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Showing 1–20 of 20 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: LCA (Leber Congenital Amaurosis) Clear all
Completed No phase listed Observational

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

NCT02575430
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Interventions
No treatment: retrospective chart review
Other
Lead sponsor
QLT Inc.
Industry
Eligibility
8 Years and older
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Baltimore, Maryland • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Apr 28, 2016 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1 Interventional

Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)

NCT01521793
View directory record Official record
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
2
States / cities
Chicago, Illinois • Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 27, 2014 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA)

NCT02781480
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
AAV RPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Jul 11, 2021 · Synced Jul 21, 2026, 2:36 PM EDT
Completed No phase listed Observational

Natural History Study of CEP290-Related Retinal Degeneration

NCT03396042
View directory record Official record
Conditions
Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration
Interventions
Not listed
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years to 99 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
4
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated May 18, 2022 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Phase 1/2 Safety and Efficacy Study of AAV-RPE65 Vector to Treat Leber Congenital Amaurosis

NCT00749957
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
rAAV2-CB-hRPE65
Biological
Lead sponsor
Beacon Therapeutics
Industry
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2017
U.S. locations
2
States / cities
Worcester, Massachusetts • Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 27, 2017 · Synced Jul 21, 2026, 2:36 PM EDT
Completed No phase listed Observational

A Patient Registry Study for Patients Treated With Voretigene Neparvovec in US

NCT03597399
View directory record Official record
Conditions
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
12 Months and older
Enrollment
87 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
10
States / cities
Los Angeles, California • Miami, Florida • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2025 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

NCT03140969
View directory record Official record
Conditions
Leber's Congenital Amaurosis
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 14, 2024 · Synced Jul 21, 2026, 2:36 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

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

NCT07681778
View directory record Official record
Conditions
Leber Congenital Amaurosis, Leber Congenital Amaurosis (LCA)
Interventions
OPGx-RDH12
Drug
Lead sponsor
Opus Genetics, Inc
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2034
U.S. locations
3
States / cities
Phoenix, Arizona • Philadelphia, Pennsylvania • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 14, 2026 · Synced Jul 21, 2026, 2:36 PM EDT
Recruiting Phase 1Phase 2 Interventional

Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

NCT05906953
View directory record Official record
Conditions
Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations
Interventions
HG004
Drug
Lead sponsor
HuidaGene Therapeutics Co., Ltd.
Industry
Eligibility
6 Years to 50 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
2
States / cities
Sacramento, California • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 18, 2024 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1 Interventional

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

NCT01014052
View directory record Official record
Conditions
LCA (Leber Congenital Amaurosis), RP (Retinitis Pigmentosa)
Interventions
QLT091001
Drug
Lead sponsor
QLT Inc.
Industry
Eligibility
5 Years to 65 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
3
States / cities
Chicago, Illinois • Baltimore, Maryland • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 13, 2013 · Synced Jul 21, 2026, 2:36 PM EDT
Completed No phase listed Observational

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

NCT02946879
View directory record Official record
Conditions
Leber Congenital Amaurosis (LCA), Eye Diseases, Eye Diseases, Hereditary, Retinal Diseases
Interventions
AAV OPTIRPE65
Biological
Lead sponsor
MeiraGTx UK II Ltd
Industry
Eligibility
3 Years to 100 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Jul 21, 2026, 2:36 PM EDT
Recruiting Phase 3 Interventional

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

NCT06891443
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Placebo IVT
Drug · Other
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
San Francisco, California • Miami, Florida • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 21, 2026, 2:36 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

NCT00999609
View directory record Official record
Conditions
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Interventions
AAV2-hRPE65v2,voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
3 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2030
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 22, 2025 · Synced Jul 21, 2026, 2:36 PM EDT
Not listed Phase 1Phase 2 Interventional

Single Ascending Dose Study in Participants With LCA10

NCT03872479
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease, Retinal Degeneration, Vision Disorders, Eye Disorders Congenital
Interventions
EDIT-101
Drug
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
3 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
5
States / cities
Miami, Florida • Boston, Massachusetts • Ann Arbor, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 4, 2022 · Synced Jul 21, 2026, 2:36 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Extension Study to Study PQ-110-001 (NCT03140969)

NCT03913130
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 14, 2024 · Synced Jul 21, 2026, 2:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Phase 1 Follow-on Study of AAV2-hRPE65v2 Vector in Subjects With Leber Congenital Amaurosis (LCA) 2

NCT01208389
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
8 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Jul 21, 2026, 2:36 PM EDT
Active, not recruiting Phase 2Phase 3 Interventional

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

NCT03913143
View directory record Official record
Conditions
Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
Interventions
sepofarsen, Sham
Drug · Other
Lead sponsor
ProQR Therapeutics
Industry
Eligibility
8 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 16, 2022 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1 Interventional

Safety Study in Subjects With Leber Congenital Amaurosis

NCT00516477
View directory record Official record
Conditions
Leber Congenital Amaurosis
Interventions
voretigene neparvovec-rzyl
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
8 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 1, 2020 · Synced Jul 21, 2026, 2:36 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

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

NCT03920007
View directory record Official record
Conditions
Leber Congenital Amaurosis, LCA, LCA1
Interventions
ATSN-101, ATSN-101 Diluent Solution, Prednisone, Triamcinalone Acetonide, 1% Prednisolone, Trimethoprim/polymyxin B
Drug
Lead sponsor
Atsena Therapeutics Inc.
Industry
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
2
States / cities
Portland, Oregon • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 19, 2024 · Synced Jul 21, 2026, 2:36 PM EDT
Completed Phase 1 Interventional

Phase I Trial of Gene Vector to Patients With Retinal Disease Due to RPE65 Mutations

NCT00481546
View directory record Official record
Conditions
Amaurosis of Leber, Retinal Diseases
Interventions
rAAV2-CBSB-hRPE65
Genetic
Lead sponsor
University of Pennsylvania
Other
Eligibility
8 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
2
States / cities
Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 16, 2026 · Synced Jul 21, 2026, 2:36 PM EDT