bioihbjkbjnl/mcl2 huggingface dataset:
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Condition: Muscular Dystrophy, Duchenne; Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn
MeSH terms: Muscular Dystrophies; Muscular Dystrophy, Duchenne; Musculoskeletal Diseases; Muscular Diseases; Muscular Disorders, Atrophic; Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Genetic Diseases, X-Linked
Study type: Interventional
Phase: Phase 3

Summary: 
      Dystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which
      develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is
      important for maintaining normal muscle structure and function. Loss of dystrophin causes
      muscle fragility that leads to weakness and loss of walking ability. A specific type of
      mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy
      in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered,
      investigational drug that has the potential to overcome the effects of the nonsense mutation.
      The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability.
      The effect of ataluren on physical function, quality of life, and activities of daily living
      will be evaluated. This study will also provide additional information on the long-term
      safety of ataluren.
    

Detailed description: 
      This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled study to
      determine the efficacy and safety of ataluren in participants with nmDMD.
    

Intervention name(s): Ataluren; Placebo
Intervention type(s): Drug; Drug
Intervention description(s): Ataluren will be administered as per the dose and schedule specified in the arm.; Placebo will be administered as per the schedule specified in the arm.
Other intervention name(s): PTC124
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Duchenne muscular dystrophy; Dystrophinopathy; Nonsense mutation; Premature stop codon; Becker muscular dystrophy; DMD/BMD; PTC124; Ataluren
Postoperative Pain in Adolescent Idiopathic Scoliosis Surgery
Condition: Scoliosis; Adolescence
MeSH terms: Scoliosis; Pain, Postoperative
Study type: Interventional

Summary: 
      Scoliosis is a 3-dimensional, structural deformity of the spine. Idiopathic scoliosis is the
      most common type and it constitutes 75-80% of all scoliosis. Surgical methods are the most
      effective way to correct the deformity in patients who cannot achieve adequate improvement
      with supportive therapy. Adolescent idiopathic scoliosis surgeries are among the most
      invasive surgeries performed on children and adolescents. Large surgical incision and massive
      tissue damage cause severe postoperative pain. In this study, we aim to compare posterior
      instrumentation (PE) and vertebral body tethering (VBT) surgeries performed in adolescent
      idiopathic scoliosis patients in terms of anesthetic management and postoperative pain.
    


Intervention name(s): Vertebral Body Tethering
Intervention type(s): Procedure
Intervention description(s): VBT surgery is a surgery performed by thoracotomy in adolescent idiopathic scoliosis patients.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Constraint-Induced (CI) Movement Therapy for Progressive Multiple Sclerosis (MS)
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive; Sclerosis
Study type: Interventional
Phase: Phase 1

Summary: 
      This trial will evaluate whether progressive multiple sclerosis associated with hemiparesis
      may benefit from Constraint-Induced Movement Therapy.
    

Detailed description: 
      Constraint-Induced Movement Therapy was developed to treat stroke hemiparesis. Due to the
      similarity between multiple sclerosis and stroke in terms of chronic motor deficit, this
      trial will attempt to determine whether multiple sclerosis may benefit similarly from
      Constraint-Induced Movement Therapy.
    

Intervention name(s): Constraint-Induced Movement Therapy
Intervention type(s): Behavioral
Intervention description(s): Intensive practice with the hemiparetic arm for 30 contact hours
Other intervention name(s): CI Therapy
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: multiple sclerosis
Advanced MR Imaging of Osteoarthritis
Condition: Osteoarthritis
MeSH terms: Osteoarthritis
Study type: Observational

Summary: 
      The utility of conventional imaging in the development of new treatments for osteoarthritis
      (OA) is hindered by:

        1. Difficulty in non-invasively evaluating the initial response to potential new treatment
           options. OA typically develops over a long time period, so evaluating the efficacy of
           new treatment options over a timeframe relevant to experimental medicine studies is
           difficult.

        2. Inadequate methods of disease quantification and stratification. OA is a heterogeneous
           condition and identifying which subgroups of patients are most likely to benefit from
           new treatments is crucial.

      Novel magnetic resonance (MR) imaging acquisition and analysis techniques have the potential
      to solve these problems. However, their reliability needs to be fully established and
      knowledge of likely effect sizes is required to inform sample size considerations for further
      longitudinal/interventional studies.

      This study aims to help address these issues by:

        1. Calculating effect sizes for MR measurements to inform sample size calculations for
           future studies.

        2. Assessing the reliability of a multiparametric MR protocol for assessment of the knee
           joint.

      The investigators will examine a single knee of an initial 15 participants with MR.
      Participants will be drawn from two groups: (1) 10 participants aged 40-60 years old with
      clinical and x-ray features of OA and (2) 5 control subjects (matched to cases for age, sex
      and body mass index in a 1:2 ratio) who do not have clinical features of OA.

      Participants will undergo an initial (baseline) MR examination, followed by repeat MR
      examinations at approximately 1 month and 1 year following the baseline examination.

      This will allow us to assess both the reliability of our MR measurements and the expected
      progression in our MR measurements in OA subjects in the absence of any disease-modifying
      intervention.
    


Observational model: Case-Control
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        Group 1 will consist of 10 patients aged 40-60 who have presented to the Orthopaedic clinic
        with a painful knee, and have mild/moderate OA on plain radiographs (Kellgren-Lawrence
        grade 2-3).

        Group 2 will consist of 5 healthy volunteer controls, matched (1:2 ratio) to cases for age,
        sex and body mass index who do not have knee pain.
      
Biospecimen description: 
      Serum samples for cartilage turnover marker measurement
Treatment for Young Adults With Anorexia Nervosa
Condition: Anorexia Nervosa
MeSH terms: Anorexia; Anorexia Nervosa
Study type: Interventional

Summary: 
      Temple University is conducting a National Institute of Health funded research study designed
      to develop and refine a family-based treatment manual for young adults with Anorexia Nervosa
      as well as assess the feasibility of this out-patient psychotherapy.
    

Detailed description: 
      Anorexia Nervosa (AN) is a serious psychiatric illness that occurs in an estimated 0.5 to
      3.7% of women. The illness severely affects physical, emotional, and social functioning. The
      mortality rates associated with this severely disabling condition are higher than those for
      any other psychiatric disorder and substantially higher than those expected in the general
      population. Lower weight at presentation, longer illness duration, and alcohol abuse are
      associated with a higher risk of mortality.

      Treatment-outcome for adult AN is poor with a quarter of adults with AN having poor outcome.
      In AN, promising outcomes are seen in family-based treatment (FBT) for adolescent patients
      who present between the ages of 12 to 18 with a short duration of illness. A manualized
      version of FBT has now been tested in several studies and case series, showing that well over
      80% of participants had good or intermediate outcome at post-treatment. Despite its efficacy
      with adolescents, FBT has not been utilized with young adults.

      The purpose of this study is to develop and refine FBT to promote weight gain in young adults
      with Anorexia Nervosa.
    

Intervention name(s): Family Based Therapy for young adults
Intervention type(s): Behavioral
Intervention description(s): Individual and Group Therapy Sessions
Other intervention name(s): FBT; FBT-Y
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: AN
Atomoxetine for Treating Attention Deficit Hyperactivity Disorder in Young Children
Condition: Attention Deficit Disorder With Hyperactivity
MeSH terms: Hyperkinesis; Attention Deficit Disorder with Hyperactivity
Study type: Interventional
Phase: Phase 4

Summary: 
      This study will evaluate the effectiveness of atomoxetine in reducing the symptoms of
      attention deficit hyperactivity disorder (ADHD) in young children.
    

Detailed description: 
      Attention deficit hyperactivity disorder (ADHD) is one of the most common mental disorders in
      children. Children with ADHD often have impaired functioning in home and school and usually
      experience difficulty relating to peers. If left untreated, the disorder can have long-term
      adverse effects into adolescence and adulthood. Atomoxetine is a selective noradrenergic
      reuptake inhibitor that is FDA-approved for the treatment of ADHD in children, adolescents,
      and adults. Unlike most other medications for ADHD, atomoxetine is not a stimulant. Studies
      have shown that atomoxetine is effective in treating ADHD, but more information is needed on
      its effectiveness in young children. This study will evaluate the effectiveness of
      atomoxetine in reducing the symptoms of ADHD in young children.

      Participants in this double-blind study will be randomly assigned to receive either
      atomoxetine or placebo for 8 weeks. In addition, all children will receive parent training
      for the duration of the study. For the first 5 weeks, participants will report to the study
      site weekly for assessments of ADHD symptoms. Study visits will occur every other week for
      the remainder of the study.
    

Intervention name(s): Atomoxetine; Placebo; Parent Training
Intervention type(s): Drug; Drug; Behavioral
Intervention description(s): Participants will receive atomoxetine for 8 weeks up to a maximum of 1.8 mg/kg/day. Dosed once daily in capsule formulation.; Participants will receive placebo for 8 weeks. Dosed once daily, capsule formulation.; All children will receive parent training for the duration of the study.
Other intervention name(s): Strattera; Pill placebo; Psychosocial and educational intervention
Intervention MeSH terms: Atomoxetine Hydrochloride
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary purpose: Treatment
Keywords: ADHD; Atomoxetine
Relative Bioavailability Study With BMS-955176
Condition: Infection, Human Immunodeficiency Virus
MeSH terms: Acquired Immunodeficiency Syndrome; HIV Infections
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to select the best dose level (amount of drug given) and best
      formulation of the study drug (BMS-955176) to develop further.
    

Detailed description: 
      Primary Purpose: Other: This study will assess the bioavailability of BMS-955176 from a
      micronized crystalline (MC) tablet relative to a spray-dried dispersion (SDD) suspension.
      Assessment of additional experimental formulations relative to the micronized crystalline
      tablet will also be performed
    

Intervention name(s): BMS-955176
Intervention type(s): Drug
Intervention description(s): BMS-955176
Intervention MeSH terms: BMS-955176
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: None (Open Label)
Primary purpose: Other
Evaluation of Safety and Efficacy of Micro-coring Device for Treatment of Facial Wrinkles and Skin Laxity
Condition: Wrinkle; Facial and Neck Skin Laxity
MeSH terms: Cutis Laxa
Study type: Interventional

Summary: 
      A pilot study is designed to evaluate safety and efficacy of the Micro-Coring (MC) technology
      for treatment of facial wrinkles. MC device employs hollow coring needles that enable safe,
      rapid, and effective full-thickness small skin cores (200-500 microns in diameter) removal
      initiating a skin repair process through formation of new collagen and elastin fibers (skin
      rejuvenation).
    

Detailed description: 
      This is a prospective, multi-center, randomized, single blind, bilateral paired study
      evaluating the safety, tolerability and preliminary effectiveness of a micro-coring device
      for removal of excess skin using 2 needle gauges (22G, 24G,) at densities (percent of skin
      removed per 1cm2) of 2.5-10% in subjects with mid- and lower- face skin laxity manifested by
      moderate-to-severe mid and lower cheek wrinkles, deepening of the nasolabial folds at rest;
      prominence of marionette lines at rest; downturn of the oral commissures at rest, sagging of
      the skin at the jawline at rest. Subjects are blinded to needle gauge and density.

      There will be two cohorts of subjects: one group of subjects will receive a single treatment
      and the second - multiple treatments. All subjects will be followed for 180 days with several
      intermediate visits at 3, 7, 30, 60, and 90-days.
    

Intervention name(s): micro-excisional skin removal with coring needle
Intervention type(s): Device
Other intervention name(s): micro-coring, micro-excisional skin removal
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: skin rejuvenation; wrinkle removal; skin laxity reduction
HMPL-523 Food Effect and Proton Pump Inhibitor Study
Condition: Relapsed or Refractory Lymphoma
Study type: Interventional
Phase: Phase 1

Summary: 
      A Phase 1, Open-label, 4-Period, Randomized 6-Sequence Study to Evaluate the Effect of Food
      and Rabeprazole, a Proton Pump Inhibitor, on the Pharmacokinetics of HMPL-523 in Healthy
      Volunteers
    

Detailed description: 
      This study will be a single-center, open-label, 4-period, randomized,6-sequence study
      conducted with 24 healthy male or female subjects. The study will consist of a Screening
      Phase (Screening and Day -1), a Treatment Phase (Periods1, 2, 3,and 4), and an End of Study
      (EOS) Phase. Screening must occur within 28 days before the first study drug administration.
      There will be a washout of at least 7days between 4 administrations of HMPL-523.Subjects in
      Periods 1, 2, and 3 will be randomized into 1 of 6 treatment sequences, with all subjects
      then receiving the same treatment in Period 4.
    

Intervention name(s): HMPL-523; Rabeprazole
Intervention type(s): Drug; Drug
Intervention description(s): 700 mg HMPL-523 will be administered by mouth once daily on Day 1, Day 8, Day 15, and Day 26; 40 mg of rabeprazole will be administered by mouth once daily in the morning from Day 20 to Day 26
Intervention MeSH terms: Rabeprazole
Allocation: Randomized
Intervention model: Crossover Assignment
Intervention model description: Open-label, 4-Period, Randomized 6-Sequence Study
Masking: None (Open Label)
Primary purpose: Other
Evaluation of Lofexidine for Treatment of Opioid Withdrawal - 7
Condition: Opioid-Related Disorders; Substance-Related Disorders
MeSH terms: Substance-Related Disorders; Opioid-Related Disorders
Study type: Interventional
Phase: Phase 1

Summary: 
      The purpose of this study is to determine the safety of lofexidine in the treatment of opiate
      withdrawal. Preliminary data will also be obtained to assess the ability of lofexidine to
      alleviate opiate withdrawal signs and symptoms.
    


Intervention name(s): Lofexidine
Intervention type(s): Drug
Intervention MeSH terms: Lofexidine
Primary purpose: Treatment
The Impact of Polycystic Ovary Syndrome (PCOS) on Quality of Life and Healthcare Beliefs
Condition: Polycystic Ovary Syndrome
MeSH terms: Polycystic Ovary Syndrome; Syndrome
Study type: Observational

Summary: 
      Women with Polycystic Ovary Syndrome (PCOS) between the age of 18 and 55 are being recruited
      to participate in an online survey to determine the impact of PCOS on quality of life and
      interaction with the healthcare system. This online survey will take 15-20 minutes to
      complete. Participants will receive no direct benefit, but the information provided will
      contribute to the knowledge of this condition and the development of improved healthcare for
      women with PCOS. For more information or to participate in this study, please visit
      http://pcossurvey.vze.com
    


Sampling method: Non-Probability Sample
Study population: 
        Women with Polycystic Ovary Syndrome (PCOS) between the age of 18 and 55
      
Keywords: PCOS; polycystic ovary syndrome; Polycystic ovarian syndrome; infertility; ovulation
Participation Restrictions in Breast Cancer Survivors
Condition: Breast Cancer
MeSH terms: Breast Neoplasms
Study type: Interventional

Summary: 
      This project will develop and preliminarily evaluate an intervention to assist young-middle
      adult rural breast cancer survivors to overcome functional impairments related to their
      cancer treatment. Younger breast cancer survivors (below age 60) have ongoing family, social
      and vocational responsibilities and recreational interests and are at increased risk of
      developing long-term difficulties performing these valued activities (participation
      restrictions). Rural cancer survivors are medically underserved and due to distance and
      geographic isolation face significant barriers to accessing traditional rehabilitation
      services. Alternative rehabilitation approaches are sorely needed for this population. Based
      on previous research with cancer and other medical populations a working draft of a treatment
      manual using a telephone-delivered Behavioral Activation and Problem Solving (BA/PS)
      intervention has been designed. Prior to testing the intervention in a randomized clinical
      trial, additional work must be completed. The specific aims of this study are to a. refine
      the BA/PS treatment manual, b. develop and apply treatment integrity measures for the BA/PS
      manual, c. assess participation restrictions and associated outcomes, d. preliminarily assess
      the immediate and maintenance effects of BA/PS, and e. examine mediators and moderators of
      BA/PS effects based on our "Self Regulation" model of functional recovery. 188 young-middle
      adult breast cancer survivors will be screened for participation restrictions following
      cancer treatment and 40 survivors with participation restrictions will receive BA/PS
      delivered by phone. BA/PS participants will be assessed for treatment effectiveness and
      interviewed regarding their experiences during treatment to provide information for refining
      the manual. The long-term objective of this line of research is to develop effective and
      feasible treatments for the medical and psychosocial consequences of cancer and its treatment
      in medically underserved cancer survivors (e.g., rural populations).
    


Intervention name(s): Behavioral activation/problem-solving (BA/PS)
Intervention type(s): Behavioral
Intervention description(s): Six weekly telephone sessions to train participant in BA/PS procedures
Intervention model: Single Group Assignment
Masking: None (Open Label)
PEG-rhG-CSF in Patients With Breast Cancer Receiving Chemotherapy to Prevent Neutropenia
Condition: Breast Cancer
MeSH terms: Breast Neoplasms; Neutropenia
Study type: Interventional
Phase: Phase 4

Summary: 
      The purpose of this study is to estimate the efficacy and safety of PEG-rhG-CSF in patients
      with breast cancer receiving chemotherapy.
    


Intervention name(s): PEG-rhG-CSF
Intervention type(s): Drug
Other intervention name(s): pegfilgrastim
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Prevention
Keywords: PEG-rhG-CSF neutropenia
A Biobehavioral Model of Diabetes Risk in Chinese Immigrants
Condition: Diabetes Mellitus, Type 2
MeSH terms: Diabetes Mellitus; Diabetes Mellitus, Type 2
Study type: Observational

Summary: 
      This prospective, longitudinal study of Chinese American immigrants will examine whether
      psychosocial factors (e.g., acculturative stress, social isolation, discrimination) are
      associated with markers of type 2 diabetes risk over time, and whether such associations are
      mediated by inflammatory pathways.
    

Detailed description: 
      It is now well-documented that immigration to the US leads to increased risk for various
      chronic diseases, including type 2 diabetes. The increased diabetes risk observed among
      Chinese immigrants has been primarily attributed to changes in diet and weight gain following
      immigration, but these changes can only partially explain disparities in disease risk. Models
      of immigrant health suggest that the stress of adapting to life in a new country has a
      considerable impact on physical health. However, few studies have considered the psychosocial
      impact of immigration upon biomarkers of health and disease risk. Therefore, this
      longitudinal study of US Chinese immigrant men and women will examine: (1) whether
      psychosocial factors (e.g., acculturative stress, social isolation, discrimination) are
      associated with markers of type 2 diabetes risk over time; and (2) whether the association
      between psychosocial factors and diabetes risk markers is mediated by inflammatory pathways.
    

Observational model: Cohort
Time perspective: Prospective
Sampling method: Non-Probability Sample
Study population: 
        600 foreign-born Chinese Americans
      
Biospecimen description: 
      Serum, plasma, and hair samples will be obtained
Comparison of Two Exercise Programmes in Patients Undergoing Hemodialysis
Condition: End Stage Renal Disease
MeSH terms: Kidney Failure, Chronic
Study type: Interventional

Summary: 
      Although exercise training of haemodialysis patients is associated with generally positive
      outcomes few haemodialysis units routinely offer intradialytic exercise therapy. This is
      often related to financial cost and/or staffing limitations. Home-based programs could be an
      alternative means of providing exercise benefits without associated implementation costs The
      aim of this study was to compare the effects of 4 weeks intradialytic versus home based
      exercise for haemodialysis patients regarding adherence and strength gains.
    


Intervention name(s): Exercise programmes
Intervention type(s): Other
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Investigator)
Primary purpose: Treatment