Leber Congenital Amaurosis - 16 Studies Found

Completed : 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
:
  • LCA (Leber Congenital Amaurosis)
  • RP (Retinitis Pigmentosa)

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

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

: 2007-05-31
: Genetic: rAAV2-CBSB-hRPE65 One or two, uniocular, subretinal injections; relative doses: 0.3X (Cohort 1)
NOT_YET_RECRUITING : A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis
:
: 2025-08-02
: Low dose Medium dose High dose
Completed : Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
: Leber Congenital Amaurosis
: 2025-08-02
: Low dose Medium dose High dose
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