Rare Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
Group 1 – Parents/Caregivers and Patients:
- You are a parent or main caregiver of a child who has been diagnosed (or is suspected to have) a very rare genetic condition that started in childhood.
- The child is under 21 years old at the time of joining the study.
- The child is expected to live for at least one more year after joining the study.
- You are a patient aged 25 or younger who has been diagnosed with (or is suspected to have) a very rare genetic condition that began in childhood.
- You are willing to give verbal agreement to take part in the study.
Group 2 – Other Family Members:
- You are a family member of someone in Group 1 and play an active role in that child's life or care.
- You are a sibling aged 13 or older, a grandparent, or another family member who is directly affected by the child's diagnosis.
- You are familiar with the child's medical situation and the family's experience.
- You are willing to give verbal agreement to take part in the study.
Group 3 – Non-Family Stakeholders:
- You currently work, or have recently worked, in clinical care, research, advocacy, or policy related to rare childhood genetic conditions.
- This may include doctors, nurses, genetic counselors, patient advocacy group members, policy representatives, or similar roles (confirm with trial site).
- You are willing to give verbal agreement to take part in interviews or focus groups.
Who may not be able to join:
- People who are not fluent in English or cannot complete the study materials and interviews in English.
- People who are unwilling or unable to give verbal agreement (consent) in English to take part.
- People who have a condition or serious illness that, in the opinion of the study doctors, would make participation unsafe or not possible — for example, significant cognitive difficulties or a serious current illness (confirm with trial site).
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Liza M. Johnson, MD, MPH, MSB, St. Jude Children's Research Hospital
Phone: 888-226-4343
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Identifying key stakeholder preferences and recommendations for the ethical conduct of n-of-few approaches in pediatric patients with rare neurological diseases (RND) utilizing semi-structured interviews; Identify key challenges and ethics-informed best practices for the development and implementation of personalized or n-of-few genomic interventions for rare and catastrophic pediatric disorders.; To develop a best practice framework for the ethical conduct of research involving personalized interventions for children with catastrophic genetic disorders of childhood onset.
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.