Information For Caregivers
What does participation involve?
You will be responsible for ensuring that the participant attends the study visits to meet with your study doctor and other study staff. It is important to know that some days in 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 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. These assessments will accurately report 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.
It is important to know that 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, one in the morning, and one in the afternoon or evening. The dose will be dependent on your child’s weight. There should be a minimum of 8 hours between each dose, and it should be given on an empty stomach.
Providing consent on behalf of the participant
Before the participant can be enrolled into the clinical trial, they need to provide their consent. Informed consent involves providing the potential participant with all the information they need to make an informed decision about participation in a clinical trial and decide if the treatment is right for them.
Due to the nature of Pitt Hopkins syndrome, you may be required to provide informed consent on behalf of the participant. 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 decide.
Accurately completing caregiver assessments
You will be required to complete a number of caregiver assessments. These will help us understand whether individuals see improvement or benefit from taking the study drug.
This will also help us understand how the participant is progressing throughout the treatment period. It is important to make sure that you fill out each assessment as accurately as possible.
It is important you’re aware of the natural bias you will have of wanting to see an improvement in your child’s symptoms and behaviours. It’s important to recognise that this is a normal response but will, however, affect how you report changes in your child’s symptoms and behaviours.
What can you do to minimise this bias?
The good news is that merely being aware of this can help control this bias and help you to objectively assess the participant’s progress.
Here are some tips to help you minimise your unintentional bias:
- Objectively provide accurate reports at all times
- Recognize that it is acceptable to report “no change”. You won’t be letting the site staff down if the study treatment doesn’t work
- Recognise 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
Social media
It is strongly encouraged that you don’t publicly post about the participant’s response to treatment in this clinical trial on any social media platforms. This is because it may influence how you and others rate/assess a participant’s progress. You may be in similar networks or circles with other families who are participating in this trial and your perspective may influence how they assess their child’s response to the treatment in this trial, and vice versa.
