Status | Study |
NOT_YET_RECRUITING |
Study Name: A Safety and Efficacy Study of HG004 in Subjects With Leber Congenital Amaurosis Condition: Date: 2023-05-09 Interventions: Low dose Medium dose High dose |
RECRUITING |
Study Name: Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) Condition: Leber Congenital Amaurosis Date: 2023-05-09 Interventions: Low dose Medium dose High dose |
Not yet recruiting |
Study Name: 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 Condition: Leber's Congenital Amaurosis Date: 2017-05-01 Interventions: Drug: QR-110 RNA antisense oligonucleotide for intravitreal injection |
Completed |
Study Name: Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. Condition: Leber Congenital Amaurosis Date: 2016-11-17 |
Recruiting |
Study Name: Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) Condition: Leber Congenital Amaurosis (LCA) Eye Diseases Date: 2016-07-22 Interventions: Genetic: AAV2/5-OPTIRPE65 AAV2/5-OPTIRPE65 intraocular administration. |
Recruiting |
Study Name: Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) Condition: Leber Congenital Amaurosis Date: 2016-04-28 Interventions: Genetic: AAV2/5 OPTIRPE65 |
Recruiting |
Study Name: Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 Condition: Leber Congenital Amaurosis Date: 2016-03-08 |
Completed |
Study Name: Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT Condition: Leber Congenital Amaurosis (LCA) Retinitis Pigmentosa (RP) Date: 2015-10-09 Interventions: Other: No treatment: retrospective chart review |
Recruiting |
Study Name: Inherited Retinal Degenerative Disease Registry Condition: Eye Diseases Hereditary Retinal Disease Achromatopsia Date: 2015-04-03 |
Completed |
Study Name: Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01) Condition: LCA (Leber Congenital Amaurosis) RP (Retinitis Pigmentosa) Date: 2012-01-06 Interventions: Drug: QLT091001 oral QLT091001 administered once daily for 7 days |