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Results for “Inherited Retinal Dystrophy Due to RPE65 Mutations”

4 trials

Showing 4 of 4 results

Large-scale testing (Phase 3)Active Not RecruitingNCT00999609
What this trial is testing

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Inherited Retinal Dystrophy Due to RPE65 MutationsLeber Congenital Amaurosis
Spark Therapeutics, Inc. 31
Early research (Phase 1)Study completedNCT01014052
What this trial is testing

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

Who this might be right for
LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
QLT Inc. 32
Not applicableStudy completedNCT04525261
What this trial is testing

Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene

Who this might be right for
Leber Congenital Amaurosis 2Retinitis Pigmentosa 20
University of Campania Luigi Vanvitelli 60
Not applicableActive Not RecruitingNCT03602820
What this trial is testing

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Who this might be right for
Inherited Retinal Dystrophy Due to RPE65 Mutations
Spark Therapeutics, Inc. 41

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