cyrilzakka/clinical-trials huggingface dataset:
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation

The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation, and being treated with Orkambi


Eligibility Criteria:
Inclusion Criteria:

* Willing and able to use the delivery device as directed by the study manual.
* Confirmed diagnosis of CF, defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis.
* Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result is not received before randomization, a previous CFTR genotype lab report may be used to establish eligibility.
* Percent predicted forced expiratory volume at one second (FEV1) of ≥40 to \<90 percentage points adjusted for age, sex, and height according to the Global Lung Initiative (GLI) at the Screening Visit.
* Willing to discontinue physician-prescribed saline use.
* Female subjects of childbearing potential with a negative serum pregnancy test at the Screening Visit.

Exclusion Criteria:

* History of any comorbidity, which in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
* Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
* A 12 lead ECG demonstrating QTcF \>450 msec at the Screening Visit.
* History of solid organ or hematological transplantation.
* Used diuretics or renin-angiotensin aldosterone system antihypertensive drugs in the 28 days prior to Screening or an anticipated need for any of these medications during the study.
* Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit.
* Inability to withhold short-acting, long-acting, or once-daily, long-acting bronchodilator use for 4, 12, or 24 hours prior to clinic visit, respectively.
* History of significant intolerance to inhaled saline, or intolerance to the single dose of saline at Screening
* Known hypersensitivity or history of intolerance to Orkambi.
* Pregnant and nursing females.
* Subjects who have participated in Parion Sciences Study NCT02343445.
A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants

X-linked adrenoleukodystrophy is a rare, demyelinating and neurodegenerative disorder, due to a loss of function of a fatty acid transporter, the peroxisomal ABCD1protein. Its more frequent phenotype, the adrenomyeloneuropathy in adults, is characterized by axonal degeneration in spinal cord, spastic paraparesis and a disabling peripheral neuropathy. Actually, there is no efficient treatment for the disease. Our work in the last twelve years dissecting the physiopathological basis of the disorder has uncovered an involvement of the oxidative stress early in the neurodegenerative cascade. In a preclinical trial we have identified an antioxidant cocktail that efficiently reverse the clinical symptoms and the axonal degeneration in the mouse model for the disease. We propose the translation of the results to an open trial to test the tolerance and effectiveness of these drugs in the correction of the previously identified oxidative lesion biomarkers, as a first step to a randomized versus placebo, multicentric and international trial. You will be clinically explored and assessed in the Hospital Universitari of Bellvitge (HUB) using clinical scales for spasticity, disability, electroneurogram and cranial and spinal Nuclear Magnetic resonance (NMR). The information will be collected in a data base that will be of great value to improve the present attention and the future follow-up to facilitate your inclusion in therapeutic randomized, double blind, against placebo clinical trials.


Eligibility Criteria:
Inclusion Criteria:

* Symptomatic AMN patients,
* 18-64 years old,
* male and female,
* clinically and biochemically diagnosed;
* females must be obligated heterozygotes or must have gene mutation identified.

Exclusion Criteria:

* Pregnant and lactation in females,
* Cerebral inflammatory disease with cognitive disorder, and/or
* need the help of two walking sticks,
* epilepsy,
* hypersensibility to cysteine related compounds,
* transaminases 2 fold up normal values.
Nalox-Comm: Naloxone Communication Training for Pharmacists

This is a pilot Randomized Controlled Trial (RCT) in which 120 pharmacists will be randomized to an experimental or control group and data on naloxone dispensing and secondary outcomes will be collected over the course of the RCT.

This is a prospective pilot RCT that will evaluate the impact of naloxone communication training (Nalox-Comm) on 120 pharmacists' naloxone dispensing behaviors (primary outcome). Data will be collected at baseline, immediately after training is completed, and at 3-month follow up. Data sources include pharmacy records (for naloxone dispensing), simulated patient observations (to rate quality of communication), and survey data (for self-reports of knowledge and self-efficacy).

Eligibility Criteria:
Inclusion Criteria:

* currently work at a pharmacy that stocks naloxone;
* currently work at a rural community pharmacy;
* are at least 18 years of age; and
* speak English.

Exclusion Criteria:

* Non-staff pharmacists such as pharmacy "floaters" or fill-in pharmacists will not be eligible to participate.
Virtual Reality-based Rehabilitation

Vertigo / dizziness, imbalance and other symptoms related to vestibular pathology have a life prevalence of 7.4%. Peripheral and unilateral vestibulopathy are among the most common types, and in some patients the symptoms become chronic and disabling, which makes it difficult for them to lead a normal life and has a significant socio-economic impact. In recent years, the effect of vestibular rehabilitation with virtual reality has been studied and positive results have been obtained when compared with traditional rehabilitation, but despite rehabilitation there is patients who are still chronic, and the reasons for this prognostic variability are still unknown and require further treatment. Virtual reality-based stimulation can play a significant role as an adjunct to conventional vestibular rehabilitation, improving its effectiveness.


Eligibility Criteria:
Inclusion Criteria:

* Unilateral vestibular deficit
* Vestibular neuritis
* Sudden hearing loss with vestibular involvement
* Labyrinthitis
* Ménière's disease
* Vestibular Shwannoma
* Benign paroxysmal positional vertigo
* Vestibular symptoms (vertigo, dizziness, imbalance, gait instability, kinosis and / or oscillopsy) chronic (more than 6 weeks of evolution)
* Over 18 years old

Exclusion Criteria:

* Failure to meet some of the inclusion criteria
* Neurological, traumatological, rheumatological, ophthalmological or systemic pathology that may interfere with the balance
* Inability to understand participation in the study
* Non-acceptance to participate in the study
A Multinational Trial of the Efficacy of Albendazole Against Soil-transmitted Nematode Infections in Children

The three major Soil-Transmitted Helminths (STH), Ascaris lumbricoides, Necator americanus/Ancylostoma duodenal and Trichuris trichiura are among the most prevalent parasites worldwide. The objective of this multicentre international study is to define the efficacy of a single 400 milligram dose of albendazole (ALB) against these three STHs using a standardised protocol. The trial will be undertaken among school age children in seven countries - Brazil, Cameroon, Cambodia, Ethiopia, India, Tanzania (Zanzibar) and Vietnam - each with a different epidemiologic pattern of infection. A trial of this nature is urgently required because in spite of the wide usage of albendazole over the last 3 decades, there is still no key publication reporting the efficacy of the anthelmintic accurately, and to modern conventional standards, that can act as a central reference for the baseline efficacy. The latter is critically important because albendazole is now being used even more widely, as large scale mass treatment campaigns are being implemented in Africa and elsewhere, with the intention of reducing morbidity in children. Such large scale usage of a drug risks resistance developing, but resistance cannot be detected unless benchmark values for baseline efficacy are widely known.


Eligibility Criteria:
Inclusion Criteria:

* Children should be infected with a minimum of 150 eggs/gram of any of the three species of STH (i.e. Ascaris or hookworms or Trichuris).

Exclusion Criteria:

* Not willing to participate
* Unable to give samples for follow up
* Severe intercurrent medical condition
* Diarrhoea at first sampling
Invasive Candidiasis in Critical Care

The combination of acute phase marker monitoring and the "T2Candida" assay (name of the test) will represent an acceleration of the identification of the causative agent of mycotic infection, a significant improvement in the specificity and positive predictive value of this strategy in the diagnosis of invasive candidiasis and candidemia in ICU patients, thereby improving the clinical condition of patients and reducing the cost of specific antifungal therapy.

Speed of response in the treatment of sepsis is crucial for the patient. The time from the collection of a positive haemoculture to the identification of the causative agent of sepsis is around 2 days; therefore, physicians in intensive care units deploy combined empiric antibiotic and antifungal therapy immediately when acute phase markers such as procalcitonin, interleukin-6, Presepsin, C-reactive protein are elevated. A new acute phase marker is lipopolysaccharide-binding protein, which, together with Presepsin, appears to be a suitable marker to distinguish invasive candida infections from bacterial infections. But its kinetics needs to be further analyzed.

At the same time, the causative agent of sepsis, G-/G+ bacteria or yeast, must be identified as soon as possible. Haemoculture and culture of the established drain is the gold standard, but the disadvantage is the low sensitivity and the time delay to obtain the result. It is therefore advisable to combine haemoculture with molecular biology-based tests that can identify the causative organism within hours. Conversely, the disadvantage of these tests is that they identify only the most common sepsis pathogens and do not determine susceptibility to antibiotics and antifungals, but the advantage is that with prophylaxis in place, these tests are often positive when haemoculture is negative. The T2Candida test can detect Candida albicans, Candida tropicalis, Candida glabrata, Candida krusei and Candida parapsilosis, which are the more common causative agents of mycotic bloodstream infections.

Eligibility Criteria:
Inclusion Criteria:

* critically ill patients
* new onset sepsis
* rise in body temperature \>38°C according to The Third Consensus Definitions for Sepsis and Septic Shock
* colonization with Candida spp. from more than 1 non-sterile site
* body temperature \>38 °C despite 5 days of broad-spectrum antibiotic therapy with the presence of at least 1 of the following risk factors: abdominal surgery, secondary peritonitis, pancreatitis, central venous catheter (CVC) insertion, total parenteral nutrition (CPV), dialysis, steroid therapy, immunosuppressive therapy, or liver transplantation
* microbiological test results will be reviewed and categorized based on whether Candida sp. is isolated from at least 2 non-sterile sites (±3 days) and whether there is an alternative microbiological diagnosis.

Exclusion Criteria:

* not signing the informed consent with participation in the study
* administration of antifungal therapy prior to collection of the biological material required for the study
A Working Memory Training to Decrease Rumination in Depressed and Anxious Individuals

Dysfunctioning executive functioning, including working memory (WM), is related to rumination. Findings show that working memory capacity (WMC) can be increased by training. The current study explored by means of a double-blind randomized controlled trial whether an adaptive WM training could reduce rumination, anxiety and depression in a sample of 98 depressed and anxious individuals.


Eligibility Criteria:
Inclusion Criteria:

* Major depression diagnosis
* Anxiety diagnosis

Exclusion Criteria:

* Current psychosis
* Substance abuse
Virtual Reality for Non-cardiac Chest Pain

The purpose of this study is to determine if virtual reality (VR) will improve symptoms in non-cardiac chest pain (NCCP).

The primary aim of this study is to assess the overall feasibility of virtual reality for the treatment of noncardiac chest pain and identify problems, issues, or challenges that may impact the performance of a future clinical trial. As such, the study will focus on willingness to participate, patient adherence, patient retention, and burdensomeness of the intervention and survey instruments. As a lesser objective, we will assess the effectiveness of virtual reality in patients with noncardiac chest pain at improving pain severity, symptom hypervigilance, anxiety, quality of life, and resilience.

Eligibility Criteria:
Inclusion Criteria:

* Symptoms of non-cardiac chest pain (recurrent anginal-like or atypical chest pain)
* Negative prior cardiac evaluation (at a minimum, including electrocardiogram)
* Prior esophagogastroduodenoscopy and/or barium esophagram to rule out severe esophagitis, stricture, or motility abnormality

Exclusion Criteria:

* Initiation of a proton pump inhibitor (PPI) within 8 weeks
* Major concomitant illness
* Current drug or alcohol use that that would interfere with adherence to study requirements
* Symptoms of vertigo or dizziness that would limit tolerability of the VR headset
Surveillance Pelvic Examination Anxiety: Brief Coping Skills Intervention

The purpose of this study is to test the feasibility and acceptability of a brief coping skills training program addressing anxiety and pain related to surveillance pelvic examinations for female gynecologic cancer survivors.

The investigators have developed and manualized a coping skills training intervention for female gynecologic cancer patients that aims to reduce anxiety related to surveillance pelvic examinations (Pelvic Examination and Anxiety Coping Skills for Empowerment: PEACE). The investigators propose a proof-of concept pilot study to evaluate the acceptability and feasibility of the coping skills intervention. The study will be conducted by Duke University. Participants (N=16) will receive the 3-session telemedicine coping skills intervention. Participants will complete assessments at baseline, post-intervention (within 2 weeks before their next scheduled pelvic examination), and post pelvic examination (2-3 months after the baseline assessment). Study aims are: Aim 1) Assess intervention acceptability and feasibility through quantitative measurement and qualitative feedback from semi-structured interviews, and Aim 2) Graphically depict patterns of change in outcome variables and intervention targets.

Eligibility Criteria:
Inclusion Criteria:

* female sex
* diagnosis of stage I-IV cervical or endometrial cancer
* 18 years of age or older
* able to speak/read English
* able to give informed consent
* completed cancer treatments within 2-24 months prior to enrollment, and recommended to undergo surveillance pelvic examination at three month intervals
* able to commit to three 45-60 minute visits within the study period
* able to participate in the intervention via the telemedicine modality (e.g., phone or video)

Exclusion Criteria:

* unable to provide informed consent
* major untreated or uncontrolled mental illness
* hearing impairment that impedes telephone or video calls
D-amino Acid Oxidase Inhibition (DAAOI-1) add-on Treatment for Chronic Schizophrenia

Adjuvant N-methyl-D-aspartic acid (NMDA)-enhancing agents, such as GlyT-1 inhibitors and NMDA-glycine site agonists have been demonstrated to be beneficial for chronic schizophrenia patients. The purpose of this study is to evaluate efficacy and safety of add-on treatment of an inhibitor of D-amino acid oxidase (DAAOI), DAAOI-1, in chronically stable schizophrenia patients who have been stabilized with antipsychotics.

The etiology of schizophrenia remains unclear. Schizophrenia patients reveal positive symptoms, negative symptoms, and cognitive impairments. In addition to dopamine system hyperactivity, hypofunction of N-methyl-D-aspartate (NMDA) receptor plays a role in the pathophysiology of schizophrenia. Consequently, enhancing NMDA receptor neurotransmission has been regarded as a novel treatment approach. To date, several reported trials on adjuvant NMDA-enhancing agents, including glycine, D-amino acids (D-serine, D-alanine), and sarcosine (a glycine transporter I inhibitor), revealed beneficial but limited efficacy for positive and negative symptoms.

DAAOI-1 is a D-amino acid oxidase (DAAO) inhibitor which can elevate synaptic concentration of D-amino acids. The aim of this project is to examine the efficacy and safety of add-on treatment of DAAOI-1 in chronically stable schizophrenia patients who have been stabilized with antipsychotics.

In the study, 60 schizophrenic patients are recruited into the 6-week trial and randomly assigned into the two groups (1 gm/dDAAOI-1, or placebo) with a double-blind manner. Positive and Negative Syndrome Scale (PANSS), Scales for the Assessment of Negative symptoms (SANS), Global Assessment of Function (GAF), quality of life (QOL), Hamilton Depression rating scale 17(HAM-D 17), Clinical Global Impression(CGI)and side effects are evaluated every two weeks during the trial. Cognitive function ("7 domains of Measurement and Treatment Research to Improve Cognition in Schizophrenia" \[MATRICS\])are assessed at weeks 0 and 6. The efficacies of two groups are compared.

Eligibility Criteria:
Inclusion Criteria:

* Are physically healthy and have all laboratory assessments (including urine/blood routine, biochemical tests, and electrocardiograph) within normal limits
* Aged 18-65 year
* Fulfill the criteria of schizophrenia according to the Diagnostic and Statistical Manual, fourth edition (DSM-IV)
* Remain symptomatic but without clinically significant fluctuation and the antipsychotic doses are unchanged for at least 3 months
* Have a minimum baseline total score of 60 on the Positive and Negative Syndrome Scale (PANSS)
* Agree to participate in the study and provide informed consent

Exclusion Criteria:

* DSM-IV diagnosis of substance (including alcohol) abuse or dependence,
* DSM_IV diagnosis of mental retardation
* History of epilepsy, head trauma or CNS diseases
* History of epilepsy, head trauma or CNS diseases
* Pregnancy or lactation
* Inability to follow protocol
Study on the Effect of WAFF Pelvic and Abdominal Mechanics Exercises During the Puerperium on Postpartum Pelvic Floor Rehabilitation for Parturients

Ethics Review Committee, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

Currently, in order to improve women's health throughout their entire life cycle, postpartum rehabilitation for parturients has received significant attention.

Currently, clinical postpartum rehabilitation mostly adopts a combined treatment of Kegel exercises, physical electrical stimulation, and biofeedback therapy. Kegel exercises can be applied during the puerperium. However, studies have found that some parturients find it difficult to locate the muscle contraction sensation during exercise, resulting in poor technique and effectiveness; furthermore, the training process is boring and difficult to maintain. Physical electrical stimulation and biofeedback therapy have significant therapeutic effects, but this treatment should be initiated after the puerperium and requires hospital visits, often leading to discontinuation due to long distances and time-consuming commutes.

Many experts domestically and internationally have stated that earlier postpartum rehabilitation leads to better outcomes. However, studies show that only a small percentage of patients are aware of the correct timing for postpartum rehabilitation and are able to undergo rehabilitation exercises during the puerperium. Most current studies initiate postpartum rehabilitation training from 6 to 8 weeks postpartum, with relatively few reports on pelvic and abdominal muscle training during the puerperium.

Therefore, we have introduced French WAFF pelvic and abdominal mechanical exercises and applied them to the rehabilitation of women during the puerperium. WAFF pelvic and abdominal mechanical exercises are safer, more precise, and more convenient, utilizing the instability created by the WAFF air cushion to stimulate patients' self-regulatory movements, thereby achieving a tighter core and deeper muscle groups to improve training efficiency.

Currently, the application of WAFF pelvic and abdominal mechanical exercises in China is still in the preliminary exploratory stage, and a unified and mature rehabilitation model has not yet been established. This study aims to explore the effectiveness of applying WAFF pelvic and abdominal mechanical exercises during the puerperium to improve postpartum rectus abdominis diastasis and pelvic floor dysfunction. It provides a basis for establishing a new model of puerperium rehabilitation and improving continuous obstetrical nursing.


Eligibility Criteria:
Inclusion Criteria:

* primipara
* Singleton pregnancy
* With full cognitive and behavioral abilities, and voluntarily participating

Exclusion Criteria:

* Neonate with a birth weight of \>4000g
* Second-degree or higher perineal tear
* Poor healing of perineal wounds
* Individuals with severe hypertension, impaired cardiopulmonary function, and neurological diseases
* History of previous pelvic floor disorders and urogenital surgeries
* Individuals engaging in other exercises such as yoga during the puerperium
Swiss ProspectivE Cohort of TRAnsgender and Gender Diverse Individuals - the SPECTRA Study

The goal of this observational study is to understand the factors influencing the well-being and health of transgender and gender-diverse (TGD) individuals undergoing gender-affirming therapies in Switzerland. The study population includes TGD individuals aged 16 and older, at various stages of their medical transition, including those who have discontinued or detransitioned.

The main question is:

What are the key factors influencing the well-being and health outcomes of transgender and gender-diverse individuals undergoing gender-affirming therapies?

Researchers will compare health outcomes across different subgroups, such as those at different stages of transition or detransition, to examine the effects of gender-affirming therapies.

Participants will:

Complete electronic questionnaires assessing gender congruence, quality of life, mental health, and other outcomes.

Provide biological samples (e.g., blood, urine, stool, and skin swabs) for laboratory analysis.

Undergo clinical evaluations related to endocrinology, fertility, dermatology, and urology as part of their routine follow-up.


Eligibility Criteria:
Inclusion Criteria:

* Age 16 years and above.
* Established diagnosis of gender incongruence
* Indication for gender affirming therapy or ongoing or discontinued gender affirming therapy

Exclusion Criteria:

* Unable to give informed consent
* Missing commitment or ability to study protocol adherence