The document summarizes key aspects of Phase II clinical trials. It discusses that Phase II trials involve 100-400 patients and aim to establish therapeutic efficacy, determine optimal dosing, and further evaluate safety and tolerability. Randomized, cross-over, and platform trial designs may be used. Eligible patients typically have the target disease or condition. Sample size calculations are performed to ensure sufficient statistical power. Phase II trials generally last several months to 2 years.

1. Presentation
On
Phase-II Clinical Trial

2. Group members
Mahamuda Jahan Shelly (ID:11715004)
Tanvirul Islam (ID: 11715010)
Samiha Rahman (ID: 11715041)
Md Saiful Islam (ID:11715042)
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3. Mahamuda Jahan Shelly
ID:11715004
Department of Pharmacy
Comilla University

4. Introduction
■ Clinical trials are studies performed with human subject to test new drugs or combinations of
drug , new approaches to surgery or rediotherapy or procedures to improve the diagnosis of
disease the quality of life of the patient.
■ Clinical trials generate data on safety and efficacy. They are conducted only after they have
received health authority/ethics committee approval in the country where approval of the
therapy is sought.
■ Clinical trial is the mainstay for bringing out new drugs to the market.
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5. Phases of Clinical Trial
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6. Phase-II Clinical Trial
■ It is also known as therapeutic exploration and dose ranging.
■ Conducted by physician , trained as clinical investigators.
■ Based on a specific inclusion and exclusion criteria about 100 to 400 patients
are selected.
■ The main aim is establishment of therapeutic efficacy doses range and ceiling
effect in a controlled setting.
■ Tolerability and pharmacokinetics are studied.
■ Phase can be blinded or open labelled.
■ Carried out at 2-4 centre.
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7. Phase II studies: Pre-requisites
■ Review of Phase I data
. Innovator / Experts
. IRB
. DCGI
■ Prior approval by IRB and DCGI is mandatory
■ For new actions of marketed drug , start with phase II ( Phase I exemption
obtained )
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8. Objectives of Phase-II
■ Efficacy in patients (primary objective)
■ Safety issues ( secondary objective)
■ Optimum dose finding
. Dose efficacy relationship
. Therapeutic dose regiment
. Duration of therapy
. Frequency of administration
. Therapeutic window
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9. Tanvirul Islam
ID: 11715010
Departmment of Pharmacy
Comilla University

10. TYPES OF PHASE-II CLINICAL TRIALS
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11. Stages of Phase-II
Phase IIB
“Dose
response”
studies
“Proof of
concept
(PoC)”
studies
Phase IIA
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12. Types based on process design
Multi-
stage
design of
phase –II
clinical
trials
Randomiz
ed design
of phase-II
clinical
trials
Platform
design of
phase-II
clinical
trials
Cross-over
design of
phase-II
clinical
trials
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13. 13

14. Randomized design
■ Randomize patient to different drugs or dose levels of the same drug
■ Can estimate difference between treatments
■ Can pick the treatment of best response
■ Randomization produces balanced groups
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15. Platform trials
■ Multiple treatments
evaluated simultaneously
■ More efficient than
traditional RCT designs
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16. Cross-over design
■ In a crossover study
design, two or more
treatments (e.g.,
drugs, procedures)
are provided to
subjects at different
time periods, and the
sequence of
treatments is
randomized for each
subject.
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17. SAMIHA RAHMAN
ID: 11715041
Department of Pharmacy
Comilla University

18. Phase II(a) vs Phase II(b)
Phase II (a) Phase II (b)
EARLY PHASE LATE PHASE
Pilot clinical trials Pivotal clinical trials
20-200 Patients 50-300 Patients
Not multicentric Multicentric
Single Blind comparison with a standard
drug
Double Blind comparison with a placebo
or standard drug
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19. How do doctors put volunteers into groups
in phase-II trial:
■ Use computer program
■ Put volunteers into different groups
■ Each volunteer has an equal chance of going in any of the groups
■ This process is known as Randomization
■ Research staff checks the benefit of the treatment
■ If the staff change the volunteer into new treatment groups, the trial result will
be changed
■ But randomization helps to avoid this
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20. Population of Phase-II Trial
Consists of multiple subgroups
Specify the response rate and prevalence of each subgroup
Calculate the response rate for whole population
Choose standard phase 2 design to test on the response rate
Observed prevalence among the accrued patients to the study may be quite different
More high risk patients are rejected
20-300 Subjects
Individual with target disease
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21. Sample Size Determination
 Having the right sample size can ensure that if a statistically significant difference
exists, it will be uncovered.
 Effect size is small a larger sample size is required
 Effect size is large a smaller sample size is required
Correct sample size assure
Correct participants
Having a sufficient sample size can help save money and make sure the efficient use of
resources
2 groups
People receive the usual treatment
People receive the usual + new treatment
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22. ■ About 70% of potential new drugs will progress to phase 2
■ Measuring safety and evaluating side effects
■ Most research projects fail in phase 2 due to less effectiveness of product
Several months to 2 years
or
<1-2 years
Duration of Phase-II Trial
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23. MD Saiful Islam
ID:11715042
Departnent of Pharmacy
Comilla University

24. Goals of phase-ll trial
Primary goals
■ To evaluate the efficacy in the
patient with particular disease
■ To evaluate the safety occasionally
■ Finding the best dose
Secondary goals
■ To investigate pharmacokinetics of
drugs
■ To investigate pharmacodynamics
of drug
■ To evaluate immunogenicity
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25. Dose determination
■ Also known as dose finding phase
■ Maximum tolerable dose is determined in phase-l
■ Dose is determined by dose ranging study
■ Different doses of an agent are tested to establish the best dose
■ Trial starts with minimum dose but not exceed maximum tolerable dose
■ A dose ranging study includes a placebo group and a few groups receiving
different dose of the drug
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26. Eligibility of the subject
■ Patients with particular disease that the drug being studied is trying to cure
■ Patients will have short medical history
■ Patients with multiple diseases should be avoided
■ Patients should be able and willing to comply with the requirement of the trial
■ Patients should voluntarily agreed for trial
■ Pregnant and breastfeeding female should be excluded
■ Patient allergic to the drug should be excluded
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27. THANK YOU
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