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  1. Clinical Trials – Types & Design Experimental studies Submitted To – Mrs. Anuradha Submitted by – Kirti 1301 M. Pharm (Pharmacology) Maharshi Dayanand University, Rohtak
  2. Clinical Trials - Types & Design • Clinical trials – Clinical trials are research studies performed in people that are aimed at evaluation a medical, surgical or behavioural intervention. • Clinical Trials are the primary way that researchers find out if a new treatment, like a new drug or diet or medical devices is safe and effective in people. • A clinical trial is used to learn if a new treatment is more effective and has less harmful side effects than the standard treatment. • Therefore the authorities like FDA approves a clinical trial to began, scientists perform laboratory tests and studies in animals to test a potential therapy’s safety and efficacy. If these studies show favourable results, the authorities gives approval for the interventions to be tested in humans.
  3. Phases of Clinical Trials • Phase 0 (Microdosing Study) • Phase 1 (Dose Ranging & Safety ) • Phase 2 (Therapeutic Exploratory Trial) • Phase 3 (Therapeutic Confirmatory Trial) • Phase 4 (Post Marketing Surveillance) Phase 0 (Microdosing Study) Use only a few small doses of a new drug in a few people. They might test whether drug reaches tumor, how drug act in human body, how cancer cells in human body respond to the drug.
  4. Phase I • An experimental treatment on a small group of often healthy people (20-80) to judge it’s safety and side effects and to find the correct drug doses. • Objective – To determine the metabolic and pharmacological actions and the maximally tolerated dose. • Factors to be identified – Pharmacokinetics, Pharmacodynamics, Bioavailability, Metabolism, bioequivalence. • Duration – upto 1 month. • Sample Size – 20-80 • Population – Healthy volunteers or Individuals with the target disease (such as – Cancer or HIV ).
  5. Phase II • The Emphasis in phase II is on effectiveness. These trials also continue to study safety, including short term side effects • Objective – to evaluate effectiveness, determine the short term – side effects and identify common risk for a specific population & diseases. • Factors to be identified – Bioavailability, Drug – Disease interaction, Drug –Drug interactions, Efficacy at various doses, Pharmacodynamics, pharmacokinetics, patient safety. • Duration – Several months. • Sample Size – 200-300 • Population – Individuals with target disease
  6. Phase III • It gather more information about safety & Effectiveness. Studying different population & different dosages, using the drug in combination with other drugs. • If the authorities like - FDA aggrees that the trials results are positive, it will approve the experimental drug or devices. • Objective – to evaluate the overall risk – benefits ratio in a demographically diverse sample. • Factors to be identified – Drug-disease interaction’s, Drug-Drug interaction’s, risk-benefit information, efficacy & safety for sub groups, dosage intervals. • Duration- Several Years • Sample Size -100-1000 • Population – individual with target diseases.
  7. Phase IV • Used for drugs or devices takes place after the authorities like FDA approves their use. Sometimes the side effects of a drug may not become clear until more people have taken it over a longer period of time. • Objective – To monitor ongoing safety in large population’s & identify additional uses of the agent that might be approved by the FDA. • Factors to be identified – efficacy and safety within large, diverse population’s, pharmacoeconomics • Duration – ongoing (following FDA approval) • Sample Size – thousands • Population – individuals with target disease as well as new age group, genders etc.
  8. Cross sectional
  9. Experimental Studies • Experimental studies also known as interventional studies. The national institute of health (NIH) defined an interventional studies as individuals in which “participants receive specific interventions according to the research plan created by the investigator. These interventions may be drug, device , method. • It is further classified on the basis of randomization. Randomization is the heart of the experimental studies.
  10. A. Randomized Controlled Studies Randomized controlled study design is the highly reliable design and it is high level evidence to practice health profession. As the name indicates, control group is present as a comparator to the interventional group. In this study interventional group receives interventional drug and control group receive either old drug or placebo. For randomized controlled study is also named as - Clinical Trials - Gold standard study
  11. 1. Parallel Studies • A parallel Study is a type of clinical study where treatment and controls are allocated to different individuals. • In this two groups of treatments are given so that one group recieves only one type of treatment and another group receives other treatment.
  12. 2. Cross over studies • In this type of studies each patient serves as his own control. Each patient gets both treatment. • Each patient receives first treatment then wash out time is provided then other treatment is provided to the same patient.
  13. Randomized Uncontrolled Studies • In this study there is no control group present only one group present with randomization. It is so weaker when this study compared with the randomized controlled study. randomized study population Interventional group Interventional Drug Outcomes measured
  14. B. Non-randomized Studies 1. Non randomized controlled Study- • The study is same that of the randomized control study but without randomization. • The control group receive placebo or old drug and interventional group receives interventional drug and then outcomes measured. Sometimes hospital records data obtained from published literature can be used as a control group, such non- randomized or non-current studies named as historical control study.
  15. 2. Non-randomized uncontrolled study - • This study is also same as that of randomized Uncontrolled study but without randomization & control group. • Outcomes measured finally after the collection. Non Randomized Population Interventional Group Interventional Drug Outcome measured
  16. C. Others 1. Latin Square • Latin Square design helpful to study more than the one treatment simultaneously to understand the usefulness of each treatment by the comparison. For example, four treatments namely – A,B,C,D then latin Square is applied ( four replications of each treatment) as follows and do comparison.
  17. 2. Factorial Randomized Study • Studies involving two or more factors while randomizing are called factorial. • In this study, only one control group and two interventional groups are present. • The results of each group comparing with the control group, and with the help of this study we can access at a time two factors ( treatment and doses form). • Used when it is desired to study the influence of a number of factors on the treatments compared as well as their interaction with different treatments factors on a dependent variables
  18. 3. Cluster Randomized Design - • In cluster Design group of participants are randomized & made into clusters • Individuals are randomized in a group. • Example- A group include, school in a city, religion, occupation in an area, hospitals in a city. • It is simple to randomized the participants by groups. • The data are also collected group wise.
  19. References - 1.Berrnard Begaud. Dictionary of epidemiology. Published by John Wiley & Sons Ltd, UK, 2000. 7 inventi Rapid: Clinical Research Vol. 2015, Issue 3 [ISSN 0976-383X] 2015 pcr 16325 © Inventi Journals (P) Ltd. Published on Web 19/06/2015, REVIEW ARTICLE 2. G Parthasarathi, Karin Nyfort Hansen, Milap Nahata. A text book of clinical pharmacy. Published by Universities Press (P) Ltd, Hyderabad, 2013 3. D Pathak. Pharmcoepidemiology: A compement to therapeutic trails, IJPT, 2:1, 2012. 4. R C Goyal. Research methodology for health professionals. Published by Jaypee Brothers medical publishers (P) Ltd, New Delhi, India, 2013. 5. Ranjan Das, P N Das. Biomedical research methodology. Published by Jaypee Brothers medical publishers (P) Ltd, New Delhi, India; 2011. 6. Verhamme K M C, Sturkenboom M C J M. Study designs in Paediatric Pharmacoepidemiology. 7. Hilary Anne Wynne, Julia Blagburn. Drug treatment in an ageing population: practical issue implications, Maturitas, 66:246 – 250, 2010. : falling into a rut. IJP, 35:137-138, 2003. 8.Bernd Rohrig, Jean-Baptist Du Prel, Maria Blettner. Study design in medical research part 2 of a Series on the Evaluation of Scientific Publications. Dtsch Arztebl Int, 106(11): 184–9, 2009.
  20. 9. M N Parikh, Nithya Gogtay. ABC of research methodology and applied biostatistics a primer for clinicians and researchers. Published by Jaypee Brothers medical Publishers (P) Ltd, New Delhi, India, 2009. 10. P S S Sundar Rao, J Richard. Introduction to biostatistics and research methodology. Published by Phi Learning (P) Ltd, New Delhi, India, 2009. 11. R Raveendran, B Gitanjali, S Manikandan. A practical approach to PG dissertation. Published by Pharma Med Press, Hyderabad, 2014. 12. Center for Disease Control and Prevention Principles of Epidemiology in Public Health Practice, 3rd ed, Published by U.S Department of Health and Human Services, 2010. 13. K G Revi Kumar, B D Miglani. A text book of pharmacy practice. Published by Career Publications, Maharashtra, India, 2012. 14. Justin Besen, Stephanie D Gan. A critical evaluation of clinical research study designs. Journal of Investigative Dermatology, 134, 2014. 15.
  21. 16. Jesper Hallas, pharmacoepidemiolog-current oppurtunities and challenges, Norwegian Journal of Epidemiology, 11(1):7-12, 2001. 17. Brian L.Strom, Stephen E.Kennel, Sean Hennessy. Text book of pharmacoepidemiology. Published by Wiley Black Well, UK, 2013. 18. Mohit Bhandari, Parag Sachet. Clinical research made easy. Published by Jaypee Brothers medical Publishers (P) Ltd, New Delhi, India, 2010. 19. D Sudheer Kumar, Dr.J.Krishna Veni, Dr.P.Manjula. Fundamentals of clinical pharmacy practice. Published by Pharma Med Press, Hyderabad, 2010. 20. Dr.H.P.Tipnis, Dr.Amrita Bajaj, Clinical pharmacy, Published by Carrer Publications, India, 2013. 21. httpwww.iarc.frenpublicationspdfs-onlineepicancerepiCancerEpi-5.pdf 22. B Gitanjali, Pharmacoepidemiology in India: falling into a rut. IJP, 35:137-138, 2003.
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