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Results for “Leber Congenital Amaurosis (LCA)”

24 trials

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Showing 20 of 24 results

Early research (Phase 1)Study completedNCT01521793
What this trial is testing

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

Who this might be right for
LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
QLT Inc. 27
Not applicableStudy completedNCT03597399
What this trial is testing

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

Who this might be right for
Confirmed Biallelic RPE65 Mutation-associated Retinal Dystrophy
Spark Therapeutics, Inc. 87
Large-scale testing (Phase 3)Active Not RecruitingNCT00999609
What this trial is testing

Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Inherited Retinal Dystrophy Due to RPE65 MutationsLeber Congenital Amaurosis
Spark Therapeutics, Inc. 31
Not applicableStudy completedNCT02575430
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis (LCA)Retinitis Pigmentosa (RP)
QLT Inc. 59
Large-scale testing (Phase 3)Looking for participantsNCT06891443
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
Laboratoires Thea 32
Early research (Phase 1)Study completedNCT00516477
What this trial is testing

Safety Study in Subjects With Leber Congenital Amaurosis

Who this might be right for
Leber Congenital Amaurosis
Spark Therapeutics, Inc. 12
Testing effectiveness (Phase 2)UnknownNCT03872479
What this trial is testing

Single Ascending Dose Study in Participants With LCA10

Who this might be right for
Leber Congenital Amaurosis 10Inherited Retinal DystrophiesEye Diseases, Hereditary+4 more
Editas Medicine, Inc. 34
Testing effectiveness (Phase 2)Study completedNCT00749957
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis
Beacon Therapeutics 12
Not applicableStudy completedNCT03396042
What this trial is testing

Natural History Study of CEP290-Related Retinal Degeneration

Who this might be right for
BlindnessLeber Congenital Amaurosis 10Vision Disorders+5 more
Editas Medicine, Inc. 26
Early research (Phase 1)Study completedNCT00821340
What this trial is testing

Clinical Trial of Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations

Who this might be right for
Leber Congenital Amaurosis
Hadassah Medical Organization 3
Testing effectiveness (Phase 2)Looking for participantsNCT05906953
What this trial is testing

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

Who this might be right for
Leber Congenital AmaurosisInherited Retinal Diseases Caused by RPE65 Mutations
HuidaGene Therapeutics Co., Ltd. 20
Large-scale testing (Phase 3)Active Not RecruitingNCT03913143
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
ProQR Therapeutics 36
Very early researchUnknownNCT03566147
What this trial is testing

Treatment of RP and LCA by Primary RPE Transplantation

Who this might be right for
Leber Congenital Amaurosis, Retinitis Pigmentosa
Eyecure Therapeutics Inc. 30
Testing effectiveness (Phase 2)Study completedNCT02781480
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis
MeiraGTx UK II Ltd 15
Not applicableStudy completedNCT02970266
What this trial is testing

Genetic Decryption of Leber Congenital Amaurosis (LCA) in a Large Cohort of Independent Families.

Who this might be right for
Leber Congenital Amaurosis
Assistance Publique - Hôpitaux de Paris 659
Testing effectiveness (Phase 2)Study completedNCT03140969
What this trial is testing

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

Who this might be right for
Leber's Congenital Amaurosis
Laboratoires Thea 11
Testing effectiveness (Phase 2)Active Not RecruitingNCT03920007
What this trial is testing

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

Who this might be right for
Leber Congenital AmaurosisLCALCA1
Atsena Therapeutics Inc. 15
Not applicableStudy completedNCT02946879
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis (LCA)Eye DiseasesEye Diseases, Hereditary+1 more
MeiraGTx UK II Ltd 15
Early research (Phase 1)Study completedNCT01014052
What this trial is testing

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

Who this might be right for
LCA (Leber Congenital Amaurosis)RP (Retinitis Pigmentosa)
QLT Inc. 32
Early research (Phase 1)Active Not RecruitingNCT00481546
What this trial is testing

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

Who this might be right for
Amaurosis of LeberRetinal Diseases
University of Pennsylvania 15
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