clinical trials Flashcards
What is a clinical trial?
a type of research study where human subjects are prospectively assigned to one or more health-related interventions (such as medications, procedures, or behavioral modifications) to evaluate the effects on biomedical or behavioral health outcomes. The purpose is to determine the intervention’s efficacy and safety for specific conditions or diseases.
What is a clinical study?
involves using human volunteers (participants) to conduct research aimed at improving medical knowledge. Clinical studies can be interventional (clinical trials) or observational, where researchers collect health data without altering participants’ treatment.
How does a clinical trial differ from other clinical studies?
Clinical trials are specifically interventional studies that involve testing investigational medicinal products (IMPs) or procedures by actively intervening in the care or health of participants, unlike observational studies which only gather data without intervention.
What are the types of clinical trials?
Mechanistic trials: Aim to understand the biological mechanism of action of an intervention.
Exploratory/developmental trials: Early-phase trials that explore the feasibility and initial efficacy of an intervention.
Pilot/feasibility trials: Small-scale studies to determine if a full-scale trial is feasible, refining the study design.
Other interventional trials: Trials focusing on a specific therapeutic question or outcome.
Behavioral trials: Assess the effects of behavioral interventions on health outcomes.
Basic experimental trials: Aim to provide insights into fundamental biological or physiological processes.
What are the main study designs in clinical research?
Experimental studies: Actively test interventions and include randomized and non-randomized control trials.
Observational studies: Do not intervene but observe and analyze health outcomes, including cohort, case-control, cross-sectional, and ecological studies.
What are Randomized Control Trials (RCTs)?
RCTs test the effectiveness of a new treatment by eliminating bias through randomization, assigning participants to either an intervention or control group. This design minimizes confounding factors and aims for highly reliable results by isolating the effect of the treatment itself.
What are non-randomized control trials?
Non-randomized control trials allocate participants to groups without randomization. They are often used in single-arm trials (where all participants receive the intervention) and use external controls for comparison. This design may introduce selection bias and is generally less rigorous than RCTs.
Define a cohort study.
an observational study where a group (cohort) sharing a common characteristic is followed over time to assess the association between interventions or exposures and health outcomes. It helps determine risk factors and potential protective factors by tracking changes over time.
What is the purpose of case-control studies?
Case-control studies investigate the potential causes of a disease by comparing individuals with the disease (cases) to similar individuals without it (controls). By identifying differences in lifestyle or exposures, researchers can infer possible risk factors for the disease.
Describe cross-sectional studies.
a “snapshot” of a population at a single point in time, aiming to describe a particular variable or health outcome. They compare groups with and without exposure to determine disease prevalence, though they cannot establish causation and may be prone to reporting bias.
What is an ecological study?
examine the relationship between health outcomes and exposures at a population level. They analyze data from groups sharing characteristics (like geographic location or socio-economic status) rather than individuals, making it challenging to draw conclusions about individual risk.
What is the focus of laboratory studies in clinical research?
the initial phase of research where scientists conduct basic research, drug discovery, and preclinical testing. They model the drug’s biological effects (efficacy) and assess potential toxicities (safety) using in vitro (test tube) or in vivo (animal) studies before human testing.
What are the goals of Phase 1 clinical trials?
test a new drug or treatment in a small group of people (20-80) primarily to assess safety, dosage range, and identify any side effects. This phase typically lasts several months and has a 52% success rate, focusing on initial tolerability rather than efficacy.
Phase 2 clinical trials.
expand testing to a larger group (100-300) to evaluate effectiveness while continuing to monitor safety. These trials provide preliminary data on whether the drug works in patients with the targeted condition, lasting 1-4 years with a 30% success rate.
Phase 3 clinical trials?
large population (1,000-3,000) to confirm the drug’s safety and effectiveness, study different dosages, examine effects across various populations, and check for drug-drug interactions. This phase has a 58% success rate and generates comprehensive safety data.
