Langtidsopfølgningsgenterapiundersøgelse for XLRP RPGR
Langtidsopfølgningsundersøgelse af deltagere efter et åbent, multicenter, fase I/II dosiseskaleringsforsøg af en rekombinant adeno-associeret virusvektor (AAV2-RPGR) til genterapi af voksne og børn med X-linked retinitis Pigmentosa På grund af defekter i Retinitis Pigmentosa GTPase Regulator (RPGR)
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Faktiske)
Kontakter og lokationer
Studiesteder
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-
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Leeds, Det Forenede Kongerige, LS9 7TF
- Leeds Teaching Hospitals NHS Trust
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London, Det Forenede Kongerige, EC1V 9EL
- Moorfields Eye Hospital
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Massachusetts
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Boston, Massachusetts, Forenede Stater, 02114
- Massachusetts Eye and Ear Infirmary
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Michigan
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Ann Arbor, Michigan, Forenede Stater, 48105
- Univ of Michigan Medical Center
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Pennsylvania
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Pittsburgh, Pennsylvania, Forenede Stater, 15213
- University of Pittsburgh Medical Center
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Prøveudtagningsmetode
Studiebefolkning
Males receiving AAV5-hRKp.RPGR in the MGT009 study
Beskrivelse
Inclusion Criteria:
- Received AAV5-hRKp.RPGR in the MGT009 Study
Exclusion Criteria:
- Unwilling or unable to meet with the requirements of the study
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Observationsmodeller: COHORT
- Tidsperspektiver: PROSPECTIVE
Kohorter og interventioner
Gruppe / kohorte |
Intervention / Behandling |
|---|---|
|
Follow up cohort
no intervention follow up study |
Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Assess the Longer-term Safety of AAV5-hRKp.RPGR Administered to Participants
Tidsramme: 60 Months
|
Longer-term safety of subretinal AAV5-hRKp.RPGR administered to participants will be reported. |
60 Months
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Change in Functional Vision of Walk Time in Vision-guided Mobility Assessment (VMA)
Tidsramme: Up to 60 Months
|
Change in functional vision of walk time after treatment administration in VMA (Version 1.0) will be reported. |
Up to 60 Months
|
|
Change in Functional Vision of Low Luminance Questionnaire (LLQ) Domain Scores in Patient Reported Outcome (PRO)
Tidsramme: Up to 60 Months
|
Change in functional vision of LLQ domain scores in PRO will be reported. The instrument is scored by domain, computed by scaling individual items from 0 to 100 and then averaging the individual items for each domain. A higher score reflects higher functional level. |
Up to 60 Months
|
|
Change in Best Corrected Visual Acuity (BCVA) Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter Score
Tidsramme: Up to 60 Months
|
The longer-term efficacy of AAV5-hRKp.RPGR in change in BCVA using ETDRS chart letter score will be reported. |
Up to 60 Months
|
|
Change in Low Luminance Visual Acuity (LLVA) as Assessed by ETDRS Chart Under Low Luminance Conditions
Tidsramme: Up to 60 Months
|
Change in LLVA as assessed by ETDRS chart under low luminance conditions will be reported. |
Up to 60 Months
|
|
Change in Mean Retinal Sensitivity Within the Central 10 Degree Visual Field Excluding Scotoma (MRS10) in Static Perimetry
Tidsramme: Up to 60 months
|
Change in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry will be reported. |
Up to 60 months
|
|
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Full Visual Field
Tidsramme: Up to 60 months
|
Change in retinal sensitivity by pointwise comparison of data in static perimetry within the full visual field will be reported. |
Up to 60 months
|
|
Change In Retinal Sensitivity by Pointwise Comparison of Data in Static Perimetry Within the Central 30 Degrees Visual Field
Tidsramme: Up to 60 months
|
Change in retinal sensitivity by pointwise comparison of data in static perimetry within the Central 30 Degrees visual field will be reported. |
Up to 60 months
|
|
Change in Mean Retinal Sensitivity Within the Full Visual Field Excluding Scotoma (MRS90) in Static Perimetry
Tidsramme: Up to 60 months
|
Change in mean retinal sensitivity within the full visual field excluding scotoma (MRS90) in static perimetry will be reported. |
Up to 60 months
|
Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Studieleder: Study Director, MeiraGTx Ocular UK Ltd
Datoer for undersøgelser
Studer store datoer
Studiestart (Faktiske)
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
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
Andre undersøgelses-id-numre
- MGT010
- 2018 (U.S. NIH-bevilling/kontrakt)
- 2018-000425-31 (EudraCT nummer)
- MGT010 (Anden identifikator: MeiraGTx Ocular UK Ltd)
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
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