Participate

About Clinical Studies

Clinical research studies evaluate investigational therapies that may be potential treatment options.  

Even if participants don’t directly benefit from the results of the clinical study, the information collected adds to scientific knowledge and may help families like yours in the future.  

Caregivers and their children who take part in clinical trials are essential to the process of improving pediatric medical care. 

What does participation involve?

You will be responsible for ensuring the participant attends all study visits with the study doctor and study staff. You should know that some days at the clinic may be quite long and tiresome because the...

Read More

Social Media

We strongly encourage you not to publicly share information about a participant’s response to treatment received in this clinical trial on any social media platforms. Public postings of information could influence your own or others’ assessments of a participant’s progress...

Read More

Providing consent on behalf of the participant

Before participants can be enrolled into a clinical trial, they need to provide their consent. Informed consent means that the potential participant is provided with all the information they need to...

Read More

Accurately completing caregiver assessments

You will be required to complete several caregiver assessments. These assessments help the study team determine whether individuals see improvement or benefit from taking...

Read More

Minimizing Assessment Bias

The good news is that merely being aware of assessment bias can aid you in objectively assessing your child’s progress.

Here are some tips to help you minimize your...

Read More

What does participation involve?

You will be responsible for ensuring the participant attends all study visits with the study doctor and study staff. You should know that some days at the clinic may be quite long and tiresome because the study team will need to take a range of samples from the participant as well as monitor their condition. You will need to factor this time into the planning of any family or work commitments.

As the caregiver of the participant in this study, you will be required to complete a number of assessments yourself. The answers you give on these assessments should provide an accurate report of any changes you see in the participant’s condition. At the start of the trial, you will receive some training on how to complete the assessments that you’ll take home. These assessments will need to be completed within 1 day of a study visit.

You will be responsible for making sure the participant takes their study medication according to the dose prescribed by the study team. There will be 2 doses each day: 1 in the morning, and 1 in the afternoon or evening. The dose given will depend on your child’s weight. There should be a minimum of 8 hours between each dose, and each dose should be given on an empty stomach.

Social Media

We strongly encourage you not to publicly share information about a participant’s response to treatment received in this clinical trial on any social media platforms. Public postings of information could influence your own or others’ assessments of a participant’s progress. Because you may connect with other families involved in this trial, your perspective could affect how they evaluate their child’s response to the treatment and vice versa. 

Providing Consent

Before participants can be enrolled into a clinical trial, they need to provide their consent. Informed consent means that the potential participant is provided with all the information they need to make an informed decision about their participation in a clinical trial and to decide if the treatment is right for them.

Due to the nature of Phelan-McDermid syndrome, and the fact that this clinical trial involves minors, you will be required to provide informed consent on behalf of your child. Understanding that you’ll be deciding to participate in this trial on behalf of your child may take some time, and that is okay. You must make this decision independently, without the influence of the study team. However, your study doctor will be able to answer any specific questions you may have that would help you make your decision.

Assessments

You will be required to complete several caregiver assessments.  These assessments help the study team determine whether individuals see improvement or benefit from taking the study drug. This will also help the team understand how the participant is progressing throughout the treatment period. It is important to make sure that you complete each assessment as accurately as possible, and that you’re aware of the natural bias you will have of wanting to see an improvement in your child’s symptoms and behaviors. It’s important to understand that this is a normal response, but it will affect how you report changes in your child’s symptoms and behaviors.

Minimize Bias

The good news is that merely being aware of this bias aids you in objectively assessing your child’s progress.

Here are some tips to help you minimize your unintentional bias: 

  • Strive to objectively provide accurate reports at all times
  • Recognize that it is acceptable to report “no change.” You won’t be disappointing the site staff if the study treatment doesn’t work
  • Recognize that the study drug may not work or may work in ways you do not expect
  • Recognize that your own mood or level of optimism/pessimism may influence the score you give on each assessment