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Results for “Leber Congenital Amaurosis”

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Showing 17 of 0 results

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 completedNCT02714816
What this trial is testing

Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65

Who this might be right for
Leber Congenital Amaurosis
MeiraGTx UK II Ltd 37
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 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
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
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
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
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
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
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
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)Study completedNCT00481546
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
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 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
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)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