Listado de ensayos


A Safety Study of Retinal Gene Therapy for COROIDEREMIA With Administration of BIIB111

Estado: | Categoría: | Resultado:

Patología

COROIDEREMIA

Tratamiento

Drug: BIIB111

Localizaciones

Research Site, Miami, Florida, United States Research Site, Boston, Massachusetts, United States Research Site, Cincinnati, Ohio, United States Research Site, Portland, Oregon, United States Research Site, Paris, France Research Site, T bingen, Germany
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REP1 Gene Replacement Therapy for COROIDEREMIA

Estado: | Categoría: | Resultado:

Patología

COROIDEREMIA

Tratamiento

Genetic: AAV-mediated REP1 gene replacement

Localizaciones

Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom
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Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (COROIDEREMIA) Gene Mutations

Estado: | Categoría: | Resultado:

Patología

COROIDEREMIA (COROIDEREMIA) mutacion en el gen CHM

Tratamiento

Biological: AAV2-hCHM

Localizaciones

Massachusetts Eye and Ear Infirmary, Boston, Massachusetts, United States University of Pennsylvania, Philadelphia, Pennsylvania, United States Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
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Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)

Estado: | Categoría: | Resultado:

Patología

RETINOSIS PIGMENTARIA LIGADA A X

Tratamiento

Genetic: AAV2/5-RPGR

Localizaciones

Massachusetts Eye and Ear Institute, Boston, Massachusetts, United States Kellogg Eye Center, Ann Arbor, Michigan, United States UPMC Eye Center, Pittsburgh, Pennsylvania, United States Leeds Teaching Hospitals NHS Trust, Leeds, United Kingdom Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom
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Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt’s Macular Degeneration

Estado: | Categoría: | Resultado:

Patología

ENFERMEDAD DE STARGARDT

Tratamiento

MEDICAMENTO: Long term follow up in all patients who received SAR422459 in previous study TDU13583

Localizaciones

Investigational Site Number 840001, Portland, Oregon, United States Investigational Site Number 250001, Paris, France
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Safety and Efficacy of Emixustat in Stargardt Disease

Estado: | Categoría: | Resultado:

Patología

ENFERMEDAD DE STARGARDT

Tratamiento

MEDICAMENTO: Emixustat MEDICAMENTO: Placebo

Localizaciones

Retina-Vitreous Associates Medical Group, Beverly Hills, California, United States UCSF Dept. of Ophthalmology, San Francisco, California, United States Emory University, Atlanta, Georgia, United States The Wilmer Eye Institute Johns Hopkins University, Baltimore, Maryland, United States University of Michigan Kellogg Eye Center, Ann Arbor, Michigan, United States Mayo Clinic Rochester, Rochester, Minnesota, United States Duke Eye Center, Durham, North Carolina, United States Casey Eye Institute - OHSU, Portland, Oregon, United States Retina Foundation of the Southwest, Dallas, Texas, United States University of Utah John Moran Eye Center, Salt Lake City, Utah, United States and 19 more
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Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)

Estado: | Categoría: | Resultado:

Patología

Ciliopathies Nephronophthisis Senior-Loken Syndrome Joubert Syndrome Jeune Syndrome Bardet-Biedl Syndrome

Tratamiento

Other: Blood sample Other: Urine sample

Localizaciones

H pital Necker-Enfants Malades, Paris, France
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Auditory Neural Function in Implanted Patients With Usher Syndrome

Estado: | Categoría: | Resultado:

Patología

Usher Syndrome Cochlear Implantation

Tratamiento

Other: Experimental manipulation of stimulation parameters

Localizaciones

Boston children's Hospital, Boston, Massachusetts, United States Gina Hounam, Columbus, Ohio, United States The Ohio State University, Columbus, Ohio, United States Vanderbilt University Medical Center, Nashville, Tennessee, United States
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