cyrilzakka/clinical-trials huggingface dataset:
Energy Balance and Weight Gain With Ivacaftor Treatment

Ivacaftor is a novel, FDA approved new therapy that addresses Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunctions in subjects with Cystic fibrosis (CF) and "gating mutations".

The primary aim is to determine the mechanism(s) for weight gain in participants whom Ivacaftor treatment was initiated based on clinical indications by CF Care Team. This longitudinal study will assess in detail energy expenditure, weight gain, body composition, and lung function in 24 subjects ≥6 years old with CF with a gating mutation before treatment and after three months treatment with Ivacaftor. All subjects will be seen at the Children's Hospital of Philadelphia's Clinical Translational Research Center.

Cystic fibrosis (CF) is a genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR), a chloride channel. Most CF mutations either reduce the number of CFTR channels at the cell surface (synthesis or processing mutations) or impair channel function (gating or conductance mutations). Ivacaftor (Kalydeco, VX-770) is a novel, FDA approved new therapy that addresses CFTR dysfunctions in subjects with CF and "gating mutations", specifically; it potentiates CFTR channel function. For mutations like G551D that permit CFTR expression at the cell membrane but compromise its activity, Ivacaftor increases the probability that the channel is open and active. In previous randomized, double-blind, placebo controlled trials, Ivacaftor treatment resulted in clinically significantly improvements in pulmonary function, weight and body mass index (BMI), and significant decreases in sweat chloride reflective of increased CFTR activity. The improvements in lung function and weight occurred over the first 8 weeks of treatment, plateaued and were sustained over the 48 weeks of the trial. The mechanism for the rapid and sustained weight gain is not known. Several mechanisms are considered in this proposal which may result in improved energy balance and energy utilization, and weight gain. These include decreased resting energy expenditure, increased energy and fat absorption from the gut, improved pancreatic enzyme and pH secretion, and increased energy intake. Improvements in weight and BMI status are expected to result from this improvement in energy balance and utilization, with potential beneficial effects on muscle mass and function and quality of life.

Eligibility Criteria:
Inclusion Criteria:

* Cystic fibrosis with one or two CFTR gating mutations
* Age: 6 years and older
* A clinical decision has been made for the subject to start Ivacaftor treatment
* In usual state of good health
* Family and subject commitment to the 3-month study protocol with two, 3-4 day visits to CHOP

Exclusion Criteria:

* FEV1 \< 40% predicted
* Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A
* Pregnancy or breast feeding
* Other illness affecting growth or nutritional status
* Subjects receiving total parenteral nutrition
A Phase 2b/3 Study of TPX-115 on Partial-thickness Rotator Cuff Tear

Rotator cuff tear is one of the most common shoulder diseases and conservative treatment is commonly used for tears involving ≤50% of tendon thickness. Since conventional conservative treatments are not fundamental to repair tendon tissue, there is a growing need of new therapy to improve structural outcome. This study assesses the safety and efficacy of allogeneic fibroblasts on partial-thickness rotator cuff tear. The primary outcome is change in Constant Score (CS) at 24 weeks after TPX-115 injection. Secondary outcomes include changes from baseline in CS, Visual Analogue Score (VAS) pain score, American Shoulder and Elbow Surgeons (ASES) score, Quick Disabilities of the Arm, Shoulder and Hand (Quick-DASH), Simple Shoulder Test (SST), and functional evaluations including Range of Motion (ROM) at 4, 12, 24 and 52 weeks after administration and structural evaluation using MRI at 24 and 52 weeks after injection.


Eligibility Criteria:
Inclusion Criteria:

1. Be 19 years of age or older.
2. Have partial-thickness rotator cuff tear, ≤50% of tendon thickness or of Ellman grade II assessed by MRI.
3. Have unilateral shoulder pain, muscle weakness and limited active range of motion lasting more than 3 months despite conservative treatment
4. VAS pain score ≥4 at screening.
5. Understand fully the study and voluntarily sign the informed consent for participation in the study.

Exclusion Criteria:

1. Regardless of partial-thickness rotator cuff tear, have full-thickness rotator cuff tear confirmed by MRI.
2. Have been treated with the following

   * Have had painkiller within 1 week prior to screening visit.
   * Have had received systemic steroid or immunosuppressive agents within 4 weeks prior to screening visit.
   * Have had subacromial or intra-articular injections on the affected shoulder within 3 months prior to screening visit.
   * Have shoulder surgery on the rotator cuff tear or had received drug that included growth factor, within 6 months prior to screening visit.
3. Have been diagnosed with the following diseases.

   * Inflammatory joint diseases
   * Other shoulder diseases which may cause shoulder pain or functional disorder
   * Autoimmune diseases
   * Active hepatitis B or C
   * HIV Ab positive
   * Malignant tumors within the last 5 years
   * Coagulopathy
   * Genetic disorders related to fibroblasts of collagen
   * Other serious diseases deemed to affect the results of the study
4. Have allergies to bovine proteins or gentamicin.
5. Be pregnant, breastfeeding, planning pregnancy or unwilling to use of contraceptive suggested in this study.
6. Have participated in other clinical trials and received investigational agents within 4 weeks of this study.
7. Be deemed inadequate for the study by investigators.
Efficacy and Safety of Growth Hormone Treatment in Children Small for Gestational Age

This trial is conducted in Europe. The aim of this clinical trial is to evaluate the height gain during 12 months of growth hormone treatment in children born small for gestational age due to intrauterine growth retardation.


Eligibility Criteria:
Inclusion Criteria:

* Born small for gestational age (SGA) due to intrauterine growth retardation (IUGR) defined as birth length and/or weight below P10
* Chronological age at least 4 years old until bone age maturation of maximum 12 years for girls and maximum 13,5 for boys at time of inclusion in the study
* Insufficient catch-up growth (height lesser than or equal to -2.5 SDS for chronological age
* Normal response to GH stimulation test (greater than 10 ng/mL)

Exclusion Criteria:

* Diabetes
* Growth retardation associated with infections severe chronic diseases (including chromosomal anomaly or nutritional disorders)
* Treatment with any medical product (anabolic drugs, sex steroids, etc.) which may interfere with GH effects
* History or presence of severe disease that could interfere with GH treatment/participation in the trial, e.g. active malignancy
* Previous or ongoing growth hormone therapy
Safety And Efficacy Of Celecoxib Versus Sodium Diclofenac In The Treatment Of Acute Low Back Pain

To evaluate the safety and efficacy of celecoxib versus sodium diclofenac in subjects with acute low back pain


Eligibility Criteria:
Inclusion criteria:

* Aged between 18 and 65 years
* Acute low back pain that falls into 1st or 2nd class of 'Quebec Task Force' classification of moderate to severe intensity (\>50 mm in the VAS)
* Acute low back pain onset \<72 hours prior to study inclusion and \>6 weeks after the last acute low back pain episode

Exclusion criteria:

* Scoliosis or known history of inflammatory arthritis, chronic pain, metastasis, Paget's disease, or any other disease that cause pain
* Low back pain from major trauma or visceral disorder
* Esophageal ulcers, gastric or duodenal ulcers or bleeding within 30 days prior to being administered study drug
Efficacy Studies of Corticosteroid Therapy in Community-Acquired Pneumonia

The purpose of this study is to determine the efficacy of addition of corticosteroid therapy to antibiotics in patient admitted with Community-acquired pneumonia.

The hypothesis is: Prednisolone in combination with antibiotic treatments is effective in improving clinical outcome in patients hospitalized with CAP.

Community-acquired pneumonia (CAP) is a acute illness with a considerable morbidity and mortality, especially patients with severe CAP. In the past decennia, in spite of many investigations, little reduction is seen in morbidity an mortality. Corticosteroids have a immune-modulation effect, which is not completely elucidated. Most likely the immune modulation effect is due to down-regulation of pro-inflammatory cytokines. The use of corticosteroids next to antibiotics in CAP could lead to shorter time to clinical stability, length of stay and costs.

Comparison: Hospitalized patients with CAP treated with antibiotics and prednisolone versus hospitalized patients with cap treated with antibiotics and placebo

Eligibility Criteria:
Inclusion Criteria:

Clinical symptoms of community-acquired pneumonia:

* Fever, cough, sputum, pleural pain, dyspnoea
* Radiological symptoms of pneumoniä

Exclusion Criteria:

* Any conditions wich requires corticosteroid therapy.
* Pregnancy of lactation
* Malignancy
* Immune-compromised patients (eg chemotherapy or AIDS)
* Pre-treatment with macrolide for \>24 hours
Reduction of Falls in the Elderly - Parkinson's Disease

The REFINE-PD study is a controlled trial embedded within a larger cluster controlled study (the IMPACT study). The study aims to investigate the efficacy of a multifactorial falls prevention program for patients with Parkinson's Disease (PD). This program contains PD-specific elements (e.g., optimizing dopaminergic therapy), plus a generic falls prevention program. The intervention will be tailored to each individual's specific risk profile for falls, as identified during detailed baseline examination.

Falls and postural instability are common complications of advanced Parkinson's Disease (PD). Falls in PD often have devastating consequences, leading to a poor overall prognosis. In addition, falls in PD are associated with substantial medical expenses due to treatment of injuries and nursing home admission. The risk of falls in older PD patients is aggravated by "generic" age-related factors, such as sedative medication or poor vision. Observations on elderly persons without PD suggest that a multifactorial prevention program might be more effective. We propose to investigate the effectiveness of a multifactorial prevention program aimed at the prevention of falls in PD, which is based on disease-specific treatment strategies with demonstrated efficacy in PD, as well as prevention strategies with proven effectiveness for the general elderly population.

In this study an individualised multifactorial program aimed at the prevention of falls in PD containing PD-specific elements and a generic falls prevention program will be compared to usual care.

Eligibility Criteria:
Inclusion Criteria:

* Patients with idiopathic Parkinson's Disease, diagnosed according to the Brain Bank criteria of the UK Parkinson's Society
* Regular control by the neurologist
* Living independently in the community
* Able to complete the trial questionnaires
* An increased risk for falling, defined as:

  * at least one fall in the preceding 12 months OR
  * recurrent (monthly) near falls in the preceding 12 months OR
  * fear of falling OR (iv) avoidance of activities due to fear of falling OR a combination of these findings

Exclusion Criteria:

* Atypical parkinsonian syndromes
* Hoehn and Yahr stage 5
* Severe cognitive impairment (MMSE \< 24)
* Severe co-morbidity (e.g., cancer)
* Planned surgical procedure for PD within the intervention period
* Patients who have already visited the Multidisciplinary Assessment Center
Anti-Xa Guided Dosing of Low Molecular Weight Heparin for Prevention of Venous Thromboembolism Following Traumatic Injury: a Multicentre Pilot Randomized Trial

This multicentre pilot trial will assess the feasibility of a full-scale, randomized trial to determine whether bloodwork guided dosing of blood thinners reduces the risk of clotting in high-risk trauma patients. Patients will receive either standard of care dosing or dosing with adjustments based on bloodwork to achieve a minimum therapeutic threshold.

This multicentre pilot trial will assess the feasibility of a full-scale, randomized trial to determine whether anti-Xa guided dosing of low molecular heparin (LMWH) reduces the risk of venous thromboembolism (VTE) in high-risk trauma patients. Patients will receive either standard of care fixed dosing of Enoxaparin or 0.5 mg/kg twice daily with dose adjustments to achieve an anti-Xa trough level between 0.1 and 0.2 IU/mL.

Eligibility Criteria:
Inclusion Criteria:

* Patients 18 years of age or older admitted to a hospital ward or intensive care unit following a traumatic injury involving two or more body systems (head, chest, abdomen, pelvis, extremity) and meeting at least one of the following high-risk criteria previously identified in a recent systematic review (1): age ≥ 65, body mass index ≥ 30 kg/m2, injury severity score ≥ 16, pelvic injury with activity restrictions, lower extremity injury with activity restrictions, or surgery during the index hospitalization.

To be eligible, patients must be deemed appropriate for pharmacologic prophylaxis by the most responsible physician and randomized with the intention to receive prophylaxis within 48 hours of admission. Prior to randomization, there is no restriction on whether or not patients have previously received pharmacologic or mechanical prophylaxis.

Exclusion Criteria:

1. Greater than 7 days since time of injury.
2. Requirement for therapeutic anticoagulation or dual-antiplatelet therapy
3. Unable or unwilling to receive pharmacologic prophylaxis within 48 hours of admission.
4. History of allergic reaction or sensitivity to LMWH.
5. Thrombocytopenia with platelets \&lt; 30.
6. Expected discharge or transfer from hospital within 72 hours.
Male Breast Cancer: Understanding the Biology for Improved Patient Care

Rationale: Gathering medical information and tumor samples from patients with male breast cancer may help doctors learn more about the disease.

Purpose retrospective part: to perform a large international retrospective analysis of clinical and biological data of male BC patients treated in the participating centers from 1990 to 2010.

Purpose prospective part: to create a registry of men with breast cancer for a period of 30 months (starting early 2014).

Objectives retrospective part (closed to patients registration in September 2013):

* To perform a large international joint retrospective analysis of clinical and biological data of male breast cancer (BC) patients treated from 1990 to 2010.
* To create a database of patient characteristics, disease features, treatments received, and clinical outcomes of a large series of men diagnosed with BC from 1990 to 2010 in centers in Europe and USA.
* To perform a central pathological review of the corresponding large series of male BC tumors to determine their biologic characteristics and identify relevant prognostic and predictive markers.

Objectives prospective part (opened early 2014):

* To run a prospective registry of all male BC patients (including newly diagnosed) irrespective of the stage and treatment, in the participating institutions for a period of 30 months; collection of FFPE, fresh frozen and blood samples is optional but highly encouraged.
* Quality of Life sub-study

The two parts of this study will provide important information regarding male BC biology and clinical evolution. The collected follow-up information will provide an overview of the current practice in the early and the advanced disease and also offer precious information of the disease evolution in such rare patients' population.

Eligibility Criteria:
Retrospective part (closed to patients registration):

* Men with histologically proven invasive breast carcinoma diagnosed since 1990 to 2010.
* A FFPE tissue sample from the primary tumor (e.g. biopsy or surgery) is mandatory

Prospective part:

* All men, with histologically proven invasive breast cancer, newly presenting at the center irrespective of the stage of disease, initial diagnosis date or treatment received.

  * patients cannot be registered in both the retrospective and protective parts of the study
  * patients who newly present at the center in the 3 months prior to center activation are still eligible as long as they were not previously included in the retrospective part of the study
* Before patient registration, written informed consent must be obtained according to ICH/GCP, and national/local regulations.
* Collection of left-over FFPE and frozen tumor samples as well as blood is optional.

Both parts:

* Concomitant DCIS or LCIS are allowed only if invasive cancer is present.
* Patients should be 18 years or over at the time of diagnosis.
* The study will accept all stages of disease (e.g. early BC, locally advanced and metastatic disease) independent of the treatment received. Patients with past or concurrent other malignancies are eligible.
Hypertonic Modulation of Inflammation Following Injury

This project seeks to determine the effect of prehospital resuscitation with hypertonic saline vs. conventional crystalloids on the inflammatory response after injury. The leading cause of late mortality following injury is multiple organ dysfunction syndrome (MODS), which results from a dysfunctional inflammatory response after injury. Previous studies suggest that hypertonic saline may be beneficial by modulating this initial response and decreasing subsequent organ injury. This project takes advantage of a unique opportunity, afforded by an NIH-funded multi-center clinical trial of hypertonic resuscitation (conducted by the Resuscitation Outcomes Consortium), to obtain blood samples from patients enrolled in this trial to analyze inflammatory responses early after hypertonic vs. conventional resuscitation. This study was an ancillary study to the main randomized clinical trial and thus prospective observational in nature

The proposed study will be carried out in experiments grouped in three Specific Aims: Aim 1 provides a thorough investigation of the immunomodulatory response following hypertonic resuscitation with regard to neutrophil, monocyte, and T cell responses at serial time points after injury and resuscitation. Aim 2 comprises experiments to investigate the mechanisms by which hypertonicity may alter inflammatory cell signaling. Aim 3 seeks to correlate the laboratory findings with clinical endpoints reflective of immune dysfunction including inflammation, organ failure, nosocomial infection, and sepsis.

The investigators hypothesize that hypertonic resuscitation will be associated with modulation of the excessive inflammatory response seen after injury and thus will result in reduced rates of inflammatory organ injury.

This was an ancillary study to the larger clinical trials of prehospital hypertonic resuscitation conducted by the resuscitation outcomes consortium. This study investigated markers of immune function and coagulation in a subset of patients enrolled in the larger trials at two of the clinical sites. Four publications detail the results please see references

Eligibility Criteria:
Inclusion Criteria:

* Blunt or Penetrating trauma with prehospital systolic blood pressure \< 70 or 70-90 mmHg with Heart rate \> 108 OR Blunt trauma with prehospital Glasgow coma score \<= 8

Exclusion Criteria:

* Age \< 15 yrs
* Known prisoners
* Pregnancy
* Ongoing Cardiopulmonary resuscitation (CPR)
* Burns \< 20%
* Hypothermia \< 28 C
* \> 2 liters intravenous fluid prior to study fluid administration
* \> 4 hour from time of dispatch
The Safety of Boostrix Following Routine Immunization of Pregnant Women

The purpose of this study was to assess the safety of Boostrix administered on or after the first day of the 27th week of pregnancy by conducting a post-marketing study that provided safety information to the public and healthcare providers. This was one of the largest cohorts of pregnant women vaccinated with Boostrix in the U.S. Through partnership between Kaiser Permanente Southern California (KPSC) and the sponsor, GlaxoSmithKline (GSK), information about the safety of maternal vaccination with Boostrix and maternal and infant adverse events (AEs) in a community setting was gained.


Eligibility Criteria:
Inclusion Criteria:

* Pregnant women with prenatal care and continuous membership (allowing up to a 31-day gap) at KPSC between the 1st day of the 27th week of pregnancy and the index (vaccination) date.
* Exposed cohort (from the 27th week of gestation): Pregnant women vaccinated with Boostrix on or after the 1st day of the 27th week of pregnancy; who were not vaccinated with any other Tdap vaccine at any other time during the pregnancy in scope of this study.
* Unexposed cohort: Women matched to the exposed cohort and pregnant sometime during the approximate estimated period between January 1, 2012-December 31, 2014 and did not receive any Tdap vaccine during the pregnancy in scope of this study.

For the analysis of congenital anomalies among live births, at birth and through six months of age, the following additional inclusion criteria for infants will be applied:

* Live born
* Born in KPSC hospitals

Note: Pregnant women vaccinated with Boostrix during pregnancy before the 27th week of gestation, with membership at the date of vaccination, and who were not vaccinated with any other Tdap vaccine at any other time during the pregnancy in scope of this study will be part of a descriptive analysis (secondary objective).
Music Therapy for Post-cardiac Surgery Pain Management

Music therapy as a supplement to post surgery pain management in cardiac surgery patients following median sternotomy.

2. Specific Aims:

1. Primary Aim: To assess whether music therapy can decrease the amount of opioid type pain medications used.

   Hypothesis: Music therapy reduces the amount of opioid type pain medication used in management of post-operative pain and discomfort.
2. Secondary Aims:

i. To identify the gender and age groups of patients who can benefit from music therapy as a supplement to post-operative pain management.

Hypothesis: Majority of the population of patient benefits from music therapy as a supplement to postoperative pain management.

ii. To assess whether music therapy can increase patient satisfaction.

Hypothesis: Music therapy increases patient satisfaction.

Eligibility Criteria:
Inclusion Criteria:

* Patients undergoing cardiac surgery via median sternotomy

Exclusion Criteria:

* Emergency cases.
* Patients with hearing impairment.
* Patients with dementia or other mental status changes.
* Patients who are clinically unstable postoperatively.
* Patients who develop postoperative complications.
* Patients who are non-English speaking.