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Filter by:An Open, Dose-escalation, Phase 1b Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A (CMT1A)
Brief Summary: This is a Phase I, first-in-human, open-label, multi-center study designed to explore the safety, tolerability, PK, and anti-tumor antitumor activity of ANS014004 monotherapy in subjects with locally advanced or metastatic solid tumors. - The study consists of two parts: a dose-escalation part (Part 1) and a dose-expansion part (Part 2). For each subject, the study will consist of a screening period (Day -28 to Day -1), a treatment period (until discontinuation of treatment) and a follow-up period (including safety follow-up and survival follow-up). - During the Treatment Period, subjects will receive ANS014004 single-agent oral administration until the subject meets any treatment termination criteria. 1, The dose-escalation part (Part 1) will consist of a single-dose period and a multiple-dose period (28 days per cycle), participants during dose escalation will receive a single dose of ANS014004 on Day 1 of the single-dose period in order to obtain complete PK parameters for the single dose. There will be a 7-day washout period between the single dosing period and the multiple dosing period at the same dose level. If no dose-limiting toxicity (DLT) occurs during the 7-day washout period, participants will begin Cycle 1 (28 days per cycle) multiple dosing treatment on Day 8, receiving ANS014004 once daily (QD). participants in the backfill cohort will enter the multiple dosing period directly. The specific dose which participant receives will depend on the different cohort assignments. The dose-escalation part (Part 1) will use the initial accelerated titration design (ATD) and the traditional "3 + 3" design to determine the MTD of ANS014004 in subjects with locally advanced or metastatic solid tumors. 2. In the Dose Expansion (Part 2), participants, will receive oral administration of ANS014004 QD at each treatment cycle (28 days per cycle). An end-of-treatment (EOT) visit will be conducted within 7 days of the final dose or investigator decision to discontinue. All subjects will undergo imaging evaluations of their tumors every 8 weeks until disease progression is confirmed by the investigator, the subject begins new antitumor therapy, dies, is lost to follow-up, or withdraws from the study, whichever occurs first.
The goal of this study is to develop an algorithm using artificial intelligence (AI) to assist identification of potential ATTR-CM cases using routine transthoracic echocardiography. The main questions it aims to answer are: - is the algorithm able to diagnose ATTR-CM - is the algorithm able to diagnose different types of ATTR-CM (ATTRv, ATTRwt) This is a non interventional study. Participant' echocardiographies will be, after deidentification, used to train, valid and test the algorithm.
This prospective observational study evaluates the relationship between the objective oral health status and the patient reported diet in 64 adult patients undergoing elective major orthopaedic surgery (hip, knee, or spine surgery).
Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) is a challenging disease to treat. Targeted KIT inhibitors have been approved for this indication based on their ability to control the mastocytosis portion of the disease, but patients frequently experience progression of the concomitant myeloid malignancy (i.e. the AHN). Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. In this study, investigators aim to study the safety and tolerability of combined avapritinib and decitabine for the treatment of SM-AHN.
Coronary atherosclerosis is the most common cause of ischaemic heart disease. About 40-50% of patients with symptoms and documented ischaemia on non-invasive tests do not show obstructive coronary artery disease on coronary angiography. This cause of ischaemic heart disease called INOCA (Ischemic Non-Obstructive Coronary Artery), far from having a benign prognosis, is associated with an increase in major adverse cardiac events (MACE) as well as increased functional limitation. The current European Society of Cardiology clinical practice guidelines for the management of chronic coronary syndrome establish for the first time a IIa recommendation for the invasive analysis of coronary flow reserve (CFR) and microvascular resistance index (MRI) in symptomatic patients with INOCA. The acetylcholine (Ach) test, based on intracoronary (ic) administration, is established as indication IIb for the assessment of micro or macrovascular vasospasm in patients with suspected vasospastic angina (VSA) (4). A national multicentre registry would allow us to determine the prevalence of INOCA and its different endotypes in our setting.
Trial purpose: To evaluate the safety and effectiveness of using a new remote rehabilitation platform for postoperative rehabilitation in patients with anterior cruciate ligament rupture compared to conventional rehabilitation care through a randomized controlled trial. Test subjects: This study is a multicenter study that recruited 252 participants from three centers (aged ≥ 18 years) for cruciate ligament rupture reconstruction; Exclude patients who have undergone revision and bilateral surgery, and exclude patients with collateral ligament and posterior cruciate ligament injuries in the knee joint. Trial plan: The subjects were randomly enrolled and randomly divided into two groups: the remote rehabilitation group and the traditional rehabilitation group, using a mixed block randomization method. The probability of being divided into both groups was 50%. All subjects received routine outpatient diagnosis and treatment, with the same content. The remote rehabilitation group uses software and sensors to provide remote postoperative rehabilitation guidance. The traditional rehabilitation group receives routine rehabilitation diagnosis and treatment, with face-to-face rehabilitation twice a week. All subjects are required to undergo regular follow-up (1 day before surgery, 3 months after surgery, 6 months). Outcome measures: The main outcome measures were knee isokinetic muscle strength and Lysholm knee joint score . the secondary outcome measures were Y-balance test, Tegner activity scale, health survey summary table (SF-36),The other outcome measures were Anterior Cruciate Ligament Return to Sport After Injury (ACL-RSI) Scale, vas pain score.
This is a prospective, randomized, controlled, non-inferiority study of patients undergoing tonsil surgeries at Boston Children's Hospital Waltham. The overall aim is to evaluate the efficacy of an opioid anesthetic plan (morphine, ketorolac, and acetaminophen versus an opioid sparing anesthetic plan (dexmedetomidine, ketorolac and acetaminophen) for perioperative analgesia and recovery time in patients undergoing tonsillectomies and tonsillotomies at Boston Children's Hospital Waltham. Secondary measures include rescue opioids administered in post-anesthesia care unit (PACU), re-operation secondary to bleeding, emergence delirium, post-operative nausea and vomiting, intraoperative hemodynamics, intraoperative vasopressor administration, and length of procedure.
ETN101 is a multiple tyrosine kinase inhibitor (mTKI) targeting fms-like tyrosine kinase 3 (FLT3), receptor tyrosine kinase (KIT), vascular endothelial growth factor receptor 2 (VEGFR2), and platelet-derived growth factor receptor beta. Both in vitro and in vivo studies showed that ETN101 treatment/administration inhibited cancer cell survival and proliferation. In animal models, ETN101 had antitumor activity when administered to animals that did not respond to conventional targeted anticancer agents.
The objective of this study is to characterize the genetic architecture of a large cohort of CIDP patients to evaluate whether specific alleles/haplotypes are implicated in the risk of CIDP, in its clinical and immunological variability, severity, therapeutic response, and association with diabetes and other autoimmune diseases. We will genotype >700,000 single nucleotide polymorphisms (SNPs) by using the Illumina Global Screening Array (GSA), of approximately 1000 patients with CIDP. About 3500 healthy controls from the Italian population have been already genotyped using GWAS from our genetic department. Alleles/haplotypes will be also compared between patients with typical CIDP and its variants, between CIDP patients with and without specific antibodies, between CIDP patients with and without comorbidities, between CIDP patients with low and high levels of disability and between CIDP patients with and without response to each individual treatment (IVIg, steroids, plasma exchange)