Promoteur(s) :
St. Jude Children's Research Hospital
Recrutement : ouvert
Centres participants
1
Dernière modification : 2025-10-08
DESCRIPTION DE L'ÉTUDE
Résumé de l'étude
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.
Source : Importé depuis le centre
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 patient, caregiver, 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.
Source : Importé depuis le centre
RECRUTEMENT
Profil des participants
Limites d'âge
minimum : 18 ans
maximum : 35 ans
Sexe(s) des participants
ALL
Source : Importé depuis le centre
Condition médicale (spécialité visée)
Domaine de recherche
Donnée non disponible
Critères de sélection
Cohortes
Nom
Condition médicale
Traitement
État du recrutement
Young Adult
15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy
Donnée non disponible
Inconnu
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.
Donnée non disponible
Inconnu
Healthcare Worker
10-20 health care workers' who provide care to patients receiving gene therapy.
Donnée non disponible
Inconnu
Young Adult
État du recrutement
unknown
15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy
Parent/caregiver
État du recrutement
unknown
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.
Healthcare Worker
État du recrutement
unknown
10-20 health care workers' who provide care to patients receiving gene therapy.
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.
Exclusion Criteria:
* 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).
Source : Importé depuis le centre
Cohortes
Thérapie ou Intervention proposée
Cohortes
Nom
Condition médicale
Traitement
État du recrutement
Young Adult
15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy
Donnée non disponible
Inconnu
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.
Donnée non disponible
Inconnu
Healthcare Worker
10-20 health care workers' who provide care to patients receiving gene therapy.
Donnée non disponible
Inconnu
Young Adult
État du recrutement
unknown
15-25 parents/families of children (patients aged 8 and above) with rare genetic diseases, who have recently received gene therapy
Parent/caregiver
État du recrutement
unknown
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.
Healthcare Worker
État du recrutement
unknown
10-20 health care workers' who provide care to patients receiving gene therapy.
Données à jour depuis :
8 octobre 2025
Description de l'étude
Description de l'étude
Résumé de l'étude
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.
Source : Importé depuis le centre
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 patient, caregiver, 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.
