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Komunikacja w zakresie terapii genowej: wykorzystanie oceny potrzeb do podjęcia decyzji o pomocy w terapii genowej rzadkich chorób (GENETX)

5 sierpnia 2026 zaktualizowane przez: St. Jude Children's Research Hospital
To prospektywne badanie z wywiadem metodą mieszaną ma na celu jakościowe opisanie przekonań, postaw i potrzeb informacyjnych dotyczących terapii genowej rzadkich chorób pediatrycznych wśród pacjentów i rodziców dzieci z rzadką chorobą, które mają być leczone przy użyciu technik terapii genowej. Wykorzystując zdobytą wiedzę, zespół opracuje platformę internetową udostępniającą treści edukacyjne i pomoce w podejmowaniu decyzji dla pacjentów i ich rodzin.

Przegląd badań

Status

Rekrutacyjny

Warunki

Interwencja / Leczenie

Szczegółowy opis

Zespół badawczy stara się zrozumieć wyjątkowe potrzeby pacjentów i rodziców dzieci, którzy rozważają nowatorskie eksperymentalne terapie genowe. Zespół badawczy zaangażuje interesariuszy pacjentów, opiekunów i pracowników służby zdrowia, aby zrozumieć ich postawy, przekonania i obawy związane z tymi metodami leczenia. Na podstawie tych spostrzeżeń zespół, przy wsparciu powyższych interesariuszy i zewnętrznego dostawcy, opracuje platformę internetową oferującą treści edukacyjne i pomoce decyzyjne, aby zwiększyć ich doświadczenie i ogólną satysfakcję z procesu podejmowania decyzji dotyczących leczenia. Jest to szczególnie ważne, ponieważ nowe terapie SCD będą coraz częściej obejmować edycję genów w badaniach pediatrycznych w ciągu najbliższych dwóch lat. Wysiłki te zajmą się istotnymi lukami w materiałach edukacyjnych dostępnych dla osób rozważających terapię genową i (zgodnie z wiedzą zespołu) stworzą zasób, który będzie pierwszym tego rodzaju.

Typ studiów

Obserwacyjny

Zapisy (Szacowany)

145

Kontakty i lokalizacje

Ta sekcja zawiera dane kontaktowe osób prowadzących badanie oraz informacje o tym, gdzie badanie jest przeprowadzane.

Kontakt w sprawie studiów

Lokalizacje studiów

Kryteria uczestnictwa

Badacze szukają osób, które pasują do określonego opisu, zwanego kryteriami kwalifikacyjnymi. Niektóre przykłady tych kryteriów to ogólny stan zdrowia danej osoby lub wcześniejsze leczenie.

Kryteria kwalifikacji

Wiek uprawniający do nauki

  • Dziecko
  • Dorosły
  • Starszy dorosły

Akceptuje zdrowych ochotników

Tak

Metoda próbkowania

Próbka bez prawdopodobieństwa

Badana populacja

Około 45-50 młodych dorosłych (w wieku 18-35 lat) lub rodziców dzieci, u których zdiagnozowano rzadką chorobę skierowaną do leczenia za pomocą technik terapii genowej oraz 10-20 pracowników służby zdrowia, którzy opiekują się pacjentami poddawanymi terapii genowej.

Opis

Kryteria przyjęcia:

  1. Tylko dla uczestników z grupy 1 (przeszli terapię genową):

    • Rodzic/opiekun, którego dziecko przeszło terapię genową. LUB Młody dorosły w wieku od 18 do 35 lat, który przeszedł terapię genową.
    • Gotowość do wzięcia udziału w indywidualnej rozmowie wideo z członkiem zespołu badawczego za pomocą osobistego urządzenia mobilnego lub komputera z działającym połączeniem internetowym.
    • Musi być gotów udzielić ustnej świadomej zgody.
    • Zwolnienie formularza informacyjnego podpisane przez uczestnika, dające naszemu zespołowi badawczemu pozwolenie na skontaktowanie się z dostawcą opieki zdrowotnej w celu zweryfikowania diagnozy i otrzymania terapii genowej (jeśli została otrzymana).
    • Pomyślna weryfikacja rozpoznania rzadkiej choroby genetycznej kierowanej do leczenia z wykorzystaniem terapii genowej.
    • Pozytywne potwierdzenie otrzymania terapii genowej i rodzaju otrzymane od lekarza (tylko dla tych, którzy otrzymali terapię genową).

  2. Tylko dla uczestników z grupy 2 (oferowane, ale nie poddane terapii genowej):

    • Rodzic/opiekun dzieci (lub młodych dorosłych w wieku od 18 do 35 lat) z rzadką chorobą genetyczną, którym zaproponowano, ale nie kwalifikowali się do badania lub zrezygnowali z terapii genowej.
    • Gotowość do wzięcia udziału w indywidualnej rozmowie wideo z członkiem zespołu badawczego za pomocą osobistego urządzenia mobilnego lub komputera z działającym połączeniem internetowym.
    • Musi być gotów udzielić ustnej świadomej zgody.
    • Podpisany formularz informacyjny dający zespołowi badawczemu GeneTx pozwolenie na kontakt z lekarzem uczestnika w celu zweryfikowania diagnozy.
    • Pomyślna weryfikacja rozpoznania rzadkiej choroby genetycznej kierowanej do leczenia z wykorzystaniem terapii genowej.

  3. Tylko dla uczestników z grupy 3 (wywiady z dostawcami):

    • Pracownik służby zdrowia, który sprawował opiekę nad ≥ 2 pacjentami otrzymującymi terapię genową.
    • Gotowość do wzięcia udziału w indywidualnej rozmowie wideo (lub osobistej) z członkiem zespołu badawczego za pomocą osobistego urządzenia mobilnego lub komputera z działającym połączeniem internetowym.
    • Świadoma zgoda uczestnika badania.

Kryteria wyłączenia:

  • Uczestnicy, którzy nie będą w stanie płynnie porozumiewać się w języku angielskim, zostaną wykluczeni.
  • Niezdolność lub niechęć uczestnika badania do wyrażenia ustnej świadomej zgody.
  • Uczestnicy, którzy nie mają dostępu do komputera lub urządzenia mobilnego obsługującego komunikację wideo, zostaną wykluczeni.
  • Stan lub choroba przewlekła, która w opinii PI/Co-I sprawia, że ​​uczestnictwo jest niebezpieczne lub nie do utrzymania (tj. upośledzenie funkcji poznawczych, współistniejąca ostra choroba).

Plan studiów

Ta sekcja zawiera szczegółowe informacje na temat planu badania, w tym sposób zaprojektowania badania i jego pomiary.

Jak projektuje się badanie?

Szczegóły projektu

Kohorty i interwencje

Grupa / Kohorta
Interwencja / Leczenie
Rodzic/opiekun
10-20 pacjentów/rodzin dzieci z rzadkimi chorobami genetycznymi, którym zaproponowano terapię genową, ale zrezygnowali z niej lub którzy ostatecznie nie zostali zakwalifikowani do badania klinicznego.
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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).
Pracownik służby zdrowia
10-20 pracowników służby zdrowia, którzy sprawują opiekę nad pacjentami poddawanymi terapii genowej.
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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).
Młody dorosły
15-25 rodziców/rodziny dzieci (pacjentów w wieku 8 lat i więcej) z rzadkimi chorobami genetycznymi, którzy niedawno otrzymali terapię genową
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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.
Inny: 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).

Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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).
Inny: 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)
Inny: 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).

Co mierzy badanie?

Podstawowe miary wyniku

Miara wyniku
Opis środka
Ramy czasowe
Wykorzystanie częściowo ustrukturyzowanych wywiadów do oceny przekonań, postaw i potrzeb informacyjnych związanych z terapią genową wśród pacjentów i rodzin z rzadkimi chorobami genetycznymi.
Ramy czasowe: 2 lata
Wyszkoleni eksperci przeprowadzą wywiady z uczestnikami badania, aby wyjaśnić ich przekonania, postawy i potrzeby informacyjne dotyczące terapii genowej rzadkich chorób pediatrycznych wśród interesariuszy tych chorób. Wywiady te zostaną przeanalizowane za pomocą dobrze opisanej, rygorystycznej metodologii semantycznej analizy treści w celu zidentyfikowania tematów w ramach systematycznego i znormalizowanego procesu.
2 lata

Współpracownicy i badacze

Tutaj znajdziesz osoby i organizacje zaangażowane w to badanie.

Sponsor

St. Jude Children's Research Hospital

Współpracownicy

National Heart, Lung, and Blood Institute (NHLBI)

Śledczy

  • Główny śledczy: Liza-Marie Johnson, MD, MPH, MSB, St. Jude Children's Research Hospital

Daty zapisu na studia

Daty te śledzą postęp w przesyłaniu rekordów badań i podsumowań wyników do ClinicalTrials.gov. Zapisy badań i zgłoszone wyniki są przeglądane przez National Library of Medicine (NLM), aby upewnić się, że spełniają określone standardy kontroli jakości, zanim zostaną opublikowane na publicznej stronie internetowej.

Główne daty studiów

Rozpoczęcie studiów (Rzeczywisty)

1 czerwca 2023

Zakończenie podstawowe (Szacowany)

1 grudnia 2027

Ukończenie studiów (Szacowany)

1 grudnia 2027

Daty rejestracji na studia

Pierwszy przesłany

30 marca 2023

Pierwszy przesłany, który spełnia kryteria kontroli jakości

30 marca 2023

Pierwszy wysłany (Rzeczywisty)

12 kwietnia 2023

Aktualizacje rekordów badań

Ostatnia wysłana aktualizacja (Rzeczywisty)

10 sierpnia 2026

Ostatnia przesłana aktualizacja, która spełniała kryteria kontroli jakości

5 sierpnia 2026

Ostatnia weryfikacja

1 sierpnia 2026

Więcej informacji

Terminy związane z tym badaniem

Dodatkowe istotne warunki MeSH

Inne numery identyfikacyjne badania

  • GENETX
  • U01HL163983 (Grant/umowa NIH USA)
  • R01HL156647 (Grant/umowa NIH USA)

Informacje o lekach i urządzeniach, dokumenty badawcze

Bada produkt leczniczy regulowany przez amerykańską FDA

Nie

Bada produkt urządzenia regulowany przez amerykańską FDA

Nie

Te informacje zostały pobrane bezpośrednio ze strony internetowej clinicaltrials.gov bez żadnych zmian. Jeśli chcesz zmienić, usunąć lub zaktualizować dane swojego badania, skontaktuj się z register@clinicaltrials.gov. Gdy tylko zmiana zostanie wprowadzona na stronie clinicaltrials.gov, zostanie ona automatycznie zaktualizowana również na naszej stronie internetowej .

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