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Leber Congenital Amaurosis - 16 Studies Found

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Completed Nombre del estudio: Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Mutations
Condición:
  • LCA (Leber Congenital Amaurosis)
  • RP (Retinitis Pigmentosa)

    • Fecha: 2009-11-12
    Intervenciones: Drug: QLT091001 oral QLT091001 administered once daily for 7 days
Completed Nombre del estudio: Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
Condición: Leber Congenital Amaurosis
Fecha: 2011-11-24
Intervenciones: Drug: rAAV2/4.hRPE65 One injection in on eye
Completed Nombre del estudio: Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
Condición:
  • Leber Congenital Amaurosis (LCA)
  • Retinitis Pigmentosa (RP)

    • Fecha: 2015-10-09
    Intervenciones: Other: No treatment: retrospective chart review
Active, not recruiting Nombre del estudio: Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations
Condición:
  • Amaurosis of Leber
  • Retinal Diseases

Fecha: 2007-05-31
Intervenciones: Genetic: rAAV2-CBSB-hRPE65 One or two, uniocular, subretinal injections; relative doses: 0.3X (Cohort 1)
NOT_YET_RECRUITING Nombre del estudio: A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis
Condición:
Fecha: 2025-11-04
Intervenciones: Low dose Medium dose High dose
Completed Nombre del estudio: Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
Condición: Leber Congenital Amaurosis
Fecha: 2025-11-04
Intervenciones: Low dose Medium dose High dose
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