Comunicazione sulla terapia genica: uso di una valutazione dei bisogni per guidare la decisione-AIDS per la terapia genica per le malattie rare (GENETX)
5 agosto 2026 aggiornato da: St. Jude Children's Research Hospital
Questo studio prospettico di intervista a metodo misto mira a descrivere qualitativamente le convinzioni, gli atteggiamenti e le esigenze informative sulla terapia genica per le malattie pediatriche rare tra i pazienti e i genitori di bambini con una malattia rara destinati al trattamento utilizzando tecniche di terapia genica.
Utilizzando le intuizioni apprese, il team svilupperà una piattaforma online che fornisce contenuti educativi e aiuti decisionali per i pazienti e le loro famiglie.
Panoramica dello studio
Stato
Reclutamento
Condizioni
Intervento / Trattamento
Descrizione dettagliata
Il team di studio cerca di comprendere le esigenze uniche dei pazienti e dei genitori di bambini che stanno prendendo in considerazione nuovi trattamenti sperimentali di terapia genica.
Il team dello studio coinvolgerà le parti interessate dei pazienti, degli operatori sanitari e degli operatori sanitari per comprendere i loro atteggiamenti, convinzioni e preoccupazioni relative a questi trattamenti.
Sulla base di queste intuizioni, il team, con il supporto delle suddette parti interessate e di un fornitore esterno, svilupperà una piattaforma web che offre contenuti educativi e ausili decisionali per migliorare la loro esperienza e la soddisfazione generale del processo di prendere decisioni terapeutiche.
Ciò è particolarmente importante in quanto le nuove terapie per la SCD includeranno sempre più l'editing genetico negli studi pediatrici nei prossimi due anni.
Questi sforzi colmeranno lacune significative nel materiale educativo disponibile per coloro che prendono in considerazione la terapia genica e (a conoscenza del team) creeranno una risorsa che sarà la prima del suo genere.
Tipo di studio
Osservativo
Iscrizione (Stimato)
145
Contatti e Sedi
Questa sezione fornisce i recapiti di coloro che conducono lo studio e informazioni su dove viene condotto lo studio.
Contatto studio
- Nome: Liza-Marie Johnson, MD, MPH, MSB
- Numero di telefono: 888-226-4343
- Email: referralinfo@stjude.org
Luoghi di studio
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Tennessee
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Memphis, Tennessee, Stati Uniti, 38105
- Reclutamento
- St. Jude Children's Research Hospital
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Contatto:
- Numero di telefono: 888-226-4343
- Email: referralinfo@stjude.org
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Criteri di partecipazione
I ricercatori cercano persone che corrispondano a una certa descrizione, chiamata criteri di ammissibilità. Alcuni esempi di questi criteri sono le condizioni generali di salute di una persona o trattamenti precedenti.
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
Sì
Metodo di campionamento
Campione non probabilistico
Popolazione di studio
Circa 45-50 giovani adulti (età 18-35) o genitori di bambini affetti da una malattia rara mirata al trattamento mediante tecniche di terapia genica e 10-20 operatori sanitari che prestano assistenza ai pazienti sottoposti a terapia genica.
Descrizione
Criterio di inclusione:
Solo per i partecipanti del gruppo 1 (sottoposti a terapia genica):
- Genitore/caregiver il cui bambino è stato sottoposto a terapia genica. OPPURE Giovane adulto di età compresa tra 18 e 35 anni sottoposto a terapia genica.
- Disponibilità a partecipare a un colloquio video individuale con un membro del team di studio utilizzando un dispositivo mobile personale o un computer con una connessione Internet funzionante.
- Deve essere disposto a fornire il consenso informato verbale.
- Rilascio del modulo informativo firmato dal partecipante che fornisce al nostro team di studio il permesso di contattare l'operatore sanitario per verificare la diagnosi e la ricezione della terapia genica (se ricevuta).
- Verifica riuscita della diagnosi di malattia genetica rara mirata al trattamento mediante terapia genica.
- Una conferma positiva al ricevimento della terapia genica e del tipo ricevuto dal proprio operatore sanitario (solo per coloro che hanno ricevuto la terapia genica).
Solo per i partecipanti del gruppo 2 (offerti, ma non sottoposti a terapia genica):
- Genitore/tutore di bambini (o giovani adulti di età compresa tra 18 e 35 anni) con una malattia genetica rara a cui era stata offerta ma non erano idonei per una sperimentazione o avevano deciso di non ricevere la terapia genica.
- Disponibilità a partecipare a un colloquio video individuale con un membro del team di studio utilizzando un dispositivo mobile personale o un computer con una connessione Internet funzionante.
- Deve essere disposto a fornire il consenso informato verbale.
- Rilascio firmato del modulo informativo che fornisce al team dello studio GeneTx il permesso di contattare l'operatore sanitario del partecipante per verificare la diagnosi.
- Verifica riuscita della diagnosi di malattia genetica rara mirata al trattamento mediante terapia genica.
Solo per i partecipanti del gruppo 3 (colloqui con i fornitori):
- Operatore sanitario che ha prestato assistenza a ≥ 2 pazienti sottoposti a terapia genica.
- Disponibilità a partecipare a interviste video individuali (o di persona) con un membro del team di studio utilizzando un dispositivo mobile personale o un computer con connessione Internet funzionante.
- Consenso informato di un partecipante allo studio.
Criteri di esclusione:
- Saranno esclusi i partecipanti che non sono in grado di conversare fluentemente in inglese.
- Incapacità o riluttanza del partecipante alla ricerca a dare il consenso informato verbale.
- Saranno esclusi i partecipanti che non hanno accesso a un computer o dispositivo mobile che supporti le comunicazioni video.
- Condizione o malattia cronica che, a parere del PI/Co-I, rende la partecipazione non sicura o insostenibile (es. deterioramento cognitivo, concomitante morbilità acuta).
Piano di studio
Questa sezione fornisce i dettagli del piano di studio, compreso il modo in cui lo studio è progettato e ciò che lo studio sta misurando.
Come è strutturato lo studio?
Dettagli di progettazione
Coorti e interventi
Gruppo / Coorte |
Intervento / Trattamento |
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Genitore/tutore
10-20 pazienti/famiglie di bambini con malattie genetiche rare che sono stati offerti ma hanno deciso di non ricevere la terapia genica o che alla fine non erano idonei per una sperimentazione clinica.
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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).
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).
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).
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).
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).
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)
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).
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Operatore Sanitario
10-20 operatori sanitari che prestano assistenza ai pazienti sottoposti a terapia genica.
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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).
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).
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).
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).
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).
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)
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).
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Giovane adulto
15-25 genitori/famiglie di bambini (pazienti di età pari o superiore a 8 anni) con rare malattie genetiche, che hanno recentemente ricevuto la terapia genica
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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).
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).
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).
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).
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).
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)
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).
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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
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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).
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).
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).
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).
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).
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)
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).
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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
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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).
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).
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).
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).
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).
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)
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).
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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
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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).
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).
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).
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).
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).
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)
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).
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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.
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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).
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).
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).
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).
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).
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)
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).
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Uso di interviste semi-strutturate per valutare le convinzioni, gli atteggiamenti e le esigenze informative sulla terapia genica tra pazienti e famiglie con malattie genetiche rare.
Lasso di tempo: 2 anni
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Esperti qualificati intervisteranno i partecipanti allo studio per chiarire le loro convinzioni, atteggiamenti e bisogni informativi sulla terapia genica per le malattie pediatriche rare tra le parti interessate della malattia.
Queste interviste saranno analizzate attraverso la metodologia rigorosa ben descritta dell'analisi del contenuto semantico per identificare i temi attraverso un processo sistematico e standardizzato.
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2 anni
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Collaboratori e investigatori
Qui è dove troverai le persone e le organizzazioni coinvolte in questo studio.
Collaboratori
Investigatori
- Investigatore principale: Liza-Marie Johnson, MD, MPH, MSB, St. Jude Children's Research Hospital
Studiare le date dei record
Queste date tengono traccia dell'avanzamento della registrazione dello studio e dell'invio dei risultati di sintesi a ClinicalTrials.gov. I record degli studi e i risultati riportati vengono esaminati dalla National Library of Medicine (NLM) per assicurarsi che soddisfino specifici standard di controllo della qualità prima di essere pubblicati sul sito Web pubblico.
Studia le date principali
Inizio studio (Effettivo)
1 giugno 2023
Completamento primario (Stimato)
1 dicembre 2027
Completamento dello studio (Stimato)
1 dicembre 2027
Date di iscrizione allo studio
Primo inviato
30 marzo 2023
Primo inviato che soddisfa i criteri di controllo qualità
30 marzo 2023
Primo Inserito (Effettivo)
12 aprile 2023
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
10 agosto 2026
Ultimo aggiornamento inviato che soddisfa i criteri QC
5 agosto 2026
Ultimo verificato
1 agosto 2026
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Malattie genetiche, congenite
- Malattie ematologiche
- Anemia, emolitica, congenita
- Anemia, emolitico
- Anemia
- Emoglobinopatie
- Malattie e anomalie congenite, ereditarie e neonatali
- Malattie emiche e linfatiche
- Anemia, anemia falciforme
- Qualità, accesso e valutazione dell'assistenza sanitaria
- Tecniche investigative
- Metodi epidemiologici
- Raccolta dei dati
- Meccanismi di valutazione dell'assistenza sanitaria
- Qualità dell'assistenza sanitaria
- Sanità pubblica
- Ambiente e salute pubblica
- Interviste come argomento
Altri numeri di identificazione dello studio
- GENETX
- U01HL163983 (Sovvenzione/contratto NIH degli Stati Uniti)
- R01HL156647 (Sovvenzione/contratto NIH degli Stati Uniti)
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
No
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
No
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .