Enrolling Your Child in an Angelman Syndrome Clinical Trial

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by Mary Chapman |

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Are you thinking of enrolling your child in a clinical trial for Angelman syndrome? Here’s information that may help you to decide.

What is a clinical trial?

The goal of clinical trials is to determine whether a device, procedure, or pharmaceutical therapy works and is safe. Clinical trials may also test new ways using existing treatments, and assess other aspects of care or simply record daily life with a disease over time (an observational study).

They are conducted on patients, and are controlled and monitored to ensure they are carried out as intended, and that any issue or potential risk is identified as soon as possible. They are regulated by law and require governmental approval of their approach and goals before they can begin.

Is it an experiment?

Yes. Scientists who design the trial aim to gather enough scientific evidence (evidence that is clinically meaningful and reproducible) to support an application to a regulatory body — in the U.S., the Food and Drug Administration — for approval of what is being tested.

What are the different phases of a clinical trial?

New medications typically undergo three trial “phases.” In the first phase, a relativity small number of participants are drawn from the general population to test the proposed treatment for safety, tolerability, and other properties. Phase 2 involves testing in the intended patient population. Tests here are for safety and early evidence of effectiveness in a generally small number of patients. A Phase 3 trial aims to show scientifically — with a statistically valid and representative patient group, to minimize scientific bias — that the medicine is of benefit for its target population.

What are the potential benefits?

By enrolling your child in a clinical trial, you can:

  • Gain access to new treatments before they are widely available
  • Receive regular and careful medical attention from a research team that includes physicians and other health professionals
  • Help others by contributing to knowledge about new treatments or procedures

Are there any risks?

Any experimental treatment can carry unpleasant, serious, or even life-threatening risks. Additionally, the trial may require more time and attention than standard treatment would, including study site visits, repeat blood tests, and extensive procedures that might require hospital stays. There also might be stringent and sometimes complex dosing schedules.

Questions to ask

If you are considering registering your child in an Angelman syndrome trial, you should feel free to ask any questions or broach any issues concerning the trial at any time. Make a list of your questions or concerns, so you are sure to address all of them.

Here are some general questions you might want to ask:

  • What is the purpose of the study?
  • Who will fund the study?
  • How long will the study last?
  • Who will inform me of the study results?
  • What are the possible benefits and risks?
  • Are there any other options available?
  • What kinds of therapies, procedures, or tests will my child undergo?
  • Does this trial have a placebo, or sham treatment, group?
  • Who will be in charge of my child’s care?
  • How might taking part in this trial affect my child’s and my daily life?
  • Can I talk to other people in the trial?
  • Will I have to pay for any part of the trial, like travel to and from a test site?

Who can enroll in a clinical trial?

Clinical studies have standards, called eligibility criteria, outlining who can participate. These are based on characteristics such as age, gender, the disorder’s type and stage, the patient’s previous treatment history, and the presence of other medical conditions.

Angelman syndrome trials

A number of Angelman syndrome clinical trials are taking place at universities and medical centers globally. You can also contact the Angelman Syndrome Foundation about ways to stay apprised of current and upcoming studies.


Last updated: May 4, 2020


Angelman Syndrome News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.