Understanding Experiences with Gene Therapy for Rare Diseases
This study, called GENETX, aims to understand the beliefs, attitudes, and information needs of patients and families considering gene therapy for rare diseases like Sickle Cell Disease. Researchers will conduct interviews with different groups: families whose children have received gene therapy, families who decided against it, and healthcare workers. The goal is to gather insights to create an online platform with educational materials and tools to help families make informed decisions about gene therapy. This study is observational, meaning it involves interviews rather than testing a new treatment, and plans to include 145 participants.
- Study design
- This is an observational study that will involve interviews with approximately 145 participants. It is not a treatment study but focuses on understanding experiences and needs.
- What's involved
- You would participate in a semi-structured interview to share your beliefs, attitudes, and informational needs regarding gene therapy.
- Compensation
- Not stated in the trial record.
- Follow-up
- The primary endpoint, assessing beliefs and needs, will be measured at 2 years.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05810181
Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)
Recruiting
Not specifiedAges 8+ObservationalSt. Jude Children's Research HospitalInvestigator-initiated
~145 participants
Updated 2026-08-10 on ClinicalTrials.gov
What's tested:Interview
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.
Measured over 2 years
Conditions
Where it's being run
1 sites across 1 statesTennessee1
Study leadership
- Liza-Marie Johnson, MD, MPH, MSB · PRINCIPAL_INVESTIGATOR · St. Jude Children's Research Hospital
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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 internet connection.
Must be willing to provide verbal informed consent.
Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).
Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy). 2. For Group 2 participants only (Offered, but did not Undergo Gene Therapy):
Parent/caregiver of children (or patients 8 and above ) with a rare genetic disease who had been offered but were not eligible for a trial or decided against receiving 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 internet connection.
Must be willing to provide verbal informed consent.
Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy. 3. For Group 3 participants only (Provider Interviews):
Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.
Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.
Informed consent from a study participant. 4. For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition):
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 internet connection.
Must be willing to provide verbal informed consent.
Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).
Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy). 5. For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ):
Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure disease who had been offered but were not eligible for a trial or decided against receiving 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 internet connection.
Must be willing to provide verbal informed consent.
Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy. 6. For Group 6 participants only (Never offered gene therapy for Bone Marrow Failure Condition):
Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure disease who had not been offered 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 internet connection.
Must be willing to provide verbal informed consent.
Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy. 7. For Group 7 participants only (Provider Interviews for Bone Marrow Failure Condition):
Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.
Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.
Informed consent from a study participant.
Exclusion
Participants who are unable to converse fluently in English will be excluded.
Inability or unwillingness of research participant to give verbal informed consent.
Participants who lack access to a computer or mobile device that supports video communications will be excluded.
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).
What this trial measures
- Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.2 years
Trained experts will interview study participants to elucidate their beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among the disease stakeholders. These interviews will be analyzed via the well-described rigorous methodology of semantic content analysis to identify themes through a systematic and standardized process.