Active clinical trials for "Syndrome"

Alport syndrome (AS) is the second most common monogenic cause of end-stage renal failure (ESRF). AS is caused by variants in the COL4A3, COL4A4, and COL4A5 genes, which encode for the a3, a4, and a5 chains of type IV collagen. This trial is a prospective, randomized, controlled and multicenter trial. Mainly to assess the safety and efficacy of ramipril in Alport syndrome patients with variants of COL4A3/COL4A4/COL4A5.

Insulin resistance (IR) is an important pathological feature of polycystic ovary syndrome (PCOS), with an incidence rate of up to 85%, which seriously affects the patient's fertility, quality of life, and offspring health, but the mechanism is unknown. The adaptor protein LNK is closely related to metabolic diseases. Our exome sequencing has found that the mutation rate of LNK gene in patients with PCOS and IR is high. Studies have found that LNK can affect adipose inflammation and impair glucose tolerance. Whether LNK is related to fat metabolism is worth further study. Our previous research found that: LNK expression was significantly increased in adipose tissue of patients with PCOS and IR. Knockout of LNK in PCOS IR model mice can reduce serum triglycerides, free fatty acids, high-sensitivity C-reactive protein levels and reduce fatty liver occurrence, which indicates that LNK has a mitigating effect on IR. Mechanism studies have shown that LNK knockout can upregulate the glucose transporter Glut4, also LNK and insulin receptor substrate IRS-1 can form protein complexes. Based on the above research basis, we propose the following scientific hypothesis: LNK in adipose tissue can regulate insulin signaling pathway by binding to IRS-1, downregulate Glut4, and participate in PCOS IR occurrence. This project intends to clarify the specific mechanism by which LNK regulates glucose transport and participate in IR in combination with clinical specimens, animal models and cell experiments, and provide scientific basis for LNK as a potential therapeutic target for PCOS IR.

The primary endpoint is to determine if ECP induces a decrease in % of tumor cells after treatment. 15 patients with Sezary Syndrome will receive ECP weekly x4, then bi-weekly for 5 months. Each patient will donate 5 samples to determine immune responses in peripheral blood. Additional clinical assessments will be a modified skin weighted assessment and flow cytometry at baseline and months 3 and 6. A CT scan will be obtained at baseline and only repeated if pathology is present at baseline. The tumor microenvironment will be studied by comparing transcriptomics of the blood samples before, 1 day after first ECP treatment, cycle 1, 1, 3 and 6 months after ECP treatment by scRNAseq (5 samples total per patient ).

Identify circulating protein-coding (mRNAs) or non-coding (ncRNAs) transcripts (ACS_signature) predictive of ventricular dysfunction in ACS patients undergoing PCI.

ARDS is an acutely induced respiratory failure characterized by the appearance of bilateral alveolar opacities on imaging and hypoxemia Etiologies are divided into two classes: pulmonary, including all infectious pathologies, aspiration pneumonia, and drowning, and extra-pulmonary, induced by sepsis or acute pancreatitis. The mortality rate of ARDS remains high in unselected patient populations Among strategies that have proven beneficial in terms of patient outcome, prone positioning (PP) is associated with the greatest impact in terms of reduction in mortality. PP is currently recommended in the European guidelines for ARDS associated with a PaO2/FiO2 ratio < 150 mmHg in patients in whom ventilatory settings have been optimized beforehand, The failure of early PP studies to demonstrate a survival benefit in ARDS was attributed to insufficient session duration. The PROSEVA study was the first to demonstrate that a PP duration of 17 h is associated with a reduction in mortality During the COVID-19 pandemic, several centers have reported the implementation of longer PP sessions. Two strategies have emerged from these studies. In one case, the patient was left in the prone position until the criteria for stopping PP were met. Thus, the PP/supine position alternation was completely suppressed. In another published strategy, PP sessions were maintained for a period covering two nights. Furthermore, in a multicenter retrospective study, PP sessions were maintained until clinical improvement was associated with reduced mortality. In this study of 263 patients, the median duration of PP in the extended duration group was 40 h, and 75% of the sessions lasted 48 h or less. Using a propensity score, the authors showed that patients treated with an extended PP duration had a lower 3-month mortality rate than patients in the standard duration group . This protocol was also associated with a 29% cumulative incidence of pressure sores, similar to the 25% cumulative incidence reported in the PROSEVA study Other data published on pressure sores and PP of duration > 24 hours are also reassuring. Finally, a recent review recently reported that an extended PP session of > 24 h had also been used before the COVID-19 pandemic. PP sessions had a median duration of 47-78 hours and were applied mainly to ARDS secondary to community-acquired pneumonia. All pre-COVID studies were retrospective, monocentric, without a control group.

TMS has been safely and reliably delivered at the Harquail Centre for over 5 years, with a primary focus on conventional rTMS protocols for treatment-resistant depression. Recently, the investgator team has gained the capability to deliver sham-controlled intermittent theta-burst stimulation (iTBS) rTMS. Unlike conventional high frequency rTMS, which was used in the previous sham-controlled rTMS PCS pilot study, iTBS is a patterned form of stimulation that recapitulates endogenous activity patterns of neural circuits pairing gamma frequency (50Hz) burst pulses coupled in a theta frequency rhythm (5Hz).12 iTBS delivers 600 pulses in just over 3 minutes with similar or greater effects on neural plasticity compared to conventional rTMS (taking over 30-45 minutes) and similar tolerability and efficacy in trials of depression. Furthermore, novel accelerated iTBS protocols stimulating the left dorsolateral prefrontal cortex (dlPFC) over 8-10 treatments, 50 minutes apart over a 5 day interval has recently demonstrated robust efficacy in depression and received recent FDA approval. Thus, accelerated iTBS can be delivered in a single week of treatment compared to 6 weeks with conventional rTMS methods. Finally, the investigators recently acquired the technology to integrate MRI neuroimage-guided stimulation, which would allow to target specific brain regions/networks implicated in PCS at high spatial resolution. No studies to date have investigated image-guided accelerated iTBS rTMS for the treatment of PCS.

Over the last years a rising medical need for treatment of chronic pain was identified. Based on previous findings indicating the pain modulating effects of cannabinoids in chronic pain disorders, this clinical trial investigates the long term efficacy and tolerability of the THC-focused nano endocannabinoid system modulator AP707 in patients with pain disorders due to diabetic polyneuropathy. Patients receive AP707 or placebo over the course of 14 weeks as an add-on to the standard of care. Changes in pain intensity, quality of life and sleep and others measures are monitored through different scales to assess the efficacy of AP707 in patients with chronic pain due to diabetic polyneuropathy.

The goal of this study is to examine the safety and treatment effects of sirolimus for targeting social communication deficits in people with genetic disorders associated with PTEN germline mutations, which are often referred to as PTEN Harmartoma Tumor Syndrome (PHTS). The mechanism of sirolimus in the body has shown promise for helping to improve social communication skills in case reports of people with PHTS. Everolimus, a closely related compound, also showed benefits in social communication skills in a previous pilot trial in people with PHTS. This is a 6 month double-blind trial followed by at 6 month open label extension trial.

The goal of this study is to learn about the effect of licorice root powder on parameters of metabolic syndrome in those women diagnosed with PCOS with age range of 15-49 years of age. The study aims to answer the following questions: Will licorice root powder consumption will have an impact on parameters of metabolic syndrome which are HDL levels, triglycerides, fasting blood pressure and blood glucose levels and abdominal obesity related to PCOS. Will licorice help in lowering stress scores and improve quality of life. Females with diagnosed PCOS will be recruited in the study and will be randomly allotted to two groups. One group will be intervention group and second will be control group. Participants in intervention group will be given 1000mg of licorice root powder in form of capsules (1 per day). Control group will not take anything. For both groups, SF-12 health survey, and readings of lipid profile, fasting blood glucose and blood pressure and central obesity will be taken. Researcher will compare readings of both groups at baseline and after 3 months of intervention to assess the effect of licorice root powder.

Platelet-rich plasma (PRP) is autologous blood which has been centrifuged to separate the supernatant resulting in a solution with high concentration of platelets. Injection of PRP stimulates cell growth and can has been proven safe and effective for uses in orthopedics, dermatology, and gynecology. Despite the relative paucity of data, the use of PRP as a therapy for sexual dysfunction and genitourinary symptoms in the setting of menopause has been patented as the O-Shot and V-Shot. This is a single-blinded randomized control trial to determine if PRP injections into the anterior vaginal wall improve sexual functioning in women diagnosed with the genitourinary syndrome of menopause (GSM) characterized by vaginal dryness/irritation.