phase zero clinical trials Dr Shivansh Verma.pptx

1. Phase Zero Clinical
Trials
P R E S E N T E D B Y – D R . S H I VA N S H V E R M A
P G 1 D E PA RT M E N T O F P H A R M A C O L O G Y
S H R I R A M M U RT I S M A R A K I N S T I T U T E O F M E D I C A L S C I E N C E S

2. What is Clinical Trials
 Clinical trials are studies to test new drugs, already
approved drugs, devices, or other forms of treatments.
 It also look at new ways to detect, diagnose, or measure the
extent of disease.

3. Why Clinical Trials Are Needed ?
To Determine weather :-
Is the new treatment safe? No treatment or procedure –even
one already in common use – is without risk. But do the
benefits of the new treatment outweigh the risks?
Is this treatment better than the standard treatment given for
this disease? Clinical trials help show if a new drug or
treatment, or a new treatment combination, works better than
what is now used.

4. Phases Of Clinical Trials
Phase 0 clinical trials: Exploring if and how new drug may work.
Phase I clinical trials: Is the treatment safe?
Phase II clinical trials: Does the treatment work?
Phase III clinical trials: Is it better than what’s already available?
Phase IV clinical trials: What else do we need to know?

5. Phase Zero Clinical Trials
Phase 0 trial is the first human clinical trial of a new, un tested drug and is also
called a Pre-phase1trial or a "proof-of-concept" trial.(Proof-of-concept .These are
the studies are typically early-stage clinical trials conducted to understand
whether an investigational product elicits a pathophysiologic signal, that is, does
it produce the expected response in individuals.)
Due to the unknown risk posed by the new drug, the sample of volunteers
selected for this phase is very small, usually between 10 and 12 individuals.
At this early stage of testing, the people chosen are usually healthy volunteers
rather than individuals suffering from the condition the drug is indicated for.

6. Classification of phase zero clinical trials
Determination of drug pharmacokinetics (micro dose trials).
Phase 0 trials involves strict pharmacokinetics investigations which further establishes the data related to various
pharmacokinetics parameters such as bioavailability, distribution, and metabolism of drug or its metabolites.
Determination of pharmacologically significant doses of drug.
These phase 0 trials are designed with a view to strictly determine the dose regimen for a molecular target
compounds or biomodulators that are used along with other drugs. This phase 0 trial cannot be used for
determination of maximum tolerated dose but can be used to derive the administration sequence and range of the
doses for further combination investigations.
Determination Drugs mechanism of action.
This phase 0 trial explores the relation of mechanism of action of drugs to drug efficacy, which assists while
evaluating the pharmacodynamic effect of a drug? For e.g. These phase 0 trials play a special role in the field of
cancer therapy for the evaluation of the chemotherapeutic drugs.

7. The Aim Of Phase Zero Clinical Trials
1. The aim of Phase 0 trials is not to test the therapeutic effects but to
check that the drug behaves as expected in humans and to gather
preliminary data regarding what the drug does to the body and what
the body does to the drug .
2. Phase 0 trial is needed to increase the safety and success of new
drug trials.
3. Drugs that progress to Phase 1 trials are tested in escalating doses to
help establish a safe dosing regimen.

8. Advantages Of Phase Zero Clinical Trials
 Saves the investment
 Shift resource utilization towards promising candidate
 Shortens Development Timeline .
 Requires less preclinical data .
 Limited Number of Subjects/Participants.(10-15)
 Limited Dosing Duration.(7 Days)

9. Disadvantages Of Phase Zero Clinical Trials
Non -linear Pharmacokinetics ie plasma conc
changes more or less than the expectations on
change in dosing rate.
False negative results can lead to discontinuation of
selected drug.
Every drug may not be suitable for phase zero trial.