Stakeholders of Rare Diseases Informing Values In Neuroethics

Conditions

• Disorder, Neurologic
• Rare Disorder

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Study Details

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 Dates

Start date

Jun 30, 2026

Status verified

May 2026

Primary completion

Jan 31, 2031

Completion

Jan 31, 2031

Study Design

Enrollment

385 participants (estimated)

Arms

• Arm: Primary Caregivers/Patients
  Primary caregivers of children/young adults (age 21 or younger) and patients (age 25 or younger) diagnosed with a super-rare, catastrophic neurologic disorder without definitive FDA-approved treatment.
• Arm: Other Family Stakeholders
  Siblings (13 years or older) and extended family members (e.g., grandparents) and primary caregivers who prefer not to participate in the longitudinal component
• Arm: Non-Family Stakeholders
  Non-family stakeholders involved in the clinical care or investigational treatments of children with catastrophic illnesses.

Primary Outcome Measure

Identifying key stakeholder preferences and recommendations for the ethical conduct of n-of-few approaches in pediatric patients with rare neurological diseases (RND) utilizing semi-structured interviews [ Time Frame: At baseline and every 6 months until end of study, approximately 5 years (Group 1); Once, within 60 days of enrollment (single interview per Group 2 and 3 participant) ]

Central Contacts

• Liza M. Johnson, MD, MPH, MSB
  888-226-4343
  [email protected]

Locations (1)

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