Development Process

Considering Participating in a Clinical Trial?

Participation in a clinical trial contributes to scientific, medical, and patient communities’ knowledge about a condition. The results of clinical trials can make a difference in the care of patients by providing information about the benefits and risks of a potential treatment. There is a possibility that clinical trial participants will experience both benefits and risks associated with an investigational product, though there are some clinical trials where participants may have no expectation of benefit.

Trial participation often requires participants to show up for many visits, according to a pre-set schedule. At those visits, participants may undergo procedures, tests, and assessments, all based on the trial protocol, and its schedule of events. These visit requirements will be described prior to enrollment, in an informed consent document. Participants may not enroll in any clinical trial, even if there is no investigational product involved, until signed informed consent is provided.

What is the informed consent process?

Before enrolling in a clinical trial, it is important that a clinical trial participant (sometimes called a “subject”) or their parent/guardian receives information about the clinical trial and acknowledges that they understand and accept that information. This is called providing informed consent. The information given to a clinical trial participant or their parent/guardian includes key known risks and the potential for unknown risks of the procedures in the trial and investigational product, logistical considerations, and other details about participating in the clinical trial.

Providing informed consent is a process which begins with an explanation of the clinical trial and review of an informed consent form (ICF) that is unique to the clinical trial. After reviewing all of the material in the ICF and getting answers to all of your questions, the participant or their parent/guardian decide whether to participate or not participate in the trial. The informed consent process continues throughout the trial as investigators continue providing participants with new information available about the drug and the clinical trial.

Remember, even if you decide to participate in a clinical trial, and provide informed consent, you always have the right to say you want to take back your consent, and drop out of the study at any time, without any penalty. That said, it’s important to understand that an investigational gene therapy trial is a bit different from other kinds of trials. In most investigational gene therapy studies, the investigational product is administered just one time, and can’t be removed from the body. This is in contrast to clinical trials of other types of investigational drugs that may be taken daily or weekly and will eventually wash out of your body if you stop taking it.

The Informed Consent Form (ICF):

Provides important information about participating in the clinical trial, including known and potential risks, time/travel commitments, compensation and other details including the ability to withdraw consent at any time

Must be reviewed in detail with the clinical trial doctor and must be clearly understood by the trial participant or parent/guardian

Must be signed by the clinical trial participant or parent/guardian to acknowledge that they have been informed of and understand the risks and commitments of participating in the clinical trial

If you are considering participating in a clinical trial, you should speak with your doctor.
You can search for clinical trials that may be available on

Currently participating in a clinical trial?

Communication is a shared responsibility

When participating in a clinical trial, it is critical that all communication about you or your child’s healthcare go through the clinical trial doctor conducting the clinical trial, and his/her appointed staff.

DO speak
with the clinical trial doctor and his/her staff.

For a doctor to fulfill their responsibility as a primary investigator for a clinical trial, the doctor and their trial site staff must be your point of contact for any medically related questions or concerns about your child’s health or the clinical trial.

DO ask
as many questions as you would like and as often as you would like.

The clinical trial doctor and trial site staff want to communicate with you and want to be informed of any questions or concerns you might have. They are prepared to receive your questions and you should feel comfortable approaching them for clarity at any point in the trial, not just at the beginning.

The clinical trial doctor will be supported by nurses or other healthcare professionals who may be referred to as the “clinical trial coordinator” or “site coordinator.” You may be asked to contact these individuals with any questions or concerns. Depending on the clinical trial site, the staff may ask you to reach out to the clinical trial coordinator first and maintain that person as your first point of contact, rather than the clinical trial doctor. You will help maintain a well-run clinical trial by following the procedures suggested by the clinical trial doctor and their staff.

discuss anything about the clinical trial with anyone other than the clinical trial doctor, trial site staff, or a designated care doctor.

Discussing any aspects or outcomes of the clinical trial could influence the interpretation of trial results and threaten other patients’ access to investigational product by delaying a potential approval.

This can be especially true in clinical trials that are “un-blinded”, which means everyone in the study knows what treatment or drug they are getting. This type of clinical trial design is fairly common in the development of new drugs for rare diseases.

Social Media Considerations

While social media is an everyday tool that people use to communicate with family and friends, clinical trial participants and families must use discretion about what they share in public forums and should avoid making any references to clinical trial experiences and outcomes.

Why does it matter if clinical trial participants or families share information on social media?

  • Sharing clinical trial progress on social media may be distressing for patients and families who were unable to enroll in the clinical trial.
  • Posting on social media has the potential to bias clinical trial results, thereby undermining the integrity of the clinical trial.
  • Posting on social media could have unintended consequences, including harming clinical trial enrollment, biasing data, misrepresenting safety or efficacy outcomes, or creating other potential complications that could delay or prevent the potential regulatory approvals required to make the investigational product available to all appropriate patients.

What else may a parent want to consider when it comes to social media?

  • It is important to educate your family and friends about the potential risks of sharing medical progress of a family member or acquaintance that is participating in a clinical trial.
  • If you or your child is in a clinical trial and you want to discuss medical status, contact your clinical trial doctor before anyone else.
  • It is also important to refrain from proactively asking active clinical trial participants or their families for any information regarding their medical status. This is critical in helping to maintain the integrity of the safety and efficacy data coming out of a clinical trial.

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