cyrilzakka/clinical-trials huggingface dataset:
Study of Pituitary Size and Function in Familial Dwarfism of Sindh

OBJECTIVES: I. Assess pituitary size and anatomic configuration by magnetic resonance imaging (MRI) in 4 affected dwarfs in the province of Sindh, Pakistan.

II. Evaluate ultradian growth hormone (GH) secretory patterns in 4 affected dwarfs.

PROTOCOL OUTLINE: Pituitary size and anatomy and growth hormone response are evaluated.

All patients undergo MRI of the pituitary fossa on day 1. On day 2, homozygous patients receive a slow infusion of normal saline with blood sampling every 10 minutes.

Eligibility Criteria:
* Familial Dwarfism of Sindh
* Severe short stature but proportionate without dysmorphic features
* Normal body size and weight at birth
* Bone age is severely delayed
* Puberty is somewhat delayed (age 15-16)
* Fertility present in at least 3 dwarfs
Virtual vs. Traditional Physical Therapy Following Total Knee Replacement

The goals of this research study are the following:

1. To compare the effects of tele-rehabilitation-supported physical therapy versus traditional home and/or clinic-based physical therapy for total knee replacement (TKR) on 90-day health service use costs.
2. To compare tele-rehabilitation-supported physical therapy and traditional physical therapy on patient-centered outcomes
3. To explore whether individual patient characteristics are associated with differential improvement from 6 to 12 weeks assessed by patient-reported outcomes.

This study will compare the effects on health service use costs of tele-rehabilitation-supported physical therapy versus traditional home and/or clinic-based physical therapy following total knee replacement (TKR) surgery. It will also examine standard post-surgical clinical measures to confirm non-inferiority of outcomes between groups. A total of 300 patients will receive standard pre- and post-surgical medical care from their healthcare providers, and be randomized to receive pre-surgery rehabilitation exercises ("pre-hab") and post-discharge physical therapy provided via either a home-based tele-rehabilitation platform (intervention group, n=150) or home-health and clinic-based physical therapy regimen (control group, n=150). Data will be collected by sites at enrollment, baseline, hospital discharge, and 6 weeks following surgery. Patients will complete telephone surveys regarding their health and knee function at baseline, 6 weeks, and 3 months after surgery. Patients will also keep a diary from hospital discharge through 3 months documenting healthcare utilization and progress toward a personal recovery goal. Diary information will be collected over the phone concurrently with administration of the 6-week and 3-month surveys. The episode for analysis will be from surgery through 3 months post-discharge, in alignment with the Centers for Medicare and Medicaid Services Comprehensive Care for Joint Replacement (CJR) bundled payment model. Physical risks or benefits to patients for participating in this study are as expected from standard pre- and post-surgical physical therapy associated with total knee replacement surgery. The low risk of loss of confidentiality for all subjects will be minimized using appropriate safeguards.

Eligibility Criteria:
Inclusion Criteria:

1. ≥ 18 years of age
2. Scheduled to have a non-traumatic TKR
3. Can be enrolled a minimum of 10 days prior to surgery (in-person visit)
4. Have a Risk Assessment and Prediction Tool (RAPT) score of ≥ 6 indicating expected discharge home after surgical hospitalization

Exclusion Criteria:

1. Unable or unwilling to provide informed consent, including but not limited to cognitive or language barriers (comprehension)
2. Scheduled for staged bilateral TKR
3. Living in a nursing home prior to surgery
Anesthetic Effects in Mitochondrial Disease

Summary. At the present, the investigators do not have the perfect anesthetic for mitochondrial patients. When possible, consideration should be given to the use of local anesthetics in small amounts. When a general anesthetic is necessary, they each carry significant risks and have been associated with poor outcomes. At present it is not possible to eliminate one group as less safe than others. What is clear is that these patients must be monitored more closely than other patients. The advent of the bispectral index (BIS) monitor may allow us to monitor their depth of anesthesia more closely and thus expose these patients only to the minimum amount of drug necessary to carry out the surgical procedure.

Purpose. The investigators hypothesize that specific mitochondrial diseases, in particular those that decrease complex I function, make certain children hypersensitive to volatile anesthetics. These same patients may be at increased risk for adverse outcomes following general anesthesia. The specific aims of this application are:

1. Determine which molecular defects in mitochondrial function lead to alter sensitivity to the VA sevoflurane.
2. Establish the relative safety of sevoflurane in treatment of patients with mitochondrial disease.

The investigators plan to monitor patients with mitochondrial disease using expanded measures of cardiovascular stability and measurements of brain electrical activity while slowly inducing general anesthesia. The investigators will use those measurements to limit the amount of anesthetic these patients receive in an attempt to minimize their risk. In addition, the investigators will correlate their sensitivity to the type of mitochondrial defect so that the investigators may be able to predict which patients are likely to have an increased sensitivity.


Eligibility Criteria:
Inclusion Criteria:

* Patients presenting to the operating room for muscle biopsy as part of their diagnostic workup for possible mitochondrial disease.

Exclusion Criteria:

* Patients more than 16 years of age.
* Patients with concurrent acute infectious disease.
* Patients not tolerating a slow induction for emotional reasons.
* Initial BIS measurement of less than 95.
* Documented pulmonary disease.
PRIME: Pilot Study Evaluating Percutaneous Vaccination of BCG for Bladder Cancer

Approximately 25 subjects will be enrolled and receive a standard WHO adult potency BCG immunization (1cc/50mg live mycobacilli) in the deltoid. Following a wait period after BCG vaccination, patients will then receive standard strength BCG intravesical therapy once a week for a total of 6 weeks. The BCG is instilled and held in the bladder for approximately 2 hours. While the BCG is retained in the bladder the patient should be repositioned every 30 minutes to maximize bladder surface exposure to the agent. Patients will undergo a cystoscopy every 3 months following most recent TURBT. Study duration last approximately 6 months.

Approximately 25 subjects will receive a standard WHO adult potency BCG immunization (1cc/50mg live mycobacilli) in the deltoid. Following a wait period after BCG vaccination, patients will then receive standard strength BCG intravesical therapy once a week for a total of 6 weeks. The BCG is instilled and held in the bladder for approximately 2 hours. While the BCG is retained in the bladder the patient should be repositioned every 30 minutes to maximize bladder surface exposure to the agent. Patients will undergo a cystoscopy every 3 months following most recent TURBT. Study duration last approximately 6 months.

Eligibility Criteria:
Inclusion Criteria:

The patient must:

* Be 18 years of age or older
* Be able to give informed consent
* Have newly diagnosed or recurrent multi-focal Ta, Large Ta, High Grade Ta, CIS or T1 Bladder Cancer
* Determined by treating urologist to be a good candidate for BCG Induction Therapy
* Have an adequate marrow function (defined as white blood cells greater than 1.5 x 103/µl (or 1,500 cells/mm3) and platelets greater than 150,000 cells/mm3; these results can be within last 60 days from the day of signing informed consent

Exclusion Criteria:

The patient cannot:

* Be less than18 years of age
* Unable to give informed consent
* Have a history of muscle invasive bladder cancer
* Be self-reported to be immune-compromised (HIV, chronic immunomodulators, chronic corticosteroids)
* Have a history of tuberculosis and/or received BCG Percutaneous Vaccination
* Pregnant or planning to become pregnant.
Betaine Supplementation and Lipid Metabolism in Overweight and Obese Pre-menopausal Women

Obesity represents a serious global health issue with significant consequences, including an increased risk of chronic diseases. Statistics indicate a growing trend of obesity, highlighting the need to seek methods that improve fat tissue metabolism and reduce obesity-related complications. Previous research on animals has shown that betaine, a substance engaged in one-carbon metabolism, may enhance fat oxidation and lower adipose tissue. Therefore, the aim of the research will be to assess the impact of 8-week betaine supplementation on body composition and lipid metabolism markers, as well as expression of genes related to lipid metabolism, in a group of adult women with abdominal obesity.

This study is designed in a placebo-controlled, double-blinded, randomized fashion. The participants will be overweight or obese pre-menopausal females. Upon enrollment, participants will be randomly assigned to one of two parallel groups: betaine (3g/d) or placebo. The supplementation period will last for 8 weeks. There will be three study meetings: T1 before supplementation, T2 after 4 weeks of supplementation, and T3 after 8 weeks of supplementation. Blood will be drawn and body composition measured, and adipose tissue biopsy taken at meetings T1 and T3. The T2 meeting will involve only body composition measurement. Study outcomes will include body mass and composition (including body fat percent), lipid profile, and the expression of genes related to lipid metabolism in adipose tissue and peripheral blood mononuclear cells.


Eligibility Criteria:
Inclusion Criteria:

* females
* age: 18-45 years
* pre-menopausal
* overweight or obese: BMI \>25 kg/m2
* waist circumference \> 80 cm

Exclusion Criteria:

* males
* age \<18 or \>45 years
* perimenopausal or postmenopausal
* BMI \< 25 kg/m2
* waist circumference \< 80 cm
* betaine administration for 3 months prior to study
* pregnant or breastfeeding
Efficacy and Safety of Telbivudine in Treatment naïve Patients With Hepatitis B e Antigen (HBeAg)-Positive Chronic Hepatitis B (CHB)

To evaluate the combination of telbivudine 600 mg orally (PO) once daily and peginterferon alpha-2a 180 ug subcutaneous (sq) injection weekly for antiviral efficacy in comparison to peginterferon alpha-2a monotherapy.


Eligibility Criteria:
Inclusion Criteria:

Documented Chronic hepatitis B (CHB) defined by all of the following:

* Clinical history compatible with CHB
* Detectable serum Hepatitis B Surface Antigen (HBsAg) at the Screening visit and at least 6 months prior
* HBeAg-positive at the Screening visit
* Hepatitis B 'e' Antibody (HBeAb)-negative at the Screening visit
* History of evidence of chronic liver inflammation,
* Elevated serum Alanine aminotransferase (ALT) level (1.3 - 10 x upper limit of normal (ULN)) at the Screening visit
* Serum HBV DNA level ≥ 6 log10 copies/mL,
* Chronic liver inflammation on previous liver biopsy within the previous 24 months.

Exclusion Criteria:

* Co-infection with Hepatitis C Virus (HCV), Hepatitis D Virus (HDV), or Human Immunodeficiency Virus (HIV).
* Has any of the following drug therapy:

  * Previously been treated in a trial with telbivudine
  * Received nucleoside or nucleotide therapy whether approved or investigational
  * Received any immunomodulatory treatment in the 12 months before Screening for this study.
  * Has a medical condition that required prolonged or frequent use of systemic acyclovir or famciclovir.
  * Has a medical condition that requires frequent or prolonged use of systemic corticosteroids although inhaled or intra-articular corticosteroids are allowed.
  * Has a medical condition requiring the chronic or prolonged use of potentially hepatotoxic drugs or nephrotoxic drugs.
  * Is currently abusing alcohol or illicit drugs or has a history of alcohol abuse illicit substance abuse within the preceding two years.
  * Uses other investigational drugs at the time of enrollment, or within 30 days or 5 half-lives of enrollment, whichever is longer.
  * Is currently receiving methadone.
* Patient has any of the following:

  * History of or clinical signs/symptoms of hepatic decompensation such as ascites, esophageal variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis.
  * History of malignancy of any organ system, treated or untreated, within the past 5 years whether or not there is evidence of local recurrence or metastases, with the exception of localized basal cell carcinoma of the skin. Patients with previous findings suggestive of possible HCC should have the disease ruled out prior to entrance into the study.
  * One or more additional known primary or secondary causes of liver disease other than hepatitis B, including steatohepatitis.
  * History of clinical and laboratory evidence of chronic pancreatitis, or demonstrates a clinical and laboratory course consistent with current pancreatitis.
* Has laboratory values during screening visit not within normal limits.
* Is pregnant or breastfeeding.
* Is a women of child-bearing potential that is unwilling to practice birth control.
Oral Omega-3 Fatty Acids in the Treatment of Dry Eye Syndrome

The investigators hypothesize that oral omega-3-acid ethyl esters (Lovaza, GlaxoSmithKline, Research Triangle Park, NC) will decrease dry-eye related symptoms as well as clinical markers associated with dry eye disease (Schirmer-1 test values, positive vital staining with lissamine green, and fluorescein tear break-up time) when compared to administration of placebo.


Eligibility Criteria:
Inclusion Criteria:

* Age \> 18 years
* Typical symptoms of dry eye (photophobia, burning, foreign body sensation, blurred vision improved with blinking)
* Schirmer Test \< 8 mm/5 minutes
* Fluorescein tear break-up time \< 8 seconds
* No current use of dry eye treatment (except artificial lubrication)
* Signature on consent form

Exclusion Criteria:

* Infectious keratoconjunctivitis or inflammatory disease unrelated to dry eye
* Eyelid or eyelash abnormalities
* Alteration of the nasolacrimal apparatus
* Treatment with drugs affecting tearing
* Concomitant ocular therapies
* Topical ophthalmic steroids taken during the 4 weeks before the study
* Pregnant/breast-feeding women
* History of liver disease
* History of fish and/or shellfish allergy or hypersensitivity
* History of corn allergy or hypersensitivity
* Treatment with systemic anticoagulation therapy
* Patients with bleeding disorders or those receiving anticoagulation (e.g., warfarin, enoxaparin, dipyridamole, clopidogrel)
Sustainability Via Active Garden Education

The goal of SAGE is to determine if a garden-based curriculum can increase physical activity and improve nutrition in young children at early care and education centers (ECEC) throughout Phoenix

SAGE will be delivered over a four month period using a cluster randomized controlled trial adapted crossover design, along with testing a sustainability action plan (SAP) at the organizational level. In this study, the investigators will determine the efficacy of the SAGE intervention on health in 3-4 year-old children. This study will pair match and randomize 20 ECEC from neighborhoods with high proportions of Hispanic or Latino residents. ECEC will receive either the SAGE intervention or the safety attention comparison. Then, ECEC will cross over and receive the treatment that they did not receive to ensure that all ECEC receive both curricula. The investigators will determine improvement in accelerometry measured PA and sedentary behaviors, fruit and vegetable consumption and explore eating in the absence of hunger compared to those in a child safety attention comparison. The investigators will explore secondary impacts on parenting practices that promote PA and fruit and vegetable consumption, home fruit and vegetable availability and improved household food security. This study will explore the process of delivery of the SAGE intervention on dimensions of reach, adoption, and implementation. This study will also develop, implement, and evaluate a SAP in the SAGE arm to determine replicability and institutionalization (sustainability) of the SAGE intervention and explore the relationship between of sustainability to child outcomes at follow-up. The development and success of the community Partnership and SAGE Community Advisory Board will also be evaluated using indicators of participation, representativeness, and collaboration. This study will rely on CBPR strategies and an established theoretical model to implement an engaging and translational multilevel intervention linking policy and practice. This work will test strategies for implementing IOM guidelines at the ECE level and will guide future efforts aimed at scaling up efficacious interventions for broad dissemination in vulnerable populations to reduce health disparities.

Eligibility Criteria:
ECEC Inclusion Criteria:

* Census tracts with \>30% Hispanic-Latino population
* ECEC must be licensed and enroll at least 35 children aged 3-4 years old
* Space for garden install

Exclusion Criteria:

* Not participating in CACFP
* No space for a garden
* Less than 35 children aged 3-5 years old enrolled
Technical Feasibility Study on Screening Sleep Related Breathing Disturbances and Sleep Apnoea With Wearable Sensors

In this study the feasibility of detecting sleep apnoeas with unobtrusive wearable sensors and sounds recorded with a smartphone is studied by making an overnight recording to patients with high probability of sleep apnoeas. The data acquired with the aforementioned devices is: ECG, acceleration, bioimpedance of thorax and processed and raw audio. In data analysis phase it will be studied which combinations of these signals would enable detecting sleep apnoeas with high enough sensitivity and specificity when compared to a night polygraphy reference (Nox T3 device using airflow, breathing movements, audio, position, movement, oxygen saturation, pulse and leg EMG).


Eligibility Criteria:
Inclusion Criteria:

* High a priori probability for SA based on previous PG findings, BMI, and ESS and medical history
* Clinical reason i.e. potential benefit of repeating the PG due to e.g. technical challenges in the previous PG and/or unclear findings or diagnosis. Participation may be offered also in cases where first PG is diagnostic. In these cases accuracy of sleep apnoea grading will be improved.
* Adequate Finnish language skills to comprehend study-related instructions and questionnaires. The study materials are available only in Finnish.
* Signed written informed consent

Exclusion Criteria:

* Medical history of a major cardiovascular event (myocardial infarction, coronary artery bypass graft (CABG), percutaneous coronary intervention (PCI), stroke, or transient ischemic attack) within the previous 6 months
* Use of cardiac pacemaker or history of atrial fibrillation
Analysis of Differential Proteins and Maternal Influencing Factors of Septic Shock in Neonates

1. Explore 32 weeks of premature infants or maternal pregnancy infection of newborn septic shock maternal influence factors. 2. Through maternal blood samples before delivery and labor cord blood sample screening and septic shock related proteomics analysis, explore the relationship between the difference protein and septic shock and prognosis, and explore the pathway mechanism, in order to prevent and reduce the occurrence of septic shock in premature infants

The birth cohort was established with 32 weeks of premature infants or maternal infection during pregnancy.Whole proteomic analysis was performed on maternal blood before delivery and umbilical cord blood samples at delivery,and screscreen differential proteins associated with septic shock and classify these proteins as leak factor into exposed and non-exposed groups for cohort study.The incidence and mortality of shock,severe brain injury and BPD in the two groups were compared, and the mechanism of the differential protein was studied to provide early warning in children with septic shock.

Eligibility Criteria:
Inclusion Criteria:

* All ≤32-week premature infants admitted to our NICU; All newborns delivered by the mother infected during pregnancy

Exclusion Criteria:

* Not delivered in our hospital; congenital cardiovascular development malformation, fetal malformation, genetic metabolic diseases, etc
Identifying Modifiable PAtient Centered Therapeutics (IMPACT) Frailty

Frailty, the decline in physical and cognitive reserves leading to vulnerability to stressors is increasingly being recognized as a public health concern. Although multiple measures exist that can identify frail patients, very little is known about how or when to intervene. Sarcopenia, or the degree of muscle wasting, is closely correlated to frailty and patient outcomes. This is a prospective cohort study of critically ill patients to identify modifiable risk factors of sarcopenia, as potential targets to reduce frailty.

Frailty is the decline in physical and cognitive reserves leading to increased vulnerability to stressors such as surgery or disease states. Frailty is not a disease, but a syndrome with a distinct frail phenotype that includes decreased status in mobility, muscle mass, nutritional status, strength, and endurance. Frail patients are at greater risk of adverse outcomes, such as functional decline, prolonged hospitalization with associated increases in healthcare costs and death. Multiple measures of frailty exist and although they are important for understanding risk for a given patient population or resource utilization, they do not provide any insight as to how to manage or treat frail patients.

In critically ill patients, sarcopenia has long been tied to poor outcomes, poor nutrition status, and decreased ability to perform activities of daily living (ADLs). We hypothesize that sarcopenia as a marker for frailty in critically ill patients can be used to track development and recovery of frailty.

The objective of this proposal to create a prospective cohort study of critically ill patients to identify modifiable risk factors of sarcopenia as potential targets for therapeutic measures to improve or reverse frailty.

The primary aim of the study is to track sarcopenia in critically ill patients. Sarcopenia is a measure of frailty and is associated with worse outcomes in critically ill patients. The aim to understand how the kinetics of sarcopenia differ in critically ill population given the heterogeneity of with various disease process which may affect the degree and rate of muscle wasting. Understanding the disease process is important in identifying when or how to intervene to obtain meaningful recovery.

Secondary aims are to assess the role biomarkers in patients across the frailty spectrum to understand their role frailty. Additionally nutrition is well known to affect sarcopenia and nutritional status is a key component in frailty. Nutrition status will be tracked to understand development of sarcopenia.

Eligibility Criteria:
Inclusion Criteria:

* Adult patients admitted to the Surgical, Cardiothoracic or Neuro ICU
* ICU stay longer than 24 hours

Exclusion Criteria:

* Patients with muscular or mitochondrial diseases affecting muscle quality or mass
* Preexisting paralysis
* Absence of lower limbs
* Ongoing discussions about goals of care
* Transfers from a skilled nursing facility
* Children under the age of 18