Stakeholders of Rare Diseases Informing Values In Neuroethics
St. Jude Children's Research Hospital
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).
Description
Primary Objectives * To utilize semi-structured interviews to identify key stakeholder preferences and gather recommendations for the ethical conduct of n-of-few approaches in pediatric patients with rare neurological diseases (RND). * Identify key challenges and best practices for informed consent and communication strategies for personalized interventions in pediatric RND trials. * To develop a best practice framework for the ethical conduct of research involving personalized interventions for children with catastrophic RND. Secondary Objectives * To use surveys to quantitatively measure…
Eligibility
- Age range
- Not specified
- Sex
- All
- Healthy volunteers
- Not specified
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 (o…
Location
- St. Jude Children's Research HospitalMemphis, Tennessee