The Institute offers the interdisciplinary expertise for designing, conducting, and analyzing early phase, confirmatory and comparative effectiveness trials. This includes:
- Expertise in the design and analysis of early phase and confirmatory human trials that use adaptive trial designs and Bayesian statistics. Proficiency in designing comparative effectiveness trials that use pragmatic designs with point of care data collection to evaluate the effectiveness of interventions in routine clinical practice for optimizing physician and patient clinical decision-making.
- Experience with a broad array of endpoints, including biomarkers, imaging outcomes, surrogate endpoints, morbidity and survival outcomes, as well as composite clinical endpoints. Another important focus of the Institute is the use of patient-centered outcomes, including quality of life, functional status, and frailty. The Institute has the expertise to analyze complex datasets and endpoints, such as biomarker, genetic and imaging data that require machine learning and AI methods.
- Cost effectiveness analyses and decision analytical models that enrich trials by including economic endpoints. Expertise in developing tools and risk models that can tailor the inferences drawn from clinical trials to subgroups or individuals.
- Innovation in data collection processes for multicenter trials by leveraging data routinely collected by national registries and by practitioners using the electronic health record to avoid duplicate data collection. Mobile apps and wearable sensors are especially valuable in the collection of patient-reported outcomes. In addition, the Institute has expertise in linking the data from trial populations to large data sets to collect long-term outcomes at reasonable cost. As such, we are able to improve efficiency and reduce costs of clinical trials.
- Expertise in collection and storage of biospecimens, and identification of genomic and biomarkers that will enhance our ability to understand the biological underpinnings of disease and treatment, which will, in turn, facilitate creating “personalized” treatments.
- Novel strategies to recruit and retain patients, and to ensure the involvement of diverse and vulnerable populations.
- A wide range of Core Labs, including echocardiography, IVUS, neurocognition, MRI and CT imaging, and biorepository.
- Regulatory expertise with the FDA, Health Canada and the European regulatory bodies needed to conduct trials of investigational products and their approval.
Selected High-Priority Trials from Institute Investigators include:
Treatment of Ischemic Mitral Regurgitation
- Acker MA, Parides MK, Perrault LP, et al. A Randomized Trial of Surgical Treatment for Severe Ischemic Mitral Regurgitation: Repair versus Replacement. New England Journal of Medicine 2014 Jan 2; 370(1):23-32.
- Goldstein D, Moskowitz AJ, Gelijns AC, et al. Two-Year Outcomes of Surgical Treatment of Severe Ischemic Mitral Regurgitation. New England Journal of Medicine. 2016 Jan 28; 374(4):344-53.
- Smith PK, Puskas JD, Ascheim DD, et al. Surgical Treatment of Moderate Ischemic Mitral Regurgitation. New England Journal of Medicine. 2014; 371:2178-88
- Michler RE, Smith PK, Parides MK, et al. Two-Year Outcomes of Surgical Treatment of Moderate Ischemic Mitral Regurgitation. New England Journal of Medicine 2016; 374:1932-1941.
Treatment of Atrial Fibrillation
- Gillinov AM, Bagiella E, Moskowitz AJ, et al. Rate Control versus Rhythm Control for Atrial Fibrillation after Cardiac Surgery. New England Journal of Medicine. 2016; 374:1911-1921
- Gillinov AM, Gelijns AC, Parides MK, et al. Surgical Ablation of Atrial Fibrillation during Mitral-Valve Surgery. New England Journal of Medicine. 2015 Apr 9; 372(15):1399-409.
Averting Complications of Cardiac Surgery
- Mack MJ, Acker MA, Gelijns AC, et al. Effect of Cerebral Embolic Protection Devices on CNS Infarction in Surgical Aortic Valve Replacement. JAMA 2017; 318: 536-547.
- Gelijns AC, Moskowitz AJ, Acker MA, et al. For the Cardiothoracic Surgical Trials Network (CTSN). Management Practices and Major Infections after Cardiac Surgery. J Am Coll Cardiol. 2014 Jul 29;64(4):372-81.
Treatment of Neurovascular Disorders
- Mohr JP, Parides MK, Stapf C. Medical management with or without interventional therapy for unruptured brain arteriovenous malformations (ARUBA): a multicentre, non-blinded, randomised trial. Lancet. 2014 Feb 15; 383(9917):614-21.
New Therapies for Advanced Heart Failure
- Rose EA, Gelijns AC, Moskowitz AJ, et al. Long-Term use of a left ventricular assist device for end-stage heart failure. New England Journal of Medicine 2001 Nov; 345(20):1435-43.
- Ascheim DD, Gelijns AC, Goldstein D, Mesenchymal Precursor Cells as Adjunctive Therapy in Recipients of Contemporary LVADs. Circulation. 2014 Mar 28. [Epub ahead of print] PubMed PMID: 24682346.
- Yau TM, Pagani F, Mancini DM, et al. Effect of Intramyocardial Injection of Mesenchymal Precursor Cells on Successful Temporary Weaning from Left Ventricular Assist Device Support among Patients with Advanced Heart Failure: A Randomized Clinical Trial. JAMA 2019;321:1176-1186.