Sperimentazione di terapia genica per il trattamento della retinite pigmentosa legata all'X associata a varianti del gene RPGR
Studio di fase 3 randomizzato e controllato di AAV5-RPGR per il trattamento della retinite pigmentosa legata all'X associata a varianti nel gene RPGR
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 3
Contatti e Sedi
Luoghi di studio
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Ghent, Belgio, 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, Danimarca, 2600
- Rigshospitalet Glostrup
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Paris, Francia, 75012
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
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Jerusalem, Israele, 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, Olanda, 1105AZ
- VUMC Amsterdam
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Nijmegen, Olanda, 6525EX
- Radboudumc
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Edinburgh, Regno Unito, EH3 9HA
- NHS Lothian
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Glasgow, Regno Unito, G12 0YN
- Gartnavel General Hospital
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Leeds, Regno Unito, LS9 7TF
- St James University Hospital
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London, Regno Unito, EC1V 2PD
- Moorfields Eye Hospital
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Madrid, Spagna, 28040
- Hosp Univ Fund Jimenez Diaz
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California
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La Jolla, California, Stati Uniti, 92093 0946
- Shiley Eye Institute Jacobs Retina Center
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Los Angeles, California, Stati Uniti, 90027
- Childrens Hospital
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Palo Alto, California, Stati Uniti, 94303
- Stanford Health Care
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Florida
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Gainesville, Florida, Stati Uniti, 32607
- VitreoRetinal Associates, PA
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Georgia
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Atlanta, Georgia, Stati Uniti, 30322
- Emory University
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Massachusetts
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Boston, Massachusetts, Stati Uniti, 02114
- Massachusetts General Hospital - Center for Celiac Research and Treatment
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Michigan
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Ann Arbor, Michigan, Stati Uniti, 48105
- Univ of Michigan Medical Center
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North Carolina
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Durham, North Carolina, Stati Uniti, 27705
- Duke Eye Center
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Pennsylvania
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Pittsburgh, Pennsylvania, Stati Uniti, 15213
- University of Pittsburgh Medical Center (UPMC)
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Texas
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Bellaire, Texas, Stati Uniti, 77401
- Retina Consultants of Houston
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Basel, Svizzera, 4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
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Lausanne, Svizzera, 1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Randomizzato
- Modello interventistico: Assegnazione parallela
- Mascheramento: Separare
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Altri nomi:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Altro: Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Altri nomi:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Sperimentale: Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Altri nomi:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
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
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
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
Lasso di tempo: 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
|
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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
Lasso di tempo: 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
|
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Change in Retinal Function as Assessed by Pointwise Response in Full Visual Field at Week 52
Lasso di tempo: From Baseline to Week 52
|
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
Lasso di tempo: 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
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Change in Retinal Function as Assessed by Pointwise Response in the Central 30 Degrees Visual Field at Week 52
Lasso di tempo: 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
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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
Lasso di tempo: 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
Lasso di tempo: 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
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Change in Functional Vision by Using Vision-guided Mobility Assessment (VMA) Response in the "Worse-seeing Eye" at Week 52
Lasso di tempo: 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
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Change From Baseline in the Modified Low Luminance Questionnaire (mLLQ) Extreme Lighting Domain score at Week 52
Lasso di tempo: 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
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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
Lasso di tempo: 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
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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
Lasso di tempo: 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
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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
Lasso di tempo: 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
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Number of Participants with Ocular and Non-ocular Adverse Events
Lasso di tempo: 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
Lasso di tempo: Day 1 - 52 Weeks
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Number of participants with abnormalities in laboratory assessments will be assessed. |
Day 1 - 52 Weeks
|
Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Study Director, MeiraGTx Ocular UK Ltd
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Altri numeri di identificazione dello studio
- CR109258
- 2020-002873-88 (Numero EudraCT)
- MGT-RPGR-021 (Altro identificatore: MeiraGTx Ocular UK Ltd)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Informazioni su farmaci e dispositivi, documenti di studio
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