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Results for “Leber Congenital Amaurosis”

36 trials

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Showing 20 of 36 results

Not applicableNot Yet RecruitingNCT06024057
What this trial is testing

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

Who this might be right for
To Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine 3
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 completedNCT00422721
What this trial is testing

Genetic Study of Patients Suffering From Congenital Amaurosis of Leber or From an Early Severe Retinal Dystrophy

Who this might be right for
AmaurosisRetinal Diseases
Nantes University Hospital 360
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
Large-scale testing (Phase 3)UnknownNCT04855045
What this trial is testing

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.

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+8 more
ProQR Therapeutics 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
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
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
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)Active Not RecruitingNCT01208389
What this trial is testing

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

Who this might be right for
Leber Congenital Amaurosis
Spark Therapeutics, Inc. 12
Testing effectiveness (Phase 2)Study completedNCT01496040
What this trial is testing

Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65

Who this might be right for
Leber Congenital Amaurosis
Nantes University Hospital 9
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
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
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
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
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
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
Not applicableActive Not RecruitingNCT03602820
What this trial is testing

Long-term Follow-up Study in Subjects Who Received Voretigene Neparvovec-rzyl (AAV2-hRPE65v2)

Who this might be right for
Inherited Retinal Dystrophy Due to RPE65 Mutations
Spark Therapeutics, Inc. 41
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)Study completedNCT00643747
What this trial is testing

Safety Study of RPE65 Gene Therapy to Treat Leber Congenital Amaurosis

Who this might be right for
Retinal Degeneration
University College, London 12
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