DM Clinical Research’s Post

Exciting News from DM Clinical Research! DM Clinical Research is proud to announce that we are a founding member of the newly launched Association of MultiSite Research Corporations (AMRC). This groundbreaking trade association unites 14 leading multisite organizations to tackle inefficiencies in clinical trials and create a more efficient, patient-focused research ecosystem. At DM Clinical Research, we leverage 24+ sites across the U.S. to deliver high-quality, patient-centric studies across diverse therapeutic areas, including Infectious Disease, Cardio-metabolic, CNS, Immunology, and Vaccines. By integrating advanced technology and centralized management, we are at the forefront of driving innovation in clinical research.  Why AMRC Is a Game-Changer: - Addresses the reality that trial numbers grew by 19% from 2012 to 2022, while recruitment rates per site dropped 14% in oncology and 54% in non-oncology. - Provides scalable solutions to meet the growing complexity of clinical trials - Streamlines operations to combat inefficiencies, ensuring that life-saving treatments can be delivered faster and more reliably. We are excited to collaborate with AMRC to advance the industry, champion innovation, and improve the clinical trial process for patients, sponsors, and investigators alike. Together, we’re shaping the future of medical research. Learn more about AMRC and join their launch webinar on Thursday 30 January, where panelists will discuss the cost of inefficiencies in clinical trials to Sponsors and CROs and the critical role of multisite organizations in addressing these issues www.amrc.org/news. #DMClinicalResearch #AMRC #ClinicalTrials #Innovation #TechnologyInResearch #DiversityInClinicalTrials #HealthcareLeadership #PatientCentricity

I am so proud of our clinical pharmacy specialist, Kim Nguyen, PharmD, APh, BCOP, for her recent publication in collaboration with City of Hope’s esteemed physicians titled, “Genomic and molecular alterations associated with primary resistance to immune checkpoint inhibitors.” #clinicalpharmacy #oncologypharmacy #cityofhopeorangecounty

'There is a need to improve patient access to HCC treatment options known to extend survival rates, such as atezolizumab + bevacizumab' 👆 This is just one of the key takeaways from our survey into HCC management in the Asia Pacific region. The findings have been published in the Journal of Gastrointestinal Cancer in a paper titled: 'Hepatocellular Carcinoma in Asia: Physician and Patient Perspectives on Surveillance, Diagnosis, and Treatment' 📜 You can read the full, open-access article (and download the accompanying slide set) here >> https://ow.ly/f6C150SFVfY Read more about the scientific committee and the details of the survey ⬇️⬇️⬇️ A group of international and multidisciplinary clinical experts and Patient Advocacy Group representatives conducted the survey. They investigated the screening, diagnosis, therapeutic strategies, and patient journeys of hepatocellular carcinoma (HCC) across the Asia Pacific region. As liver cancer is the leading cause of cancer deaths in several Asian countries, these data can provide insight into the national approach to HCC management for each country, potential areas for improvement, and what to prioritise to shape national policies to improve outcomes for patients. The findings provided valuable insights into: 💠 Patient and HCP awareness of risk factors 💠 Current surveillance programs and diagnostic tools and whether they could be enhanced 💠 How HCPs can strengthen support for patients throughout their journey 💠 How accessible HCC treatment options are for patients in this region #MedicalEducation #Oncology This programme was supported by an Independent Medical Education Grant from Roche.

New topic, starting with Estimands ***************************************************************************** Estimands: Bringing Clarity to Clinical Trial Objectives In clinical trials, estimands are transforming the way we define what we’re measuring. Introduced by ICH E9 (R1), they ensure that trial objectives are clear and aligned across stakeholders. An estimand consists of four elements: Treatment: What interventions are being compared? Population: Who is included in the analysis? Outcome (Variable): What specific effect are we measuring (e.g., survival, disease progression)? Intercurrent Events: How are events like treatment discontinuation or switching handled? Why does this matter? Without a clear estimand, trial results can be interpreted in different ways. For example, in a diabetes study, do we include all patients who started the treatment or only those who stayed on it? Estimands make this explicit, leading to more consistent, actionable insights. In complex trials, particularly in oncology or rare diseases, estimands provide a framework to handle unpredictable events like patients switching therapies, ensuring robust, interpretable results that are relevant for regulators and clinicians. By defining the right estimand, we can address real-world questions with greater precision and clarity, improving the value of trial data for clinical decision-making. Practical Example In a trial for a cancer therapy, a key intercurrent event might be patients switching to another treatment. Depending on how the estimand is framed, the trial could focus on: The treatment effect on all patients, regardless of whether they switched therapies, or Only the patients who stayed on the initial treatment, giving insight into the drug's effect in the ideal scenario. ********************************************************************************* 👉 Next up, we’ll dive into how sensitivity analyses support estimands in ensuring reliable conclusions from trials. #Estimands #ClinicalTrials #ICHGuidelines #Biostatistics #DrugDevelopment #OncologyResearch #RareDiseases #RegulatoryScience #TrialDesign #Pharma

📃Scientific paper: Clinical endpoints in allergen immunotherapy: State of the art 2022 Abstract: 110 years after the classical study by Noon, numerous studies have confirmed the efficacy of allergen immunotherapy. A variety of clinical endpoints have been used in these trials. This review gives an overview of clinical endpoints for randomized clinical trials on allergen immunotherapy (AIT) in rhinitis and asthma. In addition, real-life studies have been carried out with the same kind of endpoints. In general, AIT studies are characterized by a lack of standardized and validated outcome measures. For allergic rhinoconjunctivitis, digital tools have been developed to monitor patients. Such tools are particularly useful to obtain real-world evidence for AIT. Finally, well-accepted outcome measures are available for cost-effectiveness studies. Continued on ES/IODE ➡️ https://etcse.fr/DN4AY ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

N Engl J Med | Professors Lei Zhang and Renchi Yang’s Team Unveils Efficacy, Safety, and Mechanism of a Novel CD38 Monoclonal Antibody in ITP Patients Immune thrombocytopenia (ITP) is a common autoimmune bleeding disorder characterized by accelerated platelet destruction and impaired platelet production due to antibody-mediated mechanisms. Current first-line therapies for ITP include glucocorticoids and immunoglobulins, while second-line options often involve thrombopoietin receptor agonists (TPO-RAs), CD20 monoclonal antibodies, and splenectomy. The introduction of CD20 monoclonal antibody therapy over the past decade has transformed ITP treatment. However, heterogeneity in treatment response and duration leaves a subset of patients at risk of severe bleeding, reduced quality of life, and increased mortality. Following the success of CD20 antibodies, researchers have been exploring other potent therapeutic targets to address these unmet needs. CD38-positive long-lived plasma cells (LLPCs) play a critical role in ITP by continuously producing pathogenic antibodies, leading to disease relapse or refractory cases. Targeting CD38 to eliminate LLPCs and other antibody-secreting cells offers a promising therapeutic strategy for achieving durable clinical benefits in ITP. On June 19, 2024, Professors Lei Zhang and Renchi Yang’s team from the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, published a groundbreaking study in The New England Journal of Medicine (Impact Factor: 158.5) titled "A Novel Anti-CD38 Monoclonal Antibody for Treating Immune Thrombocytopenia." This study represents the first global report evaluating the efficacy, safety, and mechanism of CM313, a novel CD38-targeting monoclonal antibody for ITP treatment. The study marks a major innovation in ITP research and has the potential to reshape international treatment guidelines. #ITP #ImmuneThrombocytopenia #CD38Antibody #MonoclonalAntibodies #AutoimmuneDiseases #PlateletDisorders #HematologyResearch #BloodDisorders #CM313 #InnovativeTherapies #PrecisionMedicine #AutoimmuneTreatment #HematologyAdvancements #NEJMPublication #MedicalBreakthroughs

#ClinicalTrials are essential for developing new treatments for heart disease, stroke, cancer, and more. They offer hope to patients when standard treatments fail and provide early access to innovative therapies. For example, one North Carolina woman recently traveled to MD Anderson Cancer Center in Texas for recommendations after the initial treatment for her metastatic colon cancer had failed. There, her physicians checked a database for clinical trials where she might access a promising yet experimental new drug. They eventually found a trial with an open slot at, you guessed it, Novant Health. At Novant Health, we’re leading the charge in clinical research with nearly 1,500 patients participating in over 300 trials statewide. Our efforts, spearheaded by dedicated physician investigators like Dr. Franklin Chen, ensure that patients can receive cutting-edge treatments close to home, surrounded by their loved ones. Clinical trials at Novant Health are about more than just testing new treatments—they’re about offering hope and potentially life-changing outcomes. As Dr. Chen aptly said, clinical trials allow us to access the drugs of tomorrow today. #Research #Healthcare #PatientCare

📃Scientific paper: Serum miR-96-5P and miR-339-5P Are Potential Biomarkers for Multiple System Atrophy and Parkinson's Disease Abstract: Parkinson's disease (PD) and Multiple System Atrophy (MSA) are progressive neurodegenerative diseases with overlap of symptoms in early stages of disease. No reliable biomarker exists and the diagnosis is mainly based on clinical features. Several studies suggest that miRNAs are involved in PD and MSA pathogenesis. Our goal was to study two serum circulating microRNAs (miR-96-5p and miR-339-5p) as novel biomarkers for the differential diagnosis between PD and MSA. Serum samples were obtained from 51 PD patients, 52 MSA patients and 56 healthy controls (HC). We measured levels of miRNAs using quantitative PCR and compared the levels of miR-96-5p and miR-339-5p among PD, MSA and HC groups using a one-way analysis of variance. Correlations between miRNA expression and clinical data were calculated using Pearson's rho test. We used the miRTarBase to detect miRNA targets and STRING to evaluate co-expression relationship among target genes. MiR-96-5p was significantly increased in MSA patients compared with HC (Fold change (fc): 3.6; p = 0.0001) while it was decreased in PD patients compared with HC (Fold change: 4; p = 0.0002). Higher miR-96-5P levels were directly related to longer disease duration in MSA patients. We observed a significant increase of miR-339-5p in MSA patients compared with PD patients (fc: 2.5; p = 0.00013). miR-339-5p was increased in MSA patients compared with HC (fc: 2.4; p = 0.002). We identified 32 target genes of miR-96-5p and miR-339-5p, s... Continued on ES/IODE ➡️ https://etcse.fr/5CZkq ------- If you find this interesting, feel free to follow, comment and share. We need your help to enhance our visibility, so that our platform continues to serve you.

We are delighted to announce further success in our collaboration with AstraZeneca. Using BenevolentAI’s AI-drug discovery platform, a novel target for systemic lupus erythematosus (SLE) has been identified. 🎯 The target has been experimentally validated by AstraZeneca and added to its discovery portfolio. Following the heart failure milestone, it’s the second target to be selected this year from our extended collaboration. 📣 Prof Maria Belvisi, SVP and Head of Research and Early Development Respiratory and Immunology at AstraZeneca said: “By combining our immunology disease area expertise and BenevolentAI’s AI-driven discovery platform, we are increasing our ability to identify new targets based on patient insights, complementing our portfolio of potential treatments for this debilitating disease.” SLE, or lupus, can cause inflammation, pain and damage – often affecting the organs and joints in severe cases. Extreme fatigue and cognitive issues are also common, along with comorbidities including cardiovascular disease. Read more here: https://lnkd.in/e2mq8haw

🧬RNA Leaders Industry Update🧬 ⭐Great to see that Atalanta Therapeutics has secured a $97M Series B funding round to progress two siRNA therapeutics for Huntington's disease and KCNT1-related epilepsy to the clinic. 🔗https://lnkd.in/ePpjAgQH ⭐ AIRNA appoints Matthew Hawryluk as Chief Business Officer - congratulations! 🔗https://lnkd.in/eCQYBVQD ⭐Mirvie's RNA platform has uncovered a unique molecular signal for severe fetal growth restriction, progressing standard of risk monitoring during pregnancy. 🔗https://lnkd.in/ehBEahKQ Join our next in-person meeting, RNA Leaders Europe (Basel, 4-6th March), to hear more exciting updates!... 🎤 Christopher O'Donnell, Novartis Institute For Biomedical Research - Delving into the role of siRNA in targeting lipoprotein(a) in cardiovascular risk assessment and clinical practice 🎤Dr. Myriam Mendila, CureVac - Sharing insights on the development of an off-the-shelf mRNA cancer vaccine for glioblastoma 🎤 Paul Nioi, Alnylam Pharmaceuticals - Exploring results from Phase 3 study of Vutrisiran for treatment of cardiomyopathy, and regulatory pathway 🎤 Jean-François Toussaint, Sanofi - Discussing optimisation of mRNA-based vaccine clinical candidates via an integrative iterative translational science framework 🔶 See the full 2025 agenda here: https://lnkd.in/ev-7M-H4 🔶 See the full speaker line up here: https://lnkd.in/gNxMA8Y8 #RNALeaders #RNA #siRNA #ADAR #RNAi #mRNA