Research Studies

The objective of this Phase II trial is to compare the efficacy and safety of 6 months of treatment with thymalfasin plus trans arterial chemoembolization (TACE) with TACE alone in adult patients with non-surgical hepatocellular carcinoma (HCC).

This trial is conducted in China. The aim of the trial is to evaluate the efficacy and safety of BGM0504 versus semaglutide as add-on to metformin and/or sulfonylureas in patients with type 2 diabetes

This study looks at how participants with type 2 diabetes take Ozempic® and if the Ozempic® app helps participants to stay on this treatment. Participants will already be prescribed with Ozempic® by the study doctor. Participants may be asked to use a device called Mallya®, which participants must attach to their Ozempic® injection pen. Participants might also be asked to install an Ozempic® app on their mobile phone which supports the participants in the use of Ozempic®. At the beginning and at the end of this study, the participants will have to fill out some questionnaires about their diabetes treatment. Participants may also be chosen to participate in a voluntary non-mandatory interview after the study has ended. The total duration of study is approximately 10 months.

The purpose of this study is to determine the effect of topical aminocaproic acid on the immune system by assessing the levels of antimicrobial peptides in the skin of patients with rosacea. It is hypothesized that aminocaproic acid applied topically will alter the body's immune system in patients with rosacea by inhibiting activation of antimicrobial peptides.

This research study is an open-label Phase 1 Exploratory/Pilot clinical trial to measure the effects of the incretin mimetic, tirzepatide, on tissue, urine, blood, and microbiome biomarkers associated with colorectal cancer risk and to understand the feasibility of this precision prevention trial approach for a future larger study. In this research study, we are: * Investigating the effects of tirzepatide on biomarkers of colorectal cancer risk in patients with a recent diagnosis of adenoma, a type of intestinal polyp that can precede the development of cancer. * Planning to measure the potential protective effects associated with tirzepatide within biological samples (biospecimens) including stool, urine, blood, and oral swab samples collected prior to, during, and after treatment with tirzepatide. * Tirzepatide is a part of the incretin mimetic (GLP-1 receptor agonist) family, which are typically used for managing diabetes and/or obesity. * Tirzepatide may prevent colorectal cancer through multiple possible biological mechanisms. This includes weight loss which can reduce the risk of developing obesity-associated cancers, such as colorectal cancer. * Tirzepatide has been shown to effectively induce weight loss and improve glycemic control. The exact mechanism by which tirzepatide acts to prevent colorectal cancer is still unknown. By performing this research study, we will study the mechanisms of its anti-cancer effect, which may lead to the discovery of novel specific characteristics (markers) that can be used to select patients for tirzepatide treatment to reduce risk of cancer in the future. The research procedures include screening for eligibility and study treatment and scheduling four clinical research visits: * Initial visit - immediately before starting the study drug * Week 1 visit * Midpoint visit (9-12 weeks later) (Midpoint visit) * Final visit (after completing the drug intervention) At the Initial and Final visits, a flexible sigmoidoscopy will be performed along with the collection of body measurements, questionnaire data, blood, urine, saliva, stool, and up to 24 tissue biopsy samples. The first dose of the study drug will be administered by study staff at the initial visit. Participants will self-administer the second dose of the study drug at the Week 1 visit under supervision of study staff. At the Week 1 and Midpoint visits, they will also provide body measurements, blood, urine, saliva, and stool samples. Participants will be instructed to inject tirzepatide 1 time per week for up to 24 weeks. The dose will start with a 2.5mg injection per week for the first 4 weeks. Dose will increase 2.5mg/injection every 4 weeks until 15mg/injection (or maximum tolerable dose) per week is reached. Participants will be followed weekly during this time. In the very rare occasion that there are unavoidable issues scheduling the final visit, treatment may be extended up to an additional 4 weeks. It is expected that about 20 people will take part in this research study. This research is being supported by The Cancer Grand Challenges partnership funded by Cancer Research UK, the National Cancer Institute, the Bowelbabe Fund for Cancer Research UK and Institut National Du Cancer.

This study will look at how well CagriSema helps people with obesity lose weight compared to a "dummy medicine". CagriSema is a new medicine developed by Novo Nordisk. CagriSema cannot yet be prescribed by doctors. The study has two parts: First part is called the main phase and will last for 2 years, and second part is called the extension phase and will last for 1 year. In the main phase participants will either get CagriSema or "dummy medicine". Which treatment participants get is decided by chance and is not known by participants or the study doctor. In the extension phase participants will get either CagriSema or slowly reduce participants dose of CagriSema if participants had CagriSema in the main phase. Which treatment participants get is decided by chance and is not known by participants or the study doctor in both phases. If participants had "dummy medicine" in the main phase, participants will get CagriSema in the extension phase. Like all medicines, the study medicine may have side effects.

This trial is conducted in Europe. The aim of this trial is to investigate the influence of omeprazole on the pharmacokinetics (the exposure of the trial drug in the body) of oral semaglutide in healthy subjects.

Total joint replacement (TJR) is an increasing effective procedure in orthopedics. However, TJR failure due to aseptic or septic loosening remains an important problem, often due to predisposing factors of the patient, which determine the need to perform a revision surgery. In light of the recent conclusions emerged on the still open problems concerning the diagnostic accuracy of serum and synovial fluid markers in the diagnosis of peri-prosthetic joint infection (PJI), the project aims at evaluating the diagnostic accuracy and cost-effectiveness of the combination of serum and/or synovial markers in the diagnosis of PJI. Through a diagnostic clinical study on patients hospitalized for revision surgery the project would provide evidences on the potentiality of the combination of some markers in accelerating the PJI diagnosis for the best selection of surgical strategy, choosing the suitable cutoff thresholds to mitigate the effect of some factors on markers' discriminatory capability. Total joint arthroplasties (TJA) are constantly increasing in older and multi-morbid patients; knee and hip joints are the most frequently treated due to high incidence of osteoarthritis in these joints. The indication to TJA is now extended to younger and active patients with high functional demands and longer life expectancy. Because of these, an increase in revision surgeries and a PJI risk is also expected. The type of revision surgery (RS) approach and of antimicrobial treatments is based on the presence or absence of infection: one-stage revision with maintenance/replacement of prosthesis in combination with debridement and irrigation (aseptic RS); and two-stage revision with prosthesis removal, debridement and irrigation, the possible use of a temporary spacer, and the potential reimplantation after weeks or months (septic RS). Today, PJI remains an important dangerous and devastating complication; it has been reported for hip and knee joints an incidence of 0.2-1.5%, prosthesis failure of 15-25%, revision success rate of 80-95% and annual cost per infection of $20,000-$40,000. The timely and most accurate diagnosis is essential for the correct treatment of PJI as patients undergoing revision need extensive physical, psychological and economic supports. Following the definition and standardization of diagnostic major and minor criteria for PJI by the Musculoskeletal Infection Society (MSIS) an improvement in the diagnosis and treatment of PJI was observed. Recently, the International Consensus Group on PJI revised MSIS criteria defining that patients should be considered to have PJI in the presence of one of the major criteria or three of five minor criteria. Finally, an evidence-based and validated criteria for hip and knee PJI definition to diagnose patients within the preoperative period was published in 2018. Patients with an aggregate score of more than or equal to 6 are considered infected, while a 2-5 score requires the inclusion of intraoperative findings (positive histology, purulence and single positive culture) for confirming or refuting the diagnosis. A 4-5 score is inconclusive, and a score of less than or equal to 3 is not infected. During the last International Consensus Meeting on Musculoskeletal Infection, various questions on PJI were discussed and among them the working groups (WG) provided recommendations based on limited (4 - due to small numbers) and moderate (12 and 19) levels of evidence: WG4, on which patient-specific factors influence the thresholds for serum and synovial markers in acute and chronic PJI; WG12, on which serum tests have the best diagnostic accuracy for PJI and if the combination of any number of tests increase the diagnostic accuracy; WG19, if the profile of organisms causing surgical site infection or PJI following orthopaedic procedures changed over recent years. The WG4 highlighted that no inflammatory arthritis (IA)-specific factors are known to influence the thresholds for serum and synovial markers in PJIs. It appears that in IA patients there are overlaps in values of synovial markers such as alphadefensin, white blood cell count (sWBC) and C-reactive protein (CRP), limiting their usefulness in differentiating septic from aseptic patients. The WG12 stated that it is evident that diagnosis of PJI cannot be based solely on serological tests and that CRP and erythrocyte sedimentation rate (ESR) are well-researched screening tests and have high sensitivity when used alone. However, the combination of serological tests has shown to improve diagnostic accuracy, but the identification of the optimal combination needs further investigations. Finally, the WG19 recommended to perform further investigations regarding the profile of microorganism with next generation sequencing (NGS), as it may confer significant antibiotic selection implications. Among pre-operative test on synovial fluid, alpha-defensin showed the highest degree of accuracy in the diagnosis of PJI when measured by laboratory-based immunoassay rather than lateral flow test, suggesting that care must be taken with interpretation of the lateral flow test when relying on its results for the intra-operative diagnosis. Synovial fluid could be tested for other newly markers such calprotectin and cathelicidin LL-37, whose sensitivity and specifity are to be defined or for microbiological tests. Synovial fluid analysis for WBC and %PMN has high sensitivity and specificity in diagnosing PJI, and it represents an easy and widespread available method. Our meta-analysis evaluated: 1) accuracy in terms of sensitivity and specificity of synovial fluid WBC and %PMN tests in the diagnosis of PJI after total knee (TKA) and hip (THA) arthroplasty; 2) which test yielded superior test performance; and 3) the influence of study characteristics such as anatomic site and threshold value on the diagnostic accuracy of these tests. The meta-analysis results revealed that both synovial fluid WBC and %PMN have a high specificity and sensitivity in detecting PJI, and no clear superiority of one test over the other exists. In addition, an anatomic site effect was demonstrated on sensitivity of synovial fluid %PMN, suggesting that cutoff thresholds should be identified separately for TKA and THA. The differences in bearing surfaces and the possibility of fretting corrosion in dual taper stem designs might substantially alter automated synovial fluid analysis in THA compared to TKA. The aim of the project is to evaluate the diagnostic accuracy and cost-effectiveness of the combination of serum and/or synovial markers in the pre- or intraoperative (lateral flow test)/post- diagnosis of PJI. To such purpose a clinical diagnostic study on patients hospitalized for revision surgery based on the guidelines of MSIS will be carried out; the combinations of two or more markers with the highest diagnostic accuracies will be identified and evaluated in term of cost-effectiveness towards individual markers whose thresholds are based on the MSIS 2013 ICM. The significance of this research is to provide evidences on the potentiality of the combination of some markers in accelerating the PJI diagnosis for the best selection of surgical strategy to be followed. A prompt diagnosis and recognition of the etiological agent are crucial to define the appropriate antimicrobial therapy and to reduce to a minimum the cases of erroneous diagnosis and of consequent erroneous surgical conduct, with the need for subsequent re-operations, and multiplication of risk for patients and of costs for the health care system.

The purpose of this study is to test whether Direct Instruction - Language for Learning (DI-LL) is an effective way to teach language skills to children with autism spectrum disorder (ASD) and moderate language delay. Direct Instruction - Language for Learning (DI-LL) uses face to face instruction and specific lessons to teach children language skills. This method has been used previously in children with language delays, but it has not been carefully studied in children with autism spectrum disorder. This study will compare DI-LL and ongoing treatment as usual to treatment as usual (speech therapy, language services, etc.) alone. Autism spectrum disorder (ASD) is a chronic condition of early childhood onset defined by social impairment and repetitive behavior, and affecting 6 to 14 children per 1000 worldwide. Language and communication impairments are among the most common parental concerns about their children with ASD. Untreated language impairments are also predictive of negative long-term outcomes for children with ASD. Direct Instruction-Language for Learning (DI-LL) is a commercially available intervention package that has demonstrated effectiveness in children with language delays due to disadvantaged backgrounds, learning disabilities, or a primary language disorder - but it has not been carefully studied in ASD. This study will test the efficacy of DI-LL in young school-age children with ASD and moderate language delay. DI-LL is a structured, relatively inexpensive intervention designed to promote a range of language skills. It offers a comprehensive, carefully sequenced, brisk-paced program designed to teach a range of language skills to children with language impairments. A key strength of DI-LL is that it can be implemented by educators, psychologists, speech pathologists, behavior therapists without requiring deep expertise in behavioral interventions for ASD. 100 participants, ages 4 to 7 years, 11 months, will be randomly assigned to either DI-LL or Treatment as Usual (TAU). Children randomly assigned to DI-LL will be allowed to continue in ongoing treatments as well. Forty to 42 treatment sessions will occur across 24 weeks with post-treatment follow up visits at Weeks 36 and 48 for subjects in DI-LL. Negative responders to TAU at Week 24 will be offered treatment with DI-LL for 24 weeks. This study is designed to compare DI-LL and TAU on two standardized tests of language function; overall improvement rated by a blinded clinician; and the number of spoken words in a structured laboratory setting via direct observation - again blind to treatment assignment. The intent to treat approach will be used in efficacy analysis and adverse events will be monitored throughout the trial.

To determine the efficacy of GS300 when administered for 24 weeks in patients with Nonalcoholic Fatty Liver Disease (NAFLD). This is a multicenter, randomized, placebo-controlled, double-blinded, parallel-group study. Patients will be randomized 1:1 to receive either GS300 or placebo. The study includes an up to 6 weeks screening period, a 24-week treatment period, and a 1-week follow-up period. Approximately 250 patients (125 patients per arm) will be enrolled (at least 40% from each gender and at least 40% from US and 40% from Europe).