Komunikace v rámci genové terapie: Využití posouzení potřeb k podpoře rozhodování-AIDS pro genovou terapii vzácných onemocnění (GENETX)
5. srpna 2026 aktualizováno: St. Jude Children's Research Hospital
Tato prospektivní studie rozhovorů se smíšenou metodou si klade za cíl kvalitativně popsat názory, postoje a informační potřeby týkající se genové terapie vzácných dětských onemocnění mezi pacienty a rodiči dětí se vzácným onemocněním, u nichž je léčba pomocí technik genové terapie zaměřena.
Na základě získaných poznatků tým vyvine online platformu poskytující vzdělávací obsah a pomůcky pro rozhodování pacientů pro pacienty a jejich rodiny.
Přehled studie
Postavení
Nábor
Podmínky
Detailní popis
Studijní tým se snaží porozumět jedinečným potřebám pacientů a rodičů dětí, kteří zvažují novou experimentální léčbu genovou terapií.
Studijní tým zapojí pacienty, pečovatele a zdravotnické pracovníky, aby porozuměli jejich postojům, názorům a obavám souvisejícím s touto léčbou.
Na základě těchto poznatků tým, s podporou výše uvedených zainteresovaných stran a externího dodavatele, vyvine webovou platformu nabízející vzdělávací obsah a pomůcky pro rozhodování, aby zvýšil jejich zkušenosti a celkovou spokojenost s procesem rozhodování o léčbě.
To je zvláště důležité, protože nové terapie SCD budou v příštích dvou letech stále více zahrnovat úpravy genů v pediatrických studiích.
Toto úsilí se zaměří na značné mezery ve vzdělávacím materiálu dostupném těm, kdo uvažují o genové terapii, a (podle znalostí týmu) vytvoří zdroj, který bude první svého druhu.
Typ studie
Pozorovací
Zápis (Odhadovaný)
145
Kontakty a umístění
Tato část poskytuje kontaktní údaje pro ty, kteří studii provádějí, a informace o tom, kde se tato studie provádí.
Studijní kontakt
- Jméno: Liza-Marie Johnson, MD, MPH, MSB
- Telefonní číslo: 888-226-4343
- E-mail: referralinfo@stjude.org
Studijní místa
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Tennessee
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Memphis, Tennessee, Spojené státy, 38105
- Nábor
- St. Jude Children's Research Hospital
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Kontakt:
- Telefonní číslo: 888-226-4343
- E-mail: referralinfo@stjude.org
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Kritéria účasti
Výzkumníci hledají lidi, kteří odpovídají určitému popisu, kterému se říká kritéria způsobilosti. Některé příklady těchto kritérií jsou celkový zdravotní stav osoby nebo předchozí léčba.
Kritéria způsobilosti
Věk způsobilý ke studiu
- Dítě
- Dospělý
- Starší dospělý
Přijímá zdravé dobrovolníky
Ano
Metoda odběru vzorků
Vzorek nepravděpodobnosti
Studijní populace
Asi 45–50 mladých dospělých (ve věku 18–35 let) nebo rodičů dětí, u kterých bylo ověřeno, že mají vzácné onemocnění cílené na léčbu pomocí technik genové terapie, a 10–20 zdravotnických pracovníků, kteří poskytují péči pacientům podstupujícím genovou terapii.
Popis
Kritéria pro zařazení:
Pouze pro účastníky skupiny 1 (podstupující genovou terapii):
- Rodič/pečovatel, jehož dítě prošlo genovou terapií. NEBO Mladý dospělý ve věku od 18 do 35 let, který podstoupil genovou terapii.
- Ochota zúčastnit se individuálního video rozhovoru se členem studijního týmu pomocí osobního mobilního zařízení nebo počítače s funkčním připojením k internetu.
- Musí být ochoten poskytnout ústní informovaný souhlas.
- Uvolnění informačního formuláře podepsaného účastníkem, který poskytuje našemu studijnímu týmu povolení kontaktovat poskytovatele zdravotní péče za účelem ověření diagnózy a přijetí genové terapie (pokud byla přijata).
- Úspěšné ověření diagnózy vzácného genetického onemocnění cílené na léčbu pomocí genové terapie.
- Pozitivní potvrzení o přijetí genové terapie a typu obdržené od poskytovatele zdravotní péče (pouze pro ty, kteří podstoupili genovou terapii).
Pouze pro účastníky skupiny 2 (nabízeno, ale nepodstoupilo genovou terapii):
- Rodič/pečovatel dětí (nebo mladých dospělých ve věku od 18 let do 35 let) se vzácným genetickým onemocněním, které bylo nabídnuto, ale nebylo způsobilé pro zkoušku nebo se rozhodlo nepodstoupit genovou terapii.
- Ochota zúčastnit se individuálního video rozhovoru se členem studijního týmu pomocí osobního mobilního zařízení nebo počítače s funkčním připojením k internetu.
- Musí být ochoten poskytnout ústní informovaný souhlas.
- Podepsané uvolnění formuláře s informacemi poskytující týmu studie GeneTx povolení kontaktovat poskytovatele zdravotní péče účastníka za účelem ověření diagnózy.
- Úspěšné ověření diagnózy vzácného genetického onemocnění cílené na léčbu pomocí genové terapie.
Pouze pro účastníky skupiny 3 (rozhovory s poskytovatelem):
- Zdravotnický pracovník, který poskytl péči ≥ 2 pacientům léčeným genovou terapií.
- Ochota zúčastnit se individuálního video (nebo osobního) rozhovoru se členem studijního týmu pomocí osobního mobilního zařízení nebo počítače s funkčním připojením k internetu.
- Informovaný souhlas účastníka studie.
Kritéria vyloučení:
- Účastníci, kteří nebudou schopni plynule konverzovat anglicky, budou vyloučeni.
- Neschopnost nebo neochota účastníka výzkumu dát verbální informovaný souhlas.
- Účastníci, kteří nemají přístup k počítači nebo mobilnímu zařízení, které podporuje video komunikaci, budou vyloučeni.
- Stav nebo chronické onemocnění, které podle názoru PI/Co-I činí účast nebezpečnou nebo neudržitelnou (tj. kognitivní porucha, souběžná akutní morbidita).
Studijní plán
Tato část poskytuje podrobnosti o studijním plánu, včetně toho, jak je studie navržena a co studie měří.
Jak je studie koncipována?
Detaily designu
Kohorty a intervence
Skupina / kohorta |
Intervence / Léčba |
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Rodič/pečovatel
10–20 pacientů/rodin dětí se vzácnými genetickými chorobami, kterým bylo nabídnuto, ale rozhodli se nepodstoupit genovou terapii nebo kteří nakonec nebyli způsobilí pro klinickou studii.
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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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Zdravotnický pracovník
10-20 zdravotnických pracovníků, kteří poskytují péči pacientům podstupujícím genovou terapii.
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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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Mladý dospělý
15-25 rodičů/rodin dětí (pacienti ve věku 8 a více let) se vzácnými genetickými chorobami, kteří nedávno dostali genovou terapii
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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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Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
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Použití polostrukturovaných rozhovorů k posouzení názorů, postojů a informačních potřeb v oblasti genové terapie mezi pacienty a rodinami se vzácnými genetickými chorobami.
Časové okno: 2 roky
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Vyškolení odborníci povedou rozhovory s účastníky studie, aby objasnili jejich přesvědčení, postoje a informační potřeby týkající se genové terapie vzácných dětských onemocnění mezi zúčastněnými stranami.
Tyto rozhovory budou analyzovány pomocí dobře popsané rigorózní metodologie analýzy sémantického obsahu, aby bylo možné identifikovat témata prostřednictvím systematického a standardizovaného procesu.
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2 roky
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Spolupracovníci a vyšetřovatelé
Zde najdete lidi a organizace zapojené do této studie.
Spolupracovníci
Vyšetřovatelé
- Vrchní vyšetřovatel: Liza-Marie Johnson, MD, MPH, MSB, St. Jude Children's Research Hospital
Termíny studijních záznamů
Tato data sledují průběh záznamů studie a předkládání souhrnných výsledků na ClinicalTrials.gov. Záznamy ze studií a hlášené výsledky jsou před zveřejněním na veřejné webové stránce přezkoumány Národní lékařskou knihovnou (NLM), aby se ujistily, že splňují specifické standardy kontroly kvality.
Hlavní termíny studia
Začátek studia (Aktuální)
1. června 2023
Primární dokončení (Odhadovaný)
1. prosince 2027
Dokončení studie (Odhadovaný)
1. prosince 2027
Termíny zápisu do studia
První předloženo
30. března 2023
První předloženo, které splnilo kritéria kontroly kvality
30. března 2023
První zveřejněno (Aktuální)
12. dubna 2023
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
10. srpna 2026
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
5. srpna 2026
Naposledy ověřeno
1. srpna 2026
Více informací
Termíny související s touto studií
Další relevantní podmínky MeSH
- Genetické choroby, vrozené
- Hematologická onemocnění
- Anémie, hemolytická, vrozená
- Anémie, hemolytika
- Anémie
- Hemoglobinopatie
- Vrozené, dědičné a neonatální nemoci a abnormality
- Hemická a lymfatická onemocnění
- Anémie, srpkovitá anémie
- Kvalita zdravotní péče, přístup a hodnocení
- Vyšetřovací techniky
- Epidemiologické metody
- Sběr dat
- Mechanismy hodnocení zdravotní péče
- Kvalita zdravotní péče
- Veřejné zdraví
- Životní prostředí a veřejné zdraví
- Rozhovory jako téma
Další identifikační čísla studie
- GENETX
- U01HL163983 (Grant/smlouva NIH USA)
- R01HL156647 (Grant/smlouva NIH USA)
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Ne
Studuje produkt zařízení regulovaný americkým úřadem FDA
Ne
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