Ensaio de Terapia Gênica para o Tratamento da Retinite Pigmentosa Ligada ao X Associada a Variantes no Gene RPGR
Estudo randomizado e controlado de fase 3 de AAV5-RPGR para o tratamento de retinite pigmentosa ligada ao cromossomo X associada a variantes no gene RPGR
A clinical trial of AAV5-RPGR vector for participants with X-linked retinitis pigmentosa (XLRP)
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Real)
Estágio
- Fase 3
Contactos e Locais
Locais de estudo
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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, Espanha, 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, França, 75012
- Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts
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Amsterdam, Holanda, 1105AZ
- VUMC Amsterdam
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Nijmegen, Holanda, 6525EX
- Radboudumc
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Jerusalem, Israel, 91120
- Hadassah Medical Center
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Florence, Itália, 50134
- Azienda Ospedaliero Universitaria Careggi
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Milan, Itália, 20142
- Ospedale San Paolo
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Naples, Itália, 80131
- Azienda Ospedaliera Univ.- Università Degli studi della Campania - Luigi Vanvitelli
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Roma, Itália, 00198
- IRCCS Fondazione G.B. Bietti per lo Studio e la Ricerca in Oftalmologia ONLUS
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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, Suíça, 4031
- University Hospital Basel, Eye Clinic/Institute of Molecular and Clinical
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Lausanne, Suíça, 1004
- Universite de Lausanne, Hopital ophtalmique Jules-Gonin
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
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
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
- Finalidade Principal: Tratamento
- Alocação: Randomizado
- Modelo Intervencional: Atribuição Paralela
- Mascaramento: Solteiro
Armas e Intervenções
Grupo de Participantes / Braço |
Intervenção / Tratamento |
|---|---|
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Experimental: Experimental - Immediate Treatment
Intermediate dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Outros nomes:
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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Outro: Deferred Treatment
Deferred Treatment |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Outros nomes:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
|
Experimental: Experimental Immediate Treatment
Low dose. |
Bilateral, sub-retinal administration of AAV5-hRKp.RPGR - immediate treatment group
Outros nomes:
No intervention - deferred treatment group (Bilateral, sub-retinal administration of AAV5-hRKp.RPGR to be administered in the follow-up study) |
O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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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
Prazo: 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 resultados secundários
Medida de resultado |
Descrição da medida |
Prazo |
|---|---|---|
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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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
Prazo: 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
- Diretor de estudo: Study Director, MeiraGTx Ocular UK Ltd
Datas de registro do estudo
Datas Principais do Estudo
Início do estudo (Real)
Conclusão Primária (Real)
Conclusão do estudo (Real)
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Outros números de identificação do estudo
- CR109258
- 2020-002873-88 (Número EudraCT)
- MGT-RPGR-021 (Outro identificador: MeiraGTx Ocular UK Ltd)
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
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