Understanding patient needs for gene therapy in sickle cell disease

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

This study talks to patients and their families about what they think and need to know about gene therapy for sickle cell disease to help create better educational resources for them.

Quick facts

• Study type: Observational
• Enrollment: 145 (estimated)
• Ages: 8 Years and up
• Sex: All
• Sponsor: St. Jude Children's Research Hospital (Academic / other)
• Locations: 1 site (Memphis, Tennessee)
• Trial ID: NCT05810181 on ClinicalTrials.gov

What this trial studies

This observational study aims to qualitatively explore the beliefs, attitudes, and informational needs of patients and parents regarding gene therapy for rare pediatric diseases, specifically sickle cell disease. Through interviews with stakeholders including patients, caregivers, and healthcare workers, the study seeks to gather insights that will inform the development of an online platform. This platform will provide educational content and decision aids to assist families in making informed treatment decisions. The initiative addresses significant gaps in available educational resources and aims to enhance the overall experience of those considering gene therapy.

Who should consider this trial

Good fit: Ideal candidates include parents or caregivers of children who have undergone gene therapy or patients aged 8 and above who have received gene therapy for a rare genetic disease.

Not a fit: Patients who have not undergone gene therapy or do not have a rare genetic disease targeted for treatment using gene therapy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable educational resources and decision-making tools for families considering gene therapy for sickle cell disease.

How similar studies have performed: While this approach is innovative in its focus on patient and caregiver needs for gene therapy, similar studies have shown success in enhancing patient education and decision-making in other therapeutic areas.

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).

Where this trial is running

Memphis, Tennessee

• St. Jude Children's Research Hospital — Memphis, Tennessee, United States (Recruiting)

Study contacts

• Principal investigator: Liza-Marie Johnson, MD, MPH, MSB — St. Jude Children's Research Hospital
• Study coordinator: Liza-Marie Johnson, MD, MPH, MSB

Conditions Sickle Cell Disease