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

NCT05810181 | Recruiting

• 1 other identifier: GENETX
• Study Type: observational
• Target: 145
• Locations: 1 country
• Sites: 1

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.

Conditions

Sickle Cell Disease

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (7)

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

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

Healthcare Worker

10-20 health care workers' who provide care to patients receiving gene therapy.

Other: Interview

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

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

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

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

Interventions

Interview OTHER

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).

Young Adult

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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) .

You may qualify if:

• 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).
• 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.
• For Group 3 participants only (Provider Interviews):
• Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.

You may not qualify if:

• 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).

Sponsors & Collaborators

• St. Jude Children's Research Hospital: lead

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Interventions

Interviews as Topic

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Liza-Marie Johnson, MD, MPH, MSB

  St. Jude Children's Research Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Liza-Marie Johnson, MD, MPH, MSB

CONTACT

888-226-4343; referralinfo@stjude.org

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 30, 2023
• First Posted: April 12, 2023
• Study Start: June 1, 2023
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027
• Last Updated: May 7, 2026

Record last verified: 2026-05

Locations

US (1)