Gen Tedavisinde İletişim: Nadir Hastalıklarda Gen Tedavisi Karar Destek Araçları İçin İhtiyaç Analizi Kullanımı (GENETX)

Sade dilde özet

Bu çalışma bir ilaç veya tedavi denemesi değildir; görüşmelere dayalı karma yöntemli bir araştırmadır. Amaç, gen tedavisi hedeflenen nadir çocukluk çağı hastalıklarında hastaların ve ebeveynlerin gen tedavisine ilişkin inanç, tutum ve bilgi ihtiyaçlarını tanımlamaktır. Elde edilen bulgularla, hastalar ve aileleri için eğitim içeriği ve karar destek araçları sunan çevrimiçi bir platform geliştirilmesi planlanıyor; çalışmaya gen tedavisi almış çocukların ebeveynleri/bakım verenleri gibi farklı gruplar dahil ediliyor.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

N/A

Durum

Katılımcı alıyor

Sponsor

St. Jude Children's Research Hospital

Müdahale

Interview, Interview, Interview, Interview, Interview, Interview, Interview

Başlangıç

01.06.2023

Tahmini bitiş

01.12.2027

Ülkeler

United States

İlgili genler

Merkezler

• Memphis, United States

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

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).