Clinical Trials – Awareness, Barriers & Perceived Value

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About You

Please indicate which country you reside:(Required)

Are you:(Required)

A person living with homocystinuria

A person caring for someone living with homocystinuria

Which homocystinuria are you, or someone you care for, living with?(Required)

Classical homocystinuria / cysthathionine beta-synthase (CBS) deficiency

Methylenetetrahydrofolate reductase (MTHFR) deficiency

Cobalamin C (CblC) deficiency

Cobalamin D (CblD) deficiency

Cobalamin F (CblF) deficiency

Cobalamin J (CblJ) deficiency

Other

If other, please specify the type of HCU:(Required)

What is your current treatment? (select all that apply)(Required)

Protein restricted diet

Amino acid formula

Betaine

Pyridoxine (B6)

Other

If other, please specify:(Required)

Awareness & Understanding

Have you heard about clinical trials for homocystinuria?(Required)

Yes

How did you first learn about clinical trials?(Required)

Doctor/Specialist

Patient Group

Social Media

Online search

Other

If other, please specify:(Required)

How well do you feel you understand what participating in a clinical trial involves?(Required)

Not at all well	Slightly well	Moderately well	Very well	Extremely well

What aspects of clinical trials are unclear or confusing for you?(Required)

Previous Experience

Have you ever participated in a clinical trial?(Required)

Yes

How did you make the decision to participate in a clinical trial?(Required)

What motivated you to take part in the clinical trial?(Required)

How would you rate your overall experience?(Required)

Very negative	Negative	Neutral	Positive	Very positive

What worked well, and what could have been improved?(Required)

Would you participate again? Why or why not?(Required)

Have you ever been offered the opportunity to join a clinical trial?(Required)

Yes

If yes, what influenced your decision not to participate?(Required)

Motivators, Barriers & Concerns

What would encourage you to participate in a clinical trial?(Required)

How important are the following in your decision?(Required)

	Not at all important	Somewhat important	Moderately important	Very important	Extremely important
Access to new treatments
Helping others and contributing to research
Reducing long term complications
Improving quality of life
Reducing dietary restrictions

What concerns would stop you from participating in a clinical trial?(Required)

How concerned are you about the following?(Required)

	Not at all concerned	Somewhat concerned	Moderately concerned	Very concerned	Extremely concerned
Side effects and unknown risks
Receiving a placebo
Travel to trial site
Costs (transport, time off work, childcare)
Lack of information
Trial interfering with metabolic control

Please rate your overall level of concern:(Required)

Very low	Low	Moderate	High	Very high

What is the furthest you would be willing to travel to participate in a clinical trial?(Required)

0-10 km

10-25 km

25-50 km

50-100 km

100+ km

Interstate

International

How do logistics (time, travel, cost) affect your decision?(Required)

What would make participation easier? Select all that apply:(Required)

Telehealth visits

Flexible scheduling

Travel reimbursement

Local trial sites

Home visits

Support services

Other

If other, please specify:(Required)

How would you prefer to receive information about clinical trials?(Required)

What type of information would help you feel confident in deciding whether or not to participate?(Required)

Would connecting with other patients who have participated in clinical trials be helpful in making your decision?(Required)

Yes

Unsure

What is the main reason you would or would not participate in a clinical trial?(Required)

Is there any further information you require for you to consider participating in a clinical trial?(Required)

Is there anything else you would like researchers to understand about your decision to participate, or not participate, in clinical trials?(Required)

If you could change one thing about clinical trials, what would it be?(Required)