and included the following periods:

        -  A baseline period of 4 weeks between the screening visit (Visit 1) and randomization (4
           weeks of baseline period were not needed in well-known adult subjects with stable
           Tourette syndrome history over the past at least 4 weeks). A school holiday/annual leave
           period of 2-6 weeks where no study-related activities were done.

        -  A randomized treatment period of 12 weeks

        -  A safety follow-up period of 4 weeks
    


Intervention name(s): Sepranolone
Intervention type(s): Drug
Intervention description(s): Treatment with 10 mg Sepranolone s.c. twice weekly for 12 weeks alongside patient's standard of care Tourette treatment.
Other intervention name(s): Isoallopregnanolone
Intervention MeSH terms: Pregnanolone
Allocation: Randomized
Intervention model: Parallel Assignment
Intervention model description: Interventional, open-label, multicenter, randomized, parallel group study
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Tourette Syndrome in Adults
The Change of Coagulation Markers in Children With β-thalassemia Disease After Stem Cell Transplantation
Condition: Thalassemia
MeSH terms: Thalassemia; beta-Thalassemia
Study type: Observational

Summary: 
      Hypercoagulable state is well recognized in patients with β-thalassemia. Evidences of
      hypercoagulability include abnormal expression of phosphatidylserine on red blood cell (rbc)
      surface and consequent increased platelet activation and thrombin generation. In addition, a
      reduction of anticoagulants i.e. proteins C and S and antithrombin (AT) was demonstrated.
      However, coagulable state in patients with β-thalassemia following stem cell transplantation
      (SCT) has not been characterized.
    

Detailed description: 
      Hypercoagulable state is well recognized in patients with β-thalassemia. Evidences of
      hypercoagulability include abnormal expression of phosphatidylserine on red blood cell (rbc)
      surface and consequent increased platelet activation and thrombin generation. In addition, a
      reduction of anticoagulants i.e. proteins C and S and antithrombin (AT) was demonstrated.
      However, coagulable state in patients with β-thalassemia following stem cell transplantation
      (SCT) has not been characterized.Therefore, the objective is to compare coagulation markers
      and anticoagulants among β-thalassemics with and without SCT and normal control (NC).The
      subjects will be classified into 3 groups; β-thalassemia post SCT (Thal-SCT), β-thalassemia
      treated with regular transfusion (Thal-RT) and NC. Blood samples will be tested for annexin V
      (an index of abnormal expression of phosphatidylserine on rbc surface), markers of activation
      of coagulation system (thrombin antithrombin complex (TAT), prothrombin fragment (F1+2), and
      D-dimer) and anticoagulants (proteins C and S and AT).
    

Observational model: Case-Control
Time perspective: Cross-Sectional
Sampling method: Probability Sample
Study population: 
        The subjects were classified into 3 groups; β-thalassemia post SCT (Thal-SCT),
        β-thalassemia treated with regular transfusion (Thal-RT) and NC.
      
Biospecimen description: 
      Blood
    
Keywords: Coagulation markers, thalassemia disease post SCT
Vascular Risk Progression in Normal and Diabetic Control Subjects.
Condition: Diabetes; Inflammation
MeSH terms: Inflammation
Study type: Observational

Summary: 
      The purpose of this study is to look at the progression of cardiovascular disease (CVD) in
      normal control individuals and in patients with controlled diabetes.
    

Detailed description: 
      Cardiovascular disease remains the greatest cause of mortality in people with diabetes
      mellitus. This observation study will evaluate non-traditional risk factors for their
      contributions to vascular disease progression as determined by carotid intima media thickness
      and history of vascular disease events over 1 year. This study requires annual checks of
      blood, urine, history, and carotid ultrasound for carotid intimal medical thickness.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Any patient with diabetes or healthy individuals
      
Keywords: Diabetes; Vascular risk; Inflammation
US Inhaled Insulin Dose Titration Study
Condition: Diabetes Mellitus, Type 2
MeSH terms: Diabetes Mellitus, Type 2
Study type: Interventional
Phase: Phase 3

Summary: 
      This study will examine how to dose Exubera (inhaled insulin) in patients who are not well
      controlled on two or more diabetes pills. This study should show that a large number of
      patients enrolled in the study can reach the target Hemoglobin A1C levels (7% or less) that
      have been set by the American Diabetes Association (ADA). Target goals should be achieved by
      adjusting the Exubera dose either weekly or twice weekly. Patients will also receive
      nutritional counseling and diabetes education as part of the study.
    


Intervention name(s): Exubera (inhaled insulin); 8-point glucose diary (to assess pre- and post prandial glucoses); Meal Tolerance Test; 24-hour continuous glucose monitoring (at select sites); Pulmonary Function Testing; Hypoglycemic Event Monitoring; Laboratory Assessments; Nutritional Counseling and Diabetes Education
Intervention type(s): Drug; Procedure; Procedure; Procedure; Procedure; Procedure; Procedure; Behavioral
Intervention MeSH terms: Hypoglycemic Agents
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Passive Mobilization and Vascular Function
Condition: Endothelial Dysfunction; Aging; Bed Rest
Study type: Interventional

Summary: 
      With aging, vascular function (VF) declines. Inactivity and sedentary life style have been
      shown to contribute to the worsening of VF. Furthermore, bed rest, a condition commonly used
      for the management of many chronic conditions, has been proven to lead to even more
      deleterious consequences, including VF decline.

      This study evaluates the effect of passive mobilization of the lower limbs on VF in bedridden
      oldest-old. Half of the participants will undergo passive mobilization treatment in addition
      to standard therapies, while the other half will receive only standard therapies. We
      hypothesize that passive mobilization may improve nitric oxide (NO)-mediated endothelial
      function.
    

Detailed description: 
      Normally, cardiovascular function declines with age. Furthermore, cardiovascular function
      declines with immobility, promoting other adverse effects such as postural hypotension,
      impaired cardiac function, and thrombogenic events. Bed rest, a condition normally used in
      the management of many chronic pathologies in elderly people, leads to significant functional
      decline, reduction in physiologic reserve of most of the organ systems, including further
      cardiovascular decline. Fortunately, several studies have suggested interventions that may
      counteract immobility-induced cardiovascular dysfunction and ensure beneficial adaptations in
      the aging population. For example, passive mobilization of the limbs has been shown to
      improve cardiovascular function in bedridden oldest-old. However, the effect of passive
      mobilization on NO-mediated endothelial function have not been studied yet. Thus, the main
      aim of the study is to determine whether passive mobilization is an effective strategy to
      counteract endothelial dysfunction in bedridden oldest-old. For this purpose, bedridden
      individuals will be recruited and randomly assigned to two groups: one group will undergo
      passive mobilization treatment (PM) in addition to standard therapies, the other group (CTRL)
      will undergo standard therapies only. On the basis of preliminary data on a subgroup of ten
      participants, a sample of eighteen individuals will be allocated in each group so to reach a
      statistical power >0.80 and an alpha <0.05. The PM treatment will include knee
      flexion-extension and it will be performed for 30 minutes, 2 times a day, 5 days a week for 4
      weeks.

      Before, after the 4-week treatment, and 1 month after the end of the treatment NO-mediated
      endothelial function of all participants will be evaluated by means of flow-mediated dilation
      and passive limb movement tests.
    

Intervention name(s): Passive mobilization - PM
Intervention type(s): Other
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Flow-mediated dilation; Passive limb movement
Effect of Lactobacillus Rhamnosus GG (LGG) on Infant Colic
Condition: Colic; Inflammation
MeSH terms: Colic; Inflammation
Study type: Interventional

Summary: 
      This study will compare 2 currently marketed formulas in healthy full term babies: Nutramigen
      A+ (a hypoallergenic formula) and Nutramigen-Enflora (hypoallergenic formula with
      Lactobacillus GG (LGG)) during 3 months of formula feeding. The investigators' aims are to
      compare 3 outcomes in these babies: (1) normal baby crying time; (2) the composition of
      intestinal microbiota (bacteria in the stool); and (3) a lab test which measures the number
      of white blood cells in the large intestine (fecal calprotectin). The investigators predict
      that LGG supplementation (Nutramigen-Enflora) will facilitate its establishment as an
      important component of the neonatal intestinal microbial community and reduce fecal
      calprotectin.
    


Intervention name(s): Nutramigen with Enflora; Nutramigen A+
Intervention type(s): Dietary Supplement; Dietary Supplement
Intervention description(s): Hypoallergenic formula with probiotic - Lactobacillus GG; Hypoallergenic formula without lactobacillus
Other intervention name(s): (Nutramigen Lipil)
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Prevention
Keywords: Lactobacillus; Probiotic; Crying time; Fecal microbiota; Calprotectin; Colic; Infant Crying; Barr Diary; Breath Hydrogen; Clinical Design
Laryngeal Injuries After Anesthesia Induction With Sevoflurane and After Anesthesia Induction With Rocuronium
Condition: Laryngeal Injuries
MeSH terms: Wounds and Injuries
Study type: Interventional

Summary: 
      Anesthesia induction with sevoflurane is not associated with an increased incidence or
      severity of laryngeal injuries compared with an anesthesia induction with a muscle relaxant.
    


Intervention name(s): Sevoflurane; Rocuronium
Intervention type(s): Drug; Drug
Intervention description(s): anesthesia induction with propofol, remifentanil and sevoflurane; afterwards tracheal intubation; anesthesia induction with propofol, remifentanil and rocuronium; afterwards tracheal intubation
Intervention MeSH terms: Sevoflurane; Rocuronium
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary purpose: Prevention
Keywords: laryngeal injuries; vocal cord injuries; hoarseness; sore throat
ST2, a Novel Biomarker for Cardiac Remodeling in Adults With Congenital Heart Disease
Condition: Congenital Heart Disease
MeSH terms: Heart Diseases; Heart Defects, Congenital
Study type: Observational

Summary: 
      The remarkable improvement in survival of children with congenital disease has led to a
      continuously growing number of adults with congenital heart disease in the developed world.
      Many of these patients had had cardiac surgery at early age, the may live for many years with
      pressure overload, volume overload, systolic or diastolic dysfunction, cyanosis or any
      combination of the above. These past and ongoing cardiac insults often result in significant
      cardiac remodeling. A biomarker for fibrosis and remodeling may have enormous clinical and
      prognostic value for these patients.

      Serum biomarkers are now integrated in many fields in medicine. In cardiology, a number of
      biomarkers are used. In the last decade, our group has focused on the natriuretic peptides as
      markers for heart disease in infants and children. This resulted in many studies and more
      than 25 publications in the medical literature. We believe that the newly discovered cardiac
      marker, ST2, will emerge as an important addition to cardiac evaluation in the coming
      years.The aim of this study is to measure ST2 levels in patients with congenital right heart
      disease and correlate ST2 levels to clinical status, imaging and prognosis.
    


Observational model: Case-Crossover
Time perspective: Cross-Sectional
Sampling method: Non-Probability Sample
Study population: 
        Adults born with congenital heart disease in stable clinical condition
      
Biospecimen description: 
      Blood
Molecular Breast Imaging in Patients With a History of Bloody Nipple Discharge
Condition: Breast Cancer; Bloody Nipple Discharge
MeSH terms: Galactorrhea
Study type: Interventional

Summary: 
      The purpose of this study is to evaluate molecular breast imaging in the workup of patients
      with a history of bloody nipple discharge, not reproducible in the office who have a negative
      clinical breast examination, and a negative diagnostic mammogram and breast ultrasound.
    

Detailed description: 
      Although the cause of bloody nipple discharge is benign in most cases, the evaluation is
      focused on excluding a malignant cause. When bloody nipple discharge is clinically
      reproducible, management traditionally involves surgical duct excision. When bloody nipple
      discharge is not reproducible and mammogram and ultrasound are negative the management relies
      on "watchful waiting" - serial breast imaging to exclude a focal finding and continued
      observation for recurrent bloody nipple discharge. This approach provokes anxiety among
      patients who do not have a clear explanation for the bloody nipple discharge. Galactography
      is useful when positive, but has a high false-negative rate. MRI has not been studied in this
      context and is costly. Ductoscopy may play a diagnostic role in the future, but experience
      and equipment are limiting variables. The availability of a less costly method of excluding
      breast cancer would be of great value in this population.

      Molecular Breast Imaging (MBI) is a highly promising novel methodology for breast cancer
      detection developed under the leadership of Dr. Michael O'Connor at Mayo Clinic, Rochester,
      MN. Preliminary patient studies with our dual-detector system indicate that this system is
      capable of reliably detecting very small (5-10 mm) malignant lesions in the breast.
    

Intervention name(s): Molecular Breast Imaging
Intervention type(s): Procedure
Intervention description(s): A dual-detector cadmium-zinc-telluride gamma camera system mounted on a modified mammography gantry is used to image the breast. The injection dose of the radiopharmaceutical given to the patient is 20 mCi of 99m Tc-sestamibi.
Other intervention name(s): MBI
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Diagnostic
Keywords: MBI; Mammogram; Breast Cancer; Breast Ultrasound; Bloody Nipple Discharge; Molecular Breast Imaging
Retinal Detachment After Lens Surgery in the Nearsighted
Condition: Retinal Detachment; Lens Diseases; Short-Sighted
MeSH terms: Retinal Detachment; Lens Diseases; Dissociative Disorders
Study type: Observational

Summary: 
      This study aims to study the incidence of retinal detachment in the nearsighted myopic after
      phakoemulsification lens surgery and use of posterior chamber foldable soft implants with a
      follow-up of 5 years and preliminary results at 3 years.
    

Detailed description: 
      Major advances have been made in the field of lens surgery over the last 20 years which has
      significantly reduced the per and postoperative complications of this surgery. Retinal
      detachment remains the most feared complication of this surgery in the nearsighted.

      There is currently an increasing popularity in the international ophthalmic community for the
      correction of ametropia, particularly nearsightedness, by ablation of the non-cataracted
      lens, referred to as refractive lensectomy. However, no recent study reports the
      complications of this procedure with the use of modern surgical techniques.

      This study aims to study the incidence of retinal detachment in the nearsighted myopic after
      phakoemulsification lens surgery and use of posterior chamber foldable soft implants with a
      follow-up of 5 years and preliminary results at 3 years.
    

Intervention name(s): Patients having had a lens surgery
Intervention type(s): Other
Intervention description(s): Dilated fundus exam
Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Strong myopic patients with lens surgery
      
Keywords: Phacoemulsification
Face Transplantation Clinical Trial
Condition: Severe Facial Deformities
MeSH terms: Facies
Study type: Interventional

Summary: 
      The purpose of this study is to apply human facial allograft transplant to the subjects with
      severe facial deformities or disfigurements in order to provide adequate coverage, aesthetic
      appearance and functional outcome.
    

Detailed description: 
      The human scalp and face define two important body units, both functionally and
      aesthetically. Traumatic deformities of the head and neck region resulting from burn
      injuries, gun shot wounds, or ablative tumor surgeries may involve the defect of the skin,
      subcutaneous tissue, muscle or the combination of all these elements. In most cases, the
      ideal reconstruction is very difficult to achieve. Sometimes, a major part of face along with
      the ear or the nose may be involved. The extensive scalp loss due to burn or avulsion injury
      is another deforming and psychologically debilitating condition presenting as a major
      reconstructive challenge. An ideal reconstructive procedure should replace the missing
      tissues and restore the motor and sensory function. Traditional reconstructive procedures of
      facial deformities involve skin grafting, local flap applications, tissue expansion, and
      prefabrication as well as free tissue transfers. The primary goal during facial
      reconstruction is restoration of the function and aesthetic appearance. To obtain
      satisfactory results, missing parts should be replaced only by tissues of the same color and
      texture. The purpose of this study is to apply human facial allograft transplant to the
      subjects with severe facial deformities or disfigurements in order to provide adequate
      coverage, aesthetic appearance and functional outcome.
    

Intervention name(s): Surgical Procedure Composite Facial Transplant
Intervention type(s): Procedure
Intervention description(s): Single Center Prospective Clinical Study to Document the Safety and Feasibility of the Surgical Procedure for Composite Facial Allograft Transplantation
Other intervention name(s): Face Transplant
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Face Transplant; Facial Transplantation; Composite Facial Allograft Transplantation
Safety, Tolerability, Pharmacokinetics (PK), and Activity of ATYR1940 in Participants With Muscular Dystrophy - Study Extension
Condition: Facioscapulohumeral Muscular Dystrophy
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Facioscapulohumeral
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the
      treatment of adult participants with molecularly defined genetic muscular dystrophies.
    

Detailed description: 
      The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the
      treatment of adult participants with molecularly defined genetic muscular dystrophies, and to
      additionally explore the pharmacokinetics and biologic activity of ATYR1940 in adult
      participants with molecularly defined genetic muscular dystrophies. Participants who
      successfully complete the parent study (NCT02239224) are eligible for enrollment into this
      long-term extension study.
    

Intervention name(s): ATYR1940
Intervention type(s): Biological
Intervention description(s): Concentrate for solution for infusion
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
PSA Vaccine Therapy in Treating Patients With Advanced Prostate Cancer
Condition: Prostate Cancer
MeSH terms: Prostatic Neoplasms
Study type: Interventional
Phase: Phase 2

Summary: 
      RATIONALE: Vaccines may make the body build an immune response to kill tumor cells.

      PURPOSE: Randomized phase II trial to study the effectiveness of different regimens of PSA
      vaccines in treating patients who have advanced prostate cancer.
    

Detailed description: 
      OBJECTIVES: I. Evaluate the effect of prime and boost strategies using recombinant fowlpox
      prostate specific antigen (PSA) vaccine and recombinant vaccinia PSA vaccine on biochemical
      PSA progression in patients with stage D0 prostate cancer who have completed local treatment.
      II. Assess the tolerability and toxicity of these prime and boost treatment regimens in these
      patients. III. Evaluate the effects of these prime and boost treatment regimens on cellular
      immunity in these patients.

      OUTLINE: This is a randomized study. Patients are stratified according to HLA-2 typing
      (positive vs negative vs unknown). Patients are randomized to one of three treatment arms.
      Arm I: Patients receive intramuscular fowlpox prostate specific antigen (PSA) vaccine at
      weeks 0, 6, 12, and 18. Arm II: Patients receive intramuscular fowlpox PSA vaccine at weeks
      0, 6, and 12 and intradermal vaccinia PSA vaccine at week 18. Arm III: Patients receive
      intradermal vaccinia PSA vaccine at week 0 and intramuscular fowlpox PSA vaccine at weeks 6,
      12, and 18. Patients are followed at week 24, then every 3 months until year 2, every 6
      months until year 5, and annually thereafter.

      PROJECTED ACCRUAL: A total of 60 patients (20 per treatment arm) will be accrued for this
      study.
    

Intervention name(s): fowlpox virus vaccine vector; recombinant vaccinia prostate-specific antigen vaccine
Intervention type(s): Biological; Biological
Intervention MeSH terms: Vaccines
Allocation: Randomized
Primary purpose: Treatment
Keywords: stage IV prostate cancer; recurrent prostate cancer