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Recruiting
NCT NCT06891443
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Not yet reported
Laboratoires Thea
32 participants
Est. completion 2028-10
18 sites
Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
View official trial listing →
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Completed
NCT NCT03140969
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
Not yet reported, Not yet reported
Laboratoires Thea
11 participants
Est. completion 2019-10-02
3 sites
Stand-alone
Other conditions: Leber's Congenital Amaurosis
View official trial listing →
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Terminated
NCT NCT03913130
Extension Study to Study PQ-110-001 (NCT03140969)
Not yet reported, Not yet reported
Laboratoires Thea
9 participants
Est. completion 2022-10-03
3 sites
Stand-alone
Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
View official trial listing →
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Active, not recruiting
NCT NCT03913143
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
Not yet reported, Not yet reported
ProQR Therapeutics
36 participants
Est. completion 2023-03
14 sites
Stand-alone
Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
View official trial listing →
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Status unknown
NCT NCT04855045
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.
Not yet reported, Not yet reported
ProQR Therapeutics
15 participants
Est. completion 2023-12
9 sites
Stand-alone
Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
View official trial listing →