bioihbjkbjnl/mcl2 huggingface dataset:
A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris
Condition: Pemphigus Vulgaris
MeSH terms: Pemphigus
Study type: Interventional
Phase: Phase 2

Summary: 
      Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus
      vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy,
      and with conventional immunosuppressive "rescue treatment", if indicated. The duration of
      therapy in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase,
      Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.
    

Detailed description: 
      Primary Objectives:

      To evaluate the safety of PRN1008 in patients with pemphigus vulgaris (PV)

      To evaluate the clinical activity of PRN1008 in patients with PV, per criteria in the
      European Academy of Dermatology and Venereology (EADV) 2014 Pemphigus S2 Guideline (Hertl et
      al. 2015)

      Secondary Objectives

      To evaluate the pharmacokinetics (PK) and the pharmacodynamics (PD) of multiple doses of
      PRN1008 in patients with PV

      To evaluate the relationship of PK and PD to each other and to efficacy and safety in this
      patient population
    

Intervention name(s): PRN1008
Intervention type(s): Drug
Intervention description(s): Part A dosing was initiated administering 400 mg BID. Intrapatient dose adjustments (reductions and increases) were permitted based upon tolerability and clinical response with the maximum dose allowed up to 600 mg BID for 12 weeks.
Part B initial dosing was 400 mg QD for 2 weeks with dose escalation to 400 mg BID at the discretion of the Investigator for the purposes of investigating dose response and identifying the minimal efficacious dose of rilzabrutinib for 24 weeks.
Other intervention name(s): BTK inhibitor; Rilzabrutinib
Intervention model: Single Group Assignment
Intervention model description: Part A with 12 weeks treatment and 12 weeks follow-up. Part B with 24 weeks treatment and 4 weeks follow-up.
Masking: None (Open Label)
Primary purpose: Treatment
Efficacy and Tolerability of PEG-only Laxative for Fecal Impaction and Chronic Constipation in Children
Condition: Constipation; Faecal Impaction
MeSH terms: Fecal Impaction; Constipation
Study type: Interventional
Phase: Phase 4

Summary: 
      The aim of this study is to compare the efficacy, tolerability, acceptance and compliance of
      a PEG-only formulation compared to a reference PEG-EL formulation in resolving faecal
      impaction and in the treatment of chronic constipation
    

Detailed description: 
      Constipation is a very common complaint in childhood. Inadequate daily fiber intake,
      insufficient fluid intake, and withholding behavior are considered as factors leading to
      constipation.

      Painful defecation is considered a common trigger to faecal retention wich leads to
      reabsorption of fluids and increase in the size and consistency of stools.

      It is often necessary to use laxative therapy to achieve comfortable defecation. PEG-based
      laxatives are considered today the gold standard for treatment of constipation in children.
      PEG formulations differ for composition in inactive ingredients which may have an impact on
      acceptance, compliance and adherence to treatment.
    

Intervention name(s): Polyethylene glycol with electrolytes; Polyethylene glycol
Intervention type(s): Drug; Device
Intervention description(s): Constipation treatment: 1-4 sachets/day according to the patient age. 1 sachet contains 6.9 g PEG-EL. Treatment period: 4 weeks.
Faecal impaction resolution: 4 sachets as initial dose and increasing 2 sachets a day until resolution or up to 7 days.; Constipation: 0.7 g/kg/day in 2 divided doses for children of less than 20 kg. For children > 20 kg the daily dose was up to PEG 30 g daily. Treatment period: 4 weeks.
Faecal impaction resolution: 1.5 g/kg/day in 2 doses until resolution or up to 6 days.
Other intervention name(s): Movicol bambini; Onligol
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Investigator)
Primary purpose: Treatment
Keywords: chronic constipation; faecal impaction; PEG-only laxative
Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
Condition: Rubinstein-Taybi Syndrome
MeSH terms: Rubinstein-Taybi Syndrome; Hand Deformities, Congenital; Osteochondrodysplasias; Craniofacial Abnormalities; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      An exploratory phase 2 therapeutic trial in children from 6 to 21, RTS carriers, randomized
      to be treated either with sodium valproate with the usual pediatric dosage (30 mg/kg/j), or
      by placebo for one year.

      The investigator would like to include children because they could best profit from it due to
      their neuronal plasticity as CBP and EP300 take effect through neuronal and synaptic
      plasticity.

      The therapeutic effect of sodium valproate in RTS patients will be assessed thanks to a
      clinical approach (learning and memory neuropsychological evaluation, fine motor skills
      assessment by pointing), to a biological approach (histone acetylation functional tests), and
      to imaging (morphological and functional MRI).
    

Detailed description: 
      Such a multidisciplinary approach has never been presented for this syndrome; it should
      enable us identifying specific cognitive and motor deficits and their association thanks to
      imaging markers. Assessing the various aspects of motor skills, i.e. primary (movements,
      standing at ease position, immobility) and complex motor skills (actions and behaviours,
      motor aspects of feelings and language expressions) is an integral part of the clinical
      examination in psychiatrics.

      The main interest of a functional capacity analysis is to be used as an index allowing the
      assessment of the nervous system's global capacities and the study of the connections between
      cognitive functions and motor skills Main objective: Evaluate long term memory with subtest
      "point location" - CMS and "image recognition" RBMT A patient is said to be responder if
      after one year his or her test result increase for one point at least one of the two tests :
      a increase of one point to "point location" or "image recognition".

      Double-blind phase 2 clinical trial, designed as a one-step Fleming design but with a control
      group. Rubival is a two-parallel group randomized trial:

        -  a placebo group of 20 patients

        -  a group of 40 patients taking sodium valproate (HDAC inhibitor) with an oral dosage of
           30 mg/kg/jour
    

Intervention name(s): sodium valproate; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): sodium valproate (HDAC inhibitor) with an oral dosage of 30 mg/kg/jour; Placebo with an oral dosage of 30 mg/kg/jour
Intervention MeSH terms: Valproic Acid
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: RTS; Rubinstein Taybi; explorative phase 2; clinical approach; biological approach; motor skills
S1P1 Receptor Subtype (S1PR1) Axis in Patients With Interstitial Cystitis (IC) Pain
Condition: Interstitial Cystitis
MeSH terms: Cystitis; Cystitis, Interstitial
Study type: Observational [Patient Registry]

Summary: 
      For each patient in the study, blood, and pain scores will be collected to look for markers
      for pain, at the start of treatment and 6 months after surgery of treatment.
    

Detailed description: 
      The study is investigator-initiated, prospective, study. There will be no change in care.

      Patients who have had a diagnosis of IC made by cystoscopy and hydrodistension and still have
      pain will be recruited for this study.

      Patients will have blood samples taken at the visit they sign the consent form and 6 months
      later.

      The study staff will call and remind the patient of their 6 month visit.
    

Intervention name(s): Questionnaires; Collection of Blood samples
Intervention type(s): Other; Other
Intervention description(s): QOL and pain questionnaires will be given to patients at 3, 6, and 12 months post surgery; Blood for PBLs will be collected at 3, 6, and 12 months post surgery
Observational model: Other
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Women are included. The onset of interstitial cystitis usually occurs between 30 and 70
        years of age, with a median age of 43.

        .
      
Biospecimen description: 
      PERIPHERAL BLOOD LEUKOCYTE (PBLS) ANALYSIS (Research Related):

      Using immunoblotting, we will determine if the level of S1PR1 expression in peripheral blood
      leukocytes (PBLs) is increased in patients with IC pain. Patients will have blood samples
      taken, at the visit they sign the consent form 3 and 6 months later. A total of 4 teaspoons
      of blood will be taken for the study.
Drug-drug Interaction Study
Condition: Pompe Disease
MeSH terms: Glycogen Storage Disease Type II
Study type: Interventional
Phase: Phase 2

Summary: 
      This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant
      human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe
      Disease.
    

Detailed description: 
      This was a multi-center, international, open-label, two-period, fixed-sequence crossover
      study to evaluate the safety and pharmacokinetic effect of single ascending doses of
      duvoglustat on rhGAA administered 1 hour before initiation of a single rhGAA infusion. During
      Period 1, participants received a single intravenous infusion of rhGAA. During Period 2, each
      participant received a single oral dose of duvoglustat 1 hour prior to initiation of a single
      rhGAA infusion.
    

Intervention name(s): duvoglustat; rhGAA
Intervention type(s): Drug; Drug
Intervention description(s): Single oral dose; Single intravenous infusion
Other intervention name(s): AT2220; duvoglustat hydrochloride; alglucosidase alfa; recombinant human alpha-glucosidase; Myozyme; Lumizyme
Intervention MeSH terms: 1-Deoxynojirimycin
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Amicus Therapeutics; duvoglustat; AT2220; alpha-glucosidase; alglucosidase alfa
Bioequivalence of Flurbiprofen Lozenge vs Strepfen
Condition: Healthy
Study type: Interventional
Phase: Phase 1

Summary: 
      The present study has been designed to compare flurbiprofen pharmacokinetic profile after
      administration of flurbiprofen 8.75 mg lozenges, marketed by ACRAF, S.p.A., Italy (test
      product), and the marketed Strepfen 8.75 mg honey and lemon lozenges reference product.
      (Reckitt Benckiser Portugal SA).
    

Detailed description: 
      This is a single dose, open-label, randomised, 2-way cross-over bioequivalence study aimed to
      compare the bioavailability and the plasma pharmacokinetic profile of flurbiprofen after
      single dose administration of test and reference formulations.
    

Intervention name(s): Flurbiprofen Test 8.75 MG; Flurbiprofen Reference 8.75 MG
Intervention type(s): Drug; Drug
Intervention description(s): Subjects will be asked to swallow saliva before dosing. For each administration, subjects will move the lozenge around in their mouth, suck on the lozenge continually for 15 min and swallow saliva at any time during the product administration procedures, being very careful not to swallow the lozenge.; Subjects will be asked to swallow saliva before dosing. For each administration, subjects will move the lozenge around in their mouth, suck on the lozenge continually for 15 min and swallow saliva at any time during the product administration procedures, being very careful not to swallow the lozenge.
Other intervention name(s): Strepfen
Intervention MeSH terms: Flurbiprofen
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: Single dose, open-label, randomised, 2-way cross-over bioequivalence study
Masking: None (Open Label)
Primary purpose: Other
Keywords: flurbiprofen; lozenge; Strepfen; bioequivalence
Study of Ablation Versus antiaRrhythmic Drugs in Persistent Atrial Fibrillation
Condition: Atrial Fibrillation
MeSH terms: Atrial Fibrillation
Study type: Interventional
Phase: Phase 4

Summary: 
      The purpose of this study is to compare the effectivity and safety of atrial fibrillation
      ablation, in comparison to antiarrhythmic drug therapy in patients with refractory,
      persistent atrial fibrillation.
    


Intervention name(s): Atrial fibrillation ablation; Antiarrhythmic drug
Intervention type(s): Procedure; Drug
Intervention description(s): Atrial fibrillation ablation with radiofrequency, guided by 3D navigation map. Either 4mm or cooled tip-catheters will be used.
It will include, at least, pulmonary veins isolation; all other radiofrequency lines, including roof and mitral isthmus line, and complex fractioned electrograms ablation will be performed according to each Hospital protocol. Radiofrequency lines block will be tested.; Usual drug therapy for atrial fibrillation. In patients with structural heart disease, amiodarone is recommended, while flecainide plus diltiazem or beta-blockers are encouraged in patients without structural heart diseases.
Intervention MeSH terms: Anti-Arrhythmia Agents
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Catheter Ablation, Radiofrequency; Atrial fibrillation; Antiarrhythmic Drugs
Effect of Endothelin-1 Receptor Blockade on Circulating Endothelial Microparticles Levels in Patients With Pulmonary Hypertension
Condition: Pulmonary Hypertension
MeSH terms: Hypertension, Pulmonary; Hypertension
Study type: Observational

Summary: 
      Pulmonary hypertension is a progressive and life threatening condition. It is characterized
      by severe remodeling of the pulmonary vessel wall, obstructive plexiform lesions, multi-focal
      thrombosis, and enhanced vasoconstriction. All of these characteristics contribute to
      increased pulmonary vascular resistance.

      Circulating endothelial microparticles (EMPs) play an integral role in the pathogenesis and
      perpetuation of pulmonary hypertension. Levels of EMPs are considered a reliable biological
      parameter of endothelial injury.

      We propose to assess the evolution of both circulating and pulmonary venous EMPs in patients
      with PH. Assessments will be made before and after initiation of Endothelin-1 (ET-1) Receptor
      blocker therapy, and correlated to their patterns with the changes in mean PAP, the 6 Minutes
      Walking Distance test, and circulating Endothelin-1 values. Measurements of the endothelial
      microparticle circulating levels (assessed by flow cytometry methods) will be made before, 1
      month and 3 months after initiation of therapy.
    


Observational model: Case-Only
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Precapillary Pulmonary Hypertension
      
Keywords: To determine if there is a trans-pulmonary gradient in the levels of EMPs in patients with PH; To determine if the EMPs measured at the pulmonary capillary wedge position correlates even more strongly with the severity of PH than the circulating levels; To determine if PH therapy with ET-1 receptor blocker will modify EMPs levels
Subcutaneous Injection and Ultrasonic Dispersion of Cefazolin Into Chronic Pelvic-Region Pressure Ulcers in Persons With Spinal Cord Injury
Condition: Spinal Cord Injury; Chronic Pressure Ulcers
MeSH terms: Spinal Cord Injuries; Pressure Ulcer
Study type: Interventional

Summary: 
      Pressure ulcers (PU) are skin breakdowns that often form after blood flow in the skin is
      reduced from prolonged and repeated exposure to externally applied forces. As many as 85% of
      individuals with a spinal cord injury (SCI) report the occurrence of at least 1 PU since
      being injured. Despite the increasing attention and emphasis on prevention, PUs still
      represent a major health risk for persons with SCI. PUs and other skin breakdowns are at risk
      for becoming infected; it is not uncommon for many different types of "bugs" to be found in
      the wound. It has been assumed that the presence of these organisms did not impede wound
      healing or skin graft survival. The current proposal will use a new type of procedure that
      involves the direct injection of an antibiotic (in saline) into the skin beneath the wound;
      it is then distributed throughout the wound using a second device that uses sound waves. The
      study will determine if the antibiotic treatment and the standard of care improves the rate
      wound closure compared to the standard of care alone in persons with SCI and a chronic
      pelvic-region PU.
    

Detailed description: 
      Persons with spinal cord injury (SCI) are at increased risk of developing a Stage III or IV
      (full-thickness) pressure ulcer (PU). These wounds are associated with a tremendous personal
      and financial cost. An intervention that proves efficacious in the facilitation of healing
      this chronic medical condition would be of enormous medical, social, and economic benefit to
      persons with SCI and other cohorts with a difficult to heal PUs. From preliminary studies, it
      appears that hypodermoclysis (i.e., an interstitial infusion) with an antibiotic (i.e.,
      cefazolin; a first generation cephalosporin) and subsequent dispersion with ultrasound
      throughout the wound recently has been shown in preliminary work to be efficacious in the
      treatment of infection, both recognized pathogenic organisms and those that have colonized
      the wound bed), which then appears to facilitate wound closure and success of standard
      surgical intervention. This approach to wound care represents a paradigm shift because
      previously it was assumed that nonpathogenic organism did not impede wound healing or skin
      graft survival. The investigators will test this novel approach to wound healing in persons
      with SCI who have a chronic PU of the pelvic region.
    

Intervention name(s): Ultrasonic Distribution of Cefazolin
Intervention type(s): Device
Intervention MeSH terms: Cefazolin
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Cefazolin
Phase II Study Using CHFR Methylation Status in Patients With Metastatic Esophageal, Gastroesophageal, Gastric Cancer.
Condition: Metastatic Esophageal Cancer; Gastroesophageal Cancer; Gastric Cancer
MeSH terms: Stomach Neoplasms
Study type: Interventional
Phase: Phase 2

Summary: 
      To estimate and compare the response rates in patients treated with mDCF based on methylation
      status of CHFR.
    

Detailed description: 
      To estimate and compare the response rates in patients treated with mDCF based on methylation
      status of CHFR. The overall response rates in the un-methylated and methylated patient groups
      will be reported with a exact 95% binomial confidence interval. A chi-square test will be
      used for comparison.
    

Intervention name(s): Docetaxel; Leucovorin; Fluorouracil; Cisplatin
Intervention type(s): Drug; Drug; Drug; Drug
Intervention description(s): Modified Docetaxel 40mg/m2 on Day 1; Leucovorin 400mg/m2 on Day 1; Fluorouracil 400mg/m2 on Day 1 Fluorouracil 1000mg/m2/day on Days 1 and 2; Cisplatin (or Carboplatin) 40mg/m2 on Day 3
Other intervention name(s): Modified DCF; FU; Carboplatin
Intervention MeSH terms: Leucovorin; Carboplatin; Docetaxel; Fluorouracil
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Enhancing Diabetic Foot Education by Viewing Personal Plantar Pressures
Condition: Diabetes Mellitus; Diabetic Neuropathies; Foot Complications
MeSH terms: Diabetic Neuropathies; Diabetic Foot; Diabetes Mellitus
Study type: Interventional

Summary: 
      The purpose of this study is to examine the effectiveness of a novel patient education
      strategy, compared to a standard diabetic foot education. The proposed diabetic foot care
      education uses personal computer-animated plantar pressure data to educate patients on why
      and how they should care for their feet.
    

Detailed description: 
      Using block randomization, subjects with diabetes were assigned to either the standard or the
      enhanced education group. The effectiveness of enhanced education was evaluated as measured
      by foot care behavior score, patient's interpretation of neuropathy scores, and the incidence
      of diabetic foot complications over a course of 1-year.
    

Intervention name(s): Enhanced foot care education; Standard Foot Care Education
Intervention type(s): Behavioral; Behavioral
Intervention description(s): In addition to the standard foot care education, personalized, computer-animated plantar pressure maps in both barefoot and in-shoe conditions were demonstrated once at baseline visit. The demonstration includes diabetic foot education on the topic of diabetic neuropathy and how barefoot walking can lead to skin breakdown and ulcer formation, which can lead to infection and eventual amputation. The education also highlights the high plantar pressures experienced by individuals while barefoot versus in-shoe and how proper footwear is necessary in conjunction with other standard self-foot care measures to prevent injury and complications.; At baseline, a trained staff individually reviewed and dispensed the following brochures: "Prevent diabetes problems: Keep your diabetes under control" (NIH Publication No. 07-4349) and "Prevent diabetes problems: Keep your feet and skin healthy" (NIH Publication No. 07-4282) along with a 1-page summary of each brochure. Also, a 1-page supplementary diabetic shoe wear educational material was reviewed and dispensed. "Keep your diabetes under control" stresses "sugar, blood pressure, and medication control, and nutrition and physical activity, and checking feet daily for cuts, blisters, sores, swelling, redness, or sore toenails." "Keep your skin and feet healthy" emphasizes the importance of checking feet daily, highlighting diabetic foot complications that can arise from neuropathy, poor circulation and dry skin, and the importance of supportive, protective, and accommodative shoewear and annual foot exams.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Investigator)
Primary purpose: Prevention
Keywords: Diabetes Mellitus; Diabetic Foot; Diabetic Peripheral Neuropathy; Diabetic Foot Education