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- Ensaio Clínico NCT02926092
Observational Natural History Study of Autosomal Dominant Retinitis Pigmentosa (adRP)
A Prospective, Multicenter, Longitudinal, Observational Natural History Study to Evaluate Disease Progression in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) With Misfolded Rod Opsin Mutations
Visão geral do estudo
Status
Condições
Intervenção / Tratamento
Tipo de estudo
Inscrição (Real)
Contactos e Locais
Locais de estudo
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Texas
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Dallas, Texas, Estados Unidos, 75231
- Texas Retina Associates,
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Método de amostragem
População do estudo
Descrição
Inclusion Criteria:
- The subject has 1 documented pre-specified heterozygous rhodopsin gene (RHO) mutation confirmed by genetic testing (mutations will include P23H, T17M, and R135W).
The subject has at least 1 eye that meets all 3 of the following criteria:
- A measurable EZ area as determined by an evaluation of EZ limits on sdOCT scan, with a horizontal EZ width of greater than 3 mm
- BCVA of greater than or equal to 35 letters as measured by the Early Treatment Diabetic Retinopathy Study (ETDRS; equivalent to 20/200 on a Snellen chart).
- A kinetic VF of greater than 10 degrees diameter in the horizontal meridian with a spot size of III
- The subject has the ability to comply with the clinical protocol, in the opinion of the investigator.
- The subject has a clear ocular media and adequate pupillary dilation in both eyes to permit adequate visual assessments in the opinion of the investigator.
- The subject has agreed to abstain from any protocol-prohibited medication(s) during study participation.
- The subject is medically stable in the opinion of the investigator and able to fulfill the protocol requirements, including the ability to complete the assessments, without placing an undue burden on the subject/subject's family.
- The subject and/or subject's parent(s) or legally authorized guardian(s) has voluntarily signed an Institutional Review Board (IRB)/ ethics committee (EC)-approved informed consent and assent form(s), as applicable, after all relevant aspects of the study have been explained and discussed with the subject and/or the subject's parent(s) or legally authorized guardian(s).
- The subject, subject's parent(s), or legally authorized guardian(s) is able to understand the nature, scope, and possible consequences of the study and agrees to comply with the protocol-defined, scheduled assessments.
Exclusion Criteria:
- The subject is participating in an interventional clinical trial or has participated in an interventional clinical trial within 90 days of screening; participation in non-interventional observational studies is permitted.
- The subject has received treatment or has been in the treatment arm of a clinical trial for gene therapy, stem cell therapy, retinal progenitor cell therapy, tissue transplantation, device or drug delivery implantation, or other similar invasive therapy.
The subject has any of the following medical conditions that will interfere with consistent follow-up over any part of the study:
- Stroke
- Severe or unstable coronary disease
- End-stage or aggressive malignancy
- General poor health or uncontrolled or severe disease (eg, cardiovascular, neurological, psychological, pulmonary,renal, hepatic, endocrine, or gastrointestinal disorders) that in the opinion of the investigator would interfere with participation in the study
The subject has any of the following ocular conditions that could interfere with or confound follow-up of disease progression:
- Glaucoma
- Diabetic retinopathy
- Choroidal neovascularization
- Retinal inflammatory disease
- Cataract worse than grade 2 (nuclear, posterior subcapsular [PSC], or cortical)
- High myopia (≥8 diopters)
- Herpes simplex virus of the eye
- Acute infection or inflammation
- Any ocular condition that in the opinion of the investigator would interfere with the ability to assess retinal morphology and functionality
- The subject has had intraocular surgery within 90 days prior to screening.
The subject currently requires the following protocol prohibited medications or has ingested such medication within 30 days of screening:
Plaquenil Thioridazine Clofazimine Deferoxamine Phenothiazine Chlorpromazine Cisplatin Valproic acid Any other drugs with known visual side effects
- The subject has 3 first- or second-degree family members already enrolled in the study.
Plano de estudo
Como o estudo é projetado?
Detalhes do projeto
Coortes e Intervenções
Grupo / Coorte |
Intervenção / Tratamento |
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Arm 1
Observation of progression of disease over time.
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Observation of progression of disease over time
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O que o estudo está medindo?
Medidas de resultados primários
Medida de resultado |
Prazo |
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Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements
Prazo: Baseline to 4 years
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Baseline to 4 years
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Medidas de resultados secundários
Medida de resultado |
Prazo |
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by EZ width
Prazo: Baseline to 4 years
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Baseline to 4 years
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by visual fields (kinetic and static)
Prazo: Baseline to 4 years
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Baseline to 4 years
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by dark-adapted rod visual fields
Prazo: Baseline to 4 years
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Baseline to 4 years
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by electroretinography (ERG): dark- and light-adapted
Prazo: Baseline to 4 years
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Baseline to 4 years
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by best corrected visual acuity (BCVA)
Prazo: Baseline to 4 years
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Baseline to 4 years
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Vision-related function and quality of life as measured by 25-item National Eye Institute Visual Function Questionnaire (NEI VFQ-25) plus its additional items
Prazo: Baseline to 4 years
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Baseline to 4 years
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Diretor de estudo: Shire Director, Takeda
Publicações e links úteis
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 (Estimativa)
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
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- SHP630-001
Plano para dados de participantes individuais (IPD)
Planeja compartilhar dados de participantes individuais (IPD)?
Descrição do plano IPD
Essas informações foram obtidas diretamente do site clinicaltrials.gov sem nenhuma alteração. Se você tiver alguma solicitação para alterar, remover ou atualizar os detalhes do seu estudo, entre em contato com register@clinicaltrials.gov. Assim que uma alteração for implementada em clinicaltrials.gov, ela também será atualizada automaticamente em nosso site .
Ensaios clínicos em Observation
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IRCCS Eugenio MedeaMassachusetts Institute of Technology; Politecnico di MilanoRecrutamento