NCT07314736 Stakeholders of Rare Diseases Informing Values In Neuroethics
| NCT ID | NCT07314736 |
| Status | Recruiting |
| Phase | — |
| Sponsor | St. Jude Children's Research Hospital |
| Condition | Rare Disorder |
| Study Type | OBSERVATIONAL |
| Enrollment | 385 participants |
| Start Date | 2026-07-08 |
| Primary Completion | 2031-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 385 participants in total. It began in 2026-07-08 with a primary completion date of 2031-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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).
Eligibility Criteria
Inclusion Criteria: 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 Exclusion Criteria: * 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).
Contact & Investigator
Liza M. Johnson, MD, MPH, MSB
PRINCIPAL INVESTIGATOR
St. Jude Children's Research Hospital
Frequently Asked Questions
Who can join the NCT07314736 clinical trial?
This trial is open to participants of all sexes, studying Rare Disorder. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07314736 currently recruiting?
Yes, NCT07314736 is actively recruiting participants. Contact the research team at referralinfo@stjude.org for enrollment information.
Where is the NCT07314736 trial being conducted?
This trial is being conducted at Memphis, United States.
Who is sponsoring the NCT07314736 clinical trial?
NCT07314736 is sponsored by St. Jude Children's Research Hospital. The principal investigator is Liza M. Johnson, MD, MPH, MSB at St. Jude Children's Research Hospital. The trial plans to enroll 385 participants.