- ICH GCP
- Registro degli studi clinici negli Stati Uniti
- Sperimentazione clinica 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
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Tipo di studio
Iscrizione (Effettivo)
Contatti e Sedi
Luoghi di studio
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Texas
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Dallas, Texas, Stati Uniti, 75231
- Texas Retina Associates,
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
- Bambino
- Adulto
- Adulto più anziano
Accetta volontari sani
Sessi ammissibili allo studio
Metodo di campionamento
Popolazione di studio
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
Coorti e interventi
Gruppo / Coorte |
Intervento / Trattamento |
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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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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Lasso di tempo |
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Progression of disease over time in adRP patients with misfolded rod opsin mutations using ellipsoid zone (EZ) area measurements
Lasso di tempo: Baseline to 4 years
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Baseline to 4 years
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Misure di risultato secondarie
Misura del risultato |
Lasso di tempo |
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Progression of disease over time in adRP patients with misfolded rod opsin mutations as measured by EZ width
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: 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
Lasso di tempo: 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)
Lasso di tempo: 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
Lasso di tempo: Baseline to 4 years
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Baseline to 4 years
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Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Shire Director, Takeda
Pubblicazioni e link utili
Collegamenti utili
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 (Stima)
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
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- SHP630-001
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Descrizione del piano IPD
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .
Prove cliniche su Observation
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Fondazione Don Carlo Gnocchi OnlusIstituto di Neuroscienze Consiglio Nazionale delle RicercheReclutamento
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IRCCS Eugenio MedeaMassachusetts Institute of Technology; Politecnico di MilanoReclutamentoDisturbo dello spettro autisticoItalia