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ABOUT CLINICAL TRIALS

There is a lot to know about clinical trials and much to discuss with your health care provider when considering clinical trial participation. The following information can get you started.

What Is a Clinical Trial?

A clinical trial is a research study that helps determine if a new treatment is effective and safe. Before an investigational medication can be approved by health authorities (such as the US Food and Drug Administration or the European Medicines Agency) and prescribed by doctors, it must undergo a thorough testing process.

Who Participates in a Clinical Trial?

Depending on the type of clinical trial, participants can be either healthy volunteers or people with the disease that the investigational medicine is under evaluation to treat. For diseases that are caused by a genetic mutation, trial participants must have the particular disease-causing mutation in order to participate in the trial.

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Why Choose to Participate in a Clinical Trial?

The discovery and development of new and better medicines rely on the participation of patients and volunteers. Clinical trials benefit all people affected by a disease by advancing medical science. But the decision about whether to participate is a highly personal one.

Some people choose to participate for the chance to have early access to a potential new treatment for their disease. For people with rare and genetic diseases, there may be no approved treatments and the investigational medicine may be the only treatment option available.

A clinical trial may also give people access to a medicine that is administered in a way that makes management of their disease easier and more convenient.

Others may be motivated to join a trial by the chance to contribute to the advancement of science and potential breakthroughs that may help patients diagnosed with the disease in the future.

How Are Clinical Trials Conducted?

Before a clinical trial can start, researchers create a plan called a “protocol.” The protocol describes:

Who can participate in the trial
What information researchers will collect
How long the trial will last
What tests and procedures will be conducted during the trial
What medical treatments will be used, at what dosages, and how they will be delivered

The protocol is reviewed by scientists, regulators, and ethics committees to protect the safety of participants. Once the protocol is approved by these groups, the trial can begin. During a trial, participants receive treatments and have tests done according to the protocol. The research team collects information from the participants that helps them to evaluate the safety profile and effectiveness of a trial treatment.

Clinical Trials Risks and Benefits?

Clinical trials are not without risk—the investigational medicine being tested might prove ineffective or cause side effects that researchers did not anticipate. The decision to participate in a clinical trial should not be taken lightly and should be discussed with your healthcare provider. Together, you can review clinical trial information, and weigh the potential risks and benefits so you can make an informed decision.

What Are the Phases of a Clinical Trial?

Clinical trials of investigational medicines are conducted in phases, with each phase building on the information learned in the previous phase. Each phase of a trial has a different purpose and helps researchers answer different questions. At each phase, potential participants are provided with information learned from the previous research to help them an informed decision about joining a study.

Phase I Clinical Trials:

Phase I clinical trials are usually small trials that are the first that involve people following lab and animal studies. Evaluating the safety of the investigational medicine is the main purpose. Participants are often healthy volunteers but may also be people with the disease that the investigational medicine is intended to treat. Phase I studies primarily look at what dose of the treatment may be ideal, how the body responds to the treatment, and what the side effects are.

Phase II Clinical Trials:

Phase II clinical trials focus on confirming the ideal dose or doses of the investigational medicine and continue to study its safety. Phase II trials involve a larger number of participants, all of whom are patients with the disease or condition for which the treatment is being studied.

Phase III Clinical Trials:

Phase III clinical trials generally include the largest number of patients from multiple sites, often from all over the world. This phase is intended to produce data for health regulatory authorities to determine that the investigational medicine is safe and effective for the intended indication.

Phase IV Clinical Trials:

Phase IV clinical trials, also known as post marketing surveillance trials, are conducted after an investigational medicine is approved by health authorities and made commercially available for broad public use. Phase IV trials monitor the safety of the approved medicine in a larger group of patients over a much longer period of time.

Where Does a Clinical Trial Take Place?

Clinical trials can take place in a variety of locations, usually hospitals, doctor’s offices, or community clinics. Research teams made up of doctors, nurses, and other health care professionals follow the protocol at each location.

Why Diversity Matters in Clinical Trials?

Not all people respond to a medicine in the same way. Ensuring appropriate diversity of age, sex, race, ethnicity, and other factors in clinical trials is critical to the ability to evaluate the effectiveness and safety of potential new treatments for all people who may benefit from them.

Learn about what Alnylam is doing to increase diversity, equity, and inclusion in our clinical trials ›