Langtidsopfølgningsundersøgelse af AAVanc80-hOTOF genterapi
En evaluering af den langsigtede sikkerhed og effektivitet af AAVanc80-hOTOF genterapi hos personer med sensorineuralt høretab på grund af Otoferlin-genmutationer
This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have previously received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.
Studieoversigt
Status
Betingelser
Detaljeret beskrivelse
After completing one year of initial safety and efficacy assessments following an intracochlear administration of AAVAnc80-hOTOF in AK-OTOF-101, participants will continue to be observed for an additional approximately four years of safety and efficacy follow-up in this LTFU study, for a total of approximately five years of safety and efficacy observations after vector administration. For each participant, evaluations in the LTFU study will occur at approximately one and a half years and two years after vector administration, followed by annual visits through Year 5; the duration in this LTFU study is approximately four years, with the aim of understanding and mitigating any risks related to delayed adverse events, as well as characterizing the durability of effect of the AAVAnc80-hOTOF.
Undersøgelsestype
Tilmelding (Anslået)
Kontakter og lokationer
Studiesteder
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Iowa
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Iowa City, Iowa, Forenede Stater, 52242
- University of Iowa
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Massachusetts
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Boston, Massachusetts, Forenede Stater, 02115
- Boston Children's Hospital
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Pennsylvania
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Philadelphia, Pennsylvania, Forenede Stater, 19146
- Children's Hospital of Philadelphia
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Barcelona
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Esplugues de Llobregat, Barcelona, Spanien, 08950
- Hospital Sant Joan de Déu
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Taiwan
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Taipei, Taiwan, Taiwan, (R.O.C), 100
- National Taiwan University Hospital
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
- Barn
- Voksen
- Ældre voksen
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Prøveudtagningsmetode
Studiebefolkning
Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.
Beskrivelse
Inclusion Criteria:
- Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial
- Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate)
Exclusion Criteria:
- Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Observationsmodeller: CASE_ONLY
- Tidsperspektiver: PROSPECTIVE
Kohorter og interventioner
Gruppe / kohorte |
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Study Participants
Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)
Tidsramme: Through study completion, approximately four years.
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The following adverse events will be collected:
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Through study completion, approximately four years.
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Auditory Brainstem Response (ABR) Threshold
Tidsramme: Through study completion, approximately four years.
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Changes in ABR intensity threshold (decibels normal hearing level [dB nHL]) |
Through study completion, approximately four years.
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Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studieleder: Sarah Neuhaus, D.O., Eli Lilly and Company
Publikationer og nyttige links
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Anslået)
Studieafslutning (Anslået)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Faktiske)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- AK-OTOF-LTFU
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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