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Results for “Leber's Congenital Amaurosis”

36 trials

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Showing 20 of 36 results

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
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 completedNCT04525261
What this trial is testing

Natural History of Patients With Inherited Retinal Diseases Due to Mutations in RPE65 Gene

Who this might be right for
Leber Congenital Amaurosis 2Retinitis Pigmentosa 20
University of Campania Luigi Vanvitelli 60
Testing effectiveness (Phase 2)Active Not RecruitingNCT05203939
What this trial is testing

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

Who this might be right for
Retinitis PigmentosaLeber Congenital Amaurosis
Ocugen 22
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
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
Genentech, Inc. 41
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
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
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)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 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
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
Not applicableLooking for participantsNCT02435940
What this trial is testing

Inherited Retinal Degenerative Disease Registry

Who this might be right for
Eye Diseases HereditaryRetinal DiseaseAchromatopsia+25 more
Foundation Fighting Blindness 20,000
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 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 applicableActive Not RecruitingNCT03655223
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
Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Very early researchActive Not RecruitingNCT06088992
What this trial is testing

Leber Congenital Amaurosis Inherited Blindness of Gene Therapy Trial(LIGHT)

Who this might be right for
Leber Congenital Amaurosis
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 9
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 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
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