cyrilzakka/clinical-trials huggingface dataset:
A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease

This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.

The study is a single arm Phase 2 clinical trial to characterize the safety and efficacy of RP-A501, a recombinant adeno-associated serotype 9 (rAAV9 capsid containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene) in male patients with Danon Disease.

Male subjects ≥8 years of age will receive a single intravenous infusion of RP-A501.

Eligibility Criteria:
Key Inclusion Criteria:

1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.
2. Male.
3. Age ≥8 years.
4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:

   1. Abnormal thickening of Left ventricular wall,
   2. Left ventricular ejection fraction (LVEF) ≥ 50%.
5. New York Heart Association (NYHA) Class II to III.
6. High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)
7. Ability to comply with study procedures including investigational therapy and follow-up evaluations.

Key Exclusion Criteria:

1. Anti-AAV9 neutralizing antibody titer \>1:40.
2. Severe heart failure or requirement for advanced therapies.
3. History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.
4. Prior cardiac or other organ (lung, liver, other) transplantation.
Complication and Lung Function Impairment Prediction Using Perfusion and CT Air Trapping

The primary objective of the CLIPPCAIR study is to construct and validate a new algorithm for predicting post-operative forced expiratory volume in 1 second (FEV1) values for lung resection candidates; this new model will be based on data derived from a thoracic CT scan with injection of contrast media.

Secondarily, the predictions made using traditional scintigraphic data will be compared with those from the new algorithm in a subset of high-risk patients, and cumulative contrast media and irradiation doses associated with imaging will be presented. How other measures of pulmonary function (e.g. transfer factor of the lung for carbon monoxide (TLCO)) and the presence/absence of operative complications might be predicted will also be investigated. Additionally, the potential links between (i) pre-surgical imaging data, (ii) post-surgical changes in respiratory function, and (iii) changes in health related quality of life will also be explored.

Eligibility Criteria:
Inclusion Criteria:

* Patient diagnosed with non-small cell lung cancer
* Indication for pulmonary excision surgery
* Patient requiring a more recent pre-surgical computed tomography scan (CT scan)
* The patient has been correctly informed about the study and has signed the consent form
* The patient is affiliated with or a beneficiary of the French single-payer social security programme (national health insurance)

Exclusion Criteria:

* Patient in an exclusion period determined by another protocol
* Participation in another study that may affect the results of the present study (anti-cancer treatment studies are allowed)
* Patient under legal or judicial protection
* Contraindication to surgery or iodine injection
* Pregnant or lactating women
The Effect of Bicarbonate Profiling in Dialysis Fluid on Phosphate Removal During Hemodialysis and Blood pH

Hyperphosphatemia is still an unresolved problem among hemodialysis patients and significantly increases the risk of death from cardiovascular diseases. Research to date has not answered the question of whether dialysate bicarbonate concentration profiling can improve phosphate removal and its concentration without negative impact on the acid-base balance. This study addressed this issue.

Twenty stable hemodialysis patients will enroll to a four-week study during which different dialysate bicarbonate concentration profiles will be used each week. Each patient will undergo the following profiles (one-week periods): Treatment A - stable dialysate bicarbonate concentration Dbic 35 mmol/L during the whole HD session, Treatment B - Dbic 35 mmol/L for the first two hours and Dbic 30 mmol/L for the next two hours and Treatment C - the opposite mid-HD change Dbic from 30 to 35 mmol/L and one week wash-out period between Treatment B and C. We will collect blood samples each hour during the session and one hour after HD completion.


Eligibility Criteria:
Inclusion Criteria:

* stable general condition
* hemodialysis more than 3 months
* Dialysis 3 times a week/ 4 hours +/- 20 minutes
* Dialysis with arterio-venous fistulas
* bicarbonate titer before hemodialysis 22-24 mmol/l

Exclusion Criteria:

* diabetes mellitus
* cachexia
* inflamation processes
* usage of sevelamer last month
* utrafiltration during dialysis more than 2500 ml
Urinary Symptoms and the Omission of the Bladder Flap at the Time of Primary Cesarean Delivery

The bladder flap at the time of cesarean delivery is the term used to describe the separation of the bladder from the lower uterine segment by sharply incising the vesico-uterine peritoneum or serosa and using blunt and/or sharp dissection to develop this potential space which facilitates placement of a retractor, known as the bladder blade. Creating a bladder flap at the time of cesarean delivery is largely based on individual practice patterns and practitioners are divided in their use of this step. While creating a bladder flap has a theoretical advantage of protecting the bladder from injury, it is unknown whether this step has an effect on postoperative bladder function. The purpose of this study was to evaluate whether the omission or creation of a bladder flap results in a change in urinary symptoms as measured by the UDI-6 component of the PFDI-20.

This is a Parallel Assignment design study. The PFDI-20 symptom questionnaire was completed upon enrollment. After the patient was prepped and draped, the operating room nurse opened a sealed opaque envelope marked with the study subject number containing a card marked with the assignment to bladder flap or no bladder flap, which was shown to the surgeons prior to the skin incision. The patient did not see the allocation nor was it verbalized in the operating room. The PFDI-20 was repeated 6-8 weeks after delivery at the patient's postpartum exam clinic visit. Equal number of bladder flap and no bladder flap assignment cards were randomly shuffled and placed in envelopes marked with the study subject ID number prior to the start of the study.

The study hypothesis was that the omission of the bladder flap at the time of primary cesarean delivery would be associated with lower urinary symptoms scores in the postpartum as measured by the UDI-6 component of the PFDI-20.

Eligibility Criteria:
Inclusion Criteria:

* pregnant at 37 weeks gestation or greater
* scheduled for a non-urgent primary cesarean delivery

Exclusion Criteria:

* pre-term (defined as less than 37 weeks 0 days gestation) prior pelvic surgery involving the bladder
* a diagnosis of any of the following conditions: endometriosis, uterine leiomyomata, chronic pelvic pain, urinary incontinence prior to pregnancy, nephrolithiasis during the current pregnancy,
* any circumstance that precluded adequate informed consent at the time of recruitment (such as a need to for urgent or emergent delivery)
* if the indication for cesarean was a failed trial of operative vaginal delivery (forceps or vacuum extraction)
Epidemiology of Cardiovascular Disease in Elderly Turkish Population

This study will compose a national database of cardiovascular diseases' risk factors, concomitant diseases and the drug usage among the elderly patients in Turkey.


Eligibility Criteria:
Inclusion Criteria:

* patients signed patient informed sheet and informed consent form
* patients ≥65 years old
* patients followed-up cardiology clinics

Exclusion Criteria:

* patients under 65 years old
Pulmonary Fibrosis After Severe COVID-19 Pneumonia

Patients discharged after hospitalization for COVID-19 pneumonia were retrospectively selected by radiologically established criteria that at admission presented at chest computed tomography (CT) (i) normal lung parenchyma \<50% of total lung volume; and/or (ii) area of lung consolidation \> 10%. All At discharge and after 9 months, all subjects underwent cardiological evaluation, echocardiogram, pulmonary function tests (PFT) both atby 3 and by 12 months after discharge. Chest CT was performed by 12 months after discharge and chest CT. Specifically, the magnitude of pulmonary involvement between baseline and follow-up was considered the primary endpoint of this study. Secondary endpoints of the study were results of respiratory function testing, echocardiographic parametersparameters, and persistence of symptoms.


Eligibility Criteria:
Inclusion Criteria:

* Age ≥ 18
* Hospitalization for severe COVID-19 pneumonia
* Computed tomography during index hospitalization that showed: (i) normale lung parenchyma \< 50% of total lung volume and/or (ii) parenchymal consolidation \> 10%
* Cardiological and pneumological visit, echocardiography and pulmonary function test at 3 and 12 months from hospital discharge
* Computed tomography by 12 months from hospital discharge

Exclusion Criteria:

* Age \< 18
* Absence of previously cited test.
Evaluation of the Use of Levemir® in Insufficiently Controlled Patients With Type 1 or Type 2 Diabetes

This study conducted in Europe. The aim of this study is to evaluated the use of insulin detemir (Levemir®) in insufficiently controlled patients with type 1 or type 2 diabetes.


Eligibility Criteria:
Inclusion Criteria:

* Patients with insufficiently controlled type 1 or type 2 diabetes mellitus under their previous therapy
Feasibility Study of Duloxetine in the Treatment of Depression in Patients With Traumatic Brain Injury

The primary objective of the study is to compare the efficacy of duloxetine 60 mg by mouth daily with placebo in the prevention of depression associated with mild/moderate traumatic brain injury and to enhance cognitive function.

The primary objective of the study is to compare the efficacy of duloxetine 60 mg by mouth daily with placebo in the prevention of depression associated with mild/moderate traumatic brain injury and to enhance cognitive function. Research exploring the use of selective serotonin reuptake inhibitors in the treatment of post-traumatic depression generally validates this approach (Horsfield et al., 2002). However, the literature suggests that serotonin/norepinephrine reuptake inhibitors such as duloxetine may be more effective in the treatment of depression.

Eligibility Criteria:
Inclusion Criteria:

* Study participants will be 40 men and women between the ages of 18 and 75 who provide appropriate consent and who are agreeable to study requirements
* Diagnosed with mild to moderate traumatic brain injury as defined by an initial Mayo Traumatic Brain Injury Severity Scale
* Have memory impairments defined by a Hopkins Verbal Learning Test delayed recall score which falls less than or equal to 1.5 standard deviation below the mean.

Exclusion Criteria:

* Refusal to give informed consent
* A previous Central Nervous System illness or injury, including seizure that exhibits residual symptoms.
* Current post-traumatic seizure disorder
* A previous diagnosis of a psychotic disorder
* Current or previous (in the last 6 months) treatment history for alcohol or substance dependency
* Medications affecting noradrenergic or dopaminergic systems, alpha-adrenergic antihypertensives, antidepressant, phenobarbital, Monoamine oxidase inhibitor (MAOI), scheduled benzodiazepines, psychoactive herbal supplements (including Kava, St. John's wort), or nutritional supplements or within at least 14 days of discontinuing treatment with the above medications or supplements.
* A known suicide risk
* A pregnant or breastfeeding woman
* Uncontrolled narrow-angle glaucoma
* Serious and/or unstable medical comorbidity (e.g., AIDS, cancer, history of uncontrolled hypertension or cardiovascular disease) psychological condition, or clinically significant laboratory abnormality that in the opinion of the investigator would compromise participation in the study or be likely to lead to hospitalization during the course of the study
* Liver enzymes \> 1.5 times upper limit of normal
* Patients with end-stage renal disease (requiring dialysis) or severe renal impairment
* Known hypersensitivity to duloxetine or any of the inactive ingredients
Oxygen Therapy Remote Monitoring in COPD Patients.

Background : Long-term oxygen therapy is prescribed for patients with severe COPD. The aim of oxygen therapy is to administer oxygen at a concentration level higher than that of ambient air, in order to treat or prevent the symptoms and manifestations of arterial hypoxemia; this therapy can be applied either in an acute situation or as a long-term treatment, in cases of stabilized severe chronic hypoxemia.

Currently, pneumologists do not have the possibility, between 2 consultations spaced several months apart, to measure the patient's compliance with the treatment (adherence to oxygen therapy) nor the respect of the prescribed dosage (O2 flow and duration). In addition to the patient's adherence to the treatment, the specialist does not have the possibility to assess the patient's physical activity (walking ...).

This observational study is a e-health, prospective, multicenter study conducted in France under the control of pneumologists.

The main objective of this study is to evaluate the adherence to oxygen therapy in real life, and its evolution, in COPD patients justifying a long term oxygen therapy in stable state.

The observational study consists in collecting physiological data (cardiorespiratory and physical activity) and use of the oxygen therapy device in real life during the first months after the initiation of treatment.

For this purpose, two connected medical devices will be provided to each patient during 2 periods of 2 weeks (weeks 6 to 8 then weeks 12 to 14 after the initiation of oxygen therapy). The devices are: 1/ a connected watch that continuously measures vital parameters (heart rate and blood oxygen saturation), and physical activity parameters (no. of steps, distance, duration of effort), and 2/ a Teleox device that remotely monitors patients equipped with an oxygen source (it measures the oxygen flow rate requested by the patient, the duration of use of the oxygen source, and the breathing rate).

Study design: a cohort of 250 patients with COPD newly initiated a long term oxygen therapy will be enrolled in the study and follow-up over 6 months. Data will be collected by lung specialists and home health care provider teams at 2 months, 3.5 months and 6 months post-initiation of oxygen therapy.

Eligibility Criteria:
Inclusion Criteria:

* COPD patients who require the initiation of a long term oxygen therapy.
* Patient agreeing to use a connected watch and a TeleOx for the remote monitoring of their parameters and physical activity.
* Non-smoker, ex-smoker, or patient agreeing to stop smoking.
* Life expectancy greater than 6 months.
* Signed informed consent form,
* Subject affiliated to a health insurance system, or is a beneficiary.

Exclusion Criteria:

* Patient initiated on oxygen therapy prior to the inclusion visit.
* Patient with cognitive impairment.
* Simultaneous participation in a health intervention research,
* Vulnerable subjects.
Prognostic Value of a Diaphragmatic Endurance Test in Patients With Amyotrophic Lateral Sclerosis

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease which involves respiratory muscles and can lead at short term to respiratory failure. The occurrence of respiratory failure is associated with morbidity and an increased mortality. To date, respiratory muscle weakness is predicted from the reduction of vital capacity, maximal inspiratory force, nocturnal symptoms and hypercapnia. Even taken together, the predictive value of these indices is low.

The investigators hypothesize that an endurance test of diaphragmatic work would be more sensitive to respiratory muscle involvement than maximal respiratory force.

Consequently, the investigators assessed diaphragmatic performance through an isocapnic hyperventilation test (IHT) in patients at the onset of ALS and, then regularly up to the occurrence of respiratory failure. The investigators make the hypothesis that IHT will be altered earlier than maximal inspiratory force


Eligibility Criteria:
Inclusion Criteria for Patients :

* Older than 18 years old and younger than 80 years old
* ALS patient seen during the diagnostic assessment

Inclusion Criteria for control group :

* Older than 25 years old and younger than 80 years old
* No respiratory or neurologic active pathology

Exclusion Criteria for Patients :

* Bulbar ALS (inability to perform maximal respiratory maneuvers)
* Dementia
* Respiratory failure at diagnosis (arterial carbon dioxide partial pressure (pCO2) \> 45 mmHg)
* Respiratory or neurologic active pathology

Exclusion Criteria for control group :

* Chest wall deformation with spirometric defect
The Rate of Successful Intubation and the Required Time for Intubation, Using Vie Scope and Macintosh Laryngoscope Will be Compared, in Order to Evaluate the Efficacy of Vie Scope in Comparison With Macintosh in Expected Easy Airways.

The rate of successful intubation and the required time for intubation, using Vie Scope and Macintosh Laryngoscope will be compared, in order to evaluate the efficacy of Vie Scope in comparison with Macintosh in expected easy airways. Hence, a prospective randomized controlled single-blind superiority clinical trial will be conducted.

Inclusion criteria: patients of both sexes, positive or negative rapid test for COVID-19 or possible contamination, age ≥18 years old, BMI: 18.5 - 30 kg/m2, visualization of the glottis grade 1 or 2, according to Cormack-Lehane Classification.

Exclusion criteria: age under 18 years old, BMI under 18.5 kg/m2 and BMI over 30 kg/m2, visualization of the glottis grade 3 - 4, according to Cormack-Lehane Classification.

The laryngoscopy and intubation of the patients will be attempted by the same person-resident of Anesthesiology, under the supervision of experienced Anesthesiologist consultants.


Eligibility Criteria:
Inclusion Criteria:

* Patients of both sexes
* Positive or negative rapid test for COVID-19 or possible contamination
* Age ≥18 years old
* BMI: 18.5 - 30 kg/m2
* Visualization of the glottis grade 1 or 2, according to Cormack-Lehane Classification.

Exclusion Criteria:

* Age under 18 years old
* BMI under 18.5 kg/m2 and BMI over 30 kg/m2
* Visualization of the glottis grade 3 - 4, according to Cormack-Lehane Classification.
Evaluation of Novel Lung Function Parameters and Quantitative Computed Tomography (qCT) in Patients With Pulmonary Disease

Current diagnostic tools used in pulmonary disease often do not meet the challenges set by the respective pathophysiology. The investigators therefore aimed to evaluate novel or not widely used diagnostic approaches for the detection and therapeutic monitoring of patients with various pulmonary diseases.


Eligibility Criteria:
Inclusion Criteria:

* known or suspected pulmonary lung disease
* indication for computed tomography

Exclusion Criteria:

* pregnancy
* inability to participate
Home-based Psychoeducation for Older Adults With Frailty: A Randomised Controlled Trial

This study aims to examine the effectiveness of a 12-week home-based telerehabilitation programme on improving subjective well-being among community-dwelling older people with frailty.

The main question it aims to answer is

- Could home-based psychoeducation significantly enhance subjective well-being in older adults with frailty in the community?

Participants will receive 12 weekly online group-based sessions at their homes. The content for the experimental group and control group is different:

* Intervention group: psychoeducation
* Control group: physical health education Participants will receive three home visits for data collection. Some of participants in experimental group will receive interview for process evaluation.


Eligibility Criteria:
Inclusion Criteria:

(1) Aged 65 or older; (2) Identified as frail by the FRAIL scale (score ≥3); (3) Mentally competent as determined by the Abbreviated Mental Test (AMT ≥6); (4) Able to speak and understand Cantonese; (5) Living at home; (6) Experienced in using a smartphone (e.g., sending messages, watching videos).

Exclusion Criteria:

(1) Have visual or hearing impairments or language barriers that may impact communication or understanding; (2) Unfit for home-based exercise, as indicated by a score of 1 or higher on the 25-item HOME-FAST, a tool for identifying home fall hazards; (3) Currently engaging in exercise for at least 150 minutes per week over the past four weeks; (4) Undergoing active psychiatric or antidepressant treatment or participating in other physical exercise or rehabilitation programs.
HRQoL Keratinocyte Carcinomas

Rationale: Keratinocyte skin cancer is known to influence the HRQoL in a specific way. Derived utility values are required for cost-effectiveness analyses of new interventions. However there is no sensitive tool to capture HRQoL that translates into utilities available.

Objective: To document the exact HRQoL in patients with in KC using the generic EQ-5D-5L questionnaire, as well as the TTO, 15D and the BaSQoL questionnaire, and to develop health utilities based on these tools.

Study design: Longitudinal observational study (at time 0 and time 0 +12 months).

Study population: Patients aged ≥18 years consulting a dermatologist or their GP for diagnosis, treatment or follow-up of a (pre)malignant skin lesion(s).

See protocol

Eligibility Criteria:
Inclusion criteria

1. Patients with one or more premalignant lesion or KC diagnosed by a dermatologist or by histopathology untill 10 years previous to inclusion.

Exclusion criteria

1. Patient is younger than 18 years old or unable to provide consent.
2. Patient is unable to remember the diagnosis or symptoms of their KC or premalignant lesion upon request by the doctor.
3. Patient was diagnosed with a melanoma or another NMSC.
4. Patient is unable to understand the task and questionnaires.