NCT05810181 Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Eligibility & Interventions

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What to Expect as a Participant

This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.

This trial targets 145 participants in total. It began in 2023-06-01 with a primary completion date of 2027-12.

Brief 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.

Eligibility Criteria

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 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 Criteria (for all 7 groups): * 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).

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