Trials one

Early-phase trials Although obvious and already pursued to some degree, well-designed and controlled n-of-1 trials can be used in early-phase trials evaluating the tolerability, dosing and potential utility of an experimental compound.

Training N-of-1 trials are excellent vehicles for physician training since they would expose a physician to objective clinical decision-making and evidence-based practice on a systematic and rigorous level. Nationwide agenda in individualized medicine Recognition that the USA is in the midst of a healthcare crisis has motivated serious calls for advances in biomedical research [ 9 ].

Future perspective Coordinated n-of-1 trials have the potential to radically change the way in which evidence-based and individualized medicine is pursued.

Do n-of-1 trials have a role in clinical science? Design issues in n-of-1 clinical trials Randomization of treatment order, carryover effects, washout periods and blinding are key design elements that need to be considered in n-of-1 trials.

The analysis of n-of-1 clinical trials Methods that account for serial correlation in comparing the response to two or more treatments, such as certain time-series analyses, are necessary. Footnotes For reprint orders, please contact: moc. Jorgensen JT. Are we approaching the post-blockbuster era? Pharmacodiagnostics and rational drug development.

Expert Rev Mol Diagn. New era of personalized medicine: a year anniversary. Pharmacogenomics and personalized medicine: mapping of future value creation. Langreth R, Waldholz M. New era of personalized medicine: targeting drugs for each unique genetic profile.

Stratified medicine: strategic and economic implications of combining drugs and clinical biomarkers. Nat Rev Drug Discov. Collins FS. Research agenda. Opportunities for research and NIH. Genomic and personalized medicine: foundations and applications. Transl Res. Kraushaar LE, Kramer A. Are we losing the battle against cardiometabolic disease? The case for a paradigm shift in primary prevention.

BMC Public Health. Rich EC. The policy debate over public investment in comparative effectiveness research. J Gen Intern Med. Snyderman R, Yoediono Z. Perspective: prospective health care and the role of academic medicine: lead, follow, or get out of the way. Acad Med. Vogelstein B, Kinzler KW. Cancer genes and the pathways they control.

Nat Med. Cetuximab and chemotherapy as initial treatment for metastatic colorectal cancer. N Engl J Med. Clinically available pharmacogenomics tests. Clin Pharmacol Ther.

Topol EJ. Pharmacy benefit managers, pharmacies, and pharmacogenomic testing: prescription for progress? Sci Transl Med. The path to personalized medicine. Tsapas A, Matthews DR. Using N-of-1 trials in evidence-based clinical practice. Guyatt GH, Jaeschke R. N-of-1 randomized trials — where do we stand? West J Med. Everitt BS, Pickler A. Gerss JW, Kopcke W. Clinical trials and rare diseases. Adv Exp Med Biol. Meinert CL, Tonascia S. Strategies for Studying Behavior for Change.

Single Case Experimental Designs. Determining optimal therapy — randomized trials in individual patients. Laid the foundation for n-of-1 trials in clinical medicine. Many other papers published by this research group further describe their years of experience in implementing n-of-1 trials.

The n-of-1 randomized controlled trial: clinical usefulness. Our three-year experience. Ann Intern Med. Prioritising drugs for single patient n-of-1 trials in palliative care. Palliat Med. What ever happened to n-of-1 trials? Milbank Q. Good review of the history of n-of-1 trials, including interviews with researchers who have been leaders in the field providing valuable direct insight into challenges of n-of-1 trials.

Marketing therapeutic precision: potential facilitators and barriers to adoption of n-of-1 trials. Contemp Clin Trials. Moderators of treatment outcomes: clinical, research, and policy importance. Mediators and moderators of treatment effects in randomized clinical trials.

Arch Gen Psychiatry. Limitations of applying summary results of clinical trials to individual patients: the need for risk stratification. Using n-of-1 trials to improve patient management and save costs. Analysis using data from the clinical trial service developed in Australia of n-of-1 trials from the cost perspective.

Why are trials in palliative care so difficult? Abrahm JL. Combining single patient n-of-1 trials to estimate population treatment effects and to evaluate individual patient responses to treatment. J Clin Epidemiol. This paper and a recently published follow-up paper discuss the statistical methodology involved in combining n-of-1 trials.

Amitriptyline to relieve pain in juvenile idiopathic arthritis: a pilot study using Bayesian metaanalysis of multiple n-of-1 clinical trials. J Rheumatol. Senn S. Cross-over Trials in Clinical Research. Wiley; Chichester, UK: N-of-1 randomized trials for investigating new drugs. Control Clin Trials. Celecoxib compared with sustained-release paracetamol for osteoarthritis: a series of n-of-1 trials.

Kazdin AE. Methods for Clinical and Applied Settings. Single-Case Research Designs. Barlow DH, Hersen M. Spiegelhalter DJ. Statistical issues in studies of individual response. Scand J Gastroenterol Suppl. Rochon J. Transforming medicine via digital innovation.

Recent paper that paints a picture of the future of medicine and the new age of digital medical devices that we are entering. Plasqui G, Westerterp KR. Physical activity assessment with accelerometers: an evaluation against doubly labeled water. Obesity Silver Spring ; 15 10 — Development and testing of the mobiletype program.

Soc Psychiatry Psychiatr Epidemiol. A new device for a non-invasive, wireless, continuous monitoring of oxygen saturation and heart rate in patients with heart failure. Int J Cardiol. A wireless ECG smart sensor for broad application in life threatening event detection. Extended use of a new continuous glucose monitoring system with wireless data transmission in children with Type 1 diabetes mellitus.

Diabetes Technol Ther. Circulating nucleic acids as a potential source for cancer biomarkers. Curr Mol Med. Circulating endothelial cells and endothelial progenitors as surrogate biomarkers in vascular dysfunction.

Clin Lab. Individual n-of-1 trials can be combined to give population comparative treatment effect estimates: methodologic considerations. Randomized clinical trials in single patients during a 2-year period. They review the feasibility of n-of-1 trials from a patient, physician and cost perspective.

Randomised study of n-of-1 trials versus standard practice. Published by a research group that implemented an n-of-1 service in Australia to treat attention deficit and hyperactivity disorder. The efficacy and cost effectiveness of n-of-1 studies with diclofenac compared with standard treatment with nonsteroidal antiinflammatory drugs in osteoarthritis. Are n-of-1 trials an economically viable option to improve access to selected high cost medications?

The Australian experience. Value Health. Chow SC, Chang M. Adaptive Design Methods in Clinical Trials. The development of an interoperable roadmap for medical devices. An n-of-1 trial as an aid to decision-making prior to implanting a permanent spinal cord stimulator. Pain Med. A pilot study of ondansetron plus metopimazine vs. Support Care Cancer. We're super proud of Caleb, Joshua, and all the other kids who have given of themselves so that vaccination could move forward and everybody can benefit.

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As the coronavirus spread across the country, I became increasingly alarmed. Kids helping other kids This trial couldn't have happened without the kids. I wanted to fight back against the pandemic By Caleb Chung It was a great opportunity that I was able to participate in a vaccine clinical trial, especially since I had just turned 12 and barely made it into the group to qualify.

The information contained on this Web site should not be used as a substitute for the medical care and advice of your pediatrician. Cell Host Microbe. Anthelmintic drugs for treating ascariasis.

Cochrane Database Syst Rev. World Health Organization. Geneva, Switzerland; Accessed January 26, Safety of oral ivermectin during pregnancy: a systematic review and meta-analysis. Lancet Glob Health. Drug Res Stuttg. Safety of high-dose ivermectin: a systematic review and meta-analysis. J Antimicrob Chemother.

Over 25 Years of clinical experience with ivermectin: an overview of safety for an increasing number of indications. J Drugs Dermatol. Review of the emerging evidence demonstrating the efficacy of ivermectin in the prophylaxis and treatment of COVID Res Square.

National Institute of Health. Geneva, Switzerland: WHO; Accessed April 8, Heidary H, Gharebaghi R. J Antibiot. Antivir Res. Ivermectin as a broad-spectrum host-directed anti-viral: the real deal? The approved dose of ivermectin alone is not the ideal dose for the treatment of Covid Clin Pharmacol Ther. Nat Commun Biol. Ivermectin may be a clinically useful anti-inflammatory agent for late-stage covid Open Heart.

Lehrer A, Rheinstein PH. Scheim D. From cold to killer: how SARS-CoV-2 evolved without hemagglutinin esterase to agglutinate, then clot blood Cells in pulmonary and systemic microvasculature.

Accessed February 21, A five day course of ivermectin for the treatment of covid may reduce the duration of illness. Int J Infect Dis. The effect of early treatment with ivermectin on viral load, symptoms and humoral response in patients with non-severe COVID a pilot, double-blind, placebo-controlled, randomized clinical trial.

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The arcsine difference as a measure of treatment effect in meta-analysis with zero cells. Exact and efficient inference procedure for meta-analysis and its application to the analysis of independent 2 x 2 tables with all available data but without artificial continuity correction.

Meta-analysis for rare events. Trial sequential analysis reveals insufficient information size and potentially false positive results in many meta-analyses. J Clin Epidemiol. Estimating required information size by quantifying diversity in random-effects model meta-analyses. Available at: www. Fonseca AJ. Accessed January 20, Chapter interpreting results and drawing conclusions.

Accessed February 1, Efficacy and safety of ivermectin for treatment and prophylaxis of covid pandemic. A case series of covid positive patients treatedwith combination of ivermectin and doxycycline. J Bangladesh Coll Physicians Surgeons. Role of ivermectin in the prevention of covid infection among healthcare workers in India: a matched case-control study. PLoS One. Br J Dermatol. Early covid therapy with azithromycin plus nitazoxanide, ivermectin orHydroxychloroquine in outpatient settings significantly reduced SymptomsCompared to known outcomes in untreated patients.

Lack of efficacy of standard doses of ivermectin in severe covid patients. Safety and efficacy of the combined use of ivermectin, dexamethasone, enoxaparin and aspirin against covid Use of ivermectin is associated with lower mortality in hospitalized patients with coronavirus disease Effects of Ivermectin-azithromycin-cholecalciferol combined therapy on COVID infected patients: a proof of concept study.

Biomed Res. Effectiveness of ivermectin as add-on therapy in covid management pilot trial. Hellwig MD, Maia A. A covid Prophylaxis? Lower incidence associated withprophylactic administration of Ivermectin. Int J Antimicrob Agent. Ivermectin treatment may improve the prognosis of patients with covid The use of compassionate ivermectin in the management of symptomatic outpatients and hospitalized patients with clinical diagnosis of covid at the medical center bournigal and the medical center punta cana, rescue group, Dominican Republic.

Antiviral and anti-inflammatory properties ofivermectin and its potential use in Covid Arch Bronconeumol. Shokati Z. Accessed January Spoorthi V, Sasank S. Int Arch Integrated Med. Abd-Elsalam S. Ivermectin as a Novel Therapy in Covid Treatment. Alam MT. Arnold S. Centenario Hospital Miguel Hidalgo.

Hydroxychloroquine and Ivermectin for the Treatment of Covid Infection ; Ashraf S. Ataee Z. Bisoffi Z. Perez A. Echeverri E. Elalfy H. New Antiviral Drugs for Treatment of Covid ; Exman P. Fathalipour M. George B. Gheibi N. Temple University. Outpatient Use of Ivermectin in Covid ; Use of ivermectin in the treatment of Covid a pilot trial. Toxicol Rep. Kamal E. Ivermectin in Treatment of Covid 19 Patients ; Saibannavar A.

Effect of ivermectin on time to resolution of symptoms among adults with mild COVID a randomized clinical trial. J Am Med Assoc. Okasha K. Ivermectin and Nitazoxanide Combination Therapy for Covid ; Ivermectin Nasal Spray for Covid19 Patients ; Rathi S.

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Mahmud R. Ivermectin as an adjunct treatment for hospitalized adult COVID patients: a randomized multi-center clinical trial. Ravikirti, Roy R, Pattadar C, et al. Ivermectin as a potential treatment for mild to moderate COVID - a double blind randomized placebo-controlled trial. Rezai M. Int J Sci. Raad H.