Sample Size for Clinical Trials Martin Bland Prof
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Developing a Protocol
FACILITATOR/MENTOR GUIDE Developing a Protocol Created: 2013 Developing a Protocol. Atlanta, GA: Centers for Disease Control and Prevention (CDC), 2013. DEVELOPING A PROTOCOL Table of Contents LEARNING OBJECTIVES ................................................................................................... 3 ESTIMATED COMPLETION TIME ........................................................................................ 3 TARGET AUDIENCE ......................................................................................................... 3 PRE-WORK AND PREREQUISITES ..................................................................................... 3 MATERIALS .................................................................................................................... 4 OPTIONS FOR FACILITATING THIS TRAINING ....................................................................... 4 ICON GLOSSARY ............................................................................................................ 5 ACKNOWLEDGEMENTS .................................................................................................... 5 FACILITATOR RESPONSIBILITIES ...................................................................................... 7 SECTIONS 1 & 2: INTRODUCTION AND OVERVIEW .............................................................. 8 SECTION 3: WRITING A PROPOSAL OR CONCEPT PAPER ..................................................... 9 SECTION 4: WRITING A DRAFT OF THE PROTOCOL ........................................................... -
Phase 3 Clinical Study Protocol
ModernaTX, Inc. 20 Aug 2020 Protocol mRNA-1273-P301, Amendment 3 mRNA-1273 CLINICAL STUDY PROTOCOL Protocol Title: A Phase 3, Randomized, Stratified, Observer-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Immunogenicity of mRNA-1273 SARS-CoV-2 Vaccine in Adults Aged 18 Years and Older Protocol Number: mRNA-1273-P301 Sponsor Name: ModernaTX, Inc. Legal Registered Address: 200 Technology Square Cambridge, MA 02139 Sponsor Contact and Tal Zaks, MD, PhD, Chief Medical Officer Medical Monitor: ModernaTX, Inc. 200 Technology Square, Cambridge, MA 02139 Telephone: 1-617-209-5906 e-mail: [email protected] Regulatory Agency Identifier Number(s): IND: 19745 Amendment Number: 3 Date of Amendment 3: 20 Aug 2020 Date of Amendment 2: 31 Jul 2020 Date of Amendment 1: 26 Jun 2020 Date of Original Protocol: 15 Jun 2020 CONFIDENTIAL All financial and nonfinancial support for this study will be provided by ModernaTX, Inc. The concepts and information contained in this document or generated during the study are considered proprietary and may not be disclosed in whole or in part without the expressed written consent of ModernaTX, Inc. The study will be conducted according to the International Council for Harmonisation (ICH) Technical Requirements for Registration of Pharmaceuticals for Human Use, E6(R2) Good Clinical Practice (GCP) Guidance. Confidential Page 1 ModernaTX, Inc. 20 Aug 2020 Protocol mRNA-1273-P301, Amendment 3 mRNA-1273 PROTOCOL APPROVAL – SPONSOR SIGNATORY Study Title: A Phase 3, Randomized, Stratified, Observer-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Immunogenicity of mRNA-1273 SARS-CoV-2 Vaccine in Adults Aged 18 Years and Older Protocol Number: mRNA-1273-P301 Protocol Version Date: 20 Aug 2020 Protocol accepted and approved by: See esignature and date signed on last page of document. -
Chapter 13*: EPIDEMIOLOGY
Monitoring Bathing Waters - A Practical Guide to the Design and Implementation of Assessments and Monitoring Programmes Edited by Jamie Bartram and Gareth Rees © 2000 WHO. ISBN 0-419-24390-1 Chapter 13*: EPIDEMIOLOGY * This chapter was prepared by D. Kay and A. Dufour Epidemiological data are frequently used to provide a basis for public health decisions and as an aid to the regulatory process. This is certainly true when developing safeguards for recreational waters where hazardous substances or pathogens discharged to coastal and inland waters may pose a serious risk of illness to individuals who use the waters. Epidemiological studies of human populations not only provide evidence that swimming-associated illness is related to environmental exposure, but also can establish an exposure-response gradient which is essential for developing risk- based regulations. Epidemiology has played a significant role in providing information that characterises risks associated with exposure to faeces contaminated recreational waters. The use of epidemiological studies to define risk associated with swimming in contaminated waters has been criticised because the approach used to collect the data is not experimental in nature. This perception is unlikely to change, given the highly variable environments where recreational exposures take place. Although the variables may be difficult to control, it is possible to carry out credible studies by following certain standard practices that are given below. This Chapter discusses the place of epidemiological investigations in providing information to support recreational water management and the scientific basis of "health-based" regulation. 13.1 Methods employed in recreational water studies Epidemiology is the scientific study of disease patterns in time and space. -
Sample Research Protocol
Protocol REVITA 2018 April 9, 2018 A Clinical Investigation Evaluating Efficacy of a Full- Thickness Placental Allograft (Revita®) in Lumbar Microdiscectomy Outcomes Primary Investigator: Thomas Morrison III, MD Northside Hospital 1000 Johnson Ferry RD. N.E., Atlanta, GA 30342 Phone: 404-256-2633 Fax: 404-255-6532 Sponsor: StimLabs 1225 Northmeadow Parkway, Suite 104, Roswell, GA 30076 Phone: 888-346-9802 Fax: 470-719-2085 Confidential Page 1 of 19 Protocol REVITA 2018 April 9, 2018 STUDY SUMMARY A Clinical Investigation Evaluating Efficacy of a Full-Thickness Title Placental Allograft (Revita) in Lumbar Microdiscectomy Outcomes Methodology Randomized, controlled trial, blind study Estimated duration for the main protocol (e.g. from start of Study Duration screening to last subject processed and finishing the study) is approximately 2.5 years Multi-center (Polaris Spine and Neurosurgery Center, Atlanta, GA Study Center(s) and Northside Hospital, Atlanta, GA) Primary Objective: To evaluate the efficacy of Revita full thickness placental allograft in improving back and leg pain post- microdiscectomy Objectives Secondary Objectives: To evaluate post-microdiscectomy reherniation rate in patients treated with Revita Number of 182 randomized patients in two arms; Treatment and Control Subjects Inclusion Criteria - Male and female patients, 18-70 years old, in any distribution. - Symptomatic with radiating low back and leg pain for greater than 6 months prior to surgery with a clinical diagnosis of lumbar protruding disc. Diagnosis and - Consent and compliance with all aspects of the study Main Inclusion protocol, methods, providing data during follow-up contact Criteria - See methods section for full list of inclusion criteria Exclusion Criteria - Male and female patients younger than 18 years old or older than 70 years old. -
A Simplified Protocol for Reversing Phenotypic Conversion of Ralstonia
International Journal of Environmental Research and Public Health Article A Simplified Protocol for Reversing Phenotypic Conversion of Ralstonia solanacearum during Experimentation Pramod Kumar Sahu 1,* , Shailendra Singh 1 , Amrita Gupta 1, Udai B. Singh 1 , Surinder Paul 1 , Diby Paul 2 , Pandiyan Kuppusamy 3, Harsh V. Singh 1 and Anil Kumar Saxena 1 1 ICAR-National Bureau of Agriculturally Important Microorganisms, Maunath Bhanjan UP-275103, India; [email protected] (S.S.); [email protected] (A.G.); [email protected] (U.B.S.); [email protected] (S.P.); drharsh2006@rediffmail.com (H.V.S.); [email protected] (A.K.S.) 2 Pilgram Marpeck School of Science, Technology, Engineering and Mathematics, Truett McConnel University, 100 Alumni Dr. Cleveland, GA 30528, USA; [email protected] 3 ICAR-Central Institute for Research on Cotton Technology, Ginning Training Centre, Nagpur, Maharashtra 440023, India; [email protected] * Corresponding author: [email protected] Received: 16 April 2020; Accepted: 11 June 2020; Published: 15 June 2020 Abstract: Background: Ralstonia solanacearum has the problem of losing the virulence in laboratory conditions, during prolonged experimentation. Since pure colonies of R. solanacearum contain cell fractions differing in virulence, it was considered worthwhile to find a way of selecting the cells with lower attenuation. Therefore, a methodology for inducing virulent-type colonies occurrence in Ralstonia solanacearum was developed. Methods: Nutrient gradient was created by swabbing R. solanacearum culture in a slanted KMTTC medium, and Phyllanthus emblica extract was given by well diffusion. Live–dead cell imaging using BacLight, effects of ascorbic acid on cell viability, and production of virulence factors (exopolysaccharides, cellulase, and pectinase) supported this hypothesis. -
Protocol Template (Protocol)
Protocol template (Protocol) NN This is a reprint of a Cochrane protocol, prepared and maintained by The Cochrane Collaboration and published in The Cochrane Library 2013, Issue 9 http://www.thecochranelibrary.com For Preview Only Protocol template (Protocol) Copyright © 2013 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd. TABLE OF CONTENTS HEADER....................................... 1 ABSTRACT ...................................... 1 BACKGROUND .................................... 1 OBJECTIVES ..................................... 1 METHODS ...................................... 1 Figure1. ..................................... 3 REFERENCES ..................................... 5 ADDITIONALTABLES. 6 APPENDICES ..................................... 7 CONTRIBUTIONSOFAUTHORS . 21 DECLARATIONSOFINTEREST . 21 For Preview Only Protocol template (Protocol) i Copyright © 2013 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd. [Intervention Protocol] Protocol template NN1 1Not specified Contact address: N N, Not specified. Editorial group: Cochrane Metabolic and Endocrine Disorders Group. Publication status and date: New, published in Issue 9, 2013. Citation: N N. Protocol template. Cochrane Database of Systematic Reviews 2013, Issue 9. Art. No.: CDXXXXXX. DOI: 10.1002/14651858.CDXXXXXX. Copyright © 2013 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd. ABSTRACT This is the protocol for a review and there is no abstract. The objectives are as follows: To assess the effects of ... How the intervention -
Protocol Template: Observational Research Study
OCR Interventional_Drugs/Device_Protocol_Template Version: 3.0 OCR Template Date: 09-03-13 [TITLE PAGE – REVISED IRB PROTOCOL] PROTOCOL TITLE: A Randomized, Double-Blinded, Placebo-Controlled Study to Determine if Caffeine Citrate Accelerates Emergence from Anesthesia PI: Name: Aaron Fox, Ph.D. Telephone: (773) 702-2667 Fax: E-mail: [email protected] CO-I(S): Name: Zheng (Jimmy) Xie, M.D., Ph.D. Telephone: (773) 702-2667 Fax: E-mail: [email protected] FUNDING: NIH 1R01GM116119 – 01 09/01/2015 – 08/30/2019 PI: Fox Reversing Anesthesia with Caffeine Patients who are anesthetized, wake after their bodies clear the anesthetic. Our preliminary studies found that in rodents that caffeine could dramatically accelerate emergence from anesthesia. Preliminary data from a Pilot study in human subjects suggest that humans show a similar response to caffeine. The goal in this application is to determine the mechanism for the emergence and to test whether caffeine can accelerate emergence from anesthesia in human subjects. SPONSOR: Name: Principal Investigator, Aaron Fox, Ph.D. Address: The University of Chicago Neurobiology, Pharmacology and Physiology 947 E. 58th Street Telephone: (773) 702-2667 TYPE OF RESEARCH: Interventional, drug studies INTERVENTION: Study Drug Caffeine Citrate MANUFACTURER OF INTERVENTION (if applicable): Bedford Labs IND OR IDE # (applicable for IND/IDE-regulated studies): 127863 NAME OF PERSON WHO HOLDS IND OR IDE (if applicable): Aaron Fox, Ph.D. Page 1 of 71 November 3, 2016 Confidential IRB Approval Date: Author(s) IRB approved Version#: OCR Interventional_Drugs/Device_Protocol_Template Version: 3.0 OCR Template Date: 09-03-13 PROTOCOL NUMBER: PROTOCOL VERSION: 2.0 PROTOCOL VERSION DATE: Page 2 of 71 November 3, 2016 Confidential IRB Approval Date: Author(s) IRB approved Version#: OCR Interventional_Drugs/Device_Protocol_Template Version: 3.0 OCR Template Date: 09-03-13 TABLE OF CONTENTS PAGE 1. -
Sample Size Determination for Clinical Trials
Sample Size Determination for Clinical Trials Paivand Jalalian Advisor: Professor Kelly McConville May 17, 2014 Abstract An important component of clinical trials is determining the smallest sample size that provides accurate inferences. The Frequentist approach to determining sample size is most common; however there has been a recent shift towards using a Bayesian ap- proach due to its flexibility. This paper will review both the Frequentist and Bayesian branches of statistics and their respective approaches to sample size determination. As well, the advantages and disadvantages of using each method will be considered. Finally, along with the Bayesian approach to sample size determination, we will also discuss a Bayesian adaptive design for clinical trials that allows for sample size adjust- ments during the trial. 1 Introduction Clinical trials are research studies used to gain insight about new treatments or interventions, for example drugs, procedures, medical devices, etc. Clinical trials are an important part of the development process of new interventions because they determine and confirm the efficacy, as well as the safety, of an intervention. Conducting a clinical trial requires a lot of preparation, and an important aspect of de- signing a clinical trial is determining the correct sample size for that trial. Having the correct sample size limits unneeded exposure to possible harmful treatments, while ensuring accu- rate results. Additionally, determining the correct sample size saves money and resources. There are many ways in which sample size can be calculated, and all these methods aim to find the \best" sample size, or the smallest sample size necessary to get accurate and inference worthy results. -
Treating the Field As a Lab: a Basic Guide to Conducting Economic
Angelino C. G. Viceisza TREATING the FIELD as a LAB A Basic Guide to Conducting Economics Experiments for Policymaking ABOUT IFPRI The International Food Policy Research Institute (IFPRI®) was established in 1975 to identify and analyze alternative national and international strat- egies and policies for meeting food needs of the developing world on a sus- tainable basis, with particular emphasis on low-income countries and on the poorer groups in those countries. While the research effort is geared to the precise objective of contributing to the reduction of hunger and malnu- trition, the factors involved are many and wide-ranging, requiring analy- sis of underlying processes and extending beyond a narrowly defined food sector. The Institute’s research program reflects worldwide collaboration with governments and private and public institutions interested in increas- ing food production and improving the equity of its distribution. Research results are disseminated to policymakers, opinion formers, administrators, policy analysts, researchers, and others concerned with national and inter- national food and agricultural policy. IFPRI is a member of the CGIAR Consortium. ABOUT IFPRI FOOD SECURITY IN PRACTICE The Food Security in Practice technical guide series is designed for devel- opment practitioners. The guides are meant to enrich the research skills of project personnel in the field. Each volume addresses informational and methodological issues that practitioners may confront during the life of a project, explaining relevant research and operational concepts in easy- to-understand ways. All manuscripts submitted for publication as IFPRI technical guides undergo an extensive review procedure that is managed by IFPRI’s Publications Review Committee (PRC). -
FDA Guidance for Clinical Trial Sponsors
Guidance for Clinical Trial Sponsors Establishment and Operation of Clinical Trial Data Monitoring Committees For questions on the content of this guidance, contact the Office of Communication, Training, and Manufacturers Assistance (CBER) at 800-835-4709 or 301-827-1800. U.S. Department of Health and Human Services Food and Drug Administration Center for Biologics Evaluation and Research (CBER) Center for Drug Evaluation and Research (CDER) Center for Devices and Radiological Health (CDRH) March 2006 OMB Control No. 0910-0581 Expiration Date: 10/31/2021 See additional PRA statement in Section 8 of this guidance Contains Nonbinding Recommendations Guidance for Clinical Trial Sponsors Establishment and Operation of Clinical Trial Data Monitoring Committees Additional copies of this guidance are available from: Office of Communication, Training and Manufacturers Assistance, HFM-40 Center for Biologics Evaluation and Research Food and Drug Administration 1401 Rockville Pike, Rockville, MD 20852-1448 Phone: 800-835-4709 or 301-827-1800 Internet: http://www.fda.gov/cber/guidelines.htm or Office of Training and Communication Division of Communications Management Drug Information Branch, HFD-210 Center for Drug Evaluation and Research Food and Drug Administration 5600 Fishers Lane, Rockville, MD 20857 Phone: 301-827-4573 Internet: http://www.fda.gov/cder/guidance/index.htm or The Division of Small Manufacturers, International, and Consumer Assistance (DSMICA) Center for Devices and Radiological Health Food and Drug Administration 1350 Piccard Drive, Rockville, MD 20850 Phone: 800-638-2041 or 301-443-6597 Internet: http://www.fda.gov/cdrh Email: [email protected] Facts-on-Demand (faxback): 800-899-0381 or 301-827-0111 Contains Nonbinding Recommendations Table of Contents 1. -
Registration of Observational Studies
Registration of Observational Studies July 2013 Deborah A. Zarin, M.D. Director, ClinicalTrials.gov National Library of Medicine http://ClinicalTrials.gov1 ClinicalTrials.gov Definitions • Observational Study: “studies in human beings in which biomedical and/or health outcomes are assessed in pre- defined groups of individuals. Subjects in the study may receive diagnostic, therapeutic, or other interventions, but the investigator does not assign specific interventions to the subjects of the study.” • Interventional Study: “studies in human beings in which individuals are assigned by an investigator based on a protocol to receive specific interventions. Subjects may receive diagnostic, therapeutic or other types of interventions. The assignment of the intervention may or may not be random. The individuals are then followed and biomedical and/or health outcomes are assessed.” 2 http://prsinfo.clinicaltrials.gov/definitions.html Key Policies for Interventional Studies • ICMJE (Registration only) – Interventional trials • All intervention types – All phases • FDAAA (Registration and Results) – Interventional trials • Drugs, biologics, devices – Not phase 1 – US FDA jurisdiction (e.g., IND/IDE or US site) – Specific enforcement mechanisms • European Medicines Agency (EMA) (Registration and Results) 3 Reasons to Register Clinical Trials and Report Results • Human Subject Protections – Allows potential participants to find studies – Assists ethical review boards and others to determine appropriateness of studies being reviewed (e.g., harms, benefits, redundancy) – Promote fulfillment of ethical responsibility to human volunteers – research contributes to medical knowledge • Research Integrity – Facilitates tracking of protocol changes – Increases transparency of research enterprise • Evidence Based Medicine – Facilitates tracking of studies and outcome measures – Allows for more complete identification of relevant studies • Allocation of Resources – Promotes more efficient allocation of resources 4 4 5 Williams et al. -
Systematic Review Protocol and Data-Analysis Plan
Systematic review protocol and data-analysis plan A systematic review of the placebo effect of GLP-1 receptor agonists versus new class oral glucose-lowering agents in type 2 diabetes Helena M de Wit Maarten te Groen Cees J Tack Department of general internal medicine, section Diabetes Radboud University Nijmegen Medical Centre, Nijmegen, the Netherlands Contact person: H.M. de Wit Department of Internal Medicine 463, Section Diabetes Radboud University Medical Centre PO Box 9101, 6500 HB Nijmegen, the Netherlands telephone: +31 24 36 18 819 fax: +31 24 36 14 327 e-mail: [email protected] TRIAL PROTOCOL meta-analysis placebo-effect injectables versus new class OADs for T2DM V1.1 2014-8-27 This systematic review and meta-analysis will be reported according to the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) Statement.1,2 Methods of the analysis and inclusion criteria are pre-specified and documented in this protocol, and will be registered at the International prospective register of systematic reviews (PROSPERO; accessible through http://www.crd.york.ac.uk/PROSPERO/). Background and aim Every treatment is associated with a placebo-effect, which has been a focus for research for many years, especially in the area of pain relief. One of the determinants of the placebo- effect is the route of drug-administration. In migraine treatment, for example, sham surgery or sham acupuncture resulted in higher responder ratios than oral placebos.3 The role of the placebo-effect in different type 2 diabetes (T2DM) treatments is completely unknown. In the past decade, a number of new T2DM treatments have been introduced: the oral dipeptidyl peptidase-4 (DPP-4) inhibitors have no effect on weight, while the injectable glucagon-like peptide 1 receptor agonists (GLP-1 RAs) are associated with weight loss.