Krupanidhi College of Pharmacy, Bangalore

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Definition Clinical trials are prospective

biomedical or behavioral research studies on human subjects that are designed to answer specific questions about biomedical or behavioral interventions (novel vaccines, drugs, treatments, functional foods, dietary supplements, devices or new ways of using known interventions), generating safety and efficacy data

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WHO Definiton Any research study that prospectively

assigns human participants or groups of humans to one or more health related measures obtained in patients or participants, including pharmacokinetic measures and adverse events

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Objectives of Clinical Trials To diagnose or detect disease

To treat an existing disorder

To prevent disease or early death

To change behaviour, habits or other lifestyle factors

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Types of Clinical Trials Treatment Trials

Prevention Trials

Diagnostic Trials

Screening Trials

Quality of life trials

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Phases of Clinical Trials

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Phase 0: Studies conducted before the traditional phase 1

studies It is also called as the Exploratory IND studies Helps to take the go Vs no-go decision-making 10-15 number of subjects Limited dosing duration (<7 days) Microdosing is done (1/100 th dose of the

pharmacological effect) No therapeutic or diagnostic intent Only to study pharmacokinetic and

pharmacodynamic characteristics

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Phase I: Phase I trials are the first stage of testing

in human subjects  A small group of 20–100 healthy

volunteers is taken This phase is designed to assess the

safety (pharmacovigilance), tolerability, pharmacokinetics, and pharmacodynamics of a drug

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Phase II: Once a dose or range of doses is

determined, the next goal is to evaluate whether the drug has any biological activity or effect

 Phase II trials are performed on larger groups (100-300) and are designed to assess how well the drug works, as well as to continue Phase I safety assessments in a larger group of volunteers and patients

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Phase II studies are sometimes divided into Phase IIA and Phase IIB.

Phase IIA is specifically designed to assess dosing requirements (how much drug should be given).

Phase IIB is specifically designed to study efficacy (how well the drug works at the prescribed dose(s))

When the development process for a new drug fails, this usually occurs during Phase II trials when the drug is discovered not to work as planned, or to have toxic effects

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Phase III: This phase is designed to assess the

effectiveness of the new intervention and, thereby, its value in clinical practice

Phase III studies are randomized controlled multicenter trials on large patient groups (300–3,000 or more depending upon the disease/medical condition studied) 

Because of their size and comparatively long duration, Phase III trials are the most expensive, time-consuming and difficult trials to design and run

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Phase IV: Phase IV trial is also known

as postmarketing surveillance Trial Phase IV trials involve the safety

surveillance (pharmacovigilance) and ongoing technical support of a drug after it receives permission to be sold

The safety surveillance is designed to detect any rare or long-term adverse effects over a much larger patient population and longer time period than was possible during the Phase I-III clinical trials

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Importance of Clinical Trials The purpose of clinical trials is research, so the studies

follow strict scientific standards. These standards protect patients and help produce reliable study results.

Clinical trials are one of the final stages of a long and careful research process. The process often begins in a laboratory (lab), where scientists first develop and test new ideas.

If an approach seems promising, the next step may involve animal testing. This shows how the approach affects a living body and whether it's harmful. However, an approach that works well in the lab or animals doesn't always work well in people. Thus, research in humans is needed.

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