Natural History Study of Advanced RCD (ECSTASI)
External Control, preScreening, and naTurAl hiStory of Advanced retInal Degeneration Due to Rod-cone Dystrophy (ECSTASI)
ECSTASI is a Natural History Study, which will be monitoring how standard of care (SoC) affect advanced rod-cone dystrophy (RCD), a type of eye disease also known as retinitis pigmentosa. No experimental, investigational or any other drug or treatment will be tested during the study.
This study collects information in 3 parts:
Part 1: retrospective health information from medical records (up to past 10 years), Part 2: prospective health information from in-person visits (up to 1 year), and Part 3: prospective health information from medical records (up to 1 year). Participants will decide if they wish to participate in all or only to some of these parts.
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Anslået)
Fase
- Ikke anvendelig
Kontakter og lokationer
Studiekontakt
- Navn: Medical Director
Undersøgelse Kontakt Backup
- Navn: SparingVision
- Telefonnummer: +33143462060
- E-mail: info@sparingvision.com
Studiesteder
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Ghent, Belgien
- Rekruttering
- Ghent University Hospital
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Kontakt:
- Ophthalmic Clinical Trials Unit
- E-mail: OCTU@uzgent.be
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Ledende efterforsker:
- Bart Leroy, Prof
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Paris, Frankrig
- Ikke rekrutterer endnu
- CHNO XV-XX Paris - CIC 1423
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Kontakt:
- Isabelle Audo, Prof.
- Telefonnummer: 00 33 1 40 02 14 57
- E-mail: isabelle.audo@inserm.fr
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Ledende efterforsker:
- Isabelle Audo, Dr
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Dublin, Irland
- Ikke rekrutterer endnu
- Royal Victoria Eye And Ear Hospital
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Kontakt:
- Telefonnummer: 00 353 1 664 4600
- E-mail: Emma.duignan@rveeh.ie
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Ledende efterforsker:
- Emma Duignan, Dr
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- Males and females 18 years of age or older, with history of formed vision
- Clinical diagnosis of advanced retinal degeneration due to RCD
- BCVA no better than (≥) 1.0 LogMAR (≤35 Early-Treatment Diabetic Retinopathy Study [ETDRS] letters, or 20/200 Snellen equivalent) in at least one eye
- Documented preservation of foveal Outer Nuclear Layer (ONL) in at least one eye
Exclusion Criteria:
- Individuals who are receiving or have received an investigational medicinal product (IMP) in another clinical trial are excluded from the prospective part . However, they can participate in the retrospective part of the study.
- Individuals with a medical condition (systemic or ophthalmic), psychiatric condition (including substance abuse disorder), physical examination or laboratory findings that may in the opinion of the Investigator preclude adherence to the scheduled study visits, safe participation in the study, or affect study results are excluded from participating in in-person study visits. However, they can participate in the collection of data reported during both retrospective and prospective SoC routine eye examinations.
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Andet
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
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Eksperimentel: Prospective and/or retrospective data collection
This Natural History study is designed as a combination of prospective and retrospective evaluations in patients diagnosed with advanced RCD. Prospective data will be collected during in-person study visits and/or SoC routine eye examinations (not planned per protocol) as they are reported in the medical records of the participant for up to 1 year after study enrollment. Retrospective data will be collected from the medical records of the participant for up to 10 years before enrollment. |
Routine eye examinations (including slit lamp exam and dilated fundus exam), standard visual assessments (such as best corrected visual acuity test and ocular coherence tomography), evaluation of visual function (such as microperimetry). Patient-reported quality of life questionnaire. |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
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Best-corrected visual acuity (BCVA)
Tidsramme: At 6 months
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Change in BCVA from Baseline to Month 6 |
At 6 months
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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To evaluate the effects of SoC on visual function at 1 year of follow up
Tidsramme: At 1 year
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Change in BCVA from baseline to Year 1 |
At 1 year
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To determine the precision and reliability of the assessments of visual function measured in ECSTASI
Tidsramme: 1 year
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Test-retest variability and signal-to-noise ratio of visual function measurements such as BCVA and microperimetry. |
1 year
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To evaluate participant potential eligibility for the Phase 1/2a clinical trial of SPVN20 (NYRVANA).
Tidsramme: 1 year
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Percentage of participants potentially eligible to participate in the Phase 1/2a clinical trial of SPVN20 (NYRVANA), based on data collected in the ECSTASI study. |
1 year
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To evaluate the effects of SOC on visual function at 6 months and 1 year of follow-up for FST
Tidsramme: 1 year
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Change in FST from baseline to Month 6 and Year 1 |
1 year
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To evaluate the effects of SOC on visual function at 6 months and 1 year of follow-up for microperimetry
Tidsramme: 1 year
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Change in microperimetry foveal sensitivity from baseline to Month 6 and Year 1 |
1 year
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To evaluate the effects of SOC on visual function at 6 months and 1 year of follow-up for eye dominance
Tidsramme: 1 year
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Change in eye dominance from baseline to Month 6 and Year 1 |
1 year
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Samarbejdspartnere og efterforskere
Sponsor
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
Yderligere relevante MeSH-vilkår
- Genetiske sygdomme, medfødte
- Øjensygdomme
- Øjensygdomme, arvelig
- Nethindesygdomme
- Nethindedystrofier
- Nethindedegeneration
- Medfødte, arvelige og neonatale sygdomme og abnormiteter
- Keglestangsdystrofier
- Retinitis Pigmentosa
- Sundhedsvæsenets kvalitet, adgang og evaluering
- Undersøgelsesteknikker
- Epidemiologiske metoder
- Dataindsamling
- Evalueringsmekanismer til sundhedsvæsenet
- Sundhedskvalitet
- Folkesundhed
- Miljø og folkesundhed
- Undersøgelser og spørgeskemaer
Andre undersøgelses-id-numre
- SPVN-NHS-03
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
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