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

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.

Study Overview

• Status: Recruiting
• Conditions: Sickle Cell Disease
• Intervention / Treatment: Other: Interview; Other: Interview; Other: Interview; Other: Interview; Other: Interview; Other: Interview; Other: Interview

Detailed 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 is especially important as novel therapies for SCD will increasingly include gene editing in pediatric trials over the next two years. These efforts will address significant gaps in the educational material available to those considering gene therapy and (to the team's knowledge) will create a resource that will be the first of its kind.

• Study Type: Observational
• Enrollment (Estimated): 145

Contacts and Locations

• Study Contact: Name: Liza-Marie Johnson, MD, MPH, MSB; Phone Number: 888-226-4343; Email: referralinfo@stjude.org

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • Recruiting
      • St. Jude Children's Research Hospital
      • Contact: Phone Number: 888-226-4343; Email: referralinfo@stjude.org

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

About 105 patients (age 8 and above) or parents of children verified to have a rare disease targeted for treatment using gene therapy techniques and 10-20 healthcare workers who provide care to patients receiving gene therapy (including an additional 10-20 healthcare workers treating patients with a bone marrow failure condition) .

Description

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).

Study Plan

How is the study designed?

Number of groups / cohorts

7

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Parent/caregiver; 10-20 patients/families of children with rare genetic diseases who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial.	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Healthcare Worker; 10-20 health care workers' who provide care to patients receiving gene therapy.	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Young Adult; 15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Bone marrow failure condition (received gene therapy); 5-10 parents/families 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	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Bone marrow failure condition (did not receive gene therapy); 5-10 parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical trial	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Bone marrow failure condition (never offered gene therapy); 30-40 parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).
Healthcare Workers (for bone marrow failure condition); 10-20 health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions.	Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (5) parent/caregiver of children (or patients 8 and above) with bone marrow failure conditions who were offered but have decided against receiving gene therapy or who were ultimately not eligible for a clinical 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).; Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (6) parent/caregiver of children (or patients 8 and above) with a bone marrow failure condition who were never offered gene therapy (to elicit feedback and to evaluate whether their informational needs and communication expectations about participating in a clinical trial using gene therapy); Other: Interview; The following groups of stakeholders will be interviewed to assess the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases: (7) health care workers who provide care to patients receiving gene therapy for bone marrow failure conditions (to assess their perspective regarding patient/family needs for gene therapy education and communication).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Use of semi-structured interviews to assess the beliefs, attitudes, and informational needs around gene therapy among patients and families with rare genetic diseases.	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.	2 years

Collaborators and Investigators

• Sponsor: St. Jude Children's Research Hospital
• Investigators: Principal Investigator: Liza-Marie Johnson, MD, MPH, MSB, St. Jude Children's Research Hospital

Study record dates

Study Major Dates

• Study Start (Actual): June 1, 2023
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: March 30, 2023
• First Submitted That Met QC Criteria: March 30, 2023
• First Posted (Actual): April 12, 2023

Study Record Updates

• Last Update Posted (Actual): May 7, 2026
• Last Update Submitted That Met QC Criteria: May 1, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hemoglobinopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Anemia, Sickle Cell; Health Care Quality, Access, and Evaluation; Investigative Techniques; Epidemiologic Methods; Data Collection; Health Care Evaluation Mechanisms; Quality of Health Care; Public Health; Environment and Public Health; Interviews as Topic
• Other Study ID Numbers: GENETX

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No