Clinical Trial Basics: What Parents Need to Know About Trial Participation

Quick Overview

In this presentation, Jennifer Panagoulias discusses the basics of clinical trials and what parents need to know about trial participation. She emphasizes the importance of understanding the different types of studies, such as observational and interventional trials, as well as the phases of clinical trials. Panagoulias also explains the role of control groups and the significance of informed consent. She provides insights into eligibility criteria, enrollment processes, and resources for finding clinical trials. Ultimately, she advises parents to consult with their doctors and study investigators to make informed decisions about trial participation.


In this talk, we will discuss the basics of clinical trials and what parents need to know about participating in them. As a co-director of ABOM and someone with extensive experience in biotech and clinical trial applications, I aim to provide insights and considerations for parents who are considering trial participation for their children with Angelman Syndrome.

Understanding Clinical Trials

Clinical trials are research studies that evaluate the safety and effectiveness of new drugs or treatments. There are different types of studies, including observational studies and interventional studies. Observational studies involve enrolling subjects for observation without any intervention or drug administration. On the other hand, interventional studies, which are the focus of this talk, assign participants to receive an intervention such as a drug or device to evaluate its safety and efficacy.

Clinical trials are conducted in phases, which represent the progression of research over time. Phase 1 trials primarily focus on safety and involve a small number of participants. Phase 2 trials assess the effectiveness of the drug or treatment and enroll more participants. Phase 3 trials aim to confirm the safety and efficacy of the drug in a larger study population to seek approval. In rare diseases like Angelman Syndrome, these phases may overlap due to the limited number of patients available for study.

Considerations for Early Phase Studies

Many ongoing trials for Angelman Syndrome are in the early or Phase 1/2 stages. These studies often explore dose levels and use an ascending dose evaluation approach. Participants are assigned to different cohorts to assess the safety and efficacy of increasing doses. It’s important to ask the study investigator about the dose group being enrolled and the safety profile of earlier participants in the trial.

The Importance of Control Groups

Control groups play a crucial role in clinical trials as they provide a basis for comparison. In most Angelman Syndrome studies, there are no control groups, meaning all participants receive the active drug. However, control groups can take various forms, including placebo-controlled studies, active control groups, sham procedures, and historical controls. Each type serves a specific purpose in evaluating the safety and effectiveness of the drug being studied.

Placebo-controlled studies involve a group receiving a dummy treatment with no active drug. Active control groups compare a new drug to an already approved drug. Sham procedures create a placebo effect by making participants believe they have undergone a procedure when they haven’t. Historical controls use external groups of patients not enrolled in the study for comparison. Control groups help researchers understand the true effects of the drug and minimize bias in the results.

Understanding Consent and Risks

Consent is a critical part of participating in a clinical trial. During the consent process, participants are fully informed about the research, its purpose, duration, involved procedures, risks, benefits, confidentiality, compensation, and who to contact for questions or adverse events. Phase 1 trials may have less known about the drug’s safety, while late-stage trials have more clinical experience to inform the consent process. It’s essential to ask questions about safety data from prior studies and understand the risks involved.

Enrolling in a Clinical Trial

To enroll in a clinical trial, participants must meet the eligibility criteria set by the study. These criteria ensure patient safety and may also consider the study design’s requirements for measuring the drug’s effects. It’s important to note that the company does not enroll participants; it is the study doctor who determines eligibility and enrolls individuals. Resources like and advocacy organizations can provide information about ongoing trials and study contacts.

Making the Decision to Enroll

Deciding whether to enroll in a clinical trial is a personal and medical decision that should be made in consultation with the study doctor. It involves considering the family’s risk tolerance, meeting the study’s conditions, and the doctor’s assessment of the best course of action. Taking the time to ask questions and gather all necessary information is crucial before making a decision.


Participating in a clinical trial is a significant decision for parents of children with Angelman Syndrome. Understanding the basics of clinical trials, the importance of control groups, the consent process, and eligibility criteria can help parents make informed choices. Consulting with the study doctor and utilizing resources like and advocacy organizations can provide further guidance and support in the decision-making process.

Talk details

  • Title: Clinical Trial Basics: What Parents Need to Know About Trial Participation
  • Author(s): Jennifer Panagoulias
  • Author(s)’ affiliation: FAST
  • Publication date: 2022-12-03
  • Collection: 2022 FAST Science Summit