Actively Recruiting
Stakeholders of Rare Diseases Informing Values In Neuroethics
Led by St. Jude Children's Research Hospital · Updated on 2026-04-08
385
Participants Needed
1
Research Sites
243 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this research study is to learn more about the perspectives of key stakeholders-patients, families, healthcare providers, and researchers-on the ethical challenges of small-scale, personalized treatment trials for rare neurological diseases (RND).
CONDITIONS
Official Title
Stakeholders of Rare Diseases Informing Values In Neuroethics
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parental or primary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with suspected genetic cause
- Child is under 21 years old at enrollment
- Child has an expected survival of at least one year after enrollment
- Patients age 25 or younger with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic cause
- Willingness to provide verbal informed consent or assent to participate
- Family member of a Group 1 participant who plays an active role in the child's life or care
- Includes siblings 13 years or older, grandparents, or other non-primary caregivers directly affected by the child's diagnosis
- Demonstrated familiarity with the child's medical and family experience
- Individuals currently or recently engaged in clinical care, research, advocacy, or policy work related to pediatric-onset rare genetic disorders
- Willingness to provide verbal informed consent to participate in interviews or focus groups
You will not qualify if you...
- Limited English proficiency
- Unable to complete surveys or interviews in English
- Inability or unwillingness to give verbal informed consent in English
- Condition or chronic illness that makes participation unsafe or untenable, such as cognitive impairment or acute illness, as judged by the study investigators
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
Actively Recruiting
Research Team
L
Liza M. Johnson, MD, MPH, MSB
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
3
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