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Gentherapie-Kommunikation: Verwendung einer Bedarfsanalyse zur Förderung von Entscheidungshilfen für die Gentherapie für seltene Krankheiten (GENETX)

5. August 2026 aktualisiert von: St. Jude Children's Research Hospital
Diese prospektive Mixed-Method-Interviewstudie zielt darauf ab, die Überzeugungen, Einstellungen und Informationsbedürfnisse bezüglich der Gentherapie für seltene pädiatrische Erkrankungen bei Patienten und Eltern von Kindern mit einer seltenen Erkrankung, die für eine Behandlung mit gentherapeutischen Techniken vorgesehen sind, qualitativ zu beschreiben. Unter Verwendung der erlernten Erkenntnisse wird das Team eine Online-Plattform entwickeln, die Aufklärungsinhalte und Patientenentscheidungshilfen für Patienten und ihre Familien bereitstellt.

Studienübersicht

Status

Rekrutierung

Bedingungen

Intervention / Behandlung

Detaillierte Beschreibung

Das Studienteam versucht, die einzigartigen Bedürfnisse von Patienten und Eltern von Kindern zu verstehen, die neuartige experimentelle Gentherapiebehandlungen in Betracht ziehen. Das Studienteam wird Patienten, Pflegekräfte und Beschäftigte im Gesundheitswesen einbeziehen, um ihre Einstellungen, Überzeugungen und Bedenken im Zusammenhang mit diesen Behandlungen zu verstehen. Basierend auf diesen Erkenntnissen wird das Team mit Unterstützung der oben genannten Interessengruppen und eines externen Anbieters eine Webplattform entwickeln, die Bildungsinhalte und Entscheidungshilfen anbietet, um ihre Erfahrung und allgemeine Zufriedenheit mit dem Prozess der Behandlungsentscheidung zu verbessern. Dies ist besonders wichtig, da neuartige Therapien für SCD in den nächsten zwei Jahren zunehmend die Genbearbeitung in pädiatrischen Studien umfassen werden. Diese Bemühungen werden erhebliche Lücken im verfügbaren Schulungsmaterial für diejenigen schließen, die eine Gentherapie in Betracht ziehen, und (nach Kenntnis des Teams) eine Ressource schaffen, die die erste ihrer Art sein wird.

Studientyp

Beobachtungs

Einschreibung (Geschätzt)

145

Kontakte und Standorte

Dieser Abschnitt enthält die Kontaktdaten derjenigen, die die Studie durchführen, und Informationen darüber, wo diese Studie durchgeführt wird.

Studienkontakt

Studienorte

Teilnahmekriterien

Forscher suchen nach Personen, die einer bestimmten Beschreibung entsprechen, die als Auswahlkriterien bezeichnet werden. Einige Beispiele für diese Kriterien sind der allgemeine Gesundheitszustand einer Person oder frühere Behandlungen.

Zulassungskriterien

Studienberechtigtes Alter

  • Kind
  • Erwachsene
  • Älterer Erwachsener

Akzeptiert gesunde Freiwillige

Ja

Probenahmeverfahren

Nicht-Wahrscheinlichkeitsprobe

Studienpopulation

Etwa 45–50 junge Erwachsene (18–35 Jahre) oder Eltern von Kindern, bei denen nachgewiesen wurde, dass sie an einer seltenen Krankheit leiden, die mit Gentherapietechniken behandelt werden soll, und 10–20 medizinisches Personal, das sich um Patienten kümmert, die eine Gentherapie erhalten.

Beschreibung

Einschlusskriterien:

  1. Nur für Teilnehmer der Gruppe 1 (unterzogene Gentherapie):

    • Elternteil/Betreuer, dessen Kind sich einer Gentherapie unterzogen hat. ODER Junger Erwachsener im Alter zwischen 18 und 35 Jahren, der sich einer Gentherapie unterzogen hat.
    • Bereitschaft zur Teilnahme an einem persönlichen Videointerview mit einem Mitglied des Studienteams über ein persönliches Mobilgerät oder einen Computer mit funktionierender Internetverbindung.
    • Muss bereit sein, eine mündliche Einverständniserklärung abzugeben.
    • Veröffentlichung des vom Teilnehmer unterzeichneten Informationsformulars, das unserem Studienteam die Erlaubnis erteilt, den Gesundheitsdienstleister zu kontaktieren, um seine Diagnose und den Erhalt der Gentherapie (falls erhalten) zu überprüfen.
    • Erfolgreiche Überprüfung der Diagnose einer seltenen genetischen Erkrankung, die für eine Behandlung mit Gentherapie vorgesehen ist.
    • Eine positive Bestätigung über den Erhalt der Gentherapie und Art, die von ihrem Gesundheitsdienstleister erhalten wurde (nur für diejenigen, die eine Gentherapie erhalten haben).

  2. Nur für Teilnehmer der Gruppe 2 (angeboten, aber ohne Gentherapie):

    • Eltern/Betreuer von Kindern (oder jungen Erwachsenen im Alter zwischen 18 und 35 Jahren) mit einer seltenen genetischen Erkrankung, denen angeboten worden war, die jedoch nicht für eine Studie infrage kamen oder sich gegen eine Gentherapie entschieden hatten.
    • Bereitschaft zur Teilnahme an einem persönlichen Videointerview mit einem Mitglied des Studienteams über ein persönliches Mobilgerät oder einen Computer mit funktionierender Internetverbindung.
    • Muss bereit sein, eine mündliche Einverständniserklärung abzugeben.
    • Unterzeichnete Freigabe des Informationsformulars, das dem GeneTx-Studienteam die Erlaubnis erteilt, den Gesundheitsdienstleister des Teilnehmers zu kontaktieren, um die Diagnose zu überprüfen.
    • Erfolgreiche Überprüfung der Diagnose einer seltenen genetischen Erkrankung, die für eine Behandlung mit Gentherapie vorgesehen ist.

  3. Nur für Teilnehmer der Gruppe 3 (Anbieterinterviews):

    • Gesundheitspersonal, das ≥ 2 Patienten betreut hat, die eine Gentherapie erhielten.
    • Bereitschaft zur Teilnahme an einem Einzelinterview per Video (oder persönlich) mit einem Mitglied des Studienteams über ein persönliches mobiles Gerät oder einen Computer mit funktionierender Internetverbindung.
    • Einverständniserklärung eines Studienteilnehmers.

Ausschlusskriterien:

  • Teilnehmer, die nicht fließend Englisch sprechen können, werden ausgeschlossen.
  • Unfähigkeit oder mangelnde Bereitschaft des Forschungsteilnehmers, eine mündliche Einverständniserklärung abzugeben.
  • Teilnehmer ohne Zugang zu einem Computer oder Mobilgerät, das Videokommunikation unterstützt, werden ausgeschlossen.
  • Zustand oder chronische Krankheit, die nach Meinung des PI/Co-I eine Teilnahme unsicher oder unhaltbar macht (z. B. kognitive Beeinträchtigung, gleichzeitige akute Morbidität).

Studienplan

Dieser Abschnitt enthält Einzelheiten zum Studienplan, einschließlich des Studiendesigns und der Messung der Studieninhalte.

Wie ist die Studie aufgebaut?

Designdetails

Kohorten und Interventionen

Gruppe / Kohorte
Intervention / Behandlung
Elternteil/Betreuer
10-20 Patienten/Familien von Kindern mit seltenen genetischen Erkrankungen, denen eine Gentherapie angeboten wurde, die sich aber gegen eine Gentherapie entschieden haben oder die letztendlich nicht für eine klinische Studie in Frage kamen.
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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).
Gesundheitspfleger
10-20 Mitarbeiter des Gesundheitswesens, die sich um Patienten kümmern, die eine Gentherapie erhalten.
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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).
Junger Erwachsener
15-25 Eltern/Familien von Kindern (Patienten ab 8 Jahren) mit seltenen genetischen Erkrankungen, die kürzlich eine Gentherapie erhalten haben
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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.
Sonstiges: 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).

Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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).
Sonstiges: 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)
Sonstiges: 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).

Was misst die Studie?

Primäre Ergebnismessungen

Ergebnis Maßnahme
Maßnahmenbeschreibung
Zeitfenster
Verwendung halbstrukturierter Interviews zur Beurteilung der Überzeugungen, Einstellungen und Informationsbedürfnisse rund um die Gentherapie bei Patienten und Familien mit seltenen genetischen Erkrankungen.
Zeitfenster: 2 Jahre
Ausgebildete Experten werden die Studienteilnehmer befragen, um ihre Überzeugungen, Einstellungen und Informationsbedürfnisse in Bezug auf die Gentherapie für seltene pädiatrische Krankheiten unter den Krankheitsakteuren zu erläutern. Diese Interviews werden mit der gut beschriebenen strengen Methodik der semantischen Inhaltsanalyse analysiert, um Themen durch einen systematischen und standardisierten Prozess zu identifizieren.
2 Jahre

Mitarbeiter und Ermittler

Hier finden Sie Personen und Organisationen, die an dieser Studie beteiligt sind.

Sponsor

St. Jude Children's Research Hospital

Mitarbeiter

National Heart, Lung, and Blood Institute (NHLBI)

Ermittler

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

Studienaufzeichnungsdaten

Diese Daten verfolgen den Fortschritt der Übermittlung von Studienaufzeichnungen und zusammenfassenden Ergebnissen an ClinicalTrials.gov. Studienaufzeichnungen und gemeldete Ergebnisse werden von der National Library of Medicine (NLM) überprüft, um sicherzustellen, dass sie bestimmten Qualitätskontrollstandards entsprechen, bevor sie auf der öffentlichen Website veröffentlicht werden.

Haupttermine studieren

Studienbeginn (Tatsächlich)

1. Juni 2023

Primärer Abschluss (Geschätzt)

1. Dezember 2027

Studienabschluss (Geschätzt)

1. Dezember 2027

Studienanmeldedaten

Zuerst eingereicht

30. März 2023

Zuerst eingereicht, das die QC-Kriterien erfüllt hat

30. März 2023

Zuerst gepostet (Tatsächlich)

12. April 2023

Studienaufzeichnungsaktualisierungen

Letztes Update gepostet (Tatsächlich)

10. August 2026

Letztes eingereichtes Update, das die QC-Kriterien erfüllt

5. August 2026

Zuletzt verifiziert

1. August 2026

Mehr Informationen

Begriffe im Zusammenhang mit dieser Studie

Zusätzliche relevante MeSH-Bedingungen

Andere Studien-ID-Nummern

  • GENETX
  • U01HL163983 (US NIH Stipendium/Vertrag)
  • R01HL156647 (US NIH Stipendium/Vertrag)

Arzneimittel- und Geräteinformationen, Studienunterlagen

Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt

Nein

Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt

Nein

Diese Informationen wurden ohne Änderungen direkt von der Website clinicaltrials.gov abgerufen. Wenn Sie Ihre Studiendaten ändern, entfernen oder aktualisieren möchten, wenden Sie sich bitte an register@clinicaltrials.gov. Sobald eine Änderung auf clinicaltrials.gov implementiert wird, wird diese automatisch auch auf unserer Website aktualisiert .

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