RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2023-06-01

Study Completion:2026-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05810181

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 35 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:ADULT

Inclusion Criteria	Exclusion 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.	* 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).

Inclusion Criteria

Exclusion 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.

* 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).

Contacts and Locations

Study Contact

Liza-Marie Johnson, MD, MPH, MSB

CONTACT

8662785833

referralinfo@stjude.org

Principal Investigator

Liza-Marie Johnson, MD, MPH, MSB

PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital

Study Locations (Sites)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105

United States

Collaborators and Investigators

Sponsor: St. Jude Children's Research Hospital

Liza-Marie Johnson, MD, MPH, MSB, PRINCIPAL_INVESTIGATOR, St. Jude Children's Research Hospital

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-06-01

Study Completion Date2026-12

Study Record Updates

Study Start Date2023-06-01

Study Completion Date2026-12

Terms related to this study

Additional Relevant MeSH Terms

Sickle Cell Disease