LCA1 | Clinical Trials Search

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110 results for “LCA1”

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Active, not recruiting

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

15 participants · First posted Apr 18, 2019

Active, not recruiting

Single Ascending Dose Study in Participants With LCA10

34 participants · First posted Mar 13, 2019

Active, not recruiting

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

36 participants · First posted Apr 12, 2019

An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

15 participants · First posted Apr 22, 2021

Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients

22 participants · First posted Nov 15, 2022

Not yet recruiting

Safety and Tolerability of Subretinal OPGx-RDH12-1001 for LCA-Associated Inherited Retinal Degeneration (LCA-IRD)

10 participants · First posted Jul 2, 2026

Not yet recruiting

LCAR-M61S and LCAR-M61D in Treatment of Relapsed/Refractory Multiple Myeloma

66 participants · First posted Jun 25, 2024

LCAT (Lecithin Cholesterol Acyl Transferase) Natural History Study

40 participants · First posted Jan 22, 2024

Intravenous ACP-501 for Familial LCAT Deficiency (rhLCAT)

1 participants · First posted Feb 4, 2021

Not yet recruiting

Cerebellar DBS in SCA1 and SCA3 Study

12 participants · First posted Aug 7, 2026

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