Clinical trials: essentials for parents

FESCA•Clinical trials and scleroderma•Clinical trials: essentials for parents

If you’re a parent or legal guardian of a child living with scleroderma, you will likely have questions if you are being invited to take part in a clinical trial.

If you’re a partner, sibling, child or friend of someone living with scleroderma, you can find information here

Should I allow my child to take part in a trial?

Deciding whether to allow your child with scleroderma to take part in a clinical trial is a big decision. It needs to feel right for your family.

Joining a trial sometimes means your child can try new scleroderma treatments that are not widely available yet and be monitored more closely. If your child gets involved, they may feel proud to be contributing to medical research and helping other children.

However, clinical trials can also involve uncertainty, possible side effects and extra appointments and tests.

Getting good information and asking questions is key.

Safety and monitoring

The safety of your child is the highest priority in any clinical trial. All trials must meet strict ethical and legal standards, and clinical trials for children are designed with extra precautions to reduce the risks as much as possible.

You can expect:

  • Careful screening before your child joins
  • Close monitoring throughout the study, usually with regular check-ups
  • A clear plan so you know how to tell the research team about any side effects
  • Oversight by independent ethics committees

If at any point the scleroderma trial is no longer considered safe or suitable, your child’s doctor will talk with you about the alternative options.

Questions to ask your child’s scleroderma research team

Ask as many questions as you want to. Your child’s healthcare professionals and the research team will answer them.

Taking notes or bringing a friend or trusted family member to appointments can help you take in all the information so you can make an informed decision.

You may want to ask:

Tick the questions you want to ask, then copy or print your list.

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Consent and assent

Consent

For children to take part in a scleroderma clinical trial, a parent or legal guardian will be asked to sign an “informed consent” form. This can apply to children under 16 in some countries and to children under 18 in other countries.

Informed consent is your right to know everything about your child's role in a clinical trial and means you:

  • Have been given clear, detailed information about the trial – this is usually given both verbally and in writing on a participation information sheet
  • Understand the benefits, risks and commitments involved
  • Have had the chance to ask questions

Consent means you agree that your child can take part, but it does not take away their rights.

  • Participation is voluntary.
  • You still have the right to remove your child from the scleroderma trial at any time without giving a reason and without their care being affected.

What is your child’s assent?

Alongside your consent, children as young as seven can also be asked to give “assent” (agreement). This involves giving them basic facts about a scleroderma research study and asking them to be part of the decision.

However old they are, it’s important that your child has a say in their own healthcare and that their wishes are respected.

How can I talk with my child about taking part in a clinical trial for scleroderma?

Whenever possible, involve your child in decisions about the scleroderma trial and their care, making sure that the conversations are honest, age-appropriate and reassuring.

You may find it helpful to:

  • Explain what a clinical trial is in simple terms – for example you could say it is a project that helps doctors and scientists see if a new treatment can work well and is safe for you and other children with scleroderma.
  • Ask them how they feel and if they have any worries
  • Encourage them to ask questions
  • Ask them questions to check what they do and don’t understand – for example, “Do you understand what the doctors are telling you?” or “Do you understand how this treatment might help and how it might not help, and what the potential risks are?”
  • Keep talking with them about what will happen during and in between appointments
  • Give them details about the healthcare team who will be looking after them
  • Reassure them that they can say “no” and that their care will not be affected

How is your child going to react?

Children can behave in unexpected ways and have many different feelings about their treatment. For example, your child may:

  • Want to be part of the decision process and ask lots of questions
  • Not want to be involved in the decision or talk about their treatment
  • Be interested in helping other children
  • Be keen to try a new treatment
  • Have worries about painful tests
  • Have concerns about side effects
  • Not want to miss school because of the extra appointments
  • Feel anxious about meeting new doctors

Listening to your child’s views and concerns is just as important as explaining all the pros and cons of joining a clinical trial.

Sources (28)
  1. Center for Information and Study on Clinical Research Participation. Common myths about clinical trials https://www.ciscrp.org/wp-content/uploads/2024/09/Common-Myths-about-CT-Brochure_2024-08-23_EdCtr_Screen-Reader-Accessible.pdf“Many clinical trials use new treatments that are not available to the public.”
  2. National Institutes of Health. Why should I participate in a clinical trial? https://www.nih.gov/health-information/nih-clinical-research-trials-you/why-should-i-participate-clinical-trial“People participate in clinical trials for a variety of reasons. […] Participants with an illness or disease also participate to […] have the additional care and attention from the clinical trial staff.”
  3. National Institutes of Health. Why should I participate in a clinical trial? https://www.nih.gov/health-information/nih-clinical-research-trials-you/why-should-i-participate-clinical-trial“Participants with an illness or disease also participate to help others.”
  4. Should My Child Participate in a Clinical Trial?: Centre for Information & Study on Clinical Research Participation https://www.ciscrp.org/wp-content/uploads/2022/08/8.5x11-version-1.pdf“The trial treatment may not work, or your child might receive a placebo that has no medicine in it; Your child may experience unpleasant, serious, or even life-threatening medical problems caused by the trial treatment; Your child may have to undergo uncomfortable or painful procedures; Participation may be time consuming and change your normal routine.”
  5. ICH Guideline for Good Clinical Practice – Monitoring: ICH GCP. https://ichgcp.net/monitoring“The purposes of trial monitoring are to verify that: (a) The rights and well-being of human subjects are protected.”
  6. Guidance for best practices for clinical trials: World Health Organization https://www.who.int/our-work/science-division/research-for-health/implementation-of-the-resolution-on-clinical-trials/guidance-for-best-practices-for-clinical-trials“This guidance provides Member States with a framework for integrating robust and ethical clinical trial practices into their national health systems, enhancing the quality, transparency, and inclusivity of trials worldwide.”
  7. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“Clinical trials for kids are designed with extra precautions to minimize risk.”
  8. Eligibility Criteria: National Institutes of Health. https://clinicalinfo.hiv.gov/en/glossary/eligibility-criteria“Factors used to determine whether a person is eligible (inclusion criteria) or not eligible (exclusion criteria) to participate in a clinical trial.”
  9. International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use. Monitoring of clinical trials https://ichgcp.net/monitoring“Monitoring is carried out to ensure that the rights and well-being of participants are protected and that trials are conducted safely and correctly.”
  10. Advancing Health. Serious adverse events & safety reporting 101 https://www.advancinghealth.ubc.ca/research-in-action/serious-adverse-events-safety-reporting-101/“Trial protocols should define how all safety events should be managed throughout the study. They provide information on what events have to be reported and if events are unexpected, based on the information found in the Investigational Product (IP) Investigators Brochure or Product Monograph. […] During every study visit the investigator must document all the adverse events observed by study staff and reported by the participant during the visit.”
  11. Gaps in the ethical governance of pharmaceutical clinical trials in Europe: Frontiers in Medicine https://www.frontiersin.org/journals/medicine/articles/10.3389/fmed.2024.1507021/full“In a clinical trial the rights, safety, dignity and well-being of subjects should be protected… A clinical trial shall be subject to scientific and ethical review and shall be authorized.”
  12. Black Doctor. Can I leave a clinical trial? https://blackdoctor.com/leave-a-clinical-trial/“You can leave a clinical trial at any time. […] Your doctor or research team may help transition you to a standard treatment appropriate for your condition.”
  13. How health care companions support older adults at appointments: University of Michigan Institute for Healthcare Policy and Innovation https://ihpi.umich.edu/national-poll-healthy-aging/national-findings/how-health-care-companions-support-older-adults“Health care companions not only help with logistical needs such as transportation and mobility assistance, but also enhance communication, emotional well-being, and understanding of medical advice.”
  14. Research involving children: Health Research Authority https://www.hra.nhs.uk/planning-and-improving-research/policies-standards-legislation/research-involving-children/“Those who are able to give consent on behalf of children / young people, to take part in a CTIMP, in the UK are: parent or someone with parental responsibility (agreement of only one parent is required).”
  15. Research involving children: Health Research Authority https://www.hra.nhs.uk/planning-and-improving-research/policies-standards-legislation/research-involving-children/“The Medicines for Human Use (Clinical Trials) Regulations prohibit children under the age of 16 from giving consent to take part in a Clinical Trial of an Investigational Medicinal Product (CTIMP).”
  16. Child participation in research: European Union Agency for Fundamental Rights (FRA) https://fra.europa.eu/en/publication/2019/child-participation-research“EU Member States have different rules, regulations and guidelines regarding the participation of children in research, in particular concerning ethics approval and informed consent. […] There is no clear regulation about parental consent and age groups.”
  17. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“What is informed consent? It's your right to know everything about your child's role in a clinical trial.”
  18. Nclusiv. Patient information sheets for research studies: compliance guide https://nclusiv.co.uk/blog/f/patient-information-sheets-for-research-studies-compliance-guide“The Health Research Authority (HRA) in the UK provides specific guidance on content requirements, including: The purpose of the research and why the participant has been invited; What participation will involve in terms of procedures, duration, and expectations.”
  19. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“What is informed consent? It's your right to know everything about your child's role in a clinical trial. Through the informed consent process, you will receive key information about […] the potential benefits and risks.”
  20. Baer AR, Good M, Schapira L. A new look at informed consent for cancer clinical trials. J Oncol Pract. 2011;7(4):267–270. https://pmc.ncbi.nlm.nih.gov/articles/PMC3140452/“Prospective participants need to be adequately informed and given the opportunity to ask questions and consult with relatives or friends without feeling pressured or rushed.”
  21. Informed Consent of Trial Subjects: International Council for Harmonisation Good Clinical Practice https://ichgcp.net/publications/informed-consent-of-trial-subjects“None of the oral and written information concerning the trial, including the written informed consent form, should contain any language that causes the subject or the subject's legally acceptable representative to waive or to appear to waive any legal rights.”
  22. Health Research Authority. General principles of consent https://www.hra-decisiontools.org.uk/consent/principles-general.html“ It is important that you clearly present the voluntary nature of participation to potential participants during the consent process and in your Participant Information Sheet. […] Consent must be given voluntarily: not under any form of duress or undue influence from health professionals, family or friends.”
  23. Oxford University Hospitals NHS Foundation Trust. Consent https://www.ouh.nhs.uk/researchers/conduct/consent/“Written and verbal versions of the information will be presented to the subject detailing no less than […] the fact that they are free to withdraw from the trial at any time for any reason without prejudice to future care, and with no obligation to give the reason for withdrawal.”
  24. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“Children may also be asked to assent, or agree, to participate. This involves giving them basic facts about a research study and asking them to be part of the decision. Children as young as 7 years old can be asked to give assent.”
  25. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“At any age, the important thing is that your child is comfortable and their questions are answered. The research team may be able to help if you and your child disagree about participation. Some studies have advocates and ethics experts involved who can help with questions.”
  26. Your child and clinical trials – guide for CF parents: Cystic Fibrosis Foundation https://www.cff.org/research-clinical-trials/your-child-and-clinical-trials-guide-cf-parents“At any age, the important thing is that your child is comfortable and their questions are answered. The research team may be able to help if you and your child disagree about participation. Some studies have advocates and ethics experts involved who can help with questions.”
  27. Oxford University Hospitals NHS Foundation Trust. Consent https://www.ouh.nhs.uk/researchers/conduct/consent/“Written and verbal versions of the information will be presented to the subject detailing no less than […] the fact that they are free to withdraw from the trial at any time for any reason without prejudice to future care, and with no obligation to give the reason for withdrawal.”
  28. Babies, children and young people’s experience of healthcare: National Institute for Health and Care Excellence (NICE) https://www.nice.org.uk/guidance/ng204/chapter/Recommendations“Healthcare professionals should involve children and young people in decisions about their healthcare in ways that are appropriate to their maturity and understanding. Some children and young people will be able to give informed consent themselves, some will be able to contribute to the discussion, and others may not be able to be involved at all.”