Transcript Clinical Trials - PHASE II

Clinical Trials - PHASE II
Introduction




Important part of drug discovery process
Why important??
Therapeutic exploratory trial
First time in patients
OBJECTIVES
Efficacy in patients
 Pharmacodynamic action
2.

Safety issues
Common short term side effects
OBJECTIVES
3.





Optimum dose finding
Dose efficacy relationship
Therapeutic dose regimen
Duration of therapy
Frequency of administration
Therapeutic window
OBJECTIVES
Define target patient population

Types of patients with specific indication
5.
Evaluation of potential study points

Measurement of efficacy
6. Idenditify the promising drug candidate
4.
SUBDIVISION OF PHASE II
A. Phase II a
 Pilot clinical trial
B. Phase II b
 Pivotal clinical trial
PHASE II a
PILOT CLINICAL TRIAL

Small scale

Often unblind and open label

Intended to provide experience to investigator

Feasibility trial
PILOT CLINICAL TRIAL
OBJECTIVES
1.
2.
3.
4.
To confirm that trial medicine, procedure are safe,
suitable, and operational.
Dose range of new drug
Initial efficacy evaluation of a new drug, or for
new indication
Determine the duration required
PILOT CLINICAL TRIAL
OBJECTIVE
5.
6.
7.
8.
9.
Evaluation of variables related to clinical
pharmacology
Estimation of required sample size
For evaluation of methodology
Determination of availability of patient
Exploration of ethical questions
PHASE II b
PIVOTAL CLINICAL TRIAL





Well planned, well controlled trial, in full scale
Most rigorous demonstration of drug efficacy
Conducted in units with specialist investigators with
experience of particular indication
Adequate investigational facilities to monitor safety
and efficacy
Doses are usually less than the highest doses used in
phase I
PHASE II b
PIVOTAL CLINICAL TRIAL






Usually, only 3-4 centers are included
Normally, 10-12 patients should be
studied at each dose-level
May or may not randomized
Open or double blinded trial
Placebo or comparator controlled
Further evaluation of safety,
pharmacokinetic data
SUBJECT REQUIREMENTS
Approximately 50-300 patients
required
 Narrow inclusion criteria
 Homogenous population
 Preferably not suffering from
confounding comorbities
DURATION
Commonly takes 2-3 years

REGULATORY REQUIREMENTS
Phase I and Preclinical studies must be
completed before commencing phase
II trial -FDA
 Scheduled meeting at the end of
phase II
with FDA
 Permission from DCGI before Phase II
 Fee 25,000 at DCGI

Journey to cover

Phase III trial