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Results for “Leber Congenital Amaurosis 10”

7 trials

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Showing 7 of 7 results

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
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
Testing effectiveness (Phase 2)Ended earlyNCT03913130
What this trial is testing

Extension Study to Study PQ-110-001 (NCT03140969)

Who this might be right for
Leber Congenital Amaurosis 10BlindnessLeber Congenital Amaurosis+7 more
Laboratoires Thea 9
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 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)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
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