Brief overview of Clinical Trials

1. Clinical Trials
Overview

2. Introduction
• Sets of tests in medical research and drug
development that generate safety and efficacy
data for health intervention
• i.e : information about adverse drug reactions
and adverse effects of other treatments.
e.g.: drugs, diagnostics, devices, therapy
protocols

3. Classification – Intervention
• Observational
• Interventional

4. Classification - Purpose
• Prevention trials
– medicines, vitamins, vaccines, minerals, or lifestyle changes
• Screening trials
– best way to detect certain diseases or health conditions
• Diagnostic trials
– Find better tests or procedures for diagnosing
• Treatment trials
– experimental treatments, new combinations of drugs, new
approaches to surgery or radiation therapy
• Quality of life trials/supportive care trials
• Compassionate use trials / expanded access trials
– partially tested, unapproved therapeutics to a small number of
patients who have no other realistic options
• (U.S. N.I.H. classification)

5. Phases – Drug development
T0
T1

6. Pre-Clinical
• Nonhuman
– Lab
– Animal (Rats , Primates , etc)
• Efficacy
• Toxicity
• Pharmacokinetics

7. Phases…
• Phase 0: Pharmacodynamics and
Pharmacokinetics
• Phase 1: Screening for safety
• Phase 2: efficacy of the drug, usually against a
placebo
• Phase 3: Final confirmation of safety and efficacy
• Phase 4: Post Market Surveillance
• Phase 5: Translational Research (Meta Analysis)

8. Phase 0
• First in Human Studies (FIH) /Proof of concept
studies (POC)
• Healthy subjects (10-15)
• Single sub therapeutic dose (human microdosing
studies)
• Preliminary Pharmacodynamics &
Phamacokinetics
• Usually skipped (asses with Phase 01)

9. Phase 1
• Volunteers(Healthy and Unhealthy) (20-80)
• 1st stage of testing human subjects
– Safety (Pharmacovigillance )
– Tolerance
– Pharmacodynamics and Pharmacokinetics
• Safe dosage
• Half life
• Types
– Single ascending dose studies
– Multiple ascending dose studies
– Food effect assements
• Therapeutic misconception

10. Phase 2
• Phase 2A (IIA)
– Assess dosing requirements
• Phase 2B (IIB)
– Study efficacy
• Study Design – Case Series ,RCT
• 100 -300 subjects

11. Phase 3
• Compare new treatments with the best currently
available treatment
– Confirms its effectiveness
– Monitor side effects
• Randomized controlled multicenter trials
• Drug licensing studies (Pre-marketing Phase)
• Large study groups (300- 3000 subjects)

12. Phase 4
• Postmerketing Surveillance
• Safety Surveillance
• Rare or long-term adverse effects over a much
larger patient population
• Harmful effects discovered by Phase IV trials
may result in a drug being no longer sold, or
restricted to certain uses
– Eg: Roficoxib

13. Phase 5
• Comparative effectiveness research and
community-based research

14. THANK YOU