Stakeholders of Rare Diseases Informing Values In Neuroethics

Recruiting now

Conditions studied: Rare Disorder, Disorder, Neurologic

In brief

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).

Key facts

Study ID

NCT07314736

Run by

St. Jude Children's Research Hospital

People needed

385

Starts

2026-07-08

Expected to finish

2031-01-01

Last updated by the study team

2026-07-13

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Group 1 (Parental Caregiver and Patient Participants)
• Parental/primary caregiver with a child who has a genetic diagnosis of an ultrarare disorder with pediatric onset, or a clinical diagnosis with a suspected genetic etiology.
• Child is under 21 years of age at the time of enrollment.
• Child has an expected survival of at least one year following study enrollment.
• Patients (age ≤ 25 years) with a genetic diagnosis of an ultrarare disorder with pediatric onset, or clinical diagnosis with suspected genetic etiology.
• Willingness to provide verbal informed consent (or assent, as appropriate) to participate
• Group 2 (Other Family)
• Family member of a Group 1 participant who plays an active role in the child's life or care.
• Includes siblings (≥ 13 years of age), grandparents, or other non-primary caregivers directly affected by the child's diagnosis.
• Demonstrated familiarity with the child's medical and family experience.
• Willingness to provide verbal informed consent (or assent, as appropriate) to participate.
• Group 3 (Non-Family Stakeholders)
• Individuals currently engaged, or recently active, in clinical care, research, advocacy or policy work related to pediatric-onset rare genetic disorders.
• May include clinicians (e.g., neurologists, genetic counselors, nurses, child-life specialists, home-health staff), members of patient-advocacy organizations, institutional-review-board (IRB) members, payers, sponsors, funders, or representatives of hospital systems or regulatory agencies.
• Willingness to provide verbal informed consent to participate in semi-structured interviews or focus groups

You may not qualify if…

• Limited English proficiency
• Unable to complete the survey materials or complete the interviews in English.
• Inability or unwillingness of research participant to give verbal informed consent (in English)
• Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).

Where it is running

• St. Jude Children's Research Hospital — Memphis, Tennessee, United States (enrolling)

Full record on ClinicalTrials.gov

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