Ensayo de terapia génica para el tratamiento de la retinosis pigmentaria ligada al cromosoma X asociada a variantes en el gen RPGR
Estudio aleatorizado y controlado de fase 3 de AAV5-RPGR para el tratamiento de la retinosis pigmentaria ligada al cromosoma X asociada con variantes en el gen RPGR
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 3
Contactos y Ubicaciones
Ubicaciones de estudio
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Ghent, Bélgica, 9000
- UZ Gent
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Ontario
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Toronto, Ontario, Canadá, M5G 1X8
- Hospital For Sick Children
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Glostrup Municipality, Dinamarca, 2600
- Rigshospitalet Glostrup
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Madrid, España, 28040
- Hosp Univ Fund Jimenez Diaz
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California
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La Jolla, California, Estados Unidos, 92093 0946
- Shiley Eye Institute Jacobs Retina Center
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Los Angeles, California, Estados Unidos, 90027
- Childrens Hospital
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Palo Alto, California, Estados Unidos, 94303
- Stanford Health Care
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Florida
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Gainesville, Florida, Estados Unidos, 32607
- VitreoRetinal Associates, PA
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Georgia
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Atlanta, Georgia, Estados Unidos, 30322
- Emory University
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02114
- Massachusetts General Hospital - Center for Celiac Research and Treatment
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48105
- Univ of Michigan Medical Center
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North Carolina
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Durham, North Carolina, Estados Unidos, 27705
- Duke Eye Center
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Pennsylvania
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Pittsburgh, Pennsylvania, Estados Unidos, 15213
- University of Pittsburgh Medical Center (UPMC)
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Texas
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Bellaire, Texas, Estados Unidos, 77401
- Retina Consultants of Houston
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Paris, Francia, 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, Países Bajos, 1105AZ
- VUMC Amsterdam
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Nijmegen, Países Bajos, 6525EX
- Radboudumc
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Edinburgh, Reino Unido, EH3 9HA
- NHS Lothian
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Glasgow, Reino Unido, G12 0YN
- Gartnavel General Hospital
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Leeds, Reino Unido, LS9 7TF
- St James University Hospital
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London, Reino Unido, EC1V 2PD
- Moorfields Eye Hospital
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Basel, Suiza, 4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
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Lausanne, Suiza, 1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: Aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Único
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Experimental: Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Otros nombres:
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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Otro: Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Otros nombres:
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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Experimental: Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Otros nombres:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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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
Periodo de tiempo: 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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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: From Baseline to Week 52
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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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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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Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Study Director, MeiraGTx Ocular UK Ltd
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Otros números de identificación del estudio
- CR109258
- 2020-002873-88 (Número EudraCT)
- MGT-RPGR-021 (Otro identificador: MeiraGTx Ocular UK Ltd)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .