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Clinical Trial Process

This document provides an overview of clinical trials, including what they are, their purposes and types, personnel involved, protocols, regulations, informed consent, institutional review boards, patient recruitment, data collection, adverse events, phases, randomization, and blinding. The key points are: - Clinical trials are research studies that test new medical treatments or interventions on human volunteers to assess safety and efficacy. - They aim to answer specific questions about treatment effectiveness, safety, dosing, and quality of life effects. - Trials involve physicians, nurses, pharmacists, data managers and human subjects. They follow detailed protocols and are highly regulated internationally and by national bodies.

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Muhammad Ikram Rabbani
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57 views

Clinical Trial Process

This document provides an overview of clinical trials, including what they are, their purposes and types, personnel involved, protocols, regulations, informed consent, institutional review boards, patient recruitment, data collection, adverse events, phases, randomization, and blinding. The key points are: - Clinical trials are research studies that test new medical treatments or interventions on human volunteers to assess safety and efficacy. - They aim to answer specific questions about treatment effectiveness, safety, dosing, and quality of life effects. - Trials involve physicians, nurses, pharmacists, data managers and human subjects. They follow detailed protocols and are highly regulated internationally and by national bodies.

Uploaded by

Muhammad Ikram Rabbani
Copyright
© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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Download as PDF, TXT or read online on Scribd
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Clinical Trials

Dr. Madeeha Khalid


Department of Biochemistry
University of Okara
What is a Clinical Trials?

 Clinical trials are prospective biomedical or behavioral


research studies on human participants
 To answer specific questions
What Is a Clinical Trial?

 Effectiveness of intervention to treat a disease


 Safety of a new drug
 Defining dose administration
 Testing drug formulation
 Exploring combination therapies
 Evaluating effect of therapies on quality of life
Types of Clinical Trials

 Treatment
 Test new approaches to treat a disease
 Prevention
 What approaches can prevent disease
 Early-detection/screening
 What are new ways to find hidden disease
 Diagnostic
 How can new tests or procedures ID disease
Personnels Involved

 Human Subject Volunteers


 Physician Investigators
 Research Nurses
 Pharmacists
 Lab Techs
 Social Workers
 Data Managers
Research Protocol: Roadmap

 Detailed Research Plan that Includes:


 Objectives
 Background and Rationale
 Subject Selection Criteria
 Treatment Plan
 Study Procedures
 Response Evaluation Criteria
 Statistical Section
Human Research is Highly
Regulated
 National Regulations
 International regulations
International Regulation

 E6 Good Clinical Practice (GCP): Consolidated Guidance


 International ethical and scientific quality standard for
designing, conducting, recording and reporting trial
results.
Informed Consent

 Learning the key facts about a trial before deciding


whether to participate.
 Research study purpose
 Risks/Benefits
 Alternative treatments
 Confidentiality of records
 Medical treatment available if injury occurs
 Whom to contact for answers to questions
 Statement that participation is voluntary
Institutional Review Board
(IRB)
 All clinical trials must be approved and monitored by an
IRB.
 IRB is an independent committee of physicians, nurses,
statisticians, community advocates and others.
 The function of the IRB is to ensure that a clinical trial
is ethical and the rights welfare of study participants
are protected.
Patient Recruitment
Challenge
 Poor patient recruitment is the number one reason that
trials fail.
 Only 3 to 5 percent of newly diagnosed adult cancer
patients participate in a clinical trial.
 Reasons for this relatively low number are many.
Recruitment Strategies

 Physician trust and contact


 Study staff contact
 Speaking to community groups
 Newspaper and radio Ads
 Internet websites
 Physician referrals
Subject Data Collection

 Data is collected on case report forms (CRF)


 Much of clinical data is taken from the subjects medical
record (source documents)
 Pharmaceutical and device trials, data is verified by
multiple players
Serious Adverse Events

 Events that results in any of the following:


 Death or life-threatening
 Hospitalization or prolonged hospitalization
 Persistent or significant disability/incapacity
 Congenital anomaly/birth defect
 Events that are serious, unexpected, and related or
possibly related to participation in the research must be
reported to the Sponsor, FDA and IRB in a timely
manner.
Clinical Trial End Product

 Ideal: Unambiguous conclusion regarding the clinical


outcome of the test treatment/device.
 Always strive for the ideal, but in most cases have to
settle for the best comprise.
The Randomization Process

 The randomized clinical trial is the standard by which


all trials are judged

 In the simplest case, randomization is a process by


which each participant has the same chance of being
assigned to either intervention or control
Purposes of Randomization

 To generate comparative groups

 To enable valid statistical tests


Blindness

• Open label (unblinding)

• Single blinding (patients only)

• Double blinding (patients and investigators)

• Triple blinding (patients and investigators and


Monitoring investigators)
Phases

 Pre clinical (animal)


 Phase 1
 Phase 2
 phase 3
 Phase 4
Pre clinical

 Cell studies
 first tests done on a new treatment
 human cancer cells or animal cancer cells.
 Animal studies
 tested next on cancers in live animals
Phase 0

 a few small doses of a new drug in a few people


 Initial drug interaction and response to the drug
 extra tests such as biopsies, scans, and blood samples
 Phase 0 studies aren’t widely used
 10-15 people
 Microdosing study
Phase 1

 First few people in the study get a very low dose and
monitored closely
 what the drug does to the body and what the body does
with the drug.
 Safety is the main concern
 rare side effects may not be seen due to low sample
size
 Placebos (inactive treatments) are not used in phase I
trials.
 Include a small number of people (20-40)
 ADME studies
Phase 2

 A group of 25 to 100 patients with the same type of


disease upto 300 subjects
 using the dose and method found to be the safest in
phase I studies.
 everyone gets the same dose
 Placebos (inactive treatments) are not used in phase II
trials.
 Exploratory studies
 Hypothesis testing
 Therapeutic effect
 Proves primary hypothesis
 Efficacy
 Effect size
 Adverse events
Phase 3

 Randomized double blind


 a large number of patients, at least several hundred to
thousand
 Often done in many places across the country (or even
around the world)
 offered in local community hospitals and doctor's
offices.
 last longer than phase I and II studies.
 Placebos may be used
 Confirmatory trials
 Definitive proof of safety
 Additional safety data
 Adverse effects on prolonged uses
 Marketing of drug
Phase 4

 look at drugs or therapies that have already been


approved
 drugs are available for doctors to prescribe for patients,
 answer important questions still needed

 involve thousands of people.


 safest type of clinical trial because the treatment has
already been studied a lot
 safety over time.
 These studies may analyze effects on quality of life or
cost effectiveness.
Trials in special population

 Pregnant and nursing women


 Children
 Geriatric patients
 Organ dysfunction patients
 Ethnic group
Regulatory Body in Pakistan

 DRAP
 Clinical trials registry
 Clinical trials subject registry
Therapies currently awaiting
approval
Diseases as candidates for
gene therapy
Trends in the use of vectors

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