This document provides a comprehensive overview of clinical trials in biotechnology, detailing the processes and phases of pre-clinical and clinical trials. It outlines the importance of regulatory guidelines and ethical standards, including Good Clinical Practice (GCP) and the role of regulatory bodies in ensuring drug safety and efficacy. Key aspects include drug design, trial phases I-IV, participant inclusion, and the evaluation of drug effects in diverse populations.

1. A Comprehensive Overview
Clinical Trials
Name:- Takshal Shah
Subject:- Biotechnology
Roll no.:- 21

2. Pre-clinical trials
Clinical Trials Phases I - IV
Regulations and Guidelines
Summary
References
CONTENTS

3. PART
ONE
Pre-clinical trials

4. Drug Design:- Drug development is a multi-step process involving the
discovery, testing, and approval of new drugs. It includes pre-clinical and
clinical trials to ensure the drug’s safety and efficacy.
What Pre-clinical trial involves?:- Pre-clinical trials are conducted before human
testing. They involve laboratory and animal studies to evaluate a drug's safety,
biological activity, and potential efficacy.
How?:- The process begins with pre-clinical trials, followed by clinical trials in
humans, spanning several phases (Phase I-IV).

5. Methods:
▪ In vitro studies are performed using cell cultures or tissue samples.
▪ These studies assess the drug’s effects on cellular processes, receptor binding, and
enzyme activity.
▪ Common assays include cell viability assays, reporter gene assays, and enzyme
inhibition studies.
Importance: These studies help in understanding the drug's mechanism of action and
its potential effectiveness before moving to animal studies.
In-Vitro Studies

6. Animal Models:
▪ In vivo studies involve testing in animal models, such as rodents, rabbits, and
primates.
▪ These studies evaluate the drug's pharmacokinetics (ADMET) and
pharmacodynamics(effects).
▪ Animal studies help predict how the drug will behave in humans, providing insights
into dosing, safety, and potential efficacy.
Regulatory Guidelines:
▪ Guidelines for animal testing are established by organizations like the International
Council for Harmonisation (ICH) and local regulatory bodies.
▪ These guidelines ensure humane treatment of animals and scientifically valid
results.
In-Vivo Studies

7. Pharmacokinetics (ADMET):
▪ These studies focus on how the drug is absorbed, distributed, metabolized,
excreted and toxicity in the body.
▪ The results help in determining the appropriate dosing regimen and
frequency for further studies.
Pharmacodynamics:
▪ This involves studying the drug's biological effects and it’s mechanism of
action.
▪ It helps in understanding the relationship between the drug’s concentration
and its therapeutic effect.
Pharmacokinetics and Pharmacodynamics

8. PART
TWO
Clinical Trial Phases

9. Before a drug can move from pre-
clinical to clinical trials, it must receive
approval from regulatory bodies, which
review the pre-clinical data to ensure
the drug's safety for initial human
testing.
REGULATORY APPROVAL:
Clinical trials are conducted in
several phases (Phase I-IV),
each with specific objectives to
further assess the drug’s safety,
efficacy, and optimal use.
CLINICAL TRIALS PHASES:
Transition to Human Trials

10. DEFINITION AND PURPOSE
Definition:
Clinical trials are systematic studies in humans designed to
evaluate the safety, efficacy, and optimal use of new drugs
or treatments.
Purpose:
These trials are essential for ensuring that new treatments
are safe and effective before they are made available to the
general public.

11. PHASE I CLINICAL TRIALS
Objectives:
▪ The primary goal is to
evaluate the safety and
tolerability of the drug.
▪ Phase I trials also
assess the drug’s
pharmacokinetics in
humans.
Participants:
▪ Typically involve
20-100 healthy
volunteers or
patients.
Procedures:
▪ Participants are given
escalating doses to
determine the maximum
tolerated dose (MTD).
▪ Side effects and adverse
reactions are carefully
monitored to establish the
drug's safety profile

12. Objectives:
▪ The main objective is to evaluate the drug's efficacy in treating the target
disease or condition.
▪ Phase II trials continue to assess safety and side effects.
Participants:
▪ Involve 100-300 patients with the target disease.
Procedures:
▪ The trials help determine the optimal therapeutic dose and monitor for
adverse effects.
▪ Efficacy is measured using clinical endpoints, such as symptom improvement
or disease progression.
Phase II Clinical Trials

13. PHASE III CLINICAL TRIALS
Objectives:
▪ Phase III trials are
designed to confirm the
drug’s efficacy in a large,
diverse population.
▪ They also aim to
monitor side effects and
collect more
comprehensive safety
data.
Participants:
▪Typically involve
1,000-3,000
participants across
multiple sites.
Procedures:
▪ These trials are often
randomized and controlled,
with participants receiving the
new drug, a standard
treatment, or a placebo.
▪ The trials are usually
double-blinded, ensuring
unbiased results.
▪ The data from these trials
are used for regulatory
approval submissions.

14. Objectives:
▪ Phase IV trials occur after the drug has been approved and marketed.
▪ They monitor long-term effects and rare adverse effects not detected in
earlier phases as well as effects of drug on childrens and pregnant ladies.
Participants:
▪ These studies involve large patient populations in real-world settings.
Procedures:
▪ Phase IV studies provide additional data on the drug’s effectiveness and
safety during routine use.
▪ The results may lead to labeling changes, usage guidelines, or even
withdrawal of the drug if necessary.
Phase IV Clinical Trials

15. PART
Three
Regulations and Guidelines

16. GCP Guidelines:
▪ Good Clinical Practice (GCP) is an international standard for conducting clinical trials
ethically.
▪ GCP ensures that the rights, safety, and well-being of trial participants are protected.
Informed Consent:
▪ Participants must be fully informed about the trial, including potential risks and
benefits, and provide consent before participating.
▪ Informed consent is a critical process in clinical trials, safeguarding participant rights.
Regulatory Bodies in India:
▪ The Central Drugs Standard Control Organization (CDSCO) regulates clinical trials in
India to ensure compliance with national and international standards.
Good Clinical Practice (GCP)

17. ▪ The Clinical Trials Rules, 2019, govern the conduct of clinical trials in India.
▪ The Drug and Cosmetics Act of 1940 regulates the approval, manufacture,
distribution, and sale of drugs in India.
▪ This act ensures that drugs available in the market are safe, effective, and of high
quality.
Regulations and Guidelines in
India

18. ▪ The Drug Controller General of India (DCGI) is responsible for approving new drugs
and overseeing clinical trials in India.
▪ The office ensures that all drugs meet required safety and efficacy standards before
entering the market.
▪ The DCGI also plays a key role in formulating policies and guidelines related to drug
regulation in India.
▪ Ethics Committees (ECs) and Institutional Review Boards (IRBs) are independent
bodies that review and approve clinical trial protocols.
▪ They ensure that the rights, safety, and well-being of participants are protected.
▪ ECs/IRBs review the scientific, ethical, and legal aspects of the clinical trial before it
begins and monitor its progress.
Role of DCGI, Ethics Committee and IRBs

19. ▪ GCRI is one of the leading cancer research institutions in India, located in
Ahmedabad, Gujarat.
▪ It is a major center for conducting clinical trials, particularly in oncology.
▪ GCRI collaborates with various national and international pharmaceutical companies
to test new cancer treatments.
▪ B.J. Medical College in Ahmedabad is renowned for its clinical research activities.
▪ The college conducts a wide range of clinical trials in various therapeutic areas,
including infectious diseases, cardiology, and more.
▪ It has well-established infrastructure and experienced faculty for conducting high-
quality clinical research.
Clinical Trials in Gujarat

20. Summary:

21. TAKE AWAYS
SYSTEMATIC STUDIES IN HUMANS TO EVALUATE SAFETY,
EFFICACY, AND OPTIMAL USE OF NEW
DRUGS/TREATMENTS.
I II
IV III
Tests safety, tolerability, and
pharmacokinetics with 20-100
participants.
Post-approval monitoring of long
-term effects and effect on
Childrens and Pregnant females.
Regulations: Governed by Good Clinical Practice (GCP), Drug and Cosmetics Act (1940),
and Clinical Trials Rules (2019). Overseen by the DCGI and ethics committees.
Assesses efficacy and optimal
dose in 100-300 patients.
Confirms efficacy in 1,000-3,000
participants for regulatory
approval.

22. 1. Drug Design: Principles and Applications
2. Saeidnia, S., Manayi, A., & Abdollahi, M. (2015). From in vitro experiments
to in vivo and clinical studies; pros and cons. Current drug discovery
technologies, 12(4), 218-224.
3. Mahan, V. L. (2014). Clinical trial phases. International Journal of Clinical
Medicine, 5(21), 1374-1383.
4. Stephenson, C. M., Levin, R. D., Spector, T., & Lis, C. G. (2013). Phase I
clinical trial to evaluate the safety, tolerability, and pharmacokinetics of high-
dose intravenous ascorbic acid in patients with advanced cancer. Cancer
chemotherapy and pharmacology, 72, 139-146.
5. Lee, L. H., Wu, C. L., Lee, B. R., & Lee, C. J. Clinical trials from phase I to
phase IV. Clinical Trials of Drugs and Biopharmaceuticals, 113-9.
6. Clinical Trials and Regulations P6 M28-33
7. Images
References

23. THANK YOU
From:-
Takshal Shah