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Results for “Leber Congenital Amaurosis 2”

12 trials

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Showing 12 of 12 results

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
Spark Therapeutics, Inc. 41
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 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
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
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)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
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
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
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
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 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