Trials / Conditions / Leber Congenital Amaurosis

Leber Congenital Amaurosis

19 registered clinical trials studyying Leber Congenital Amaurosis — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) NCT06891443	Laboratoires Thea	Phase 3
Recruiting	Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR) NCT05906953	HuidaGene Therapeutics Co., Ltd.	Phase 1 / Phase 2
Active Not Recruiting	Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT) NCT06088992	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine	EARLY_Phase 1
Active Not Recruiting	Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis NCT05203939	Ocugen	Phase 1 / Phase 2
Unknown	An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerabil NCT04855045	ProQR Therapeutics	Phase 2 / Phase 3
Active Not Recruiting	Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Bia NCT03920007	Atsena Therapeutics Inc.	Phase 1 / Phase 2
Terminated	Extension Study to Study PQ-110-001 (NCT03140969) NCT03913130	Laboratoires Thea	Phase 1 / Phase 2
Active Not Recruiting	A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in NCT03913143	ProQR Therapeutics	Phase 2 / Phase 3
Completed	Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) NCT02781480	MeiraGTx UK II Ltd	Phase 1 / Phase 2
Completed	Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 NCT02714816	MeiraGTx UK II Ltd	—
Recruiting	Inherited Retinal Degenerative Disease Registry NCT02435940	Foundation Fighting Blindness	—
Active Not Recruiting	Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis NCT00999609	Spark Therapeutics, Inc.	Phase 3
Completed	Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65 NCT01496040	Nantes University Hospital	Phase 1 / Phase 2
Active Not Recruiting	Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2 NCT01208389	Spark Therapeutics, Inc.	Phase 1 / Phase 2
Completed	Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families. NCT02970266	Assistance Publique - Hôpitaux de Paris	—
Recruiting	Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford NCT01793168	Sanford Health	—
Completed	Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis NCT00749957	Beacon Therapeutics	Phase 1 / Phase 2
Completed	Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations NCT00821340	Hadassah Medical Organization	Phase 1
Completed	Safety Study in Subjects With Leber Congenital Amaurosis NCT00516477	Spark Therapeutics, Inc.	Phase 1