PTC Therapeutics - What to Expect

Know What Happens During a Clinical Trial

1. Pre-screening Process

Before joining the clinical trial, you will speak with the study team to learn more about the trial in which you are interested. This will provide you with a thorough understanding of the study, including the time frame, and any measurements or procedures that will be used throughout.

2. Provide Consent

Once you fully understand the components of the study, you or your loved one can agree to participate by signing the informed consent form. This outlines your rights and responsibilities and acknowledges that the study has been clearly explained to you. Signing a consent form is not a contract—as a participant, you may withdraw from the study at any time.

3. Study Screening

During this step, the study site team reviews your medical history and conducts a physical exam to determine if you qualify to participate. Clinical studies each have different standards or guidelines that outline who can participate called “inclusion criteria”. Studies also have factors that prohibit certain people from participating called “exclusion criteria”. The study team will inform you if you qualify for the study.

4. Start Treatment

Once you qualify for the study, you are then able to be enrolled and can begin treatment. From here, you will meet with the research team leading the study to gain an understanding of next steps, including what you will need to do and how often you will meet with the team. Typically, after enrolling in a clinical study, participants will also continue to meet with their usual healthcare providers.

5. Study Participation

The type of study a participant is in will determine how often they will meet with the study team and what is required at each visit. These visits may involve performing physical exams, answering questionnaires, or carrying out other procedures that researchers use to gather data.

6. Study Conclusion

When the clinical trial is over, you may still stay in touch with the study team. After participation in the trial has ended, the participant and their healthcare provider should work together to determine the next steps for care. Some clinical trials are designed to provide access to the study treatment after the trial is over, while others are not.

Data Review

Once the study has concluded, researchers will analyze the information provided by all of the participants over the course of the study. This will help inform researchers of the next steps for the treatment.

Assess Results

Upon reviewing the data, researchers will determine whether the test results proved the study treatment to be safe and/or effective. They will then decide whether to move on to the next phase of clinical research or stop treatment development.

Publish Results

Results from clinical trials may then be published in a scientific or medical journal and made available to the public. The outcomes of the study may also be discussed at scientific meetings and by the patient community. When a study is published in a peer-reviewed journal, it signifies that the results are reviewed for quality of research and editorial standards by other healthcare professionals in that particular field of study.

Treatment Approval

Through the different clinical trials, researchers are be able to determine whether or not the treatment was proven to work safely and effectively. From here, organizations like the U.S. Food and Drug Administration (FDA) and government agencies from other countries can assess whether there is enough evidence to approve the treatment. Upon approval, the treatment can be prescribed by healthcare providers for appropriate patients.

For helpful information about how to read and better understand a scientific publication, you can download the PTC Publication Navigation Tool below.

Download PTC Publication Navigation Tool

Before Enrolling in a Clinical Trial