A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
Status: Recruiting
Location: See all (12) locations...
Intervention Type: Other, Drug
Study Type: Interventional
Study Phase: Phase 3
SUMMARY
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6
Healthy Volunteers: f
View:
• Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
• Adults: \>=18 years / Minors: 6 to \<18 years.
• BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.
• Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baseline.
• Detectable ONL in the macular area as determined by the CRC at Screening.
Locations
United States
California
UCSF Wayne and Gladys Valley Center for Vision
RECRUITING
San Francisco
Florida
University of Miami - Bascom Palmer Eye Institute
RECRUITING
Miami
Iowa
University of Iowa
RECRUITING
Iowa City
Minnesota
University of Minnesota Medical School
RECRUITING
Minneapolis
Pennsylvania
University of Pennsylvania - Center for Advanced Retinal & Ocular Therapeutics
RECRUITING
Philadelphia
Other Locations
Belgium
Universitair Ziekenhuis Gent (UZ)
RECRUITING
Ghent
Canada
University of Alberta
RECRUITING
Edmonton
France
Centre de maladies rares CHNO des Quinze Vingt
RECRUITING
Paris
Germany
Justus-Liebig Universität - Department of Ophthalmology
RECRUITING
Giessen
University of Tuebingen - Inst. for Ophthalmic Research
RECRUITING
Tübingen
Netherlands
Radboud Universitair Medisch Centrum
RECRUITING
Nijmegen
United Kingdom
Moorfields Eye Hospital NHS Foundation Trust
RECRUITING
London
Contact Information
Primary
Sepul Bio Patient Advocacy Director
contact@sepulbio.com
+31 617060791
Backup
Sepul Bio Chief Medical Officer
Time Frame
Start Date:2025-06-04
Estimated Completion Date:2028-10
Participants
Target number of participants:32
Treatments
Active_comparator: Sepofarsen - Treatment Eye - up to Month 12
Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6.
Placebo_comparator: Placebo - Fellow Eye - up to Month 12
Subjects to receive Sepofarsen in one eye (160µg first then 40µg) and Placebo in the fellow eye at baseline and at month 6.
Active_comparator: Continued - Treatment Eye - up to Month 24
Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18.
Placebo_comparator: Continued - Fellow Eye - up to Month 24
Subjects to receive Sepofarsen (40µg) in one eye and Placebo in the fellow eye at Month 12 and Month 18.
Active_comparator: Mixed - Treatment Eye - up to Month 24
Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18.
Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18.
Placebo_comparator: Mixed - Fellow Eye - up to Month 12
Subjects to receive Sepofarsen (40µg) in one eye and Sepofarsen in the fellow eye (160µg first then 40µg) at Month 12 and at Month 18~Note: up to Month 12 these subjects receive placebo in the Fellow Eye, as all subjects do.