Status:
RECRUITING
Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
Lead Sponsor:
Laboratoires Thea
Collaborating Sponsors:
Sepul Bio
Conditions:
Leber Congenital Amaurosis 10
Blindness
Eligibility:
All Genders
6+ years
Phase:
PHASE3
Brief 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)...
Detailed Description
This is a double-masked, randomized, placebo-controlled, paired-eye study in which one eye of each subject will serve as a control. At the start of the study the two eyes of each subject will be rand...
Eligibility Criteria
Inclusion
- 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.
Exclusion
- Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
- Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
- Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollment. CME is allowed if stable for 3 months (with or without treatment).
- Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
- Any prior receipt of genetic or stem-cell therapy for ocular or non-ocular disease.
Key Trial Info
Start Date :
June 4 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
October 1 2028
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT06891443
Start Date
June 4 2025
End Date
October 1 2028
Last Update
January 9 2026
Active Locations (13)
Enter a location and click search to find clinical trials sorted by distance.
1
UCSF Wayne and Gladys Valley Center for Vision
San Francisco, California, United States, 94158
2
University of Miami - Bascom Palmer Eye Institute
Miami, Florida, United States, 33156
3
University of Iowa
Iowa City, Iowa, United States, 52242
4
University of Minnesota Medical School
Minneapolis, Minnesota, United States, 55455