Stakeholders of Rare Diseases Informing Values In Neuroethics
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorSt. Jude Children's Research Hospital
About this trial
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
Qualifiers
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.
Disqualifiers
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).
Trial design
Treatments tested in this trial
- Not listed
Trial groups
385 Participants
are grouped into 3 trial groupsSponsors and collaborators
Source ClinicalTrials.gov