Genterapiforsøg til behandling af X-linked Retinitis Pigmentosa associeret med varianter i RPGR-genet
Fase 3 randomiseret, kontrolleret undersøgelse af AAV5-RPGR til behandling af X-linked Retinitis Pigmentosa associeret med varianter i RPGR-genet
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Studieoversigt
Status
Betingelser
Intervention / Behandling
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 3
Kontakter og lokationer
Studiesteder
-
-
-
Ghent, Belgien, 9000
- UZ Gent
-
-
-
-
Ontario
-
Toronto, Ontario, Canada, M5G 1X8
- Hospital For Sick Children
-
-
-
-
-
Glostrup Municipality, Danmark, 2600
- Rigshospitalet Glostrup
-
-
-
-
-
Edinburgh, Det Forenede Kongerige, EH3 9HA
- NHS Lothian
-
Glasgow, Det Forenede Kongerige, G12 0YN
- Gartnavel General Hospital
-
Leeds, Det Forenede Kongerige, LS9 7TF
- St James University Hospital
-
London, Det Forenede Kongerige, EC1V 2PD
- Moorfields Eye Hospital
-
-
-
-
California
-
La Jolla, California, Forenede Stater, 92093 0946
- Shiley Eye Institute Jacobs Retina Center
-
Los Angeles, California, Forenede Stater, 90027
- Childrens Hospital
-
Palo Alto, California, Forenede Stater, 94303
- Stanford Health Care
-
-
Florida
-
Gainesville, Florida, Forenede Stater, 32607
- VitreoRetinal Associates, PA
-
-
Georgia
-
Atlanta, Georgia, Forenede Stater, 30322
- Emory University
-
-
Massachusetts
-
Boston, Massachusetts, Forenede Stater, 02114
- Massachusetts General Hospital - Center for Celiac Research and Treatment
-
-
Michigan
-
Ann Arbor, Michigan, Forenede Stater, 48105
- Univ of Michigan Medical Center
-
-
North Carolina
-
Durham, North Carolina, Forenede Stater, 27705
- Duke Eye Center
-
-
Pennsylvania
-
Pittsburgh, Pennsylvania, Forenede Stater, 15213
- University of Pittsburgh Medical Center (UPMC)
-
-
Texas
-
Bellaire, Texas, Forenede Stater, 77401
- Retina Consultants of Houston
-
-
-
-
-
Paris, Frankrig, 75012
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
-
-
-
-
-
Amsterdam, Holland, 1105AZ
- VUMC Amsterdam
-
Nijmegen, Holland, 6525EX
- Radboudumc
-
-
-
-
-
Jerusalem, Israel, 91120
- Hadassah Medical Center
-
-
-
-
-
Florence, Italien, 50134
- Azienda Ospedaliero Universitaria Careggi
-
Milan, Italien, 20142
- Ospedale San Paolo
-
Naples, Italien, 80131
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
-
Roma, Italien, 00198
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
-
-
-
-
-
Basel, Schweiz, 4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
-
Lausanne, Schweiz, 1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
-
-
-
-
-
Madrid, Spanien, 28040
- Hosp Univ Fund Jimenez Diaz
-
-
Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
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 undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Enkelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
|---|---|
|
Eksperimentel: Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navne:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Andet: Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navne:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Eksperimentel: Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Andre navne:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
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
|
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
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
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
|
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
|
|
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
|
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
|
|
Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Tidsramme: From Baseline to Week 52
|
Pointwise response in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
|
|
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
|
Pointwise response in worse-seeing eye in full visual field at Week 52 will be assessed. |
From Baseline to Week 52
|
|
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
|
Pointwise response in the central 30 degrees visual field at Week 52 will be assessed. |
From Baseline to Week 52
|
|
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
|
|
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
|
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
|
|
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
|
Change in functional vision by using VMA assessment in the "Worse-seeing Eye" at Week 52. |
From Baseline to Week 52
|
|
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Tidsramme: From Baseline to Week 52
|
Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52. |
From Baseline to Week 52
|
|
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
|
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
|
|
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
|
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
|
|
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
|
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
|
|
Number of Participants with Ocular and Non-ocular Adverse Events
Tidsramme: Day 1 - Week 52
|
Number of participants with ocular and non-ocular adverse events will be assessed. |
Day 1 - Week 52
|
|
Number of Participants With Abnormalities in Laboratory Assessments
Tidsramme: Day 1 - 52 Weeks
|
Number of participants with abnormalities in laboratory assessments will be assessed. |
Day 1 - 52 Weeks
|
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
- CR109258
- 2020-002873-88 (EudraCT nummer)
- MGT-RPGR-021 (Anden identifikator: MeiraGTx Ocular UK Ltd)
Plan for individuelle deltagerdata (IPD)
Planlægger du at dele individuelle deltagerdata (IPD)?
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Studerer et amerikansk FDA-reguleret enhedsprodukt
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .