bioihbjkbjnl/mcl2 huggingface dataset:
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
Condition: Gaucher Disease, Type 3
MeSH terms: Gaucher Disease
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
      glucocerebrosidase (GCB). Gaucher disease has been classified into 3 clinical subtypes based
      on the presence or absence of neurological symptoms and the severity of these neurological
      symptoms. Patients with type 2 Gaucher disease present with acute neurological deterioration,
      and those with type 3 disease typically display a more sub acute neurological course. Type 1
      Gaucher disease, the most common form accounting for more than 90% of all Gaucher disease
      cases, does not involve the central nervous system.

      The purpose of this clinical research study is to investigate the safety and effectiveness of
      velaglucerase alfa in patients with type 3 Gaucher disease.
    

Detailed description: 
      Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme
      glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside
      accumulates within the macrophages leading to cellular engorgement, organomegaly, and organ
      system dysfunction. Gaucher disease has been classified into 3 clinical subtypes based on the
      presence or absence of neurological symptoms and severity of neurological symptoms. Patients
      with type 2 Gaucher disease present with acute neurological deterioration, and those with
      type 3 Gaucher disease typically display a more sub acute neurological course; type 1 Gaucher
      disease, the most common form accounting for more than 90% of all cases, does not involve the
      central nervous system.

      Velaglucerase alfa is an approved enzyme replacement therapy (ERT) for pediatric and adult
      patients with type 1 Gaucher disease. ERTs have been proven to reduce organomegaly, improve
      hematological parameters and positively impact health-related quality of life; ERTs have not
      been shown to cross the blood brain barrier and as a result have shown limited ability to
      improve the neurological (Central Nervous System; CNS) manifestations associated with Gaucher
      disease.

      This study will provide a basis for exploring the efficacy and safety of velaglucerase alfa
      in patients with type 3 Gaucher disease.
    

Intervention name(s): velaglucerase alfa
Intervention type(s): Biological
Intervention description(s): lyophilized powder, intravenous infusion, units, Every other week (EOW)
Other intervention name(s): VPRIV; Gene activated glucocerebrosidase; GA-GCB; Enzyme replacement therapy
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: VPRIV; velaglucerase alfa
Age Gene/Environment Susceptibility Reykjavik Study
Condition: Neoplasms
Study type: Observational

Summary: 
      5764 men and women in Iceland, aged 66 to 96 years entered the Ages Gene/Environment
      Susceptibility (AGES) - Reykjavik Study. Serum 25(OH)D was measured in 5,519 of all 5,764
      (96%) participants at study entry in year 2002 through 2006. The main emphasis was on
      exploring whether pre-diagnostic serum levels of 25(OH)D were associated with survival after
      cancer diagnosis.
    


Observational model: Cohort
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        In 1967, all 30,795 men and women living in the capital area of Iceland and born between
        1907 and 1935 were invited by the Icelandic Heart Association to join a prospective cohort
        study. Approximately 19,000 attended the clinic at least once and became participants of
        the Reykjavik Study. Subsequently, in 2002, a random sample of the cohort comprising 5,764
        men and women who were still alive in Iceland and aged 66 to 96 years entered the Ages
        Gene/Environment Susceptibility (AGES) - Reykjavik Study
      
Biospecimen description: 
      Serum samples
    
Keywords: Vitamin D; Cancer; Survival
Clofarabine in Treating Patients With T-Cell or Natural Killer-Cell Non-Hodgkin's Lymphoma That Has Relapsed or Not Responded to Previous Treatment
Condition: Leukemia; Lymphoma; Small Intestine Cancer
MeSH terms: Lymphoma; Leukemia; Intestinal Neoplasms
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      RATIONALE: Drugs used in chemotherapy, such as clofarabine, work in different ways to stop
      the growth of cancer cells, either by killing the cells or by stopping them from dividing.

      PURPOSE: This phase I/II trial is studying the side effects and best dose of clofarabine and
      to see how well it works in treating patients with T-cell or natural killer-cell lymphoma
      that has relapsed or not responded to previous treatment.
    

Detailed description: 
      OBJECTIVES:

      Primary

        -  Determine the maximum tolerated dose of clofarabine in patients with relapsed or
           refractory T-cell or natural killer-cell lymphoma.

        -  Determine the toxicity of this drug in these patients.

        -  Determine, preliminarily, the efficacy of this drug, in terms of response rate, in these
           patients.

      OUTLINE: This is a phase I, non-randomized, dose-escalation study followed by an open-label,
      phase II study.

        -  Phase I: Patients receive clofarabine IV over 1 hour once daily on days 1-3. Treatment
           repeats every 21 days for up to 2 courses in the absence of disease progression or
           unacceptable toxicity. Patients achieving stable disease or partial response (PR) or
           complete response (CR) may receive 2 additional courses of treatment. Patients with PR
           or CR after completing 4 courses of therapy may receive 2 additional courses.

      Cohorts of 1-6 patients receive escalating doses of clofarabine until the maximum tolerated
      dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2
      of 6 patients experience dose-limiting toxicity.

        -  Phase II: Patients receive clofarabine as in phase I at the MTD determined in phase I.

      After completion of study treatment, patients are followed every 3 months for 2 years.
    

Intervention name(s): clofarabine
Intervention type(s): Drug
Intervention MeSH terms: Clofarabine
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: anaplastic large cell lymphoma; angioimmunoblastic T-cell lymphoma; recurrent adult lymphoblastic lymphoma; recurrent adult T-cell leukemia/lymphoma; recurrent mycosis fungoides/Sezary syndrome; adult nasal type extranodal NK/T-cell lymphoma; prolymphocytic leukemia; childhood nasal type extranodal NK/T-cell lymphoma; recurrent childhood lymphoblastic lymphoma; recurrent cutaneous T-cell non-Hodgkin lymphoma; small intestine lymphoma
Replication of the DECLARE Diabetes Trial in Healthcare Claims
Condition: Diabetes
MeSH terms: Diabetes Mellitus
Study type: Observational

Summary: 
      Investigators are building an empirical evidence base for real world data through large-scale
      replication of randomized controlled trials. The investigators' goal is to understand for
      what types of clinical questions real world data analyses can be conducted with confidence
      and how to implement such studies.
    

Detailed description: 
      This is a non-randomized, non-interventional study that is part of the RCT DUPLICATE
      initiative (www.rctduplicate.org) of the Brigham and Women's Hospital, Harvard Medical
      School. It is intended to replicate, as closely as is possible in healthcare insurance claims
      data, the trial listed below/above. Although many features of the trial cannot be directly
      replicated in healthcare claims, key design features, including outcomes, exposures, and
      inclusion/exclusion criteria, were selected to proxy those features from the trial.
      Randomization is also not replicable in healthcare claims data but was proxied through a
      statistical balancing of measured covariates according to standard practice. Investigators
      assume that the RCT provides the reference standard treatment effect estimate and that
      failure to replicate RCT findings is indicative of the inadequacy of the healthcare claims
      data for replication for a range of possible reasons and does not provide information on the
      validity of the original RCT finding.
    

Intervention name(s): Dapagliflozin; DPP-4 inhibitor
Intervention type(s): Drug; Drug
Intervention description(s): Dapagliflozin dispensing claim for any dose is exposure; DPP4 inhibitor dispensing claim for any dose is reference
Intervention MeSH terms: Dapagliflozin; Dipeptidyl-Peptidase IV Inhibitors
Observational model: Cohort
Time perspective: Retrospective
Sampling method: Non-Probability Sample
Study population: 
        This study will involve a new user, parallel group, cohort study design comparing
        dapagliflozin to the DPP-4 inhibitor (DPP4i) antidiabetic class. DPP4is serve as a proxy
        for placebo, since this class of antidiabetic drugs is not known to have an impact on the
        outcome of interest. The comparison against DPP4 inhibitors is the primary comparison. The
        patients will be required to have continuous enrollment during the baseline period of 180
        days before initiation of canagliflozin or a comparator drug (cohort entry date). Follow-up
        for the outcomes (a. 3P-MACE and b. composite of heart failure hospitalization/all-cause
        mortality), begins the day after drug initiation. As in the trial, patients are allowed to
        take other antidiabetic medications during the study.
Radiofrequency Versus Laser Ablation for Hepatocellular Carcinoma
Condition: Carcinoma, Hepatocellular; Neoplasms, Liver
MeSH terms: Carcinoma; Carcinoma, Hepatocellular; Liver Neoplasms
Study type: Interventional

Summary: 
      The purpose of this study is to prospectively compare percutaneous radiofrequency ablation
      (RFA) versus percutaneous laser ablation (LA) for the treatment of small hepatocellular
      carcinoma in patients with cirrhosis.
    

Detailed description: 
      Percutaneous ablation is a safe and effective therapy for cirrhotic patients with HCC when
      resection or liver transplantation is not possible. Among the various percutaneous local
      ablative therapies, radiofrequency ablation (RFA) has attracted the greatest interest because
      of its effectiveness and safety for single HCC ≤ 5.0cm or ≤3 HCC nodules ≤3cm. Some studies
      have shown that laser ablation (LA) may be as effective as RFA in the treatment of HCC.
      However, RFA and LA are different techniques and, in different cases (for example: size or
      site of HCC nodule), each of these procedures may have some advantages or disadvantages.
      Therefore there is the need for a prospective randomized controlled study to compare RFA and
      LA in patients with small HCC.
    

Intervention name(s): percutaneous radiofrequency ablation; percutaneous laser ablation
Intervention type(s): Procedure; Procedure
Intervention description(s): Procedure: Radiofrequency ablation For RFA, we used a commercially available system (Valleylab, Tyco Healthcare, Boulder, CO, USA) and a 17-gauge "cool-tip" needle electrode with a 3cm exposed tip. The needle is inserted percutaneously under ultrasound guidance in the HCC nodule and treatment lasts 12 minutes. For nodules larger than 3cm in diameter, 2 insertions are used. In a period of six months, treatment may be repeated for no more than three times. When, after treatment, CT or MRI show a residual nodule activity of 10% or less, percutaneous ethanol injection may be done. If the residual activity is of 50% or more transarterial chemoembolization may be used.; Procedure: Laser ablation For LA, we used a commercially available system (Echolaser XVG system, Esaote El.En., Florence, Italy) and four optical fibers,inserted into the cancer through four 21-gauge needles. The treatment lasts 6 minutes. For nodules larger than 3 cm in diameter, two treatments are done. In a period of six months, treatment may be repeated for no more than three times. When, after treatment, CT or MRI show a residual nodule activity of 10% or less, percutaneous ethanol injection may be done. If the residual activity is of 50% or more transarterial chemoembolization may be used.
Other intervention name(s): Cool-tip ablation system - Valleylab, USA; Echolaser XVG system, Italy
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
A Study of Cerebral-circulatory Response to Intravenous Antihypertensive Treatment in Women With Serious Preeclampsia
Condition: Preeclampsia
MeSH terms: Pre-Eclampsia
Study type: Observational

Summary: 
      The purpose of this study is a systematical registration of hemodynamic measurements (cardiac
      output, blood pressure and systemic vascular resistance) in women with serious preeclampsia.
    


Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Pregnant women with moderate-to-severe pre-eclapmsia
      
Keywords: Preeclampsia; Trans cranial Doppler; Cardiac output; systemic vascular resistance; blood pressure
Antimicrobial Coated Sutures in Paediatric Surgery
Condition: Wound Infections
MeSH terms: Wound Infection
Study type: Interventional

Summary: 
      The purpose of this study is to determine if suture material coated by antimicrobial agent
      triclosan would decrease the incidence of surgical site infections (SSIs) in paediatric
      surgery compared to ordinary sutures.

      1500 children (age form 4 weeks to 18 years) coming for general pediatric surgery to the Oulu
      University Hospital are randomised to have sutures coated with triclosan (Vicryl Plus,
      Monocryl Plus) or ordinary sutures. The occurrence of SSIs is monitored by email
      questionnaires to the parents on days 10 and 30. The diagnosis of SSIs are made along CDC
      criteria.
    

Detailed description: 
      We have now (spring 2014) recruited almost 1500 patients to the study. When reviewing the
      recruited material, we found out that the study suture material has been used in 83% of the
      recruited patients. The reasons for this have been clinical issue during the operation where
      melting suture material may not have been needed at all.

      We have decided to prolong recruiting so that we would gather the calculated sample size of
      1500 of patient with study suture material. This will take 12-18 months (probably till autumn
      2015).
    

Intervention name(s): Triclosane; Control
Intervention type(s): Other; Other
Intervention description(s): Triclosane coated suture material; Ordinary suture material
Other intervention name(s): Vicryl plus; Vicryl
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Prevention
Lurasidone Pediatric Pharmacokinetics Study
Condition: Schizophrenia; Autism
MeSH terms: Schizophrenia
Study type: Interventional
Phase: Phase 1

Summary: 
      This is a Phase 1, open-label, multicenter, single and multiple ascending lurasidone dose
      study in subjects from 6 to 17 years old with schizophrenia spectrum, bipolar spectrum,
      autistic spectrum disorder, or other psychiatric disorders.
    

Detailed description: 
      To characterize lurasidone PK profile following single and multiple oral doses of 20, 40,80,
      120 or 160 mg/day lurasidone in the targeted pediatric/adolescent population (6-17 years
      old)with schizophrenia spectrum, bipolar spectrum, autistic spectrum disorder or other
      psychiatric disorders. Data from this study will be used to recommend pediatric doses that
      result in comparable exposures to those observed in currently approved adult doses of Latuda®
      (40, 80, 120 and 160 mg/day) in subsequent efficacy and safety studies.
    

Intervention name(s): Lurasidone
Intervention type(s): Drug
Intervention description(s): Lurasidone 20, 40,80, 120 or 160 mg/day oral single and multiple does of lurasidone for 12 days
Intervention MeSH terms: Lurasidone Hydrochloride
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Schizophrenia; autism; bipolar; Lurasidone; Latuda
Transplant-Related Mortality in Patients Undergoing a Peripheral Blood Stem Cell Transplantation or an Umbilical Cord Blood Transplantation
Condition: Acute Myeloid Leukemia; Acute Lymphoblastic Leukemia; Myelodysplastic Syndrome
MeSH terms: Leukemia; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Myelodysplastic Syndromes
Study type: Observational

Summary: 
      Study CR-AIR-006 is a part of the ATIR clinical development plan and will provide control
      data for patients treated with ATIR in clinical studies (e.g. study CR-AIR-007).
    


Observational model: Cohort
Time perspective: Retrospective
Sampling method: Probability Sample
Study population: 
        Patients having received a haploidentical hematopoietic stem cell transplantation (HSCT)
        between 1 January 2006 and 30 June 2014, an HSCT from an unrelated donor between 1 January
        2010 and 31 December 2012, or a double UCB transplantation between 1 January 2010 and 31
        December 2012 will be selected from the centers that enrolled patients in study CR-AIR-004
        and/or study CR-AIR-007.
      
Keywords: Haploidentical donor; Unrelated donor; Umbilical cord blood; Graft versus host disease; Transplant-related mortality; Hematologic malignancy; Hematopoietic Stem Cell Transplantation
Study of the Safety and Efficacy of Fixed Dose OPC-34712 as Adjunctive Therapy in the Treatment of Adults With Major Depressive Disorder (the Pyxis Trial)
Condition: Depressive Disorder; Depression; Depressive Disorder, Major; Mood Disorders; Mental Disorders
MeSH terms: Depression; Depressive Disorder; Depressive Disorder, Major; Mental Disorders; Mood Disorders
Study type: Interventional
Phase: Phase 3

Summary: 
      To compare the effect of OPC-34712 (brexpiprazole) to the effect of placebo (an inactive
      substance) as add on treatment to an assigned FDA approved antidepressant treatment (ADT) in
      patients with Major Depressive Disorder who demonstrate an incomplete response to a
      prospective trial of the same assigned FDA approved ADT
    


Intervention name(s): OPC-34712 + ADT; Placebo + ADT; Placebo + ADT
Intervention type(s): Drug; Drug; Drug
Intervention description(s): Tablet, Oral, 2 mg OPC-34712 and FDA Approved Antidepressant Therapy (ADT); Placebo + FDA Approved Antidepressant (ADT); Placebo + FDA Approved Antidepressant (ADT)
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary purpose: Treatment
Keywords: OPC-34712; brexpiprazole; Major Depressive Disorder; Adjunctive Treatment
Deep Brain Stimulation (DBS) for the Treatment of Parkinson's Disease
Condition: Parkinson's Disease
MeSH terms: Parkinson Disease
Study type: Interventional

Summary: 
      The purpose of this study is to evaluate the safety and effectiveness of Boston Scientific's
      Vercise Deep Brain Stimulation (DBS) system in the treatment of patients with with advanced,
      levodopa-responsive bilateral Parkinson's disease (PD) which is not adequately controlled
      with medication.
    

Detailed description: 
      The study is multi-center, prospective, double-blind, randomized (3:1) controlled trial.

      GUIDE XT may be used for planning of programming as needed.
    

Intervention name(s): Deep Brain Stimulation
Intervention type(s): Device
Intervention description(s): The Vercise™ DBS system will be implanted in subjects in both study arms. Stimulation parameters will vary depending on the study arm assignment. All subjects will receive therapeutic settings at the end of the blinded period.
Other intervention name(s): Vercise™ Deep Brain Stimulation (DBS) system.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Intrepid, Vercise, PD, DBS, Boston Scientific
Study of Oliceridine (TRV130) for the Treatment of Moderate to Severe Acute Pain After Bunionectomy
Condition: Acute Pain
MeSH terms: Acute Pain
Study type: Interventional
Phase: Phase 3

Summary: 
      The primary objective is to evaluate the analgesic efficacy of intravenous (IV) oliceridine
      administered as needed (PRN) compared with placebo in patients with moderate to severe acute
      postoperative pain after bunionectomy.
    


Intervention name(s): Oliceridine; Placebo; Morphine
Intervention type(s): Drug; Drug; Drug
Intervention MeSH terms: Morphine
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
The Importance of Electrical Stimulation as a Treatment for Urinary Incontinence in Patients Prostatectomy
Condition: Urinary Incontinence
MeSH terms: Urinary Incontinence; Enuresis
Study type: Interventional

Summary: 
      The use of electrical stimulation to reduce urinary incontinence in men undergoing
      prostatectomy exercises of the pelvic floor muscles (MAPs)
    

Detailed description: 
      The study will be an experiment with active control, randomized, double-blind, parallel
      intervention. After met the eligibility criteria of the patients will be randomly allocated
      into two groups: Group exercises MAPs (GEX), which is the active control group and
      electrostimulation + exercises MAPs (GEEX).
    

Intervention name(s): electrostimulation with exercises MAPs; exercises MAPs
Intervention type(s): Other; Other
Intervention description(s): this group will make the electrostimulation with exercises of the pelvic floor muscles with a focus on strengthening the pelvic floor muscles.the frequency will be 20 sessions, 2 times per week, lasting 30 minutes.; This group will focus exercises of the pelvic floor muscles with strengthening the muscles of the pelvic floor. the frequency will be 20 sessions, 2 times per week, lasting 30 minutes.
Other intervention name(s): electrostimulation and exercises of the pelvic floor muscles; exercises of the pelvic floor muscles
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Investigator, Outcomes Assessor)
Primary purpose: Treatment