bioihbjkbjnl/mcl2 huggingface dataset:
Adherence and Outcomes in Functional Constipation With a Constipation Action Plan
Condition: Adherence, Medication; Functional Constipation; Pediatric ALL; Survey, Family Life
MeSH terms: Constipation
Study type: Interventional

Summary: 
      We will be assessing for improvement in reported symptoms as well as reported quality of life
      in pediatric patients with functional constipation using a constipation action plan and an
      adherence log.
    

Detailed description: 
      Functional Constipation is one of the most common chief complaints for both the general
      pediatrician and the pediatric gastroenterologist. Treatment for this disorder is
      multifactorial, and usually involves different medications combined with behavioral
      techniques, depending on the severity of the underlying constipation. Studies have shown that
      medication compliance is one of the strongest predictors of successful treatment, but
      maintaining good medication adherence at home is uncommon for a variety of reasons. In this
      study, the investigators are aiming to improve home medication adherence for functional
      constipation to improve treatment outcomes. The investigators will be administering surveys
      for all enrolled participants to determine the child's and family's overall quality of life
      related to functional constipation. For the treatment group, the investigators will be
      providing tools to help with medication adherence. This will include a daily log to determine
      symptom severity, along with an "action plan" with instructions for how to adjust medicines,
      if necessary, depending on symptoms. By empowering families with this knowledge and
      medication roadmap, the investigators hypothesize that there will be overall symptomatic
      improvement in functional constipation, along with improved quality of life for both the
      patient and their family. The investigators will measure adherence (by reviewing symptom log
      and action plan at 2-month and 4-month follow up appointments.) with the treatment group. The
      investigators will be measuring overall quality of life and symptom improvement using a
      validated pediatric constipation quality of life survey (PedsQL GI) with both the treatment
      group and the control group at follow up appointments.
    

Intervention name(s): Action Plan
Intervention type(s): Behavioral
Intervention description(s): See arm description.
Other intervention name(s): Adherence Log
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Response Surface Pathway Design With Two Interventional- and One Response Variable in Estimating Minimum Efficacy Dose
Condition: GERD
Study type: Interventional

Summary: 
      Response Surface Pathway (RSP) design was previously developed for dose-finding studies with
      one interventional and one response variable, but RSP to higher dimensions is requested. The
      aim of this study is to introduce and evaluate the RSP design with two interventional and one
      response variable exemplified by estimating Minimum Efficacy Dose (MED) of Osteopathic Manual
      Therapy (OMT) in treatment of gastroesophageal reflux disease (GERD).
    

Detailed description: 
      15 GERD patients, divided in three design-level with three, five and seven patients. The
      study was performed as a two-dimensional randomized, between-patient RSP designed multicenter
      study with two interventional- and one response variable. The interventional variables
      "Number of OMT's" and "Treatment Interval" with common response variable formed two
      independent one-dimensional randomized between-patient RSP studies. The response variable was
      the percent reduction in sum of the five GERD score from baseline. Three GERD patients were
      allocated on the first design level given six OMT with five days' interval. Based on results
      obtained in the first and second design level, five patients were included to the second
      design level and seven to the third.
    

Intervention name(s): Osteopatic Manual Therapy (OMT)
Intervention type(s): Procedure
Intervention description(s): OMT consisting of following interventions; thoracic spine and diaphragm mobilisation, traction of the cardia and posture correction.
Allocation: Randomized
Intervention model: Single Group Assignment
Intervention model description: The study is a RSP designed multicenter study with two interventional- and one response variable. The two-interventional variable were Number of OMTs and Interval in days between each treatment. The GERD variables were degrees of Heartburn, Chest pain, Acid in mouth, Epigastric pain and "Thoracic pain". The response variable were percent reduction in sum of the GERD variables from baseline in 4 categories. Each of the two RSP designs consisted of three dose levels. Three GERD patients were allocated to the first design level given six OMT with five days' interval. Based on the results obtained from the first design level, five patients were subsequently included to the second design level. The number of OMT's and interval between each treatment was separately determined. Seven patients were included to the third design level and given number of OMT's and interval between treatments calculated based on results obtained from the second design level.
Masking: None (Open Label)
Primary purpose: Basic Science
Keywords: Two-dimensional randomized response surface pathway design; Osteopathic Manual Therapy; OMT in treatment of GERD; Dose-response
Efficacy of Pregabalin Versus Combined Pregabalin and Milnacipran in Fibromyalgia.
Condition: Fibromyalgia, Primary
MeSH terms: Fibromyalgia; Myofascial Pain Syndromes
Study type: Interventional

Summary: 
      Diagnosis of fibromyalgia is complex and treatment options are limited. Pharmacological
      management of fibromyalgia is mainly centered on the central nervous system. In particular
      there is robust evidence for the use of tricyclic antidepressants (e.g., amitriptyline),
      anti-convulsants such as gabapentin or pregabalin and agents from the serotonin
      norepinephrine reuptake inhibitor (SNRI) family such as milnacipran. Aim of the work: To
      compare the efficacy of pregabalin agent (averopreg) alone versus combined pregabalin and
      serotonin norepinephrine reuptake inhibitor (milnacipran) in the management of fibromyalgia.
    

Detailed description: 
      Subjects: The study will include 50 patients diagnosed as having fibromyalgia according to
      the ACR 2010 Fibromyalgia diagnostic criteria.

      Methods: Patients will be classified into two groups:

      1) Group 1: Patients will receive pregabalin as a monotherapy and the doses will be
      administered according to the treatment recommendations for fibromyalgia in the package
      insert, starting by 50mg twice daily increasing to 100 mg twice daily within 1 week based on
      efficacy and tolerability.

      7) Group 2: Patients will receive combined pregabalin and milnacipran and the doses will be
      administered according to the treatment recommendations for fibromyalgia in the package
      insert, starting by 50mg twice daily increasing to 100 mg twice daily within 1 week for the
      pregabalin and starting by 12.5 mg increasing gradually to reach 100 mg daily of milnacipran
      based on efficacy and tolerability.
    

Intervention name(s): Pregabalin 300mg; Combined pregabalin 300mg and milancipran 100mg
Intervention type(s): Drug; Drug
Intervention description(s): Group 1: will receive pregabalin as a monotherapy. Pregabalin will be administered according to the treatment recommendations for fibromyalgia in the package insert [Pfizer Inc., 2012] starting by 50 mg twice daily increasing to 300 mg twice daily according to the efficacy and tolerability for 3 months.; Group 2: will receive a combined pregabalin and milancipran. Pregabalin will be administered as group 1, while milancipran will be administered according to the treatment recommendations for fibromyalgia, starting by 50 mg once daily for 1 week then increased to reach 100 mg daily (50 mg twice daily) based on efficacy and tolerability for 3 month.
Intervention MeSH terms: Pregabalin
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Study for HER2-Overexpressed MBC Patients Treated by Cipterbin® Plus vinorelbinE
Condition: Progress-free Survival; Overall Response Rate; Adverse Events
Study type: Observational

Summary: 
      Breast cancer is the most common malignant disease and the most frequent causes of cancer
      mortality in females worldwide. The situation is same as the world in China. Trastuzumab has
      been proved valuable treatment for HER2-positive breast cancer patients. Cipterbin® is
      developing by Shanghai CP Guojian Pharmaceutical Co.Ltd.Now we carried on the phase III trial
      in order to prove Cipterbin® 's efficacy and safety.
    


Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        HER2-positive metastatic breast cancer patients
Efficacy of the CO-OP Approach With Brazilian Children With Developmental Coordination Disorder
Condition: Developmental Coordination Disorder
MeSH terms: Motor Skills Disorders
Study type: Interventional

Summary: 
      Children with Developmental Coordination Disorder (DCD) have difficulties performing daily
      activities which reflects negatively on participation, impacting their lives. To date, there
      are a number of interventions to improve performance of these children on activities they
      want or need to. In Brazil, there is little research on the efficacy of such approaches. Our
      main objective was to start a set of studies to examine the effects of the Cognitive
      Orientation to Daily Occupational Performance Approach (CO-OP Approach) protocol on
      occupational performance and satisfaction of Brazilian children who have DCD; to examine
      whether children were able to transfer strategies and skills learned during CO-OP to
      untrained goals.
    

Detailed description: 
      Background. Children with Developmental Coordination Disorder (DCD) have difficulties
      performing daily activities which reflects negatively on participation, impacting their
      lives. Objectives. To examine the effects of the Cognitive Orientation to Daily Occupational
      Performance Approach (CO-OP Approach) protocol on occupational performance and satisfaction
      of Brazilian children who have DCD; to examine whether children were able to transfer
      strategies and skills learned during CO-OP to untrained goals. Methods. A pre-post group
      comparison design with eight boys aged 6-10 years old. Children participated in 12 CO-OP
      sessions with their parents twice a week, with an extra session added to the protocol for
      parents´ orientation. The Canadian Occupational Performance Measure and the Performance
      Quality Rating Scale were used as outcome measures.
    

Intervention name(s): Cognitive Orientation to Daily Occupational Performance
Intervention type(s): Behavioral
Intervention description(s): In CO-OP Approach, therapists use mediational techniques to teach cognitive strategies - GOAL, PLAN, DO, CHECK - to improve occupational performance in goals chosen by the children in collaboration with them and their parents.
Other intervention name(s): CO-OP Approach
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: child; goal; intervention; motor skills disorder; occupational therapy
Phase 2 Study of Triheptanoin (UX007) for the Treatment of Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
Condition: Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
MeSH terms: Carbohydrate Metabolism, Inborn Errors; Syndrome
Study type: Interventional
Phase: Phase 2

Summary: 
      The primary objectives of the study are to evaluate the efficacy of UX007 compared to placebo
      as measured by the reduction from randomization to Week 8 in frequency of seizures and to
      evaluate the safety of UX007 via adverse event (AE) rates, laboratory values, and
      electrocardiogram (ECG).
    


Intervention name(s): UX007; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): oral liquid; oral liquid
Other intervention name(s): C7 oil; triheptanoin; glycerol triheptanoate; glycerol trienanthate; 1, 2, 3-trienanthoylglycerol; trienanthin; 2,3-di(heptanoyloxy)propyl heptanoate
Intervention MeSH terms: Glycerol
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Glucose Transporter Type 1 Deficiency Syndrome Glut1
Multiple Dose Comparison of the Effect of Two Dose Combinations of Tipranavir/Ritonavir (TPV/RTV), on the Pharmacokinetic Characteristics of Efavirenz (Sustiva®) in Healthy Adult Volunteers
Condition: Healthy
Study type: Interventional
Phase: Phase 1

Summary: 
      The objective of this study was to characterize the effects of two dose combinations of
      tipranavir/ritonavir (TPV 500 mg/RTV 100 mg and TPV 750 mg/RTV 200 mg), administered daily
      and BID, on the pharmacokinetics of efavirenz (EFV), 600 mg daily
    


Intervention name(s): EFV; TPV/RTV - low dose; TPV/RTV - high dose
Intervention type(s): Drug; Drug; Drug
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Intervention and Outcomes in Duarte Galactosemia
Condition: Duarte Galactosemia
MeSH terms: Galactosemias
Study type: Observational

Summary: 
      The purpose of this study is to learn about Duarte galactosemia (DG). This study will examine
      the possible effects of Duarte galactosemia (DG) in children, and determine whether dietary
      exposure to milk in infancy or early childhood is associated with developmental outcomes of
      school-age children with Duarte galactosemia (DG).
    

Detailed description: 
      Duarte galactosemia (DG) is an autosomal recessive genetic condition characterized by partial
      loss of galactose-1-phosphate uridylyltransferase (GALT), which results in partially impaired
      metabolism of the sugar, galactose, which is abundant in milk and also found at lower levels
      in many other foods. There is currently no consensus on long-term outcome prognosis for
      infants with Duarte galactosemia (DG) and some studies suggest these children might be at
      increased risk for developmental difficulties later in childhood. There is also no conclusive
      data on whether children with Duarte galactosemia (DG) might benefit from dietary restriction
      of galactose. The investigator will be assessing whether 6-12 year old children with Duarte
      galactosemia experience developmental disorders relative to controls, and if so, whether
      dietary exposure to milk in infancy or early childhood is associated with developmental
      outcomes in this patient population.
    

Observational model: Case-Control
Time perspective: Prospective
Sampling method: Probability Sample
Study population: 
        Study participants will be recruited in multiple collaborating states from records of
        infants born in that state who were diagnosed with Duarte Galactosemia detected by newborn
        screening (NBS). The current list of collaborating states includes: AL, CA, GA, IA, IL, MI,
        MO, NC, NJ, OR, SC, TX, WA, and WI but this list may change.
      
Biospecimen description: 
      DNA isolated from saliva samples
    
Keywords: Galactose-1-phosphate uridylyltransferase deficiency; Cognitive disorders
Effect of Baby Wipe Solutions on Fecal Enzyme-induced Irritation
Condition: Skin Health
Study type: Observational

Summary: 
      This is a single-center, prospective, single-blind pilot study to determine baby wipe
      formulations on an adult dermatitis model.
    

Detailed description: 
      This is a single-center, prospective, single-blind pilot study to determine baby wipe
      formulations on an adult dermatitis model.

      Biophysical measurements will be taken at each test sites.
    

Intervention name(s): Artificial Fecal Enzyme
Intervention type(s): Other
Intervention description(s): Artificial Fecal enzyme Trypsin/Chymotrypsin mixture
Observational model: Other
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        The study population consists of 24 Caucasian females, age 18-40, in good general health
        and free from dermatological conditions in the testing area.
Drug Interaction Study With Proton Pump Inhibitor
Condition: HIV Infections
MeSH terms: HIV Infections
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this clinical research study is to assess the effect of omeprazole at 20 mg on
      the pharmacokinetics of atazanavir administered as atazanavir with ritonavir relative to
      atazanavir or atazanavir/ritonavir in the absence of omeprazole in healthy subjects.
    


Intervention name(s): Atazanavir; Atazanavir/Ritonavir; Atazanavir/Ritonavir+Omeprazole; Atazanavir/Ritonavir+Omeprazole; Atazanavir; Atazanavir/Ritonavir; Atazanavir/Ritonavir+Omeprazole; Atazanavir/Ritonavir+Omeprazole
Intervention type(s): Drug; Drug; Drug; Drug; Drug; Drug; Drug; Drug
Intervention description(s): Capsules, Oral, 400 mg, once daily, 6 days.; Capsules/capsules, Oral, 300/100 mg, once daily, 10 days.; Capsules/capsules + Capsules, Oral, 300/100 mg + 20 mg, once daily in PM + once daily in AM, 7 days.; Capsules/capsules + Capsules, Oral, 400/100 mg + 20 mg, once daily in PM + once daily in AM, 7 days.; Capsules, Oral, 400 mg, once daily, 6 days.; Capsules/capsules, Oral, 300/100 mg, once daily, 10 days.; Capsules/capsules + Capsules, Oral, 400/100 mg + 20 mg, once daily in AM + once daily in AM, 7 days.; Capsules/capsules + Capsules, Oral, 400/100 mg + 20 mg, once daily in AM + once daily in PM, 7 days.
Other intervention name(s): Reyataz; Reyataz; Reyataz; Reyataz; Reyataz; Reyataz; Reyataz; Reyataz
Intervention MeSH terms: Ritonavir; Atazanavir Sulfate; Omeprazole
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Keywords: Protease inhibitor
Intracoronary Transplantation of Bone Marrow Derived Mononuclear Cells in Pediatric Cardiomyopathy
Condition: Cardiomyopathy
MeSH terms: Cardiomyopathies
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      According to the high morbidity and mortality of idiopathic Dilated CardioMyopathy (IDCM) in
      pediatric, new modality of treatment is emerging. There are some case reports of
      administration of stem cell therapy. The investigators design the first randomized clinical
      trial in this setting. The investigators enroll 32 pediatric IDCM patients in two groups (16
      pts. in each group including cell therapy and control). The investigators assess the safety
      and efficacy of intracoronary transplantation of autologous bone marrow derived mononuclear
      cells in this patients compared to control group.
    

Detailed description: 
      Dilated cardiomyopathy as the most common form of cardiomyopathy is a rare but
      life-threatening disorder in children. The primary cause of nearly 37 % of children with DCM
      was unknown at diagnosis. Despite of the developing in the medical and surgical treatment
      during the past several decades, standard treatments (including Digitalis, diuretics,
      inhibitors of ACE, beta blockers, antiplatelet drugs and treatments Antiarrhythmic), may
      stabilize the condition, but will not restore heart function to its previous condition.
      Therapy remains complex and expensive. For some not all children the heart transplantation is
      only option and mortality continues to be high, also. Stem cell and cell-based therapies
      offer an innovative approach to reverse cardiac structure and function towards normal,
      possibly reducing the need for aggressive therapies and cardiac transplantation. According to
      the inclusion and exclusion criteria of trial, 32 patients with left ventricular ejection
      fraction less than 45% who resistance to the standard medical therapy were randomly allocated
      in 2 groups including BM-derived mononuclear (n=16) and control (n=16). Only the MNC group
      underwent the bone marrow aspiration and intracoronary injection. The investigators followed
      all of patients at 2 weeks, 1, 2, 4 and 6 months after transplantation for cell therapy group
      or registration for placebo by physical examination, laboratory tests and imaging such as
      echocardiography, CXR and CMR.
    

Intervention name(s): Mono Nuclear Cell (MNC) transplantation
Intervention type(s): Biological
Intervention description(s): Intracoronary administration of autologous bone marrow derived mononuclear cells
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Investigator)
Primary purpose: Treatment
Keywords: Dilated cardiomyopathy pediatric heart failure cell therapy