Genterapiforsøk for behandling av X-bundet retinitis Pigmentosa assosiert med varianter i RPGR-genet
Fase 3 randomisert, kontrollert studie av AAV5-RPGR for behandling av X-koblet retinitis Pigmentosa assosiert med varianter i RPGR-genet
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Studietype
Registrering (Faktiske)
Fase
- Fase 3
Kontakter og plasseringer
Studiesteder
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Ghent, Belgia, 9000
- UZ Gent
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Ontario
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Toronto, Ontario, Canada, M5G 1X8
- Hospital For Sick Children
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Glostrup Municipality, Danmark, 2600
- Rigshospitalet Glostrup
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California
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La Jolla, California, Forente stater, 92093 0946
- Shiley Eye Institute Jacobs Retina Center
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Los Angeles, California, Forente stater, 90027
- Childrens Hospital
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Palo Alto, California, Forente stater, 94303
- Stanford Health Care
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Florida
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Gainesville, Florida, Forente stater, 32607
- VitreoRetinal Associates, PA
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Georgia
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Atlanta, Georgia, Forente stater, 30322
- Emory University
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Massachusetts
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Boston, Massachusetts, Forente stater, 02114
- Massachusetts General Hospital - Center for Celiac Research and Treatment
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Michigan
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Ann Arbor, Michigan, Forente stater, 48105
- Univ of Michigan Medical Center
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North Carolina
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Durham, North Carolina, Forente stater, 27705
- Duke Eye Center
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Pennsylvania
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Pittsburgh, Pennsylvania, Forente stater, 15213
- University of Pittsburgh Medical Center (UPMC)
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Texas
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Bellaire, Texas, Forente stater, 77401
- Retina Consultants of Houston
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Paris, Frankrike, 75012
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
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Jerusalem, Israel, 91120
- Hadassah Medical Center
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Florence, Italia, 50134
- Azienda Ospedaliero Universitaria Careggi
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Milan, Italia, 20142
- Ospedale San Paolo
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Naples, Italia, 80131
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
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Roma, Italia, 00198
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
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Amsterdam, Nederland, 1105AZ
- VUMC Amsterdam
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Nijmegen, Nederland, 6525EX
- Radboudumc
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Madrid, Spania, 28040
- Hosp Univ Fund Jimenez Diaz
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Edinburgh, Storbritannia, EH3 9HA
- NHS Lothian
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Glasgow, Storbritannia, G12 0YN
- Gartnavel General Hospital
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Leeds, Storbritannia, LS9 7TF
- St James University Hospital
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London, Storbritannia, EC1V 2PD
- Moorfields Eye Hospital
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Basel, Sveits, 4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
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Lausanne, Sveits, 1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
Inclusion Criteria:
- Male or female
- 3 years of age or older
- Has XLRP confirmed by a retinal specialist and has a predicted disease-causing sequence variant in RPGR confirmed by an accredited laboratory
Exclusion Criteria:
- Has had ocular surgery within 3 months prior to screening or is anticipated to require ocular surgery within 6 months after the study intervention administration
- Any investigational ocular treatment or any other ocular treatment that could confound the interpretation of the efficacy results or affect participant compliance with the visit schedule
- Has undergone prior retinal surgery involving the macula, macular laser photocoagulation, external-beam radiation therapy, transpupillary thermotherapy, glaucoma filtration surgery or corneal surgery
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: Randomisert
- Intervensjonsmodell: Parallell tildeling
- Masking: Enkelt
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
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Eksperimentell: Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navn:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
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Annen: Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navn:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Eksperimentell: Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navn:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
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Change From Baseline to Week 52 in Vision-guided Mobility Assessment (VMA) as Measured by the Ability of the Participant to Navigate Through a VMA Maze
Tidsramme: From Baseline to 52 Weeks
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Change from baseline to Week 52 in VMA as measured by the ability of the participant to navigate through a VMA maze. |
From Baseline to 52 Weeks
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Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
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Change From Baseline in Mean Retinal Sensitivity Within the Central 10 Degrees Excluding Scotoma (Mean Retinal Sensitivity Within the Central 10 Degree Excluding Scotoma in Static Perimetry [MRS10]) in Static Perimetry at Week 52
Tidsramme: From Baseline to Week 52
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Change from baseline in mean retinal sensitivity within the central 10 degrees excluding scotoma (MRS10) in static perimetry at Week 52 will be assessed. |
From Baseline to Week 52
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Change From Baseline in Mean Retinal Sensitivity of Worse-seeing Eye Within the Central 10 Degrees Excluding Scotoma in Static Perimetry (MRS10) at Week 52
Tidsramme: From Baseline to Week 52
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Change from baseline in mean retinal sensitivity of worse-seeing eye within the central 10 degrees excluding scotoma in static perimetry (MRS10) at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Tidsramme: From Baseline to Week 52
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Pointwise response in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in Full Visual Field at Week 52
Tidsramme: From Baseline to Week 52
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Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Tidsramme: From Baseline to Week 52
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Pointwise response in the central 30 degrees visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Retinal Function as Assessed by Pointwise Response in Worse-seeing Eye in the Central 30 Degrees Visual Field at Week 52
Tidsramme: From Baseline to Week 52
|
Pointwise response in worse-seeing eye in the central 30 degrees visual field at Week 52 will be assessed. |
From Baseline to Week 52
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Change From Baseline in Retinal Function as Assessed by Mean Retinal Sensitivity Within the Full Visual Field (MRS90) in Static Perimetry at Week 52
Tidsramme: From Baseline to Week 52
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Change from baseline in retinal function as assessed by mean retinal sensitivity within the full visual field (MRS90) in static perimetry at Week 52 will be assessed. |
From Baseline to Week 52
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Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
Tidsramme: From Baseline to Week 52
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Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52. |
From Baseline to Week 52
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Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Tidsramme: From Baseline to Week 52
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Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by Monocular Low Luminance Visual Acuity Using the Early Treatment Diabetic Retinopathy Study (ETDRS) Chart Letter score at Week 52
Tidsramme: From Baseline to Week 52
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Change from baseline in visual function as assessed by monocular low luminance visual acuity using the ETDRS chart letter score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by monocular Best Corrected Visual Acuity (BCVA) Using the ETDRS Chart Letter Score at Week 52
Tidsramme: From Baseline to Week 52
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Change From Baseline in visual function as assessed by monocular BCVA using the ETDRS chart letter score at Week 52. |
From Baseline to Week 52
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Change From Baseline in Visual Function as Assessed by Low Luminance Visual Acuity Using the ETDRS Chart Letter Score in Worse-seeing Eye at Week 52
Tidsramme: From Baseline to Week 52
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Change from baseline in visual function as assessed by low luminance visual acuity using the ETDRS chart letter score in worse-seeing eye at Week 52. |
From Baseline to Week 52
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Number of Participants with Ocular and Non-ocular Adverse Events
Tidsramme: Day 1 - Week 52
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Number of participants with ocular and non-ocular adverse events will be assessed. |
Day 1 - Week 52
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Number of Participants With Abnormalities in Laboratory Assessments
Tidsramme: Day 1 - 52 Weeks
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Number of participants with abnormalities in laboratory assessments will be assessed. |
Day 1 - 52 Weeks
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Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Study Director, MeiraGTx Ocular UK Ltd
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Faktiske)
Studiet fullført (Faktiske)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Andre studie-ID-numre
- CR109258
- 2020-002873-88 (EudraCT-nummer)
- MGT-RPGR-021 (Annen identifikator: MeiraGTx Ocular UK Ltd)
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
Legemiddel- og utstyrsinformasjon, studiedokumenter
Studerer et amerikansk FDA-regulert medikamentprodukt
Studerer et amerikansk FDA-regulert enhetsprodukt
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