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โ† Trials
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Stakeholders of Rare Diseases Informing Values In Neuroethics

Recruitingn/aAges any - anyAllSt. Jude Children's Research Hospital
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In plain words

What the trial is, and whether it could suit you

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).

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 โ‰ค 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 (โ‰ฅ 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).
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What it tests

  • Not listed
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Where

1 site
St. Jude Children's Research Hospital
Memphis, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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