Textbook of clinical trials

Jump to navigation Jump to search

Ethical Issues

Helsinki agreement

Informed consent

Investigational Review Board (IRB)

HIPAA deidentification and reidentification of patients and patient privacy

Regulatory Issues and Pathways in Clinical Trials

The 1572 form and investigator responsibilities

Investigational new drug (IND) application

Investigational new drug or device exemption

510K pathway

Designing Clinical Trials

Randomized controlled trials versus observational studies

Phase 1 trials

Pharmacokinetic (PK) and pharmcodynamic (PD) assessment

Phase 2 trials

Advancing from phase 2 to a phase 3 trial

Phase 3 trials

Parallel versus dose escalation studies

Hypothesis generation

Inclusion and exclusion criteria


Primary endpoint

Secondary endpoints and exploratory endpoints

Composite endpoints

Surrogate endpoints

Sample size and power calculations

Protocol creation

Abbreviations used in clinical trials

Operationalizing Clinical Trials

Principal investigator role

Executive committee role

Steering committee role

Nurse coordinator clinical research associate role

Monitor role

Sponsor role

Randomization service

Central laboratory facility

EKG core laboratory

Angiographic core laboratory

MRI core laboratory

Genetics core laboratory

Biomarker core laboratory

Identification of sites



Recruitment of study participants

Electronic data collection

Schedule of events

Concomitant medications

Clinical event committee or event adjudication committee (CEC)

Adverse events, serious adverse events and serious unexpected events

Data safety monitoring board (DSMB)

Study drug discontinuation, withdrawal of consent, incomplete follow up and lost to follow up patients

Protocol adherence

Storage and handling of the investigational product

Data management and designing an electronic data capture form

Source documents

Study completion and close out

Protocol amendments

Publication committee

Statistical Analysis of Clinical Trials

Cleaning a dataset

Summary statistics

Intent to treat versus modified intent to treat versus as treated

Student's t tests and analysis of variance (ANOVA)

Chi square analysis and Fisher's exact test

Logistic regression

Multivariate modeling

Survival analysis

Handling missing data

Subgroup and interaction analyses

Net clinical benefit analyses

Cost effectiveness analyses

Quality-adjusted life year analyses

Interim analyses and futility analyses

Reporting results

How to write a manuscript


Positive publication bias

Study Tools

Bleeding:  ACUITY HORIZONS bleeding criteria  ●  Bleeding Academic Research Consortium  ●  CURE bleeding criteria  ●  GRACE bleeding criteria  ●  GUSTO bleeding criteria  ●  PLATO bleeding criteria  ●  STEEPLE bleeding criteria  ●  TIMI bleeding criteria

Calculators:  GFR calculation ●  Wells score calculation ●  Caprini score calculation ●  CHA2DS2-VASc score calculation ●  DAPT score calculation ●  TIMI risk score calculation

Differential Diagnosis:  D/D of Anemia ●  D/D of CK elevation ●  D/D of Chest Pain

Other:  Abbreviations ●  Hy's law

Risk Score:  GRACE risk score ●  TIMI risk score

Course Slides

Download the slides here:

Clinical Research Course Syllabus

Lecture Topic Presenter
Lecture 1 Introduction to Clinical Trials & Study Design C. Michael Gibson
Lecture 2 Description of Trial Phases - I C. Michael Gibson
Lecture 3 and 4 Description of Trial Phases - II C. Michael Gibson
Lecture 5 Study Design - I Gerald Chi
Lecture 6 Study Design II Serge Korjian
Lecture 7 Study Design Part III Arzu Kalayci
Lecture 8 Clinical Trial Protocol Lecture 1 of 4 Clara Fitzgerald
Lecture 9 Developing a Study Protocol - II Arzu Kalayci
Lecture 10 Developing a Study Protocol - III Gerald Chi
Lecture 11 Part 1:Clinical Trial Protocol Violations & Amendments

Part 2: Introduction to Observational Studies and Cohort

Clara Fitzgerald
Lecture 12 Cross-sectional studies Gerald Chi
Lecture 13 Exploratory Data Analysis: A statistician perspective Gheorghe Doros
Lecture 14 Statistical Perspective II: power and sample size Gerald Chi
Lecture 15 Statistical Perspective III: measures of association Gerald Chi
Lecture 16 Statistical Perspective IV: Overview of regression models Arzu Kalayci
Lecture 17 Ethics of clinical trial I Clara Fitzgerald
Lecture 18 Ethics of clinical trial II Sonia Ammu
Lecture 19 Running a Clinical Trial part I & II Maria Ok
Lecture 20 Running a Clinical Trial part III Danielle Feingold
Lecture 21 Importance of missing data C. Michael Gibson
Lecture 22 The Role of Academic Research Organizations in Clinical Research Sponsor Donald E. Cutlip
Lecture 23 Clinical Event Committee David Weiss