cyrilzakka/clinical-trials huggingface dataset:
ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts.

This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5.

The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose cohorts (C1) and (C2) will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. An initial cohort C1 of three (3) patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies.

Enrollment of three (3) patients in the 2nd higher dose cohort C2 (with a 7-fold safety margin relative to the highest safe dose in the GLP toxicology study) will be initiated following review of the one-month safety data post-administration in cohort C1 by an independent Data Safety Monitoring Board (DSMB).

Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months.

All subjects will be followed up for an additional 4.5 years after completion of the evaluation period.

Eligibility Criteria:
Inclusion Criteria:

* Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
* Ambulant male or female patients aged 6 to less than 12 years of age at screening
* Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support

Exclusion Criteria:

* Detectable neutralizing antibodies against AAV8
* Cardiomyopathy with left ventricular ejection fraction (LVEF) \&lt; 50%
* Respiratory assistance
* Concomitant medical condition that might interfere with LGMDR5 evolution
* Acute illness within 4 weeks of anticipated IMP administration
* Current participation in another clinical trial with investigational medicinal product
* Previous participation in gene and cell therapy trials
* Any condition that would contraindicate immunosuppressant treatment
* Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
* Any vaccination 1 month prior to planned IMP administration
* Serology consistent with HIV exposure or active hepatitis B or C infection
* Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
Endometrial Biopsy Instrument Comparison Study

The purpose of this study is to determine the level of pain women experience during an endometrial biopsy and the effect that the biopsy tool might have on that pain. The investigators will also evaluate the adequacy of the sample each endometrial biopsy tool collects.

Is there a difference in pain perceived by patients undergoing endometrial biopsy via the Pipelle or Explora curette? Currently, the instrument selected for endometrial biopsy is solely dependent on provider preference. This study aims to elucidate advantages and/or disadvantages of the Pipelle versus the Explora curette in terms of pain perceived by the patient, adequacy of sample, and provider ease of use to better aid in the selection of the appropriate device.

Eligibility Criteria:
Inclusion Criteria:

* Women ages 18 and over who present to the Oregon Health and Science University Center for Women's Health and the Portland Veteran's Affairs Medical Center with an indication for endometrial biopsy, are literate in English or Spanish, who are willing to participate in the study.

Exclusion Criteria:

* Pregnancy, known or suspected
* Known cervical stenosis
* History of Mullerian tract anomalies
* History of uterine or cervical surgery
* Pelvic inflammatory disease (current or within the past 3 months)
* Sexually transmitted diseases (current)
* Puerperal or post abortion sepsis (current or within the past 3 months)
* Purulent cervicitis (current)
* Known clotting disorder
* Known uterine anomalies or fibroids distorting the cavity in a way incompatible with office endometrial biopsy
* Allergy to any component of the Pipelle or Explora curette
* Patients who are premedicated with analgesics or misoprostol
* Patients who require mechanical cervical dilation or receive paracervical block
Innovative Care Pathway in Physical Activity and Behavior Change in Coronary Patients Post-cardiac Rehabilitation

The objective of this 18-month research is to show the effectiveness of an individualized physical activity( PA) compared to standard PA management and voluntary PA, to achieve a change in the behavior of the coronary patient.


Eligibility Criteria:
Inclusion Criteria:

* Post-infarction coronary artery patient aged 18 years and older, with no gender difference.
* Patients engaged in a follow-up care and cardiovascular rehabilitation program at the Supervaltech center.
* Patient at low to moderate RARE risk of complication (RARE score 2 to 3)
* Patients equipped with smartphones, laptops or other equipment compatible with downloading an application with their personal ID
* Patient able to understand French for the purpose of conducting the study.
* Affiliated member or beneficiary of a social security scheme.
* Participant who has been informed and has given free, informed and written consent (no later than the day of inclusion and before any examination required by the research).

Exclusion Criteria:

* Patients with a formal contraindication to physical activity such as: unstabilized coronary syndrome, decompensated heart failure, severe ventricular arrhythmias, intracardiac thrombus at risk embolic, PAH, moderate pericardial effusion, history of thrombophlebitis with or without pulmonary embolism, severe and/or symptomatic left ventricular ejection obstruction.
* Patients with ongoing infectious disease
* Participant whose physical and/or psychological health is severely impaired, which in the opinion of the investigator may affect the study participant's compliance.
* Participant included in other research
* Participant in a period of exclusion from another research still in progress at the time of inclusion.
* Protected participant: an adult under guardianship, curatorship or other legal protection, deprived of liberty by judicial or administrative decision.
* Pregnant, breastfeeding or parturient women.
* Participant hospitalized without consent.
MOtiVating Endometrial Cancer Survivors With Activity Monitors and Tailored Feedback

Overweight and obese endometrial cancer (EC) survivors at the University of North Carolina (UNC) at Chapel Hill will be approached for tailored feedback fitness intervention. The investigators plan to enroll 36 women (18 in each arm) to evaluate if receipt of weekly tailored feedback messages can improve physical activity in EC survivors. It is hypothesized that women receiving the feedback message intervention will increase step counts from baseline more than 2,000 steps compared to women in the non-intervention arm.

Objectives:

* To assess if a tailored feedback fitness intervention can increase physical activity in EC survivors from baseline to 12 weeks post-baseline
* To determine the acceptability of the fitness intervention in EC survivors through a completion survey.
* To assess if a tailored feedback message fitness intervention can (1) decrease BMI, (2) reduce waitst to hip (W.H) ratios, (3) improve quality of life (QOL) and (4) improve serum metabolic markers (insulin, glucose and low density lipoprotein (LDL) in EC survivors.

Eligibility Criteria:
Inclusion Criteria:

* Be over the age of 18 years
* Have a confirmed diagnosis of endometrial cancer and have completed therapy (surgery, chemotherapy or radiation) within the past 6 months
* Have no current evidence of endometrial cancer
* Have a BMI 25 kg/m2 or greater
* Have approval from their treating physician to engage in moderate-intensity physical activity.
* Have a smart phone with Bluetooth capabilities turned on
* Have access to email

Exclusion Criteria:

* Are currently undergoing treatment for their cancer
* Are unable to read a sample message aloud
* Do not have a computer or smart phone with Bluetooth capabilities
* Are pregnant
* Have a history of angina or palpitations with exertion
* Have a history of uncontrolled pulmonary disease (COPD or asthma)
* Have one or more significant medical conditions that in the physician's judgment preclude participation in the walking intervention.
Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.

For each subject, only a single abdominal or facial attack was treated and evaluated. After receiving treatment, subjects were observed for a minimum of 4 hours, after which they could be discharged from the study center if they reported onset of symptom relief. Starting from 4 hours after treatment, subjects who reported insufficient or no symptom relief could receive a second dose of double-blind treatment (called "rescue medication") as follows: C1-INH 20 U/kg bw for subjects initially receiving placebo, C1-INH 10 U/kg bw for subjects initially receiving C1-INH 10 U/kg bw, and placebo for subjects initially receiving C1-INH 20 U/kg bw.

The study was defined to be successful if the primary outcome measure and at least one of the secondary outcome measures were met in the comparison between the C1-INH 20 U/kg bw group and the Placebo group.

Eligibility Criteria:
Key Inclusion Criteria:

* Documented congenital C1-INH deficiency
* Acute facial or abdominal HAE attack

Key Exclusion Criteria:

* Acquired angioedema
* Treatment with any other investigational drug within the last 30 days before study entry
* Treatment with any C1-INH concentrate within the previous 7 days
Effects of the Addition of Uterosacral Ligament Plication to Pectopexy Operation on Anatomical and Subjective Symptoms

The investigators planned to measure the effectiveness of laparoscopic pectopexy in apical prolapse and whether the addition of sacrouterine ligaments plication to pectopexy increases the effectiveness of pectopexy operation. Investigators planned to measure patients' quality of life using the female sexual function scale test 3, 6 and 12 months after the surgery and to determine how the operation affects the quality of sexual life.

In this study, the investigators planned to measure the effectiveness of laparoscopic pectopexy in apical prolapse and whether the addition of sacrouterine ligaments plication to pectopexy increases the effectiveness of pectopexy operation. Investigators planned to measure patients' quality of life using the female sexual function scale test 3, 6 and 12 months after the surgery and to determine how the operation affects the quality of sexual life.

Eligibility Criteria:
Inclusion Criteria:

* patients with stage II-IV apical compartment defect according to the POP-Q classification

Exclusion Criteria:

* having severe chronic disease that makes the operation dangerous
* patients under 25 and patients over 75 years old
Positron Emission Tomograph(PET) Imaging Explores the Role of SF-DEVD-2 in Monitoring Tumor Efficacy

This is a single arm study to determining the value of apoptotic molecular probe SF-DEVD-2 in the early evaluation of tumor efficacy and comparing it head-to-head wihe 18F-FDG(18F-2-fluoro-2-deoxy-D-glucose)


Eligibility Criteria:
Inclusion Criteria:

1. Patients voluntarily signed informed consent;
2. Age18-70, male or female;
3. Diagnosed with stage 2 or 3 breast cancer
4. Solid tumors、masses larger than 2cm with lymph node metastasis
5. Pathology is HER3+、triple negative patients
6. Patients to be treated with neoadjuvant chemotherapy

Exclusion Criteria:

1. Age greater than or equal to 70 years old
2. Abnormal liver and kidney function (more than five times the normal value)
3. Diagnosis level of breast cancer stage 4 or second time tumor patients
Effects of Hardaliye, a Fermented Grape Drink, on Oxidative Stress, Lipid Profile and Blood Pressure in Young Footballers

In the present study, the authors aimed to investigate the effects of hardaliye (grape juice) consumption on oxidative stress parameters, lipid profile and blood pressure in young football players.


Eligibility Criteria:
Inclusion Criteria:

* Being a male football player playing in a team in the Elite Development League,
* Participating in training with the team at least 5 days a week for 3 months,
* Not consuming red wine, grape juice, and/or hardaliye regularly before the study was started,
* Those who stayed in the sports facility where catering service was provided for breakfast, lunch and dinner were included in the study.

Exclusion Criteria:

* With chronic disease,
* Smoker,
* Those taking antioxidant supplements,
* Non-cooperative subjects or unwilling to sign an informed consent and participate in the study.
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease

Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. The purpose of this study is to examine the density of the lung as measured by chest computed tomography (CT) and determine if existing emphysema predicts changes in the rate of subsequent emphysema or changes in CT, serum or plasma biomarkers of interest. The overarching goal is to develop biomarkers that can be used in interventional trials since lung function changes do not typically inform disease progression in AATD.

Progression of lung disease in patients with Alpha-1 Anti-trypsin deficiency is variable and while some patients may have stable lung function over many years, some progress and deteriorate rapidly. Currently there are no predictors that would help identify patient at risk of rapid deterioration. The aim of this study is to identify markers and features in CT imaging that may allow identification of these patients early before deterioration. To achieve this, the study will follow a cohort of patients with confirmed Alpha-1 Anti-trypsin deficiency and lung disease and measure a number of biomarkers in blood and sputum and obtain high resolution CT scans at baseline and again three years later. If the study is able to determine markers that allow the identification of patients at risk early, the investigator may be able to study early interventions in later studies and possibly find ways to avoid serious complications. Patients will be followed longitudinally to assess deterioration of lung function.

Study procedures include: Review of medical history and medication history, blood draw, complete Pulmonary Function Test (PFT), induced sputum (at some sites), completion of questionnaires and CT Chest scan. All of the mentioned procedures above will be performed on enrollment and repeated at 18 months and 36 months, with the exception of monthly Alpha- net exacerbation questionnaires.

Eligibility Criteria:
Inclusion Criteria:

1. Males and females aged 18 years and older
2. Patients with known Alpha-1 Anti-trypsin deficiency (PiZZ)
3. Be an existing member of the Alpha-1 Foundation Clinical Cohort (Alpha-1 Foundation Research Registry)
4. Willingness to perform study procedures of Chest CT, blood biomarker determination, questionnaire completion, and lung function testing.

Exclusion Criteria:

1. AATD non-PiZZ status, including carriers
2. Pregnancy at the time of the screening visit
3. COPD exacerbation or other pulmonary infection within 6 weeks of baseline visit
4. Previous lung or liver transplantation or currently on the transplant list
Endocannabinoids, Stress, Craving And Pain Effects Study

Opioid use disorder (OUD) represents one of the most severe public health crises, with more than 2 million individuals affected in the United States. Existing treatments do not target and restore several key alterations triggering opioid craving and relapse, including increased response to stress, mood disturbances and greater sensitivity to pain, which are caused by prolonged exposure to opioids. This double-blind, randomized, placebo-controlled study will investigate the effects that palmitoylethanolamide (PEA), an endogenous molecule part of the endocannabinoid system available as a dietary supplement, exerts on these alterations and their underlying mechanisms, with the goal of identifying a novel therapeutic approach to reduce craving and prevent relapse in patients with OUD.


Eligibility Criteria:
Inclusion Criteria:

* Age 18 to 65
* DSM-5 diagnosis of OUD
* English speaking
* Receiving either buprenorphine or methadone for treatment of opioid use disorder for at least 3 consecutive months prior to enrollment
* Receiving a stable dose of buprenorphine or methadone for the duration of the study
* Agreeable to abstaining from using any cannabis or CBD products two weeks prior to enrollment in the study, and for the duration of the trial
* For women of childbearing potential: agreeable to use one of the following:

  * hormonal methods, such as birth control pills, patches, injections, vaginal rings, or implants
  * barrier methods (such as a condom or diaphragm) used with a spermicide (a foam, cream, or gel that kills sperm)
  * intrauterine device (IUD)
  * abstinence (no sex)

Exclusion Criteria:

* DSM-5 diagnosis of moderate-to-severe cannabis use disorder, alcohol use disorder, and/or psychostimulant use disorder \[medical record review and health history form\]
* Active, recurrent substance use within the last 3 months that will interfere with study participation and completion of study procedures \[medical record review and health history form\]
* History of psychotic, bipolar and schizoaffective disorders \[medical record review and health history form\]
* Lifetime psychiatric hospitalization or suicide attempt, as assessed by the health history form
* Recent history (within 2 years) of major depressive disorder \[health history form and clinical interview\]
* Currently pregnant or breastfeeding (female only) \[pregnancy test/ self-reported\]
* History of autoimmune or chronic inflammatory diseases \[health history form\] Current use of medications known to alter inflammatory and immune response \[health history form\] Raynaud's disease \[health history form\]
* BMI \>45
* Hepatic liver enzymes greater than 3x upper normal limit
* Vital signs: HR ≤60 or ≥100, SBP ≤90 or ≥160, DBP ≤50 or ≥100, RR \< 12 or \> 20
* Recent history of clinically significant medical conditions including, but not limited to, malignancy (and treatment for malignancy), HIV, immunological, endocrine (including uncontrolled diabetes or thyroid disease), renal, GI, or hematological abnormalities that are uncontrolled\* \[health history form and medical record review\]
Undifferentiated Embryonal Sarcoma of the Liver: Evaluation of the Relapse Profile According to the Therapies Administered

Undifferentiated embryonal sarcoma of the liver is the 3rd most common malignant liver tumor after hepatoblastoma and hepatocellular carcinoma with a peak incidence between 6 and 10 years of age. Historically, it is a tumor treated only by surgery with a poor prognosis. In the last decade, the combination of more intensive chemotherapy and, more randomly, radiotherapy, has significantly improved the survival rate of these patients. Due to its low incidence, there are few series reported in the literature and to date there is no specific treatment protocol for the management of these tumors.

It seems appropriate to review the management of these tumors in France in order to discuss the best therapeutic strategy.


Eligibility Criteria:
Inclusion criteria:

* Any patient aged 0 to 25 on the date of inclusion in the study
* Treatment carried out in France (included in NRSTS 2005, in the childhood cancer registry or in an SFCE center)
* Start of treatment from 01/01/2008 to 31/12/2018
* Diagnosis of undifferentiated embryonal sarcoma of the liver defined by a compatible histological analysis
* Adult subject who has not expressed, after being informed, his opposition to the reuse of his data for the purposes of this research
* Minor subject (and/or his parental authority) who has not expressed, after being informed, his opposition to the reuse of his data for the purposes of this research

Exclusion criteria:

* Refusal to participate in this research
* Diagnosis of rhabdoid tumor, rhabdomysarcomas, angiosarcomas, PNET
* Absence of histological and/or molecular documentation.
Micronutrients in Critically Ill Patients

Nutritional support is an important component of modern critical care. The practice of vitamin and micronutrient supplementation is variable and official guidelines are not consistent. The primary objective of this observational study is to investigate the clinical practice of micronutrient supplementation during critical illness and to explore the factors underpinning clinical decisions.

Nutritional support is an integral component of modern critical care. Malnutrition is common, but measurement of serum concentrations of most vitamins and micronutrients does not form part of routine care for most critically ill adults in the UK. When measured in a research setting, a large proportion of critically ill patients have low serum concentrations of many essential nutrients. Nutrient deficiency is associated with adverse outcomes, including delirium and increased mortality. To date, it is unclear whether there is a role for micronutrient supplementation in critically ill patients. Official guidelines from the European Society of Parenteral and Enteral Nutrition (ESPEN), American Society of Parenteral and Enteral Nutrition (ASPEN), German Intensive Care Society and Kidney Disease Improving Global Outcome (KDIGO) vary and are contradictory to some extent. Additionally, vitamins B1 and C have been administered as an intervention in the treatment of septic shock and other disease states, but methodological flaws with such studies have left many clinicians with equipoise over the place of vitamin and micronutrients in clinical practice.

There is a need for a well designed, adequately powered intervention study. In preparation, we would like to explore current practice and decision making of doctors, pharmacists and dieticians who are directly involved in prescribing nutritional support.

The primary objective of this observational study is to investigate the clinical practice of micronutrient supplementation during critical illness and to explore the factors underpinning clinical decisions.

Eligibility Criteria:
Inclusion Criteria:

* healthcare professional who is involved in prescribing vitamins or micronutrients to critically ill patients in the ICU

Exclusion Criteria:

* none