Understanding
clinical trials
Actor portrayals
What are clinical trials?
Clinical trials are research studies required by health authorities like the FDA for new treatments. The goal is to determine if a treatment is safe and effective for people like you. Before joining a clinical trial, we encourage you to consult your healthcare provider to discuss your eligibility and participation requirements.
What are the phases of a clinical trial?
A small trial focused on safety and determining a treatment’s proper dosage amount. This phase can include healthy volunteers or participants with a medical condition.
A bigger trial to establish efficacy and monitor side effects of an investigational product in participants with a specific medical condition or disease.
A large trial that evaluates safety, efficacy, different dosages, and compares the results to other treatments. This is the key phase before health authority approval.
After approval, a treatment is monitored for long-term side effects and effectiveness.
What do these clinical terms mean?
You may see and hear some complex medical language around clinical trials. Here’s some simple definitions for these terms.
Any negative change that occurs during a clinical trial, or during a certain time period afterwards. Events may or may not be due to the trial treatment
Data collected at the beginning of the study from all participants, including demographics and study-specific measures (e.g., health conditions, medications etc.)
A trial where neither the participant nor the researchers are told if the investigational treatment or placebo is given to reduce bias
Key requirements that must be met to participate in a clinical trial, including an exclusion and inclusion criteria
Reasons that prevent someone from participating in a trial
Reasons that allow someone to participate in a trial
A process used by researchers to inform potential and enrolled participants about the risks and benefits of a trial
Monitoring patients for an extended period (often years) after a trial to detect delayed adverse events and assess long-term safety and efficacy
Observational studies that are pre-planned and have no intervention. They are meant to track a disease’s progression and understand its natural course without treatment
A unique, 8-digit ID number given to each clinical trial registered on clinicaltrials.gov
A clinical trial where both the researchers and participants know whether the investigational treatment or placebo is being administered
A type of study where all participants receive the active study drug. Everyone, including the researchers and patients, knows the treatment being administered
An inactive treatment that looks and is given the same way as the trial treatment
The single most important measure for evaluating the effect of a treatment
A written description of a clinical trial that includes all details
A process where two or more alternative treatments (including placebo) are assigned by chance
A detailed evaluation process to determine if a participant meets eligibility criteria for a specific clinical trial. This step is completed after participants sign an informed consent form and before a participant receives any study medication
Other measures for evaluating the effect of a treatment
Any adverse event that puts someone in danger. It may be life-threatening, require hospitalization, interfere with normal life, cause birth defects, or result in death
The investigation methods and strategies used in a clinical trial
Who can participate in clinical trials?
Participation depends on trial-specific rules such as: types and stage of disease, age, biological sex, and lifestyle. Research staff at a study location will perform tests to determine your eligibility. Clinical trial diversity matters - all ages, sexes, races, and ethnicities are welcome to apply.
Explore Insmed clinical trialsTestimonial from a clinical trial participant
“I surprised myself in the sense that I can do all of this and still lead a normal life, and I’m grateful for that.”
