Leber Congenital Amaurosis - 16 Studies Found

Active, not recruiting : Safety Study in Subjects With Leber Congenital Amaurosis
: Leber Congenital Amaurosis
: 2007-08-13
: Biological: AAV2-hRPE65v2 Subjects will be dosed unilaterally (one eye) beginning with the lowest dose.
Completed : Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.
: Leber Congenital Amaurosis
: 2016-11-17
Active, not recruiting : Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis
: Leber Congenital Amaurosis
: 2008-09-08
: Biological: rAAV2-CB-hRPE65 Recombinant adeno-associated virus vector expressing RPE65
Not yet recruiting : Study to Evaluate QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene
: Leber's Congenital Amaurosis
: 2017-05-01
: Drug: QR-110 RNA antisense oligonucleotide for intravitreal injection
Completed : Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65
: Leber Congenital Amaurosis
: 2016-03-08
Active, not recruiting : Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2
: Leber Congenital Amaurosis
: 2010-09-22
: Biological: AAV2-hRPE65v2 One time, subretinal administration of 1.5E11 vg AAV2-hRPE65v2 vector to the c
Active, not recruiting : Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
:
  • Inherited Retinal Dystrophy Due to RPE65 Mutations
  • Leber Congenital Am
    : 2009-10-21
    : Biological: AAV2-hRPE65v2 Subretinal administration of gene therapy vector AAV2-hRPE65v2 (1.5E11 vector
Recruiting : Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)
: Leber Congenital Amaurosis
: 2016-04-28
: Genetic: AAV2/5 OPTIRPE65
Completed : Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations
: Leber Congenital Amaurosis
: 2009-01-12
: Genetic: rAAV2-hRPE65 Uniocular subretinal injections; relative doses: Cohort 1 - basic (lowest) viral d
Completed : Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)
:
  • LCA (Leber Congenital Amaurosis)
  • RP (Retinitis Pigmentosa)

    • : 2012-01-06
    : Drug: QLT091001 oral QLT091001 administered once daily for 7 days
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