Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
St. Jude Children's Research Hospital
Summary
This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.
Description
The study team seeks to understand the unique needs of patients and parents of children who are considering novel experimental gene therapy treatments. The study team will engage patients, caregivers, and healthcare worker stakeholders to understand their attitudes, beliefs, and concerns surrounding these treatments. Based on these insights, the team, with the support of the above stakeholders and an external vendor, will develop web platform offering educational content and decision aids to enhance their experience and overall satisfaction with the process of making treatment decisions. This…
Eligibility
- Age range
- 8+ years
- Sex
- All
- Healthy volunteers
- Yes
Inclusion Criteria: 1. For Group 1 participants only (Undergone Gene Therapy): * Parent/caregiver whose child has undergone gene therapy. OR Parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have undergone gene therapy. * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working…
Interventions
- OtherInterview
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (1) patients and families of children with rare genetic diseases who have received gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy).
- OtherInterview
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (2) patients and families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were never offered gene therapy or ultimately did not qualify for a trial (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy were met).
- OtherInterview
The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (3) healthcare workers who provide care to patients receiving gene therapy (to assess their perspective regarding patient/family needs for gene therapy education and communication).
- OtherInterview
The following group of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (4) parents/caregivers of children with a bone marrow failure condition, who has undergone gene therapy, OR parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment OR patients age 8 and above who have undergone gene therapy (to assess their perspectives and understand their informational needs around participating in a clinical trial using gene therapy).
Location
- St. Jude Children's Research HospitalMemphis, Tennessee