Clinical Trials on LCA1

Total 10 results

Atsena Therapeutics Inc.

Active, not recruiting

Study of Subretinally Injected ATSN-101 Administered in Patients With Leber Congenital Amaurosis Caused by Biallelic Mutations in GUCY2D

Leber Congenital Amaurosis | LCA | LCA1

United States
Opus Genetics, Inc

Recruiting

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD)

LCA5

United States
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

Familial LCAT Deficiency

United States
University of Pennsylvania

Recruiting

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study (LCAT NHS)

LCAT Deficiency

United States
Shanghai General Hospital, Shanghai Jiao Tong University...

Not yet recruiting

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
University of Pennsylvania

Suspended

Mendelian Reverse Cholesterol Transport Study

Lipid Metabolism, Inborn Errors | Cholesterol, HDL | Tangier Disease | LCAT Deficiency | Cholesteryl Ester Transfer Protein (CETP) Deficiency

United States
QLT Inc.

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)

United States, Canada, Germany, Netherlands, United Kingdom
QLT Inc.

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)

Canada, United States, Germany, Netherlands, United Kingdom
QLT Inc.

Completed

Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT

Leber Congenital Amaurosis (LCA) | Retinitis Pigmentosa (RP)

United States, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
MeiraGTx UK II Ltd
Syne Qua Non Limited

Completed

Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65)

Eye Diseases | Retinal Diseases | Eye Diseases, Hereditary | Leber Congenital Amaurosis (LCA)

United States, United Kingdom

Clinical Trials on LCA1

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