Survey on design of clinical trials to improve bone health in DMD - Parent/guardian version

Poor bone health and fractures are very common in boys with DMD. However, there is still very little information on how to treat weak bones (osteoporosis) in these boys. We would like to develop clinical trials to improve bone health in DMD and would like to know your opinion. Your opinion is extremely important and we would take your feedback into consideration when we are designing our research study. We would be very grateful if you could fill in this survey which consists of 15 questions and should take you approximately 15 minutes. If you are not a parent/guardian or primary carer of a boy with DMD, please do not fill this survey.

Thank you for taking the time to complete this survey.

Dr Shuko Joseph (Clinical Academic Fellow, Glasgow)
Ms Marina DiMarco (Princpal Neuromuscular Physiotherapist, Glasgow)
Ms Jen Dunne (Neuromuscular Nurse Specialist, Glasgow)
Dr Iain Horrocks (Consultant Paediatric Neurologist, Glasgow)
Dr Jarod Wong (Senior Clinical Lecturer Paediatric Endocrinology, Glasgow)

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* 1. Are you the parent/guardian or primary carer of a child with DMD?

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* 2. Your child's age in years 

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* 3. Has your child ever had a broken bone (fracture) or a compression fracture/collapse of his back bone (vertebral fracture)?
[Please only tick one answer]

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* 4. Has your child ever been treated with bone strengthening medicines (Bisphosphonate medicines for example Pamidronate, Zoledronate, Risedronate or Alendronate)? 
[Please only tick one answer]

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* 5. How concerned are you about your child's bone health?
[Please only tick one answer]

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* 6. Do you think it is worth doctors trying to find a better treatment to improve your child's bone health?
[Please only tick one answer]

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* 7. In a clinical trial, several outcomes will be measured. However, one outcome needs to be the main outcome measure.
What do you think is the most important outcome measure in a clinical trial to improve bone health in DMD?
[Please only tick one answer]

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* 8. Do you think that the opportunity of strengthening your child's bones is worth 6 monthly infusions via a drip into his veins as part of a clinical trial?
[Please only tick one answer]

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* 9. Do you think the opportunity of strengthening your child's bones is worth 3 monthly injections under the skin as part of a clinical trial?
[Please only tick one answer]

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* 10. In a randomised clinical trial, you are not able to choose which treatment your child would receive. Participants are put into group by chance.
Would you be happy with this?
[Please only tick one answer]

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* 11. In a randomised clinical trial, one group will be allocated to receive the new treatment and the other group will receive no treatment or usual treatment (control group).
Would you be happy with this?
[Please only tick one answer]

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* 12. In a randomised placebo clinical trial, one group will be allocated to receive the new treatment and the other group will receive placebo (a liquid which looks exactly like the active medicine but does not contain the active medicine).
Would you be happy if your child was selected to be in the group which will receive placebo to be given as an infusion via a drip into the veins?
[Please only tick one answer]

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* 13. In a randomised placebo clinical trial, one group will be allocated to receive the new treatment and the other group will receive placebo (a liquid which looks exactly like the active medicine but does not contain the active medicine).
Would you be happy if your child was selected to be in the group which will receive placebo to be given as injections under the skin?
[Please only tick one answer]

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* 14. If the randomised clinical trial to improve bone health was happening now, would you be willing for your child to join?
[Please only tick one answer]

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* 15. Did your child assist in completing this survey?
[Please only tick one answer]

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