Juvenile Dermatomyositis Study Survey

Please share your ideas with iCAN

Dear iCAN Members,

Thank you for taking the time to share your valuable feedback.

Today, we are conducting a survey to collect your feedback on a clinical study of medicine called PF-06823859, which is being developed for adults with dermatomyositis and children with juvenile dermatomyositis.

This survey contains 21 questions and should take no more than 15 minutes of your time. Your response is greatly appreciated and will help the Study Team and Researchers improve how the study is designed and the overall participant experience in the study.

Introduction for the study:

Juvenile dermatomyositis (JDM) is a rare autoimmune disorder that causes muscle weakness and skin rash but also may cause heart problems, difficulty swallowing, and arthritis. There is no known cure for JDM and the current treatments are limited to managing the symptoms and inflammation. The medications used to treat JDM are immunosuppressive drugs such as steroids. For adults, intravenous immunoglobulin (IVIG) is approved in the United States and Europe for treatment of adults with dermatomyositis, but there is no approved therapy for JDM.

An upcoming clinical trial will evaluate the safety and efficacy of a new medicine in adults and adolescents (>12 years and older) with dermatomyositis. The study will last about a year and a half, and will include intravenous (IV) infusion with the study drug or placebo every 4 weeks for 52 weeks. Participants who take corticosteroids or immunosuppressants will be able to continue to take steroid medications and/or one immunosuppressant during the study . After study completion, participants who are willing and eligible will be invited to continue in a long-term extension study of active therapy with the study medicine.

In the survey below, you will be asked to provide your feedback on this study and the procedures involved.

Thank you for your feedback and time completing this survey!

2.Are you completing this survey as: (please choose one option)

Myself

Sibling

Parent/Caregiver

3.How old are you?

6 years to less than 9 years

9 years to less than 12 years

12 years to less than 18 years

18 years to less than 29 years (young adult)

Not applicable as I am a parent completing it for my child.

4.If you are a parent, how old is your child?

5.Do you have a rare disease?

Yes

Not sure

If Yes, what type?

6.Do you have a chronic condition?

Yes

Not sure

If Yes, what type?

7.Do you have an autoimmune disorder (Autoimmune conditions are conditions where the person’s natural immune system mistakes its own healthy tissues and systems as foreign and attacks them)?

Yes

Not sure

If Yes, what type?

8.Do you have juvenile dermatomyositis (JDM)?

Yes

Other (Please provide additional explanation in the space below)

9.Do you have juvenile polymyositis (JPM)?

Yes

Other (Please provide additional explanation in the space below)

10.If you have JDM or JPM, at what age were you diagnosed?

0 to <5 years

5 years to <8 years

8 years to <11 years

11 years to <14 years

14 years to <18 years

Not applicable because I have/my child has a different dermatological or immunological condition or other condition

11.How interested would you be in participating in the study described above, if you had JDM?

12.If you answered the previous question as not interested or very uninterested in participating in this trial, please share the reasons why:

13.How likely do you think it will be that your parent/guardian would be interested in you participating in this study?

14.If the study permitted telehealth sessions (where you would use a phone or computer to talk to your doctor – like FaceTime or Zoom), would this ENCOURAGE you in participating in the study?

Yes, this would ENCOURAGE me to enroll myself/my child to participate in the study

No, this would DISCOURAGE me from enrolling myself/my child to participate in the study

Maybe (Please provide additional explanation in the space below)

15.If the study permitted home visits (where a nurse or healthcare worker comes to your home) and/or telehealth sessions, which option would you prefer?

Telehealth only

Home Health visit only

I do not have a preference for telehealth visit or home health visit

I do not have experience with telehealth or home health

Other (Please provide additional explanation in the space below)

16.The study drug is administered intravenously (IV) and the infusion lasts 1 hour, with a 1 hour observation period after the infusion is complete. If you were enrolled in this study, which visit type would you prefer?

In-clinic/in-hospital visit for all infusions

Home health visits (infusion administered in your home)

Mobile visits – the ability to have the infusion administered at a clinic that is closest to your home

Hybrid visits – infusions administered in clinic/in hospital, but at other assessments (PROs, physician assessments via telehealth)

Other (Please provide additional explanation in the space below)

17.

In this study, participants will need to wear an activity tracker, like a FitBit, to understand how the participant’s daily mobility (how much you walk, exercise, or move around) is affected by the study drug. The data will only be shared with the researchers and will show the participant’s heart rate, and physical activity daily (steps, calories, distance). The researchers will not know your child’s name and location, and will keep the data private. Data from the watch will be sent via Bluetooth using either a small data hub or mobile device app. How likely would you be able to wear the tracker every day for 52 weeks?

18.How likely would you be to wear the tracker if, instead of wearing it every day for 1 year, you wore it for 2 weeks a time, 5 times during the study?

19.How likely would your school be to allow you to wear a tracker?

20.In this study, the researchers would like to take photographs (pictures) of the participants’ skin to check for any changes in the skin rash associated with JDM during the participant’s participation in the study. These photographs will be seen only by the researchers. Would you comfortable having photographs taken for this purpose?

21.Which items would help you feel more comfortable during the study drug infusion and after the infusion observation? (a total of 2 hours) (Please check all that apply)

Other (please specify)

Current Progress,

0 of 21 answered